37 completed, 18 recruiting, 12 terminated, 6 active not recruiting. 86 Phase 2, 34 Phase 1, 2 Phase 3.
Follow DNA Repair-Deficiency Disorders: new trials by email Literature Review of DNA Repair-Deficiency Disorders trials Research Report on DNA Repair-Deficiency Disorders
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 |
|---|---|---|---|---|---|---|
| Prenatal Transplantation for Fetuses With Fanconi Anemia IUHSCT for FA-affected fetuses - Fanconi Anemia, Anemia, Hypoplastic, Congenital | Phase 1 / Phase 2 | Not yet recruiting | Agnieszka Czechowicz | 12 | 2028-01-01 | 2026-06-16 |
| Phase 2, Open-Label Study to Evaluate the Safety and Tolerability of Progerinin in Werner Syndrome Progerinin - Werner Syndrome | Phase 2 | Recruiting | PRG Science & Technology Co., Ltd. | 5 | 2026-10-01 | 2026-10-05 |
| Preventive Dendritic Cell Vaccination for Lynch Syndrome DC vaccination - Lynch Syndrome | Phase 1 / Phase 2 | Not yet recruiting | Radboud University Medical Center | 16 | 2026-10-01 | 2026-08-19 |
| A Study to Evaluate the Efficacy of VVD-133214 in Combination With Pembrolizumab Compared With Pembrolizumab Alone in Participants With Advanced Cancer of the Colon or Rectum VVD-133214, Pembrolizumab - Colorectal Adenocarcinoma | Phase 2 | Recruiting | Vividion Therapeutics, Inc. | 140 | 2026-09-11 | 2026-10-21 |
| Safety and Efficacy of Mutation-targeted Precision Genetic Therapy for Ataxia-Telangiectasia (A-T) Antisense oligonucleotide targeting the ATM gene - Ataxia Telangiectasia | Phase 1 / Phase 2 | Recruiting | Timothy Yu | 10 | 2026-08-01 | 2026-05-28 |
| Hematopoietic Stem Cell Transplantation (HSCT) for Common Variable Immunodeficiency (CVID) and Other Autoimmune Manifestations of Primary Immune Regulatory Disorders (PIRD) Hematopoietic stem cell transplant (HSCT) - Common Variable Immunodeficiency (CVID), Primary Immune Regulatory Disorder | Phase 2 | Recruiting | Paul Szabolcs | 25 | 2026-05-04 | 2026-05-15 |
| Study of Treosulfan-Based Conditioning for HSCT in Nijmegen Breakage Syndrome Treosulfan (Treo) - HSCT, Nijmegen Breakage Syndrome | Phase 2 | Not yet recruiting | Federal Research Institute of Pediatric Hematology, Oncology and Immunology | 24 | 2025-12-30 | 2026-01-05 |
| An Phase Ib/II Clinical Trial of TCC1727 Combination Therapy in Advanced Solid Tumors TCC1727 tablet 90mg, TCC1727 tablet 120mg, TCC1727 tablet 160mg - Solid Cancers, NSCLC (Advanced Non-small Cell Lung Cancer) | Phase 1 / Phase 2 | Recruiting | Beijing Tide Pharmaceutical Co., Ltd | 266 | 2025-12-03 | 2026-01-28 |
| Matched Related and Unrelated Donor Stem Cell Transplantation for Severe Combined Immune Deficiency (SCID): Busulfan-based Conditioning With h-ATG, Radiation, and Sirolimus Sirolimus, Busulfan, Horse -Anti-thymocyte - Severe Combined Immune Deficiency (SCID) | Phase 1 / Phase 2 | Withdrawn | National Institute of Allergy and Infectious Diseases (NIAID) | 2025-11-26 | 2025-12-01 | |
| A Study to Assess the Safety, Tolerability, and Efficacy of NDI-219216 in Patients With Advanced Solid Tumors. NDI-219216 - Advanced Solid Tumors Cancer, MSI-H Cancer | Phase 1 / Phase 2 | Recruiting | Nimbus Wadjet, Inc. | 134 | 2025-03-31 | 2026-05-06 |
| Afatinib in Patients With Fanconi Anemia (FA) and Advanced Head and Neck Squamous Cell Carcinoma (HNSCC) Afatinib - Fanconi Anemia, Head and Neck Squamous Cell Carcinoma | Phase 1 / Phase 2 | Recruiting | Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau | 25 | 2024-11-08 | 2026-05-27 |
| Sirolimus Treatment in Pediatric Patients With Ataxia-Telangiectasia Rapamune (sirolimus/rapamycin) - Ataxia-Telangiectasia (A-T), Ataxia-Telangiectasia | Phase 2 | Completed | Sara Elva Espinosa Padilla, MD | 6 | 2024-07-11 | 2026-09-15 |
| Arsenic Trioxide Combined With Chemotherapy for the Treatment of p53-mutated Pediatric Cancer Arsenic trioxide - Pediatric Cancer, Li-Fraumeni Syndrome | Phase 2 | Recruiting | Yang Li | 50 | 2023-12-13 | 2024-01-03 |
| Low Dose Treosulfan Based Conditioning Regimen and PTCy in HSCT for Nijmegen Breakage Syndrome Treosulfan - Nijmegen Breakage Syndrome | Phase 2 | Recruiting | Federal Research Institute of Pediatric Hematology, Oncology and Immunology | 10 | 2023-11-22 | 2024-01-10 |
| Safety and Efficacy Study of Transplantation of Autologous CD34+ Cells Transduced With the G2ARTE Lentiviral Vector Expressing the DCLRE1C cDNA in Artemis (DCLRE1C) Deficient Severe Combined Immunodeficiency Patients (ARTEGENE) ARTEGENE drug product - Artemis (DCLRE1C ) Deficient Severe Combined Immunodeficiency | Phase 1 / Phase 2 | Recruiting | Assistance Publique - Hôpitaux de Paris | 7 | 2023-07-19 | 2026-03-27 |
| A Dose Escalation Study of FP-045 in Patients With Fanconi Anemia FP-045 - Fanconi Anemia | Phase 1 / Phase 2 | Withdrawn | Foresee Pharmaceuticals Co., Ltd. | 2023-06-30 | 2025-03-06 | |
| Testing a Combination of Vaccines for Cancer Prevention in Lynch Syndrome Adenovirus 5 CEA/MUC1/Brachyury Vaccine Tri-Ad5, Biopsy Procedure, Biospecimen Collection - Colorectal Carcinoma, Colorectal Neoplasm | Phase 2 | Active not recruiting | National Cancer Institute (NCI) | 186 | 2023-05-08 | 2026-06-17 |
| A Phase IIa Randomized, Double-Blinded Clinical Trial of Naproxen or Aspirin for Cancer Immune Interception in Lynch Syndrome Naproxen, Aspirin - T Cells, Colorectal Cancer | Phase 2 | Recruiting | M.D. Anderson Cancer Center | 40 | 2023-03-21 | 2026-06-01 |
| Cancer Preventive Vaccine Nous-209 for Lynch Syndrome Patients Adenoviral Tumor-specific Neoantigen Priming Vaccine GAd-209-FSP, Biospecimen Collection, Endoscopic Biopsy - Colorectal Carcinoma, Lynch Syndrome | Phase 1 / Phase 2 | Active not recruiting | National Cancer Institute (NCI) | 45 | 2022-11-10 | 2026-03-03 |
| A Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients With Xeroderma Pigmentosum C and V Afamelanotide - Xeroderma Pigmentosum | Phase 2 | Unknown | Clinuvel Europe Limited | 6 | 2022-03-28 | 2023-09-21 |
Showing 20 of 86. Sign in to see all of them.
Fanconi Anemia (38) · Severe Combined Immunodeficiency (20) · Ataxia Telangiectasia (10) · Colorectal Neoplasms, Hereditary Nonpolyposis (10) · Anemia, Aplastic (9) · Myelodysplastic Syndromes (8) · Anemia, Diamond-Blackfan (7) · Colorectal Neoplasms (7) · Leukemia, Myeloid, Acute (7) · Leukemia (6) · Precursor Cell Lymphoblastic Leukemia-Lymphoma (6) · Anemia, Sickle Cell (5) · Bone Marrow Failure Disorders (5) · Dyskeratosis Congenita (5) · Granulomatous Disease, Chronic (5)
Metabolic Diseases (86) · Nutritional and Metabolic Diseases (86) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (82) · Genetic Diseases, Inborn (81) · Hemic and Lymphatic Diseases (45) · Bone Marrow Diseases (42) · Hematologic Diseases (42) · Anemia, Hypoplastic, Congenital (40) · Anemia (39) · Congenital Bone Marrow Failure Syndromes (39) · Bone Marrow Failure Disorders (36) · Immune System Diseases (36) · Anemia, Aplastic (32) · Immunologic Deficiency Syndromes (32)
Cyclophosphamide (15) · fludarabine (15) · fludarabine phosphate (14) · Busulfan (13) · Antilymphocyte Serum (10) · Filgrastim (9) · Granulocyte Colony-Stimulating Factor (8) · Whole-Body Irradiation (7) · Alemtuzumab (6) · Melphalan (6) · Methylprednisolone (6) · Mycophenolic Acid (6) · plerixafor (5) · Cyclosporine (4) · Peripheral Blood Stem Cell Transplantation (4)
Masonic Cancer Center, University of Minnesota (5) · University of California, San Francisco (4) · Assistance Publique - Hôpitaux de Paris (3) · Children's Hospital Medical Center, Cincinnati (3) · Federal Research Institute of Pediatric Hematology, Oncology and Immunology (3) · National Heart, Lung, and Blood Institute (NHLBI) (3) · Paul Szabolcs (3) · Bellicum Pharmaceuticals (2) · Boston Children's Hospital (2) · Clinuvel Europe Limited (2) · Emory University (2) · National Cancer Institute (NCI) (2) · Radboud University Medical Center (2) · Roswell Park Cancer Institute (2) · Sierra Oncology LLC - a GSK company (2)
United States (55) · Spain (9) · United Kingdom (7) · France (6) · Germany (6) · Italy (4) · Netherlands (4) · Belgium (3) · China (3) · Australia (2) · Brazil (2) · Mexico (2) · Poland (2) · Portugal (2) · Puerto Rico (2)
Paul Szabolcs, MD (3) · Margaret MacMillan, MD (2) · Ajay Bansal (1) · Akiko Shimamura, MD, PhD (1) · Andre Larochelle, M.D. (1) · Anita Scheuber, MD, PhD (1) · Ann-Sofie Backman, MD PhD (1) · Anwaar Saeed, MD (1) · Biljana Horn, MD (1) · Bordignon Claudio, MD (1) · Chantal Lagresle-Peyrou, MD (1) · Chris Dvorak, MD (1) · Christelle Achkar, MD (1)
PMID 25824692, PMID 16763896, PMID 16934683, PMID 18218852, PMID 18723429, PMID 19179314, PMID 19633200, PMID 19652199, PMID 19841031, PMID 22144182, PMID 22184407, PMID 22875082, PMID 22927201, PMID 24236946, PMID 25295500, PMID 25656498, PMID 26433381, PMID 26547562, PMID 26851119, PMID 28346229, PMID 28871440, PMID 28969699, PMID 29110179, PMID 30261899, PMID 30586396, PMID 30796268, PMID 30948626, PMID 31501599, PMID 31599480, PMID 32634238, PMID 32750127, PMID 33159275, PMID 33437944, PMID 33482890, PMID 33974038, PMID 33974366, PMID 34349180, PMID 34485595, PMID 34620022, PMID 34665631, PMID 34751952, PMID 35500223, PMID 35914227, PMID 36378548, PMID 37120671, PMID 39642902, PMID 40665878, PMID 40696207, PMID 40739200, PMID 40859225
UG1CA242609 (2), 2014 (1), 2019 (1), P30CA015704 (1), P30CA016672 (1), P30CA060553 (1), R01CA257375 (1), R01HL081499 (1), UG1CA242596 (1), UG1CA242632 (1), UG1CA242635 (1), UG1CA242643 (1)