91 terminated. 91 Phase 1, 32 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 91 | Terminated 91 |
| Phase 2 | 32 | Terminated 32 |
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 |
|---|---|---|---|---|---|---|
| Study to Evaluate the Pharmacokinetics and Safety Effects Following Coadministration of Carbamazepine or Itraconazole With Radiprodil in Healthy Adults Radiprodil with Carbamazepine, Radiprodil with Itraconazole - Tuberous Sclerosis Complex (TSC), Focal Cortical Dysplasia | Phase 1 | Terminated | GRIN Therapeutics, Inc. | 30 | 2025-05-08 | 2025-11-21 |
| Hero: A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics (PK) and Pharmacodynamics (PD) of ION269 in Participants With Down Syndrome (DS) at Risk for Alzheimer's Disease (AD) ION269 - Alzheimer Disease, Down Syndrome | Phase 1 | Terminated | Ionis Pharmaceuticals, Inc. | 1 | 2024-12-20 | 2026-02-20 |
| Neoadjuvant ADI-PEG 20 + Ifosfamide + Radiotherapy in Soft Tissue Sarcoma ADI PEG20, Ifosfamide, Radiotherapy - Soft Tissue Sarcoma, Sts | Phase 1 | Terminated | Washington University School of Medicine | 6 | 2024-03-14 | 2026-09-03 |
| Long-Term Follow-Up Study of Unilateral Microtia Patients Implanted With AUR-201 AUR-201 - Microtia | Phase 1 / Phase 2 | Terminated | Auregen Biotherapeutics, SA | 1 | 2024-01-26 | 2025-11-21 |
| Subcutaneous Implant Combination Product (AUR-201) in Patients With Unilateral Microtia AUR-201 - Microtia | Phase 1 / Phase 2 | Terminated | Auregen Biotherapeutics, SA | 3 | 2024-01-26 | 2025-11-21 |
| A Study to Explore Safety, Pharmacokinetics, and Early Clinical Signal of Efficacy of DS-2325a in Patients With Netherton Syndrome DS-2325a, Placebo - Netherton Syndrome | Phase 1 / Phase 2 | Terminated | Daiichi Sankyo | 9 | 2023-09-28 | 2026-05-14 |
| The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency INZ-701 - Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets | Phase 1 | Terminated | Inozyme Pharma | 9 | 2023-06-25 | 2026-10-15 |
| Study of LQT-1213 on QTc-induced Prolongation in Healthy Adult Subjects (Part1) and on Congenital Long QT in Patients Diagnosed With Type 2 or 3 Long QT Syndrome (Part 2). LQT-1213, Placebo, Dofetilide 250 μg Cap - Long QT Syndrome | Phase 1 / Phase 2 | Terminated | Thryv Therapeutics, Inc. | 42 | 2023-03-12 | 2025-07-24 |
| A Phase I Study of RGT-264 in Subjects With Advanced Solid Tumors RGT-264 phosphate tablets - Advanced Solid Tumor | Phase 1 | Terminated | Regor Pharmaceuticals Inc. | 11 | 2023-02-15 | 2024-08-01 |
| A Study to Evaluate Administration of SBT101 Gene Therapy in Adult Patients With Adrenomyeloneuropathy (AMN) SBT101 Treatment - AMN, AMN Gene Mutation | Phase 1 / Phase 2 | Terminated | SwanBio Therapeutics, Inc. | 9 | 2022-11-17 | 2026-08-26 |
| Subcutaneous Implant Combination Product (AUR-201) in Patients With Unilateral Microtia (Australia) AUR-201 - Microtia | Phase 1 | Terminated | Auregen Biotherapeutics, SA | 2 | 2022-11-04 | 2025-11-19 |
| DFF332 as a Single Agent and in Combination With Everolimus & Immuno-Oncology Agents in Advanced/Relapsed Renal Cancer & Other Malignancies DFF332, RAD001, PDR001 - Carcinoma, Renal Cell | Phase 1 | Terminated | Novartis Pharmaceuticals | 40 | 2021-11-30 | 2026-03-03 |
| Clinical Trial to Assess the Safety and Efficacy of Investigational Product in Patients Due to Hypospadias Treatment Failure "Ex vivo" expanded autologous human oral epithelium containing stem cells - Hypospadias | Phase 1 / Phase 2 | Terminated | Holostem s.r.l. | 1 | 2021-11-26 | 2024-11-12 |
| AuriNovo for Auricular Reconstruction AuriNovo - Microtia | Phase 1 / Phase 2 | Terminated | 3DBio Therapeutics | 2 | 2021-08-09 | 2024-03-04 |
| PF-07284892 in Participants With Advanced Solid Tumors PF-07284892, lorlatinib, binimetinib - Solid Tumor | Phase 1 | Terminated | Pfizer | 53 | 2021-03-17 | 2026-06-04 |
| Initiation of Acid Suppression Therapy Prospective Outcomes for Laryngomalacia Famotidine, Speech Language Therapy - Laryngomalacia, Gastro Esophageal Reflux | Phase 1 / Phase 2 | Terminated | Reema Padia | 65 | 2020-11-18 | 2025-07-20 |
| Glucagon Receptor Inhibition to Enable Breast Cancer Patients to Benefit From PI3K Inhibitor Therapy (REMD-477) REMD-477 - Hyperglycemia Drug Induced | Phase 1 | Terminated | Duke University | 1 | 2020-11-13 | 2022-11-08 |
| Derazantinib Alone or in Combination With Paclitaxel, Ramucirumab or Atezolizumab in Gastric Adenocarcinoma Derazantinib, Derazantinib-paclitaxel-ramucirumab combination - Gastric Adenocarcinoma | Phase 1 / Phase 2 | Terminated | Basilea Pharmaceutica | 47 | 2020-10-06 | 2024-04-04 |
| First in Human Trial of Topical VT30 in Pts With Venous/Lymphatic Malformations Assoc With PIK3CA or TEK Gene Mutations VT30 - Venous Malformation, Lymphatic Malformation | Phase 1 / Phase 2 | Terminated | Venthera, Inc., a BridgeBio company | 15 | 2020-10-01 | 2022-08-04 |
| Study of CYH33 in Combination With Olaparib an Oral PARP Inhibitor in Patients With Advanced Solid Tumors. CYH33 - Ovarian Cancer, Breast Cancer | Phase 1 | Terminated | Haihe Biopharma Co., Ltd. | 24 | 2020-09-28 | 2024-03-21 |
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