[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"PYC Therapeutics\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":73},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,45],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":26,"conditions":27,"keywords":4,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":33,"lastUpdatePostDateStruct":34,"startDateStruct":37,"completionDateStruct":39,"leadSponsor":41,"locationsCount":44},"100590645","phase-1-safety-of-single-and-repeat-dose-of-pyc-001-eye-injections-in-people-with-autosomal-dominant-optic-atrophy-myrtle-100590645",false,"NCT06970106","Safety of Single and Repeat Dose of PYC-001 Eye Injections in People With Autosomal Dominant Optic Atrophy (Myrtle)","A Phase 1b Open-Label, Randomized, Single Dose and Repeat Dose Study to Evaluate the Single and Repeat Dose Safety and Tolerability of Intravitreally Administered PYC-001 in Participants With Confirmed OPA1 Mutation-Associated Autosomal Dominant Optic Atrophy","PYC-001-CL-102","Inclusion Criteria:\n\n1. Must give written informed consent before any study-related activity is carried out\n2. Adult males and females, aged 18 years and above at screening;\n3. Body mass index ≥18.0 and ≤35.0 kg\u002Fm2\n4. Have a recent (within five years) genetic diagnosis of OPA1 mutation-associated (haploinsufficiency) ADOA and\u002For confirmed diagnosis during pre-screening or screening, as determined by the PI.\n5. Treatment naïve participants with best-corrected visual acuity (BCVA) of between ≤20\u002F40 (≤70 Early Treatment of Diabetic Retinopathy Study \\[ETDRS\\] letters) and ≥20\u002F200 (≥35 ETDRS letters).\n6. Treatment Naïve participants with mild to moderate visual field loss and retinal nerve fiber layer (RNFL) loss in the study eye only as determined by the Spectralis Glaucoma Module Premium Edition (GMPE) RNFL \\& visual field structure function data (map)\n7. Medically healthy (in the opinion of the PI), as determined by pre-study medical history\n8. Female participants must be of non-childbearing potential or if female participants are of childbearing potential, they must:\n\n   1. Have a negative pregnancy test at the screening visit and on study Day -1;\n   2. Agree not to attempt to become pregnant or donate ova from signing of the consent form until at least 130 days after final IVT dose administration of PYC-001;\n   3. Agree to use adequate contraception\n9. Male participants must:\n\n   1. Agree not to donate sperm\n   2. If engaging in sexual intercourse with a female partner who could become pregnant, agree to use adequate contraception\n   3. If engaging in sexual intercourse with a female partner who is not of childbearing potential or a same-sex partner, agree to use a condom\n10. Willing and able to comply with all study assessments and protocol schedule\u002F restrictions\n\nExclusion Criteria:\n\n1. Participant has a known allergy to PYC-001 or any of its excipients;\n2. Demonstrated clinically significant co-morbidities, which, in the opinion of the PI, would interfere with the participant's ability to participate in the study and\u002For confound study outcomes;\n3. Females who are breastfeeding or planning to breastfeed;\n4. Based on recent genetic testing, the participant has mutations in genes that cause ADOA, other than OPA1 (for example in case of dominant negative ADOA and ADOA Plus) or has other pathological variants that result in an ADOA-like optic atrophic phenotype or other pathologic genetic findings indicating presence of additional confounding ocular diseases based on comprehensive genetic screening.\n5. Have received any prior cell or gene therapy for a retinal condition, excluding participation in study PYC-001-101;\n6. Within three months prior to study Day -1, have undergone any vitreoretinal surgery or any other ocular surgery in the study eye.\n7. Within three months prior to study Day -1, have placement of an Ozurdex® implant. T\n8. Within three years prior to study Day -1, have placement of Retisert® of Iluvien® implants.\n9. Have ocular media opacity or poor pupillary dilation prohibiting quality ophthalmic evaluation or photography, ;\n10. Macular edema (intraretinal, sub-retinal or other fluid) in the study eye requiring treatment.\n11. History of recurrent uveitis (idiopathic or immune-related) or active ocular inflammation;\n12. Have used within 30 days of the Screening visit or is using any investigational drug or over-the-counter drug such as Idebenone, Vitamin B6, Vitamin B12, A decision will be made on a case-by-case basis by the PI in consultation with the Sponsor.\n13. Over-the-counter drugs like CoQ10 and other Nutraceutical usage will require a washout by five half-lives prior to baseline visit.\n14. Have a recent history (\\\u003C6 months) of or current excessive recreational drug or alcohol use, in the opinion of the PI.\n15. Positive alcohol breath test as assessed at screening, and on study Day -1 and study Day 1;\n16. Positive urine drugs of abuse as assessed at screening and on study Day -1 and study Day 1;\n17. Any retinal pathology other than ADOA or any other condition or prior therapy that in the opinion of the PI would make the volunteer unsuitable for this study\n18. Presence of illness or pathology that, per investigator, include symptoms and\u002For the associated treatments that can alter visual function including current ocular infection.\n19. Positive test for human immunodeficiency virus, hepatitis B or C virus;\n20. Clinically significant findings in clinical chemistry, hematology, coagulation and urinalysis tests at screening","ALL","18 Years",{"count":20,"type":21},24,"ESTIMATED","INTERVENTIONAL",[24,25],"PHASE1","PHASE2","This study aims to gather safety data and determine the optimal dosing regimen for PYC-001 in participants with confirmed OPA1 mutation-associated ADOA. Approximately 18 participants from Australia, New Zealand, and other APAC countries are expected to be enrolled, depending on safety review committee (SRC) throughout the course of the study.\n\nParticipants may be assigned to any of the following:\n\n1. A single 60ug dose of PYC-001\n2. Three doses of 10ug PYC-001 at an interval of 8 weeks\n3. Three doses of 10ug PYC-001 at an interval of 12 weeks\n4. Three doses of 30ug PYC-001 at an interval of 8 weeks\n5. Three doses of 30ug PYC-001 at an interval of 12 weeks\n\n   Following completion of the 4 week safety review of the single 60ug of PYC-001 cohort, and if the 60 μg dose level is deemed safe by the SRC, the following cohorts will also be available:\n6. Three doses of 60ug PYC-001 at an interval of 12 weeks",[28,29,30,31],"OPA1 Gene Mutation","Autosomal Dominant Optic Atrophy","Hereditary Optic Atrophies","Kjer Optic Atrophy","RECRUITING","2026-06-24",{"date":35,"type":36},"2026-06-29","ACTUAL",{"date":38,"type":36},"2025-09-30",{"date":40,"type":21},"2029-11",{"name":42,"class":43},"PYC Therapeutics","INDUSTRY",3,{"id":46,"slug":47,"hasResults":11,"nctId":48,"briefTitle":49,"officialTitle":50,"acronym":4,"eligibilityCriteria":51,"healthyVolunteers":52,"sex":17,"minAge":18,"maxAge":53,"enrollmentInfo":54,"targetDuration":4,"studyType":22,"phases":56,"briefSummary":57,"conditions":58,"keywords":60,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":64,"lastUpdatePostDateStruct":65,"startDateStruct":67,"completionDateStruct":69,"leadSponsor":71,"locationsCount":72},"100570958","phase-1-phase-1-study-to-evaluate-the-safety-and-tolerability-of-intravenously-administered-pyc-003-100570958","NCT06714006","Phase 1 Study to Evaluate the Safety and Tolerability of Intravenously Administered PYC-003","A Phase 1 Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Intravenously Administered PYC-003, a Peptide-phosphorodiamidate Morpholino Oligonucleotide Conjugate, in Healthy Adult Participants and Adult Participants With Confirmed PKD1 Mutation-associated Autosomal Dominant Polycystic Kidney Disease","Part A (SAD - Healthy Volunteers) Key Inclusion Criteria\n\n1. Adult aged 18 to 60 years (inclusive)\n2. At the discretion of the PI or designee, in good general health, with no significant medical history, and have no clinically significant abnormalities on physical examination at Screening\n3. BMI ≥ 18.0 and ≤ 32.0 kg\u002Fm2 and weight ≥ 50 kg.\n4. Non-smoker and must not have used any tobacco or nicotine products within 2 months prior to Screening.\n5. Clinical laboratory values within normal range or deemed not clinically significant by the PI\n6. eGFR ≥ 80 mL\u002Fmin\u002F1.73 m2 via the Chronic Kidney Disease Epidemiology Collaboration (CKD EPI) 2021 calculation\n7. Woman of childbearing potential (WOBCP) must agree to use an acceptable, highly effective, double barrier method of contraception from the start of Screening until study completion.\n8. Males must be surgically sterile (vasectomized for at least 6 months prior to first IP administration) or, if engaged in sexual relations with a WOCBP, must agree to use an acceptable, highly effective, double barrier method of contraception from the start of Screening until study completion.\n9. Females must agree not to donate ova from the first administration of IP until 30 days following study completion.\n10. Males must agree not donate sperm from the first administration of IP until 90 days following study completion.\n11. Able and willing to attend the necessary visits to the study site.\n12. Able and willing to adhere to caffeine, alcohol, and nicotine-containing product restrictions\n13. Able and willing to provide written informed consent after the nature of the study has been explained and prior to the commencement of any study procedures.\n\nPart A (SAD - Healthy Volunteers) Key Exclusion Criteria\n\n1. Females who are pregnant, breastfeeding, or plan to become pregnant during the course of the study.\n2. Underlying physical or psychological medical condition that, in the opinion of the PI or designee, would make the participant unlikely to comply with the protocol or complete the study per protocol.\n3. Has only 1 kidney or has received a kidney transplant.\n4. Blood donation or had significant blood loss (\\> 500 mL) within 30 days prior to the first administration of IP.\n5. Plasma donation within 7 days prior to the first administration of IP.\n6. Fever (body temperature \\> 38°C) or symptomatic viral or bacterial infection within 2 weeks prior to first administration of IP.\n7. Infections requiring parenteral antibiotics within 6 months prior to Screening.\n8. Positive test for hepatitis C antibody (HCV), hepatitis B surface antigen (HBsAg), HIV antibody.\n9. History of life-threatening infection (e.g., meningitis).\n10. Vaccination with a live vaccine within 4 weeks prior to the first administration of IP.\n11. Poor venous access.\n12. History of severe allergic or anaphylactic reactions, or sensitivity to the IP or its constituents.\n13. History of malignancy, except for non-melanoma skin cancer excised more than 1 year prior to Screening and cervical intraepithelial neoplasia that has been successfully cured more than 2 years prior to Screening.\n14. Abnormal electrocardiogram (ECG) findings at Screening, Day -3 to Day -1, or predose that are considered by the PI or designee to be clinically significant.\n15. History or presence of a condition associated with significant immunosuppression.\n16. Exposure to any drugs that cause significant immunosuppression (including experimental therapies as part of a clinical study) within 4 months or 5 half-lives (whichever is longer), prior to Screening.\n17. ALP, AST, and ALT \\> 1.5 × ULN at Screening or Day -3 to Day -1.\n18. History of borderline to low blood magnesium and potassium levels and\u002For Screening or Day -3 to Day -1 blood magnesium level \\\u003C 0.7 mmol\u002FL and potassium levels \\\u003C 3.5 mmol\u002FL.\n19. Active infection of the urinary tract (ie, kidney, bladder).\n20. Positive toxicology screening panel (urine test including qualitative identification of amphetamines, barbiturates, benzodiazepines, cocaine, methamphetamine, methadone, opiates, phencyclidine, tetrahydrocannabinol \\[THC\\], tricyclic antidepressants), or alcohol breath test.\n21. History of substance abuse or dependency or history of recreational IV drug use over the last 5 years (by self-declaration).\n22. Regular alcohol consumption defined as \\> 14 standard drinks per week for females and \\> 21 standard drinks for males (where 1 standard drink = 375 mL of mid-strength beer \\[3.5% alcohol\u002Fvolume\\], 100 mL wine \\[13.5% alcohol\u002Fvolume\\] or 30 mL of spirits \\[40% alcohol\u002Fvolume\\]) or \\> 4 standard drinks on any single day.\n23. Unwilling to abstain from alcohol for 48 hours prior to admission to the study site and for 48 hours prior to any follow-up visits.\n24. Use of any investigational medical device or investigational drug within 30 days or 5 half-lives of the investigational drug (whichever is longer) prior to the first administration of IP.\n25. Use of (or anticipated use of) the following:\n\n    1. Any prescription drugs (other than hormonal contraception; oral contraceptive pills, long-acting implantable hormones, injectable hormones, a vaginal ring, or an intrauterine device \\[IUD\\]) within 14 days prior to the first administration of IP and during the course of the study without prior approval of the PI and MM.\n    2. Any over-the-counter medication, herbal remedies, supplements or vitamins within 7 days prior to the first administration of IP and during the course of the study without prior approval of the PI and MM. Note: Paracetamol (i.e., up to 2000 mg per day) may be used for minor ailments during the study, at the discretion of the PI, without prior consultation with the MM.\n26. Unwilling to refrain from strenuous exercise (including weightlifting) for 48 hours prior to admission to the study site and for 48 hours prior to any follow-up visits.\n27. Anything that the PI considers would jeopardize the safety of the participant, prevent complete participation in the study, or compromise interpretation of study data.\n\nPart B and Part C (ADPKD Participants) Key Inclusion Criteria\n\n1. Male or female aged 18 to 65 years (inclusive) at the time of informed consent.\n2. ADPKD diagnosis as confirmed by the presence of genetic mutations associated with ADPKD, including, but not limited to, the presence of PKD1 mutation. Note: Where genotyping is not included the medical history for a participant, genotyping may be completed at Pre-Screening.\n3. Class 1C, 1D, or 1E per Mayo Imaging Classification System for Predicting Kidney Outcomes in ADPKD (Irazabal et al. 2015) (based upon prior magnetic resonance imaging \\[MRI\\] or computed tomography \\[CT\\] scan obtained prior to Screening, or MRI obtained during Pre-Screening).\n4. BMI ≥ 18.0 and ≤ 35.0 kg\u002Fm2 and weight ≥ 50 kg.\n5. Non-smoker and must not have used any tobacco or nicotine products within 2 months prior to Screening.\n6. Estimated glomerular filtration rate (eGFR) ≥ 30 mL\u002Fmin\u002F1.73 m2 via the CKD EPI 2021 calculation\n\n8\\. Hematology and serum chemistry results at Screening that meet the following criteria:\n\n1. Platelets \\> 150 × 10\\^9\u002FL\n2. Total white blood cell count \\> 3.0 × 10\\^9\u002FL\n3. Absolute neutrophil count \\> 1.5 × 10\\^9\u002FL\n4. Hemoglobin \\> 110 g\u002FL for females and \\> 120 g\u002FL for males\n5. Total and direct bilirubin \\\u003C 1.5 × ULN, unless elevated bilirubin is associated with a known benign condition (e.g., Gilbert's syndrome)\n6. Alanine aminotransferase (ALT) \\\u003C 1.5 × ULN\n7. Aspartate aminotransferase (AST) \\\u003C 1.5 × ULN\n8. Alkaline phosphatase (ALP) \\\u003C 1.5 × ULN\n9. Gamma-glutamyl transferase \\\u003C 2 × ULN\n\n   9.WOCBP must agree to use an acceptable, highly effective, double barrier method of contraception from the start of Screening until study completion\n\n   10\\. Males must be surgically sterile (vasectomized for at least 6 months prior to first administration of IP) or, if engaged in sexual relations with a WOCBP, must agree to use an acceptable, highly effective, double barrier method of contraception from the start of Screening until study completion\n\n   11\\. Females must agree not to donate ova from the first administration of IP until 30 days following study completion.\n\n   12.Males must agree not donate sperm from the first administration of IP until 90 days following study completion.\n\n   13\\. Able and willing to attend the necessary visits to the study site.\n\n   14\\. Able and willing to adhere to alcohol and nicotine-containing product restrictions\n\n   15\\. Able and willing to provide written informed consent after the nature of the study has been explained and prior to the commencement of any study procedures.\n\n   Part B and Part C (ADPKD Participant) Key Exclusion Criteria\n   1. Females who are pregnant, breastfeeding, or plan to become pregnant during the course of the study.\n   2. Presence of potentially confounding genetic mutations (per genotyping by a NATA accredited or equivalent diagnostic laboratory)including, but not limited to, the presence of PKD2, HNF1B, GANAB, IFT140, and\u002For DNAJB 11 mutations.\n   3. Use of (or anticipated use of) Tolvaptan and\u002For metformin administration within 30 days prior to the first administration of IP until study completion.\n   4. Underlying physical or psychological medical condition that, in the opinion of the PI or designee, would make the participant unlikely to comply with the protocol or complete the study per protocol.\n   5. Any renal or systemic pathology other than ADPKD or any other condition or prior therapy that in the opinion of the PI or designee would make the participant unsuitable for this study.\n   6. Has only 1 kidney or has a kidney transplant.\n   7. Blood donation or had significant blood loss (\\> 500 mL) within 30 days prior to the first administration of IP.\n   8. Plasma donation within 7 days prior to the first administration of IP.\n   9. Has received (or is anticipated to receive) cell therapy, gene therapy, or RNA therapy for any renal condition.\n   10. Fever (body temperature \\> 38°C) or symptomatic viral or bacterial infection within 2 weeks prior to first administration of IP.\n   11. Infections requiring parenteral antibiotics within 6 months prior to Screening.\n   12. Positive test for hepatitis C antibody (HCV), hepatitis B surface antigen (HBsAg), HIV antibody.\n   13. History of life-threatening infection (e.g., meningitis).\n   14. Vaccination with a live vaccine within 4 weeks prior to the first administration of IP.\n   15. Poor venous access.\n   16. History of severe allergic or anaphylactic reactions, or sensitivity to the IP or its constituents.\n   17. History of malignancy, except for non-melanoma skin cancer excised more than 1 year prior to Screening and cervical intraepithelial neoplasia that has been successfully cured more than 2 years prior to Screening.\n   18. Abnormal ECG findings at Screening, Day -3 to Day -1, or predose that are considered by the PI or designee to be clinically significant.\n   19. Abnormal vital signs findings at Screening that are considered by the PI or designee to be clinically significant.\n\n       Note: A hypertensive participant is eligible if on a stable antihypertensive regimen for ≥ 28 days prior to first administration of IP and the blood pressure adequately controlled (per PI discretion) prior to first administration of IP.\n   20. History or presence of a condition associated with significant immunosuppression.\n   21. Exposure to any drugs that cause significant immunosuppression (including experimental therapies as part of a clinical study) within 4 months or 5 half-lives (whichever is longer), prior to Screening.\n   22. History of borderline to low blood magnesium and potassium levels and\u002For Screening or Day -3 to Day -1 blood magnesium level \\\u003C 0.7 mmol\u002FL and potassium levels \\\u003C 3.5 mmol\u002FL.\n   23. Urine protein: creatinine ratio (UPCR) of \\> 50 mg\u002Fmmol.\n   24. Hematuria (urine protein: creatinine ratio \\> 30 mg\u002FmmoL, or hematuria \\> ++ on dipstick, or \\> 100 cells per high-power field on microscopy) and\u002For urinary abnormalities at Screening deemed by the PI or designee to be of moderate or higher severity.\n   25. Renal complications (eg, cyst rupture or cyst infections) within 6 weeks prior to first administration of IP.\n   26. Active infection of the urinary tract (ie, kidney, bladder).\n   27. Positive toxicology screening panel (urine test including qualitative identification of amphetamines, barbiturates, benzodiazepines, cocaine, methamphetamine, methadone, opiates, phencyclidine, tetrahydrocannabinol \\[THC\\], tricyclic antidepressants), or alcohol breath test.\n   28. History of substance abuse or dependency or history of recreational IV drug use over the last 5 years (by self-declaration).\n   29. Regular alcohol consumption defined as \\> 14 standard drinks per week for females and \\> 21 standard drinks for males (where 1 standard drink = 375 mL of mid-strength beer \\[3.5% alcohol\u002Fvolume\\], 100 mL wine \\[13.5% alcohol\u002Fvolume\\] or 30 mL of spirits \\[40% alcohol\u002Fvolume\\]) or \\> 4 standard drinks on any single day.\n   30. Unwilling to abstain from alcohol for 48 hours prior to admission to the study site and for 48 hours prior to any follow-up visits.\n   31. Use of any investigational medical device or investigational drug within 30 days or 5 half-lives of the investigational drug (whichever is longer) prior to the first administration of IP.\n   32. Unwilling to refrain from strenuous exercise (including weightlifting) for 48 hours prior to admission to the study site and for 48 hours prior to any follow-up visits.\n   33. Anything that the PI considers would jeopardize the safety of the participant, prevent complete participation in the study, or compromise interpretation of study data.\n\n   Part D will be an extension study for participants that complete Part C.",true,"65 Years",{"count":55,"type":21},166,[24],"This is a Phase 1, First-in-Human study to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and immunogenicity of PYC-003 in healthy adult participants and adult participants with confirmed PKD1 mutation-associated Autosomal Dominant Polycystic Kidney Disease (ADPKD) There are 4 parts in this study, i.e. Part A, Part B, Part C and Part D.",[59],"Autosomal Dominant Polycystic Kidney Disease (ADPKD)",[61,62,63],"PKD1","Autosomal Dominant Polycystic Kidney Disease","ADPKD","2026-06-04",{"date":66,"type":36},"2026-06-08",{"date":68,"type":36},"2025-04-07",{"date":70,"type":21},"2028-11",{"name":42,"class":43},10,""]