377 completed. 377 Phase 1, 121 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 377 | Completed 377 |
| Phase 2 | 121 | Completed 121 |
Follow Congenital Abnormalities: new trials by email Literature Review of Congenital Abnormalities trials Research Report on Congenital Abnormalities
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Effects of VX-407 on the Pharmacokinetics of Oral Contraceptives in Healthy Participants VX-407, LNG/EE, NGM/EE - Autosomal Dominant Polycystic Kidney Disease (ADPKD) | Phase 1 | Completed | Vertex Pharmaceuticals Incorporated | 74 | 2025-07-11 | 2026-03-30 |
| A Phase 1 Study Evaluating Safety, Tolerability, and Pharmacokinetics of VX-407 in Healthy Participants VX-407, Placebo - Autosomal Dominant Polycystic Kidney Disease (ADPKD) | Phase 1 | Completed | Vertex Pharmaceuticals Incorporated | 32 | 2025-06-12 | 2026-03-27 |
| Phase 1, Open-label, Drug-drug Interaction Study to Assess Effect Between Radiprodil and the Co-administered Drugs. Radiprodil + co-administered drugs - Tuberous Sclerosis Complex (TSC), Focal Cortical Dysplasia | Phase 1 | Completed | GRIN Therapeutics, Inc. | 18 | 2025-05-20 | 2025-11-18 |
| Study of Inhaled RCT1100 in Adults With PCD Caused by Pathogenic Mutations in the DNAI1 Gene to Measure Mucociliary Clearance RCT1100 - Primary Ciliary Dyskinesia (PCD) | Phase 1 | Completed | ReCode Therapeutics | 14 | 2024-10-22 | 2026-04-30 |
| PK/PD of Digoxin in Infants With SVHD PK/PD Model Based Dosing of Digoxin in Infants with Single Ventricle Heart Disease - Single-ventricle, Infant Conditions | Phase 1 / Phase 2 | Completed | Duke University | 24 | 2024-10-10 | 2026-08-14 |
| A Study to Assess the Safety, Tolerability, Ciliary Rescue, and Pharmacodynamics of RCT1100 in Adults With PCD RCT1100 - Primary Ciliary Dyskinesia | Phase 1 | Completed | ReCode Therapeutics | 7 | 2024-09-20 | 2025-10-08 |
| Assessment of the Remineralization Efficacy of Vitamin D in the Treatment of MIH. In Vitro Study and In Situ Randomized Double-Blind Placebo Clinical Trial. Vitamin D3 mouth wash - Molar Incisor Hypomineralization | Phase 1 | Completed | Cairo University | 32 | 2024-08-30 | 2026-07-06 |
| Trimetazidine Efficacy in Attenuating Paclitaxel-Induced Peripheral Neuropathy Trimetazidine, Placebo - Peripheral Neuropathy Due to Chemotherapy | Phase 1 / Phase 2 | Completed | Minia University | 60 | 2024-06-23 | 2025-03-30 |
| Evaluation of Topical ATR12-351 in Adults With Netherton Syndrome ATR12-351 - Netherton Syndrome | Phase 1 | Completed | Azitra Inc. | 6 | 2024-06-16 | 2026-07-29 |
| A Phase 1 Study to Evaluate Safety, Tolerability, and Pharmacokinetics of VX-407 in Healthy Participants VX-407, Placebo, Midazolam - Autosomal Dominant Polycystic Kidney Disease (ADPKD) | Phase 1 | Completed | Vertex Pharmaceuticals Incorporated | 159 | 2024-04-17 | 2025-07-14 |
| IC14 (Atibuclimab) in Arrhythmogenic Cardiomyopathy IC14 - Arrhythmogenic Right Ventricular Dysplasia, Arrhythmogenic Left Ventricular Cardiomyopathy | Phase 1 / Phase 2 | Completed | Implicit Bioscience | 1 | 2024-03-29 | 2026-04-22 |
| Tuvusertib (M1774) Human Mass Balance and Absolute Bioavailability Study (DDRIVER Solid Tumors 303) Tuvusertib [14C]Tuvusertib microtracer, Tuvusertib, Tuvusertib + [14C]Tuvusertib microdose bolus injection - Solid Tumor | Phase 1 | Completed | Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany | 12 | 2024-03-25 | 2026-04-23 |
| Leucettinib-21 First-in-Human Phase 1 in Healthy Volunteers and Subjects With Down Syndrome and Alzheimer's Disease Leucettinib-21 - Healthy Volunteers, Down Syndrome | Phase 1 | Completed | Perha Pharmaceuticals | 152 | 2024-01-18 | 2026-06-30 |
| Treatment of LSCD With DM transplantation of a Descemet's Membrane corneal onlay, partial LSCD, transplantation of a Descemet's Membrane corneal onlay, total/near-total LSCD - Limbal Stem-cell Deficiency, Congenital Aniridia | Phase 1 | Completed | University of Minnesota | 19 | 2023-11-30 | 2026-01-08 |
| A Study to Evaluate Setanaxib in Patients With Alport Syndrome Setanaxib, Placebo - Alport Syndrome | Phase 1 / Phase 2 | Completed | Calliditas Therapeutics AB | 20 | 2023-11-27 | 2026-04-13 |
| Tuvusertib (M1774) in Combination With Cemiplimab in Participants With Non-Squamous NSCLC (DDRiver NSCLC 322) M1774, Cemiplimab - Non-Small Cell Lung Cancer | Phase 1 / Phase 2 | Completed | EMD Serono Research & Development Institute, Inc. | 61 | 2023-09-13 | 2026-09-14 |
| Single and Multiple Ascending Dose Study of MTR-601 in Healthy Individuals MTR-601 - Muscle Spasticity, Hypertonia, Muscle | Phase 1 | Completed | Motric Bio | 89 | 2023-09-11 | 2026-06-05 |
| CTH120 First-in-Human Study: Single and Multiple Ascending Doses and Potential Food Interaction CTH120, Placebo - Fragile X Syndrome | Phase 1 | Completed | Connecta Therapeutics, S.L. | 76 | 2023-06-23 | 2025-01-23 |
| The Safety and Tolerability of CLZ-2002 in Patients With Charcot-Marie Tooth Disease. CLZ-2002 - Charcot Marie Tooth Disease, Type 1 | Phase 1 | Completed | Cellatoz Therapeutics, Inc | 9 | 2023-06-19 | 2025-02-17 |
| Assessment of KM-001 - Safety, Tolerability, and Efficacy in Patients With PPPK1 or PC KM-001 cream 1% 12 weeks treatment., Physical Examination, Vital Signs - Punctate Palmoplantar Keratoderma Type 1, Pachyonychia Congenita | Phase 1 | Completed | Orvida Pharma (formerly Kamari Pharma) | 18 | 2023-03-06 | 2025-01-15 |
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PMID 18035553, PMID 18424759, PMID 18557726, PMID 19066739, PMID 20236866, PMID 39356039, PMID 15100156, PMID 17284531, PMID 23607606, PMID 23660685, PMID 11788640, PMID 12171484, PMID 12213860, PMID 14605787, PMID 14662594, PMID 15100681, PMID 16278404, PMID 17562873, PMID 17640728, PMID 17662768, PMID 17679729, PMID 17761590, PMID 18155964, PMID 18212521, PMID 18299450, PMID 18327587, PMID 18333898, PMID 18974373, PMID 19092853, PMID 19109557, PMID 19179301, PMID 19638218, PMID 19786617, PMID 19829004, PMID 20071660, PMID 20083693, PMID 20382682, PMID 20400682, PMID 20443823, PMID 20511167, PMID 20858843, PMID 20978268, PMID 21047224, PMID 21120277, PMID 21209029, PMID 2122732, PMID 21239698, PMID 21403127, PMID 21482669, PMID 21791475
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