[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Disc Medicine, Inc\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":153},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,6,0,[8,42,54,78,101,123],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":18,"targetDuration":4,"studyType":21,"phases":22,"briefSummary":24,"conditions":25,"keywords":4,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":30,"lastUpdatePostDateStruct":31,"startDateStruct":34,"completionDateStruct":36,"leadSponsor":38,"locationsCount":41},"100053793","phase-2-study-of-disc-0974-201-in-participants-with-ibd-and-anemia-100053793",false,"NCT07368972","Study of DISC-0974-201 in Participants With IBD and Anemia","RALLY-IBD: A Phase 2 Randomized, Double-Blind Study to Evaluate the Safety, Tolerability, and Efficacy of DISC-0974 in Participants With Inflammatory Bowel Disease and Anemia of Inflammation","Inclusion Criteria:\n\nParticipants must meet all the following criteria at screening (unless otherwise specified) to be eligible for enrollment in the study:\n\n1. Aged ≥18 years at the time of signing informed consent.\n2. Established diagnosis of IBD (CD, UC, or IBD-unclassified) based on documented findings on both endoscopy and histopathology.\n3. Baseline endoscopy at screening with modified Mayo Score for UC and CDAI for Crohn's Disease to include mild disease as defined below:\n\n   a. CDAI of \\\u003C220 and SES-CD of 0 to 6 (CD\u002FIBD-unclassified) modified Mayo Score of \\\u003C5 points and Mayo endoscopic subscore of 0 to 1 (UC\u002FIBD-unclassified).\n4. Are symptomatic from anemia as assessed by the Investigator despite optimized, stable conventional IBD-directed therapy for 3 months.\n5. Hgb ≥7 AND \\\u003C12 g\u002FdL for females and ≥7 AND \\\u003C13 g\u002FdL for males (local lab) at screening.\n6. Have symptomatic anemia defined as:\n\n   1. Hgb ≤10 g\u002FdL and symptomatic as assessed by Investigator (fatigue, shortness of breath at rest or on minimal exertion, palpitations, tachycardia, orthostatic hypotension or dizziness), or\n   2. Hgb \\>10 g\u002FdL and a minimum score of 4 on the Numeric Rating Scale for Fatigue.\n7. Serum ferritin ≥75 μg\u002FL at screening (local lab).\n8. AST and ALT \\\u003C2× upper limit of normal (ULN) at screening.\n9. Total and direct bilirubin \\\u003CULN at screening.\n10. Estimated glomerular filtration rate ≥30 mL\u002Fmin\u002F1.73 m2 by the Chronic Kidney Disease-Epidemiology Collaboration (CKD-EPI) formula.\n11. If female, then EITHER postmenopausal (defined as at least 12 months of spontaneous amenorrhea, 6 months of spontaneous amenorrhea with serum follicle-stimulating hormone (FSH) \\>40 mIU\u002FmL, or at least 6 weeks following surgical bilateral oophorectomy with or without hysterectomy), surgically sterile, OR agreeable to use 1 of the following highly effective contraception methods (listed below) during the study and for at least 8 weeks after the last dose of study drug:\n\n    * Stable hormonal contraceptive (≥3 months)\n    * Intrauterine device in place for at least 3 months\n    * Tubal ligation or single male partner with vasectomy\n12. If a male with female sexual partner(s) of childbearing potential, agrees to use 1 of the following highly effective methods of contraception during the study and for at least 8 weeks after the last study drug dose:\n\n    * Stable hormonal contraceptive (≥3 months; female partner)\n    * Intrauterine device in place for at least 3 months (female partner)\n    * Surgically sterile by hysterectomy, bilateral oophorectomy, or bilateral tubal ligation (female partner)\n    * Confirmed successful vasectomy\n13. Able to understand and provide written informed consent.\n14. Able to comply with all study procedures.\n\nExclusion Criteria:\n\nParticipants meeting any of the following criteria at screening are not eligible for study enrollment:\n\n1. Treatment within 2 days prior to screening with oral iron or iron-containing supplements. Participants may be considered for the study if they undergo a 2-day washout period prior to screening labs for oral iron or iron-containing supplements. Between screening and 2 days prior to Day 1 visit, participants may continue oral iron or iron-containing supplements at the discretion of the Investigator, but any study-related lab draws will require a 48-hour washout from oral iron.\n2. Treatment within 30 days prior to screening with any of the following anemia treatments: blood transfusion, EPO-stimulating agent (ESA), or IV iron. Participants may be considered for the study if they undergo a 30-day washout period for ESAs or IV iron prior to screening labs.\n3. Planned change in IBD directed therapy within 3 months of screening.\n4. Moderate or severe IBD assessed during screening period. Defined as:\n\n   1. CD\u002FIBD-unclassified: participants with a CDAI score ≥220 or SES-CD ≥7\n   2. UC\u002FIBD-unclassified: modified Mayo Score of ≥6 or endoscopic subscore of 3\n   3. Fever, tachycardia, or anticipated need for surgery in the next 3 months\n5. Hospitalization within 30 days prior to screening.\n6. Positive direct antiglobulin test with reactive eluate at screening or medical history at screening of active hemolytic anemia.\n7. Gross gastrointestinal blood loss (eg, visible rectal bleeding, hematochezia, melena) within 4 weeks prior to screening.\n8. Active gastrointestinal bleeding requiring hospitalization, blood transfusion, or endoscopy hemostasis within 8 weeks prior to screening.\n9. Current use of Janus kinase (JAK) inhibitor.\n10. History of hereditary hemochromatosis.\n11. History of Primary Sclerosis Cholangitis.\n12. History of hemoglobinopathy or intrinsic RBC defect associated with anemia.\n13. History of total splenectomy.\n14. Hematopoietic stem cell or solid organ transplant within the past 10 years.\n15. Medical history of anemia from Vitamin B12 or folate deficiency or infection in the 3 months prior to screening.\n16. Stroke, myocardial infarction, deep venous thrombosis, pulmonary or arterial embolism within 6 months prior to screening.\n17. Medical history of clinically significant thrombotic disorder.\n18. If female, pregnant or breastfeeding.\n19. Any major surgery within 8 weeks before screening or incomplete recovery from any previous surgery.\n20. Current or recent systemic corticosteroid use (within 3 months of screening).\n21. Endoscopic abnormalities concerning for colon cancer on baseline endoscopy.\n22. History of malignancy within the last 3 years. The following history\u002Fconcurrent conditions are allowed: basal or squamous cell carcinoma skin cancer, carcinoma in situ of the cervix, carcinoma in situ of the breast, histologic finding of prostate cancer (T1a or T1b using the tumor, nodes, metastasis clinical staging system). A history of completed treatment (medical or surgical) of Stage 1-2 cancers may be permitted with prior Sponsor agreement.\n23. Participation in any other clinical protocol or investigational study that involves administration of experimental therapy and\u002For therapeutic devices within 30 days of screening.\n24. A history or known allergic reaction to any investigational product excipients.\n25. History of ADA formation with anaphylaxis.\n26. History of inadequately controlled heart failure (New York Heart Association Classification 3 or 4) and\u002For have a history of left ventricular ejection fraction \\\u003C35%.\n27. Uncontrolled fungal, bacterial, or viral infection (defined as ongoing signs\u002Fsymptoms related to the infection without improvement, despite appropriate treatment).\n28. Active infectious gastroenteritis including clostridium difficile colitis or viral enteritis (eg, cytomegalovirus).\n29. HIV positive, active hepatitis B virus surface antigen (HBV), or active hepatitis C virus antibody (HCV).\n30. Significant medical condition, laboratory abnormality, or psychiatric condition that would prevent the patient from participating in the study.\n31. Any condition or concomitant medication that would confound the ability to interpret data from the study.","ALL","18 Years",{"count":19,"type":20},21,"ESTIMATED","INTERVENTIONAL",[23],"PHASE2","This is a Phase 2, multicenter, randomized, double-blind placebo-controlled study of DISC-0974 to evaluate safety, tolerability, and efficacy in participants with IBD and anemia of inflammation.",[26,27,28],"Inflammatory Bowel Disease (IBD)","Anemia","Inflammatory Bowel Disease (IBD); Anemia","RECRUITING","2026-07-09",{"date":32,"type":33},"2026-07-13","ACTUAL",{"date":35,"type":33},"2026-02-20",{"date":37,"type":20},"2027-03",{"name":39,"class":40},"Disc Medicine, Inc","INDUSTRY",13,{"id":43,"slug":4,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":44,"targetDuration":4,"studyType":21,"phases":45,"briefSummary":24,"conditions":46,"keywords":4,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":47,"lastUpdatePostDateStruct":48,"startDateStruct":50,"completionDateStruct":51,"leadSponsor":52,"locationsCount":53},"100621311",{"count":19,"type":20},[23],[26,27,28],"2026-06-29",{"date":49,"type":33},"2026-06-30",{"date":35,"type":33},{"date":37,"type":20},{"name":39,"class":40},11,{"id":55,"slug":56,"hasResults":11,"nctId":57,"briefTitle":58,"officialTitle":59,"acronym":4,"eligibilityCriteria":60,"healthyVolunteers":11,"sex":16,"minAge":61,"maxAge":4,"enrollmentInfo":4,"targetDuration":4,"studyType":62,"phases":4,"briefSummary":63,"conditions":64,"keywords":67,"overallStatus":73,"whyStopped":4,"lastUpdateSubmitDate":74,"lastUpdatePostDateStruct":75,"startDateStruct":4,"completionDateStruct":4,"leadSponsor":76,"locationsCount":77},"100641328","expanded-access-program-of-bitopertin-for-participants-with-epp-or-xlp-100641328","NCT07603401","Expanded Access Program of Bitopertin For Participants With EPP or XLP","Expanded Access Program Protocol: Bitopertin (DISC-1459) For The Treatment of Erythropoietic Protoporphyria (EPP) and X-Linked Protoporphyria (XLP)","Inclusion Criteria:\n\n* Written informed consent (and assent where applicable).\n* Age ≥12 years at time of request.\n* Diagnosis of erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP), confirmed by ferrochelatase (FECH) or aminolevulinic acid synthase 2 (ALAS2) genotyping or by biochemical porphyrin analysis. Prior confirmation is acceptable; re-testing is not required if documentation of prior diagnosis is available.\n* Hemoglobin ≥10 g\u002FdL at baseline.\n* Females of childbearing potential must have a negative pregnancy test prior to enrollment.\n\nExclusion Criteria:\n\n* Known hypersensitivity to bitopertin or its excipients.\n* Patients with AST or ALT ≥3 × upper limit of normal (ULN), or total bilirubin ≥2 × ULN (unless due to Gilbert syndrome), or albumin below the lower limit of normal at screening.\n* Patients with a history of liver transplantation or anticipated need for liver transplantation.\n* Pregnancy, planned pregnancy during the program, or breastfeeding.\n* Inability or unwillingness to comply with contraception requirements.\n* Use of any of the following prohibited concomitant therapies:\n\n  * Strong CYP3A4 inhibitors\n  * Strong CYP3A4 inducers\n  * Grapefruit or Seville orange products\n  * Afamelanotide or dersimelagon\n  * Chronic opioid therapy (\\>7 consecutive days)\n* Patients who are currently participating in a bitopertin clinical study.\n* Patients who previously participated in a bitopertin study and discontinued the study due to an adverse event or for reasons that, in the judgement of the treating physician or Disc, would put the patient at unacceptable risk or otherwise preclude participation in the EAP.\n* Patients who have received another investigational therapy within 30 days.\n* History of suicidal ideation with intent (C-SSRS Grade 4 or 5) within the past year or suicidal behavior within the past 5 years.","12 Years","EXPANDED_ACCESS","The goal of this expanded access program is to provide bitopertin to patients with EPP and XLP who have no satisfactory treatment options available in the US and to learn if bitopertin is safe to treat EPP and XLP.",[65,66],"Erythropoietic Protoporphyria (EPP)","X-Linked Protoporphyria (XLP)",[68,69,70,71,72],"EPP","XLP","DISC-1459","RO4917838","Porphyria","AVAILABLE","2026-06-26",{"date":49,"type":33},{"name":39,"class":40},3,{"id":79,"slug":80,"hasResults":11,"nctId":81,"briefTitle":82,"officialTitle":83,"acronym":4,"eligibilityCriteria":84,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":85,"targetDuration":4,"studyType":21,"phases":87,"briefSummary":89,"conditions":90,"keywords":4,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":92,"lastUpdatePostDateStruct":93,"startDateStruct":95,"completionDateStruct":97,"leadSponsor":99,"locationsCount":100},"100607393","phase-1-a-phase-1b-open-label-study-of-disc-3405-in-participants-with-sickle-cell-disease-scd-100607393","NCT07187973","A Phase 1b, Open-Label Study of DISC-3405 in Participants With Sickle Cell Disease (SCD)","A Phase 1b Study of the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DISC-3405 in Participants With Sickle Cell Disease","Inclusion Criteria:\n\n1. Aged 18 years or older at the time of signing the informed consent form (ICF).\n2. Male or female study participants with SCD HbSC or HbSS.\n3. Participants who have been diagnosed with any of the following SCD-related complications: between 1-10 episodes of VOC in the past 12 months, any history of sickle cell related retinopathy, silent cerebral infarct, avascular necrosis, sensorineural hearing loss; or at least 1 episode of priapism, hepatic sequestration, splenic sequestration, or splenic infarct within the last 12 months as assessed locally.\n4. Hgb ≥7.0 g\u002FdL during Screening. The first 2 participants must have an Hgb ≥9 g\u002FdL.\n5. Normal alpha globin gene screen.\n6. Absolute reticulocyte count or % reticulocyte count \\>1.5 × upper limit of normal (ULN) during Screening.\n7. TSAT ≥15% at Screening.\n8. Ferritin ≥50 ng\u002FmL for HbSC or ≥100 ng\u002FmL for HbSS (ferritin must be \\\u003C1000 ng\u002FmL at Screening).\n9. For participants taking hydroxyurea, L-glutamine, or crizanlizumab, stable dose for at least 2 months prior to Screening and with no anticipated need for dose adjustments during the study.\n10. If male, not vasectomized for at least 6 months, with female sexual partner(s) of childbearing potential, agrees he and partner will use double methods of the following highly effective methods of birth control (described below) from the first dose of randomized study drug until 120 days after the last administration of study drug and must not donate sperm during their study participation:\n\n    1. Stable hormonal contraceptive (≥3 months; female partner) in conjunction with a barrier method (eg, condom \\[male or female\\] or diaphragm).\n    2. Intrauterine device, in place for at least 3 months (female partner) in conjunction with a barrier method (eg, condom \\[male or female\\] or diaphragm).\n    3. Surgically sterile by hysterectomy, bilateral oophorectomy, or bilateral tubal ligation (female partner) in conjunction with a barrier method (eg, condom \\[male or female\\] or diaphragm).\n11. If female, then EITHER postmenopausal, defined as at least 12 months natural, spontaneous amenorrhea, 6 months of spontaneous amenorrhea with serum follicle-stimulating hormone (FSH) \\>40 mIU\u002FmL at Screening, or at least 6 weeks following surgical menopause (bilateral oophorectomy with or without hysterectomy); surgically sterile, OR agree to use 1 of the following highly effective methods of birth control on Day 1 (or earlier) and for at least 120 days after the last administration of study drug:\n\n    1. Stable hormonal contraceptive (≥3 months) in conjunction with a barrier method (eg, condom \\[male or female\\] or diaphragm).\n    2. Intrauterine device, in place for at least 3 months in conjunction with a barrier method (eg, condom \\[male or female\\] or diaphragm).\n    3. Tubal ligation or single male partner with vasectomy in conjunction with a barrier method (eg, condom \\[male or female\\] or diaphragm).\n12. Negative pregnancy test (females of childbearing potential) prior to dosing.\n13. Able to understand the study aims, procedures, and requirements, and provide written informed consent.\n14. Able to comply with all study procedures.\n\nExclusion Criteria:\n\n1. Participants who are receiving regularly scheduled blood (RBC) transfusion therapy or phlebotomy or have received RBC transfusion or phlebotomy within 60 days of Screening.\n2. Hospitalized for VOC or other sickle cell related complication within 14 days of Screening.\n3. Participants with clinically significant bacterial, fungal, parasitic, or viral infection.\n4. Active HIV, hepatitis B, or C. A positive hepatitis or HIV result should be discussed between the Investigator and Sponsor prior to enrollment.\n5. Significant renal dysfunction, evidenced by estimated glomerular filtration rate of \\\u003C60 mL\u002Fmin\u002F1.73 m2 at the Screening visit, as assessed locally.\n6. Hepatic dysfunction characterized by alanine aminotransferase (ALT) \\>2.5 × ULN.\n7. Any episode of ACS in the last 6 months.\n8. Prior or planned hematopoietic stem cell transplant or gene therapy.\n9. History of unstable or deteriorating cardiac or pulmonary disease within 6 months prior to Screening.\n10. History of invasive malignancies within the last 5 years, except localized cured prostate cancer and cervical cancer, or other malignancies deemed acceptable by the Sponsor.\n11. Major surgery within 8 weeks before Screening or incomplete recovery from any previous surgery.\n12. A history or known allergic reaction to any IP excipients or history of anaphylaxis to any food or drug.\n13. History of alcohol dependence or excessive alcohol consumption, as assessed by the Investigator.\n14. Other medical or psychiatric condition or laboratory finding not specifically noted above that, in the judgment of the Investigator or Sponsor, would put the participant at an unacceptable risk or otherwise preclude the participant from participating in the study.\n15. Condition or concomitant medication that would confound the ability to interpret clinical, clinical laboratory, including a major psychiatric condition that has had an exacerbation or required hospitalization in the last 6 months.\n16. If female, pregnant or breastfeeding.\n17. Participation in any other clinical protocol or investigational study that involves administration of experimental therapy and\u002For therapeutic devices within 30 days of Screening.\n18. Participants with a history of transient ischemic attack or stroke may be considered in consultation with Sponsor.",{"count":86,"type":20},24,[88],"PHASE1","This is an open-label, multicenter, within-participant dose-escalation study examining up to 3 dose levels of DISC-3405 and will assess the safety, tolerability, PK, and PD of DISC 3405 in participants with sickle cell disease.",[91],"Sickle Cell Disease","2026-06-03",{"date":94,"type":33},"2026-06-04",{"date":96,"type":33},"2026-01-06",{"date":98,"type":20},"2027-10",{"name":39,"class":40},7,{"id":102,"slug":103,"hasResults":11,"nctId":104,"briefTitle":105,"officialTitle":106,"acronym":4,"eligibilityCriteria":107,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":108,"targetDuration":4,"studyType":21,"phases":110,"briefSummary":111,"conditions":112,"keywords":4,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":114,"lastUpdatePostDateStruct":115,"startDateStruct":117,"completionDateStruct":119,"leadSponsor":121,"locationsCount":122},"100591801","phase-2-a-phase-2-open-label-study-of-disc-3405-in-participants-with-polycythemia-vera-pv-100591801","NCT06985147","A Phase 2, Open-Label Study of DISC-3405 in Participants With Polycythemia Vera (PV)","A Phase 2, Open-Label Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of DISC-3405 in Participants With Polycythemia Vera (PV)","Inclusion Criteria:\n\n1. Aged 18 years or older at the time of signing the informed consent form (ICF).\n2. Meet revised 2022 World Health Organization (WHO) criteria for the diagnosis of PV.\n3. Complete blood count values at Screening of HCT \\\u003C45% or HCT \\\u003C48% if followed by a phlebotomy within 2 weeks, white blood cells 4000\u002FμL to 20,000\u002FμL (inclusive), and platelets 100,000\u002FμL to 1,000,000\u002FμL (inclusive).\n4. At least 3 phlebotomies in 26 weeks before Screening or at least 5 phlebotomies in 52 weeks before Screening. At least 1 phlebotomy must be within the 12 weeks prior to Screening.\n5. Participants receiving cytoreductive therapy must have been taking for at least 6 months and be on a stable PV therapy regimen for at least 2 months for hydroxyurea, interferon or ruxolitinib with no anticipated need for dose adjustments during the study, or have decreasing dose (with medical monitor approval).\n6. Participants treated with phlebotomy alone must have stopped cytoreductive therapy 6 months before Screening.\n7. Eastern Cooperative Oncology Group (ECOG) performance status 0 or 1, or with medical monitor approval, ECOG 2.\n8. If male with female sexual partner(s) of childbearing potential, agrees to use one of the following acceptable methods of contraception during the study and for at least 120 days after the last study drug dose:\n\n   1. Stable hormonal contraceptive (≥3 months; female partner) in conjunction with a barrier method (eg, condom or diaphragm \\[female partner\\])\n   2. Intrauterine device in place for at least 3 months (female partner)\n   3. Surgically sterile hysterectomy, bilateral oophorectomy, or bilateral tubal ligation (female partner) in conjunction with a barrier method (eg, condom \\[male or female\\] or diaphragm)\n   4. Confirmed successful vasectomy in conjunction with a barrier method (eg, condom \\[male or female\\] or diaphragm)\n9. If female, then EITHER postmenopausal, defined as at least 12 months of natural, spontaneous amenorrhea, 6 months of spontaneous amenorrhea with serum follicle-stimulating hormone \\>40 mIU\u002FmL at Screening, or at least 6 weeks following surgical menopause (bilateral oophorectomy with or without hysterectomy); surgically sterile, OR agreeable to use of highly effective contraception (listed below) on Day 1 (or earlier) and for at least 120 days after the last dose of study drug:\n\n   1. Stable hormonal contraceptive (≥3 months) in conjunction with a barrier method (eg, condom \\[male or female\\] or diaphragm)\n   2. Intrauterine device in place for at least 3 months\n   3. Tubal ligation or single male partner with vasectomy in conjunction with a barrier method (eg, condom \\[male or female\\] or diaphragm)\n10. Negative pregnancy test (females of childbearing potential).\n11. Able to understand the study aims, procedures, and requirements, and provide written informed consent.\n12. Able to comply with all study procedures.\n\nExclusion Criteria:\n\n1. Clinically significant laboratory abnormalities at Screening.\n2. Participants who require phlebotomy at HCT levels \\\u003C45%.\n3. Clinically significant thrombosis (eg, deep vein thrombosis or splenic vein thrombosis) within 2 months prior to study treatment.\n4. Clinically significant active or chronic bleeding, considered meaningful in consultation with the medical monitor, within 6 months prior to study treatment.\n5. Significant renal dysfunction, evidenced by estimated glomerular filtration rate of \\\u003C30 mL\u002Fmin\u002F1.73 m2 at the Screening visit, as assessed locally.\n6. History of invasive malignancies within the last 5 years, except localized cured prostate cancer and cervical cancer, or other malignancies deemed acceptable by the Sponsor.\n7. Participants with in situ or stage 1 squamous cell carcinoma of the skin, in situ or stage 1 basal cell carcinoma of the skin, or in situ melanoma of the skin identified during Screening unless the cancer is adequately treated before study entry.\n8. Received busulfan, pipobroman, or phosphorus-32 within 7 months prior to Screening.\n9. Major surgery within 8 weeks before Screening or incomplete recovery from any previous surgery.\n10. A history or known allergic reaction to any investigational product excipients or history of anaphylaxis to any food or drug.\n11. History of alcohol dependence or excessive alcohol consumption, as assessed by the Investigator.\n12. Active human immunodeficiency virus (HIV), hepatitis B or C. A positive hepatitis or HIV result should be discussed between the Investigator and Sponsor prior to enrollment.\n13. Other medical or psychiatric condition or laboratory finding not specifically noted above that, in the judgment of the Investigator or Sponsor, would put the participant at unacceptable risk or otherwise preclude the participant from participating in the study.\n14. Condition or concomitant medication that would confound the ability to interpret clinical data, including a major psychiatric condition that has had an exacerbation or required hospitalization in the last 6 months.\n15. If female, pregnant or breastfeeding.\n16. Participation in any other clinical protocol or investigational study that involves administration of experimental therapy and\u002For therapeutic devices within 30 days (or 5 half-lives for drugs, whichever is longer) of Screening. Previous use of other hepcidin inducing agents that may impact TMPRSS6 expression are not allowed. Previous use of hepcidin mimetics may be allowed in discussion with the Sponsor.",{"count":109,"type":20},60,[23],"This open-label, multicenter, within-participant dose escalation study examining up to 2 dose levels of DISC-3405 will assess the safety, tolerability, efficacy, pharmacokinetics, and pharmacodynamics of DISC-3405 in participants with polycythemia vera (PV).",[113],"Polycythemia Vera (PV)","2026-05-26",{"date":116,"type":33},"2026-05-28",{"date":118,"type":33},"2025-08-12",{"date":120,"type":20},"2029-02",{"name":39,"class":40},15,{"id":124,"slug":125,"hasResults":11,"nctId":126,"briefTitle":127,"officialTitle":128,"acronym":4,"eligibilityCriteria":129,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":130,"targetDuration":4,"studyType":21,"phases":132,"briefSummary":133,"conditions":134,"keywords":141,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":144,"lastUpdatePostDateStruct":145,"startDateStruct":147,"completionDateStruct":149,"leadSponsor":151,"locationsCount":152},"100463861","phase-1-study-of-disc-0974-rally-mf-in-participants-with-myelofibrosis-or-myelodysplastic-syndrome-and-anemia-100463861","NCT05320198","Study of DISC-0974 (RALLY-MF) in Participants With Myelofibrosis or Myelodysplastic Syndrome and Anemia","RALLY-MF: A Phase 1b\u002F2 Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Clinical Activity of DISC-0974 in Participants With Myelofibrosis or Myelodysplastic Syndrome and Anemia","Inclusion Criteria for Participants with MF and Anemia\n\nParticipants are eligible for the study if all of the following criteria apply:\n\n1. Age 18 years or older at the time of signing the informed consent form (ICF).\n2. For Phase 1b: DIPSS score of 3 to 4 (intermediate 2 risk) or ≥5 (high-risk) primary MF, post PV MF, and\u002For post ET MF, as confirmed in the most recent local bone marrow biopsy report, according to WHO 2016 criteria.55\n\n   For Phase 2: In addition to the criteria above, DIPSS score of ≥2 (intermediate 1 risk) may also be included.\n3. Washout of at least 28 days prior to Screening of the following treatments:\n\n   1. Androgens\n   2. EPO\n   3. Cladribine\n   4. Immunomodulators (lenalidomide, thalidomide)\n   5. Luspatercept\u002Fsotatercept\n   6. Systemic corticosteroids are permitted for non-hematological conditions if stable or decreasing dose for ≥28 days prior to Screening and receiving an equivalent to ≤10 mg prednisone for the 28 days immediately prior to Screening.\n\n   Screening can begin before the 28 day washout is completed, but the washout period must be completed prior to collection of Screening blood samples.\n4. Anemia:\n\n   For Phase 1b: Hgb \\\u003C10 g\u002FdL on ≥3 assessments over 84 days prior to Screening, without RBC transfusion, or Hgb \\\u003C10 g\u002FdL and receiving RBC transfusions periodically but not meeting criteria for TD participant as defined for the TD Cohort (see Section 6.3). The baseline Hgb value for these participants is the lowest Hgb level during the 84 days prior to Screening, or RBC transfusion dependence, defined as an RBC transfusion frequency of 6 units PRBC over the 84 days immediately prior to Screening There must not be any consecutive 42 day period without an RBC transfusion in the 84 day period, and the last transfusion must be within 28 days prior to Screening.\n\n   For Phase 2:\n\n   TD high transfusion burden cohort: RBC transfusion dependence, defined as an RBC transfusion requirement of 3 to 12 PRBC units over the 84 days immediately prior to Screening TD low transfusion burden cohort: RBC transfusion dependence, defined as an RBC transfusion requirement of 1 to 2 PRBC units over the 84 days immediately prior to Screening nTD cohort: Non-transfusion dependence, baseline Hgb \\\u003C10 g\u002FdL as defined on ≥3 assessments over 84 days prior to Screening, without RBC transfusion\n5. Stable dosing of MF-directed therapy:\n\n   1. Hydroxyurea, or, if taking any other treatment for MF, stable for at least 28 days prior to Screening.\n   2. Interferon alpha stable dosing for at least 12 weeks prior to Screening.\n   3. JAK inhibitors require 12 weeks of stable dosing prior to Screening. For the TD high, TD low, and nTD cohorts, JAK inhibitors allowed include momelotinib, pacritinib, fedratinib, and ruxolitinib.\n   4. If the participant discontinues JAK inhibitor (including momelotinib\u002Fpacritinib\u002Fruxolitinib\u002Ffedratinib) and\u002For hydroxyurea prior to Screening, a 60-day washout period is required.\n6. Eastern Cooperative Oncology Group (ECOG) performance score ≤2.\n7. Infusion of hematopoietic stem cell transplant not anticipated within 8 months after Screening.\n8. TSAT \\\u003C75% (local lab acceptable).\n9. Liver iron concentration by MRI \\\u003C7 mg\u002Fg dry weight within 3 months of eligibility confirmation by central review. Required for TD high participants only.\n10. Serum ferritin ≥50 µg\u002FL at Screening.\n11. Platelet count ≥25,000\u002FµL and \\\u003C1,000,000\u002FµL; neutrophils ≥1,000\u002FµL; and total white blood cell (WBC) count \\\u003C50,000\u002FµL at Screening.\n12. Estimated glomerular filtration rate (eGFR) ≥30 mL\u002Fmin\u002F1.73 m2 by the Chronic Kidney Disease-Epidemiology Collaboration (CKD-EPI) formula.\n13. Aspartate aminotransferase (AST) and ALT \\\u003C3x upper limit of normal (ULN) at Screening.\n14. Direct bilirubin \\\u003C2x ULN at Screening. Higher levels are acceptable if these can be attributed by the Investigator to ineffective erythropoiesis or Gilbert's syndrome, with approval from Sponsor.\n15. If male with female sexual partner(s) of childbearing potential, agrees to use 1 of the following highly effective methods of contraception during the study and for at least 8 weeks after the last study drug dose:\n\n    1. Stable hormonal contraceptive (≥3 months; female partner)\n    2. Intrauterine device in place for at least 3 months (female partner)\n    3. Surgically sterile by hysterectomy, bilateral oophorectomy, or bilateral tubal ligation (female partner)\n    4. Confirmed successful vasectomy\n16. If female, then EITHER postmenopausal (defined as 12 months of spontaneous amenorrhea, 6 months of spontaneous amenorrhea with serum follicle-stimulating hormone (FSH) levels \\>40 mIU\u002Fml, or 6 weeks postsurgical bilateral oophorectomy with or without hysterectomy), surgically sterile, OR agreeable to use 1 of the following highly effective contraception methods (listed below) on Day 1 (or earlier) and for at least 8 weeks after the last dose of study drug:\n\n    1. Stable hormonal contraceptive (≥3 months)\n    2. Intrauterine device in place for at least 3 months\n    3. Tubal ligation or single male partner with vasectomy\n17. Negative urine pregnancy test (females of childbearing potential) at Screening (Days 28 to 2).\n18. Able to understand the study aims, procedures, and requirements, and provide written informed consent.\n19. Able to comply with all study procedures.\n\nInclusion Criteria for Exploratory Cohort of Participants with MDS and Anemia\n\nParticipants are eligible for the MDS exploratory cohort if all of the following criteria apply:\n\n1. Age 18 years or older at the time of signing the ICF.\n2. Molecular International Prognostic Scoring System (IPSS-M) classification of very low, low, or intermediate (ie, lower risk) MDS-ringed sideroblasts (RS) negative, MDS\u002FMPN with ringed sideroblasts and thrombocytosis (RS-T), Chronic Myelomonocytic Leukemia (CMML), Atypical Chronic Myeloid Leukemia (aCML), or Myelodysplastic\u002FMyeloproliferative Neoplasms, Unclassifiable (MDS\u002FMPN-U) as confirmed in the most recent local bone marrow biopsy report according to WHO criteria.\n3. Washout of at least 28 days is required for prior anemia\u002Fneutropenia-directed therapies, including:\n\n   1. Androgens\n   2. EPO-stimulating agents\n   3. Luspatercept\n   4. Sotatercept (ACE-011)\n   5. Imetelstat\n   6. Granulocyte colony-stimulating factor (G-CSF) OR granulocyte-macrophage CSF (GM-CSF)\n   7. Systemic corticosteroids (except for participants on a stable or decreasing dose for ≥28 days prior to randomization for non-hematological conditions and receiving an equivalent to ≤10 mg prednisone for the 28 days immediately prior to Screening) Screening can begin before the 28-day washout is completed, but the washout period must be completed prior to collection of Screening blood samples.\n4. Anemia:\n\n   1. Baseline Hgb of \\\u003C10 g\u002FdL on ≥3 assessments over 84 days prior to Screening, without RBC transfusion, or Hgb \\\u003C10 g\u002FdL and receiving RBC transfusions periodically during the 84 days prior to Screening\n   2. Medical history of ≤24 units of PRBC for MDS and anemia\n5. ECOG performance score ≤2\n6. Infusion of hematopoietic stem cell transplant not anticipated within 8 months after Screening\n7. TSAT \\\u003C75% (local lab acceptable)\n8. Liver iron concentration by MRI \\\u003C7 mg\u002Fg dry weight within 3 months of eligibility confirmation by central review\n9. Serum ferritin ≥50 μg\u002FL at Screening\n10. Platelet count ≥25,000\u002FμL and \\\u003C1,000,000\u002FμL, and total WBC count \\\u003C50,000\u002FμL at Screening or otherwise approved by Sponsor\n11. eGFR ≥30 mL\u002Fmin\u002F1.73 m2 by the CKD-EPI formula\n12. AST and ALT \\\u003C3x ULN at Screening\n13. Direct bilirubin \\\u003C2x ULN at Screening. Higher levels are acceptable if these can be attributed by the Investigator to ineffective erythropoiesis.\n14. If male with female sexual partner(s) of childbearing potential, agrees to use 1 of the following highly effective methods of contraception during the study and for at least 8 weeks after the last study drug dose:\n\n    1. Stable hormonal contraceptive (≥3 months; female partner)\n    2. Intrauterine device in place for at least 3 months (female partner)\n    3. Surgically sterile hysterectomy, bilateral oophorectomy, or bilateral tubal ligation (female partner)\n    4. Confirmed successful vasectomy\n15. If female, then EITHER postmenopausal (defined as 12 months of spontaneous amenorrhea, 6 months of spontaneous amenorrhea with serum FSH levels \\>40 mIU\u002Fml, or 6 weeks postsurgical bilateral oophorectomy with or without hysterectomy), surgically sterile, OR agreeable to use 1 of the following highly effective contraception methods (listed below) on Day 1 (or earlier) and for at least 8 weeks after the last dose of study drug:\n\n    1. Stable hormonal contraceptive (≥3 months)\n    2. Intrauterine device in place for at least 3 months\n    3. Tubal ligation or single male partner with vasectomy\n16. Negative urine pregnancy test (females of childbearing potential) at Screening (Days 28 to 2).\n17. Able to understand the study aims, procedures, and requirements, and provide written informed consent.\n18. Able to comply with all study procedures.\n\nExclusion Criteria Exclusion Criteria for Participants with MF and Anemia\n\nParticipants are excluded from the study if any of the following criteria apply:\n\nMedical History, Participants with MF and Anemia\n\n1. Hereditary hemochromatosis\n2. Hemoglobinopathy or intrinsic RBC defect associated with anemia\n3. Total splenectomy\n4. Hematopoietic cell transplant within the past 2 years or graft vs host disease requiring immunosuppression\n5. Current anemia from iron deficiency, vitamin B12 or folate deficiency, infection, or bleeding\n6. Active immune-mediated hemolytic anemia\n7. Symptomatic bleeding, unrelated to surgery, in a critical area or organ and\u002For bleeding causing a decrease in Hgb of ≥2 g\u002FdL or leading to transfusion of ≥2 units of RBCs in the 6 months prior to Screening\n8. Major surgery within 8 weeks prior to Screening or incomplete recovery from any previous surgery\n9. Malignancy with the past 3 years, other than primary MF, post ET MF, or post PV MF. The following history or concurrent conditions are allowed:\n\n   1. basal or squamous cell carcinoma\n   2. carcinoma in situ of the cervix or the breast\n   3. histologic finding of prostate cancer (T1a or T1b using the tumor, nodes, metastasis \\[TNM\\] clinical staging system) A history of completed treatment (medical or surgical) of stage 1-2 cancers may be permitted with prior Sponsor agreement\n10. Stroke, deep vein thrombosis, or pulmonary or arterial embolism within 3 months prior to Screening\n11. Known allergic reaction to any study drug excipient\n12. A history of anti-drug antibody formation\n13. Inadequately controlled heart disease (New York Heart Association Classification 3 or 4) and\u002For known to have left ventricular ejection fraction \\\u003C35%\n14. Hepatitis B or C, or human immunodeficiency virus (HIV) with detectable viral load\n15. Uncontrolled fungal, bacterial, or viral infection (ongoing signs\u002Fsymptoms related to the infection, without improvement despite appropriate treatment)\n\n    Treatment History, Medical History, Participants with MF and Anemia\n16. Iron chelation therapy in the 28 days prior to Screening\n17. Change in anticoagulant therapy regimen within 8 weeks prior to Screening\n\n    Laboratory Exclusions, Medical History, Participants with MF and Anemia\n18. Peripheral blood myeloblasts ≥10% of WBC differential at most recent evaluation prior to Screening\n19. Positive direct antiglobulin test in conjunction with a reactive RBC eluate at Screening\n\n    Miscellaneous, Medical History, Participants with MF and Anemia\n20. Pregnant or lactating\n21. Condition or concomitant medication that would confound the ability to interpret study data\n22. Other medical or psychiatric condition or laboratory finding not specifically noted above that, in the judgment of the Investigator or Sponsor, would put the participant at unacceptable risk or otherwise preclude the participant from participating in the study\n23. Participation in any other clinical protocol or investigational study that involves administration of experimental therapy and\u002For therapeutic devices within 30 days prior to Screening\n\nExclusion Criteria for Exploratory Cohort of Participants with MDS and Anemia\n\nParticipants are excluded from the MDS exploratory cohort if any of the following criteria apply:\n\nMedical History, Participants with MDS and Anemia\n\n1. Secondary MDS, ie, MDS that is known to have arisen as the result of chemical injury or treatment with chemotherapy and\u002For radiation from other diseases\n2. Peripheral blasts ≥5%\n3. Prior treatment with hypomethylating agent or other acute myeloid leukemia (AML)-like combination chemotherapy\n4. Prior treatment with \\>3 anemia-directed therapies (unless otherwise approved by Sponsor) including:\n\n   1. Luspatercept\n   2. Sotatercept (ACE-011)\n   3. EPO-stimulating agent\n   4. Imetelstat\n5. Hereditary hemochromatosis\n6. Hemoglobinopathy or intrinsic RBC defect associated with anemia\n7. Total splenectomy\n8. Hematopoietic cell transplant within the past 10 years\n9. Current anemia from iron deficiency, vitamin B12 or folate deficiency, infection, or bleeding\n10. Active immune-mediated hemolytic anemia\n11. Symptomatic bleeding, unrelated to surgery, in a critical area or organ and\u002For bleeding causing a decrease in Hgb of ≥2 g\u002FdL or leading to transfusion of ≥2 units of RBCs in the 6 months prior to Screening\n12. Major surgery within 8 weeks prior to Screening or incomplete recovery from any previous surgery\n13. Malignancy within the past 3 years, other than MDS or MDS\u002FMPN without excess blasts. The following history or concurrent conditions are allowed:\n\n    1. basal or squamous cell carcinoma\n    2. carcinoma in situ of the cervix or the breast\n    3. histologic finding of prostate cancer (T1a or T1b using the TNM clinical staging system) A history of completed treatment (medical or surgical) of stage 1-2 cancers may be permitted with prior Sponsor agreement\n14. Stroke, deep vein thrombosis, or pulmonary or arterial embolism within 6 months prior to Screening\n15. Known allergic reaction to any study drug excipient\n16. A history of antidrug antibody formation\n17. Inadequately controlled heart disease (New York Heart Association Classification 3 or 4) and\u002For known to have left ventricular ejection fraction \\\u003C35%\n18. Active hepatitis B or C, or HIV with detectable viral load\n19. Uncontrolled fungal, bacterial, or viral infection (ongoing signs\u002Fsymptoms related to the infection, without improvement despite appropriate treatment)\n\n    Treatment History, Participants with MDS and Anemia\n20. Iron chelation therapy in the 28 days prior to Screening\n21. Change in anticoagulant therapy regimen within 8 weeks prior to Screening\n\n    Laboratory Exclusions, Participants with MDS and Anemia\n22. Positive direct antiglobulin test in conjunction with a reactive RBC eluate at Screening\n\n    Miscellaneous, Participants with MDS and Anemia\n23. Pregnant or lactating\n24. Condition or concomitant medication that would confound the ability to interpret study data\n25. Other medical or psychiatric condition or laboratory finding not specifically noted above that, in the judgment of the Investigator or Sponsor, would put the participant at unacceptable risk or otherwise preclude the participant from participating in the study\n26. Participation in any other clinical protocol or investigational study that involves administration of experimental therapy and\u002For therapeutic devices within 30 days prior to Screening",{"count":131,"type":20},150,[88,23],"This phase 1b\u002F2a open-label study will assess the safety, tolerability, pharmacokinetics and pharmacodynamics of DISC-0974 as well as categorize the effects on anemia response in subjects with myelofibrosis or myelodysplastic syndrome and anemia.",[135,27,136,137,138,139,140],"Myelofibrosis; Anemia","Myelofibrosis","Myelofibrosis Due to and Following Polycythemia Vera","Primary Myelofibrosis","Post-essential Thrombocythemia Myelofibrosis","Myelodysplastic Syndromes",[142,143],"Myeloproliferative Neoplasm","Myeloproliferative Disorders","2026-05-12",{"date":146,"type":33},"2026-05-13",{"date":148,"type":33},"2022-06-06",{"date":150,"type":20},"2026-09",{"name":39,"class":40},25,""]