[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"diffuse-intrinsic-pontine-gliomas-dipg\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:diffuse-intrinsic-pontine-gliomas-dipg":27},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,48],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":32,"overallStatus":35,"whyStopped":4,"lastUpdateSubmitDate":36,"lastUpdatePostDateStruct":37,"startDateStruct":40,"completionDateStruct":42,"leadSponsor":44,"locationsCount":47},"100580544","phase-2-act001-for-the-treatment-of-diffuse-intrinsic-pontine-gliomas-and-h3k27-altered-high-grade-gliomas-100580544",false,"NCT06838676","ACT001 for the Treatment of Diffuse Intrinsic Pontine Gliomas and H3K27-altered High Grade Gliomas","A Phase II Trial of ACT001 in Children and Adolescents With Diffuse Intrinsic Pontine Gliomas and H3K27-altered High Grade Gliomas","Inclusion Criteria:\n\n1. Patients must be ≥ 12 months and ≤ 39 years of age at the time of study enrollment.\n2. Diagnosis:\n\n   * Cohort A: Newly Diagnosed DIPG\n\n     * Patients with newly-diagnosed DIPG with typical MRI findings (tumors with a pontine epicenter and diffuse involvement of at least 2\u002F3 of the pons) with or without biopsy and have completed radiation therapy (RT) within 28 to 35 calendar day prior to start of therapy.\n     * Patients must have started RT \\\u003C42 calendar days from radiographic diagnosis (for non-biopsied DIPG patients only) or definitive surgery, whichever is later.\n\n       * If a biopsy was performed, the date of surgical biopsy will be considered the date of definitive diagnostic surgery; if a patient underwent two upfront surgeries \\[e.g., biopsy then debulking\\], this is the date of the second surgery)\n   * Cohort B: progressive\u002Frecurrent DIPG or H3K27-altered HGG OR refractory disease\n\n     * Patients with DIPG (no biopsy required), pathologically-confirmed (at diagnosis or recurrence) H3K27-altered DIPG, or extra-pontine H3K27-alteredHGG who have progressive\u002Frecurrent or refractory disease\n     * Progressive\u002Frecurrent: patients who have progressive or recurrent disease following frontline treatment must have included at least focal RT. New lesions since completion of frontline RT qualify as progressive disease.\n     * Refractory disease is defined as: Presence of persistent, measurable, abnormality on conventional MRI that is further distinguished by histology or advanced imaging, OR as determined by the treating physician and discussed with the Study Chair(s) prior to enrollment.\n     * Patients with H3K27-altered spinal HGG are eligible.\n     * Patients with metastatic disease are eligible.\n3. Disease Status\n\n   * Cohort A: patients may have any disease status but must have completed initial radiation therapy (RT) before enrollment.\n   * Cohort B: Patients must have measurable disease assessable by MRI. Patients may have extra neuronal disease.\n4. Performance Level: Karnofsky Performance Scale score ≥ 50% for patients \\> 16 years of age and Lansky Performance Scale score \\> 50% for patients ≤ 16 years of age (applies to all patients) Note: Patients who are unable to walk because of paralysis, but who are capable of using a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.\n5. Prior anti-cancer therapy:\n\n   * For Cohort A ONLY:\n   * Surgery, radiation (focal to disease) and\u002For steroids (dexamethasone with goal to wean dexamethasone throughout protocol therapy) are permissible. Temozolomide administered concurrently with RT is permissible. Bevacizumab use is permitted given the last dose was administered \\>\u002F= 21 days prior to enrollment. No other prior anticancer therapy for DIPG will be allowed.\n   * Patients must enroll and start treatment on study between 28 and 35 calendar days post-completion of RT.\n   * Patients must have started RT \\\u003C42 calendar days of initial diagnosis (defined as the date of diagnostic biopsy or resection; if a patient underwent two upfront surgeries \\[e.g., biopsy then resection or debulking\\], this is the date of the second surgery).\n   * Radiotherapy must have been administered at standard dose of 54 Gy for DIPG patients. Any variances in the radiotherapy dose within 10% of the standard doses outlined above will be discussed with the Study Chair to confirm eligibility prior to study enrollment.\n   * For Cohort B ONLY: Patients must have fully recovered from the acute treatment related toxicities (defined as \\\u003C\u002F= Grade 1 if not defined in eligibility criteria) of all prior chemotherapy, immunotherapy, radiotherapy, or any other treatment modality prior to entering on this study, with the exception of alopecia.\n\n   Notes to the above for Cohort B: Patients with chronic Grade 2 toxicities may be eligible per discretion of the Investigator and Sponsor (e.g., Grade 2 chemotherapy-induced neuropathy). Grade 2 or 3 toxicities from prior anti-tumor therapy that are considered irreversible - defined as having been present and stable for \\> 6 months (such as ifosfamide-related proteinuria) may be allowed if they are not otherwise described in the exclusion criteria AND there is agreement to allow by both the Investigator and Sponsor.)\n\n   The wash out period between the prior anti-cancer chemotherapy, and enrollment must be:\n   1. Myelosuppressive chemotherapy: At least 21 days after the last dose of myelosuppressive chemotherapy (42 days if prior nitrosourea).\n   2. Hematopoietic growth factors: At least 14 days after the last dose of a long-acting growth factor (e.g. Neulasta) or 7 days for short-acting growth factor.\n   3. Biologic (anti-neoplastic agent): At least 7 days after the last dose of a biologic agent. For agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur. The duration of this interval must be discussed with the study chair.\n   4. Immunotherapy: At least 42 days after the completion of any type of immunotherapy, e.g. tumor vaccines.\n   5. Monoclonal antibodies: \\> 21 days must have elapsed from the infusion of last dose of antibody\n   6. Radiation therapy:\n\n      * All Cohort B patients: Patients must have received their last fraction of focal irradiation to new sites of progressive disease \\> 14 days prior to enrollment.\n\n        * Patients who received CSI must have received their last fraction \\> 3 months prior to enrollment.\n      * Progressive\u002Frecurrent disease: Patients who received re-irradiation to primary disease must have received their last fraction \\> 3 months prior to study enrollment.\n      * Refractory Disease:\n\n        * Patients must have completed frontline RT \\> 6 months prior to enrollment.\n        * Patients who received re-irradiation to primary disease must have received their last fraction \\> 3 months prior to study enrollment.\n   7. Stem Cell Transplant: Patients must be ≥ 3 months since autologous stem cell transplant. Patients who received allogenic stem cell transplant or solid organ transplant are not eligible for study\n6. Organ Function Requirements (applies to all patients)\n\n   1. Adequate bone marrow function defined as:\n\n      * Peripheral absolute neutrophil count (ANC) \\> 1000\u002Fmm³\n      * Platelet count \\> 100,000\u002Fmm³ (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment)\n   2. Adequate renal function defined as:\n\n      * Creatinine clearance or radioisotope GFR ≥ 70 mL\u002Fmin\u002F1.73 m² or\n      * A serum creatinine based on age\u002Fgender as follows (Schwartz et al. J. Peds, 106:522, 1985): Age Maximum Serum Creatinine (mg\u002FdL) Male Female 1 to \\\u003C 2 years 0.6 0.6 2 to \\\u003C 6 years 0.8 0.8 6 to \\\u003C 10 years 1.0 1.0 10 to \\\u003C 13 years 1.2 1.2 13 to \\\u003C 16 years 1.5 1.4 ≥ 16 years 1.7 1.4\n   3. Adequate liver function defined as:\n\n      * total bilirubin must be \\\u003C\u002F=1.5X institutional ULN for age\n      * AST (serum glutamic-oxaloacetic transaminase \\[SGOT\\]) \u002F ALT (serum glutamic-oxaloacetic transaminase \\[SGPT\\]) ≤ 2.5 × institutional upper limit of normal\n      * Serum albumin ≥ 2 g\u002FdL\n   4. Adequate cardiac function defined as:\n\n      * Ejection fraction of ≥ 50% by echocardiogram\n      * QTc ≤ 450 msec (by Bazett formula)\n   5. For Cohort B: Adequate neurologic function defined as:\n\n      * Patients with seizure disorders may be enrolled if seizures are well- controlled. Well controlled is defined by no increase in seizure frequency in the 7 days prior to enrollment.\n      * Patients with neurological deficits should have deficits that are stable for at least the 7 days prior to enrollment.\n7. Informed Consent: All patients and\u002For their parents or legally authorized representatives must sign a written informed consent. Assent, when appropriate, will be obtained according to institutional guidelines.\n8. Absence of other clinically significant concomitant active medical disorder, based on the investigator's judgement.\n\nExclusion Criteria:\n\nA patient who meets any of the following exclusion criteria will not be eligible in the study (Applies to both cohorts except where noted below):\n\n1. Cohort A only: Patients with metastatic disease.\n2. Concomitant medications:\n\n   * Corticosteroids:\n\n     * Cohort A - Patients receiving corticosteroids are eligible regardless of dosing\n     * Cohort B - Patients receiving corticosteroids who have been on a stable or decreasing dose of corticosteroid for at least 7 days prior to enrollment are eligible\n   * Anti-cancer Agents: Patients who are currently receiving other anti-cancer agents are not eligible (Refer study inclusion criteria relating to anti-cancer therapies)\n\n     * Cohort A - Patients that have received any anti-cancer treatment other than surgery, RT, temozolomide concurrent with RT, and\u002For previous bevacizumab with appropriate washout period are not eligible.\n   * Investigational Drugs: Patients who are currently receiving another investigational drug are not eligible.\n   * Anticonvulsants should be used as clinically indicated. The use of enzyme inducing anticonvulsants is not permitted\n   * LHRH agonist \u002F antagonists are not permitted\n   * High Dose Biotin (B7) supplements are not permitted\n3. Concomitant medications used with caution: selective serotonin reuptake inhibitor (SSRI) such as Lexapro, Fluoxetine (Prozac), fluvoxamine (Luvox), paroxetine (Paxil), sertraline (Zoloft), citalopram (Celexa), and escitalopram should be used with caution.\n4. Infection: Patients who currently have an uncontrolled infection (in the opinion of the PI) are not eligible.\n5. Patients who have received a prior solid organ transplantation are not eligible.\n6. Pregnant or breast-feeding women will not be entered on this study due to unknown risks of fetal and teratogenic adverse events as seen in animal\u002Fhuman studies. Pregnancy tests must be obtained in girls who are postmenarchal. Males or females of reproductive potential may not participate unless they have agreed to use an effective contraceptive method.\n7. Patients of childbearing or child fathering potential must agree to use adequate contraceptive methods (hormonal or barrier method of birth control; abstinence) while being treated on this study and for 3 months after completing therapy. Note: The definition of effective contraception will be based on the judgement of the principal investigator or a designated associate.\n8. Patients who are in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study are not eligible.\n9. Patients who have previously received either ACT001 or parthenolide are not eligible.","ALL","12 Months","39 Years",{"count":20,"type":21},60,"ESTIMATED","INTERVENTIONAL",[24],"PHASE2","This is a Phase II open-label study to investigate the safety and efficacy of ACT001 in patients with DIPG and H3K27-altered HGG.",[27,28,29,30,31],"Diffuse Intrinsic Pontine Gliomas (DIPG)","Progressive DIPG","Refractory DIPG","Recurrent DIPG","H3K27-altered High Grade Glioma",[33,34],"High Grade Glioma","Diffuse Intrinsic Pontine Glioma","RECRUITING","2026-05-13",{"date":38,"type":39},"2026-05-15","ACTUAL",{"date":41,"type":39},"2025-07-10",{"date":43,"type":21},"2035-07",{"name":45,"class":46},"Nationwide Children's Hospital","OTHER",20,{"id":49,"slug":50,"hasResults":11,"nctId":51,"briefTitle":52,"officialTitle":53,"acronym":54,"eligibilityCriteria":55,"healthyVolunteers":11,"sex":16,"minAge":56,"maxAge":57,"enrollmentInfo":58,"targetDuration":4,"studyType":22,"phases":60,"briefSummary":62,"conditions":63,"keywords":68,"overallStatus":81,"whyStopped":4,"lastUpdateSubmitDate":82,"lastUpdatePostDateStruct":83,"startDateStruct":85,"completionDateStruct":86,"leadSponsor":88,"locationsCount":4},"100618395","phase-1-gallium-maltolate-for-the-treatment-of-pediatric-patients-with-relapsed-or-refractory-pediatric-high-grade-glioma-and-atypical-teratoid-rhabdoid-tumor-100618395","NCT07331064","Gallium Maltolate for the Treatment of Pediatric Patients With Relapsed or Refractory Pediatric High-Grade Glioma and Atypical Teratoid Rhabdoid Tumor","A Phase 1 Clinical Trial of Gallium Maltolate for the Treatment of Pediatric Patients With Relapsed or Refractory Pediatric High-Grade Glioma and Atypical Teratoid Rhabdoid Tumor","GABRIEL","Inclusion Criteria:\n\n1. Voluntary written consent must be obtained before performance of any study-related procedure not part of standard medical care, with the understanding that consent may be withdrawn by the subject at any time without prejudice to future medical care.\n2. Patients must have a prior histological diagnosis of pediatric high-grade glioma (WHO Grade 3 or 4, including DMG\u002FDIPG) or ATRT (WHO Grade 4) or molecular features of such tumors (per the 6th volume of Central Nervous System Tumors in the 5th edition of the WHO Classification of Tumors).\n3. Patients are required to have received standard treatment for their tumor type which is considered to include at least:\n\n   a. pHGG (including DIPG\u002FDMG): maximum safe resection, focal radiotherapy. i. Addition of temozolomide, bevacizumab, or lomustine are considered of standard of care but not a requirement for inclusion.\n\n   ii. Addition of sites of radiotherapy to include all areas of disease as needed are considered standard of care but not a requirement for inclusion.\n\n   b. ATRT: maximum safe resection, radiotherapy (focal or craniospinal), and combination chemotherapy per a nationally-accepted ATRT regimen (such as DFCI-ATRT, COG ACNS0334, or MUV-ATRT).\n4. Patients must have fully recovered from the acute toxic effects of all prior anti-cancer therapy and must meet the following minimum duration from prior anti-cancer directed therapy prior to enrollment; if after the required timeframe, the numerical eligibility criteria are met, e.g., blood count criteria, the patient is considered to have recovered adequately.\n\n   1. Cytotoxic chemotherapy (given systemically or intraventricular\u002Fintrathecal) or other anti-cancer agents known to be myelosuppressive ≥21 days after the last dose of cytotoxic or myelosuppressive chemotherapy (42 days if prior nitrosourea).\n   2. Anti-cancer agents not known to be myelosuppressive (e.g., not associated with reduced platelet or absolute neutrophil count \\[ANC\\] counts): ≥7 days after the last dose of agent.\n   3. Antibodies: ≥ 21 days must have elapsed from infusion of last dose of antibody, and toxicity related to prior antibody therapy must be recovered to grade ≤1 (for purposes of this study, bevacizumab is considered an antibody).\n   4. Corticosteroids:\n\n   i. If used to modify immune adverse events related to prior therapy, ≥14 days must have elapsed since last dose of corticosteroid.\n\n   ii. If used for symptom management related to tumor edema or elevated ICP, patient should be on a stable dose of corticosteroid for ≥7 days.\n\n   e. Hematopoietic growth factors: ≥14 days after the last dose of a long-acting growth factor (e.g., pegfilgrastim) or ≥ 7 days for short-acting growth factor.\n\n   f. Autologous stem cell infusion, including boost infusion: ≥42 days g. Cellular therapy: ≥42 days after the completion of any type of cellular therapy (e.g., modified T cells, natural killer \\[NK\\] cells, dendritic cells) h. h. Radiation therapy (XRT)\u002Fexternal beam irradiation including protons: ≥14 days after local XRT; ≥30 days after whole brain or craniospinal XRT.\n5. Patients must have measurable disease that can be assessed for response to treatment as defined by RAPNO for high-grade gliomas (59), RAPNO for DIPG (60), or RAPNO for medulloblastoma and other leptomeningeal seeding tumors (for ATRT) (61) that incorporates MRI assessment and clinical factors. In the absence of measurable disease, pathologic confirmation of recurrent disease is required (i.e., positive cerebrospinal fluid cytology).\n6. Male or female subjects must be 0-17 years of age.\n7. Lanksy\u002FKarnofsky performance status ≥50 (refer to Appendix 1). Subjects who are wheelchair-bound because of paralysis will be considered \"ambulatory\" when they are up in their wheelchair.\n8. Patients must have adequate bone marrow function as evidenced by:\n\n   1. An absolute neutrophil count (ANC) of \\>1,000\u002FµL (stable off any growth factor within one week of study drug administration.\n   2. Hemoglobin \\>8 g\u002FdL.\n   3. Platelet count \\>100,000\u002FµL without transfusion within one week.\n9. Patients must have adequate hepatic and renal function based on the following laboratory tests:\n\n   1. ALT ≤ 2 x ULN\n   2. AST ≤ 2 x ULN\n   3. Alkaline phosphatase ≤ 2 x ULN\n   4. Total bilirubin ≤ 2 x ULN\n   5. Adequate kidney function (as defined by eGFR \\>60 mL\u002Fmin\u002F1.72m2 as calculated by the Bedside Schwartz equation)\n10. Patients must be able to swallow liquid (suspension) medication, or have nasogastric or gastric tube present to give medication.\n11. Female subjects must meet one of the following:\n\n    1. Premenstrual OR\n    2. If subject is of childbearing potential, agree to practice two acceptable methods of contraception (combination methods require use of two of the following: diaphragm with spermicide, cervical cap with spermicide, contraceptive sponge, male or female condom, hormonal contraceptive) from the time of signing of the informed consent form through 21 days after the last dose of study agent, OR\n    3. Agree to practice true abstinence when this is in line with the preferred and usual lifestyle of the subject. (Periodic abstinence \\[e.g., calendar, ovulation, symptothermal, postovulation methods\\] and withdrawal are not acceptable contraception methods.)\n12. Male subjects of child-fathering potential must agree to one of the following:\n\n    1. Practice effective barrier contraception during the entire study period and through 60 calendar days after the last dose of study agent, OR\n    2. Agree to practice true abstinence when this is in line with the preferred and usual lifestyle of the subject. (Periodic abstinence \\[e.g., calendar, ovulation, symptothermal, post-ovulation methods\\] and withdrawal are not acceptable methods of contraception.)\n13. Patients taking oral iron supplements or iron chelators must discontinue these medications at least one week prior to starting GaM since these agents may impact on the efficacy of GaM. Drug-drug interactions between GaM and other concomitant medications have not been reported.\n\nExclusion Criteria:\n\n1. Presence of other active malignant disease diagnosed within 12 months.\n2. Not appropriately recovered from prior therapy as defined by time frames listed in the inclusion criteria (Section 4.5.2.1).\n3. Known hypersensitivity to or intolerance to gallium-based medications.\n4. Concurrent use of cytotoxic chemotherapy is not permitted.\n5. Unstable or severe concurrent medical conditions such as severe heart disease, renal failure, uncontrolled diabetes mellitus, or severe lung disease.\n6. History of interstitial lung disease, history of slowly progressive dyspnea and unproductive cough, sarcoidosis, silicosis, idiopathic pulmonary fibrosis, pulmonary hypersensitivity pneumonitis, or symptomatic pleural effusion.\n7. Patients who have not completed all standard-of-care treatments including surgical procedures and radiation therapy.\n8. Inability to tolerate an oral medication.\n9. Patients who are pregnant or nursing.\n10. Patients with any condition which, in the investigator's opinion, makes the patient unsuitable for study participation.","0 Months","17 Years",{"count":59,"type":21},15,[61],"PHASE1","In this study, we want to find out more about the side effects of an investigational drug for relapsed or refractory atypical teratoid rhabdoid tumor and high-grade glioma, Gallium Maltolate (GaM) and what doses of GaM are safe for people to take. Everyone in this study will receive GaM which is still experimental and is not approved by the U.S. Food and Drug Administration. We do not know all the ways that this drug may affect people. We hope the information from this study will help us develop a better treatment for relapsed or refractory atypical teratoid rhabdoid tumor and high-grade glioma in the future.",[64,65,66,67,27],"High Grade Gliomas","Atypical Teratoid Rhabdoid Tumors (ATRT)","Diffuse Midline Glioma","DIPG",[67,69,70,71,72,73,74,75,76,77,78,79,80],"HGG","ATRT","DMG","relapse","refractory","progressive","high grade glioma","diffuse midline glioma","diffuse intrinsic pontine glioma","atypical teratoid rhabdoid tumor","brain tumor","pediatric","NOT_YET_RECRUITING","2025-12-29",{"date":84,"type":39},"2026-01-09",{"date":38,"type":21},{"date":87,"type":21},"2031-05-15",{"name":89,"class":46},"Sarah Rumler"]