31 completed, 9 recruiting, 9 terminated, 4 unknown. 62 Phase 1, 34 Phase 2.
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 |
| Safety and Tolerability of Metformin and Naproxen for Cancer Prevention in Fanconi Anemia Metformin, Naproxen Sodium - Fanconi Anemia, Head and Neck Cancer | Phase 1 | Recruiting | Rockefeller University | 80 | 2026-10-14 | 2026-10-28 |
| 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 |
| 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 |
| 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Sirolimus Sirolimus, Busulfan, Horse -Anti-thymocyte - Severe Combined Immune Deficiency (SCID) | Phase 1 / Phase 2 | Withdrawn | National Institute of Allergy and Infectious Diseases (NIAID) | 2025-11-26 | 2025-12-01 | |
| First in Human Pilot Study to Assess the Safety and Efficacy of Dendritic Cells Loaded With Frameshift Derived Neopeptides for the Prevention of Cancer in of Lynch Syndrome Carriers Autologous Tolerogenic Dendritic cells - Lynch Syndrome | Phase 1 | Recruiting | Fundacion Clinic per a la Recerca Biomédica | 20 | 2025-09-04 | 2026-04-13 |
| 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 |
| 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 | |
| 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 |
| Comparative Bioavailability of Betamethasone Oral Solution Metered Spray (GTX-102) in Healthy Subjects GTX-102 medium dose fast Period 1 and Period 2, GTX-102 medium dose slow Period 1 and Period 2, GTX-102 high dose fast Period 1 and Period 2 - Ataxia Telangiectasia | Phase 1 | Unknown | Grace Therapeutics Inc. | 48 | 2022-09-13 | 2023-01-23 |
| Depleted Donor Stem Cell Transplant in Children and Adults With Fanconi Anemia After Being Conditioned With a Regimen Containing Briquilimab JSP191, CliniMACS Prodigy System, Depleted Stem Cell Transplant - Fanconi Anemia | Phase 1 / Phase 2 | Recruiting | Porteus, Matthew, MD | 18 | 2021-12-07 | 2026-01-27 |
| Haplocompatible Transplant Using TCRα/β Depletion Followed by CD45RA-Depleted Donor Lymphocyte Infusions for Severe Combined Immunodeficiency (SCID) Anti-thymocyte globulin (rabbit), Busulfan, Fludarabine - Severe Combined Immunodeficiency | Phase 1 / Phase 2 | Active not recruiting | St. Jude Children's Research Hospital | 4 | 2021-09-02 | 2026-09-16 |
| Safety and Early Efficacy Study of TBX-1400 in Patients With Severe Combined Immunodeficiency TBX-1400 - Severe Combined Immunodeficiency | Phase 1 | Unknown | Taiga Biotechnologies, Inc. | 8 | 2021-08-01 | 2020-10-08 |
| Testing the Combination of Two Immunotherapy Drugs (Nivolumab and Ipilimumab) in Children, Adolescent, and Young Adult Patients With Relapsed/Refractory Cancers That Have an Increased Number of Genetic Changes, The 3CI Study Biospecimen Collection, Ipilimumab, Nivolumab - Constitutional Mismatch Repair Deficiency Syndrome, Hematopoietic and Lymphoid Cell Neoplasm | Phase 1 | Withdrawn | National Cancer Institute (NCI) | 2021-05-11 | 2026-06-10 | |
| Phase I Study of Progerinin in Healthy Volunteers Progerinin, Placebo - Hutchinson-Gilford Progeria Syndrome, Werner Syndrome | Phase 1 | Completed | PRG Science & Technology Co., Ltd. | 64 | 2020-08-24 | 2021-09-22 |
| Study to Test How Well Patients With Advanced Solid Tumors Respond to Treatment With the Elimusertib in Combination With Pembrolizumab, to Find the Optimal Dose for Patients, How the Drug is Tolerated and the Way the Body Absorbs, Distributes and Discharges the Drug Elimusertib (BAY1895344), Pembrolizumab (Keytruda®) - Advanced Solid Tumors | Phase 1 | Completed | Bayer | 56 | 2019-09-30 | 2024-04-02 |
| Phase Ib of L-NMMA and Pembrolizumab L-NMMA, Pembrolizumab - Non-Small Cell Lung Cancer, Malignant Melanoma | Phase 1 | Completed | The Methodist Hospital Research Institute | 12 | 2018-08-27 | 2026-04-09 |
Showing 20 of 62. Sign in to see all of them.
Fanconi Anemia (30) · Severe Combined Immunodeficiency (18) · Anemia, Diamond-Blackfan (7) · Anemia, Aplastic (6) · Colorectal Neoplasms, Hereditary Nonpolyposis (6) · Anemia, Sickle Cell (5) · Thalassemia (5) · Ataxia Telangiectasia (4) · Granulomatous Disease, Chronic (4) · Hemoglobinopathies (4) · Anemia (3) · Bone Marrow Failure Disorders (3) · Dyskeratosis Congenita (3) · Hematologic Neoplasms (3)
Nutritional and Metabolic Diseases (62) · Metabolic Diseases (61) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (60) · Genetic Diseases, Inborn (59) · Hemic and Lymphatic Diseases (34) · Bone Marrow Diseases (32) · Hematologic Diseases (32) · Anemia, Hypoplastic, Congenital (31) · Congenital Bone Marrow Failure Syndromes (30) · Anemia (29) · Bone Marrow Failure Disorders (29) · Anemia, Aplastic (26) · Immune System Diseases (26) · Immunologic Deficiency Syndromes (22)
fludarabine (8) · Busulfan (6) · Filgrastim (6) · Cyclophosphamide (5) · Cyclosporine (5) · Granulocyte Colony-Stimulating Factor (5) · fludarabine phosphate (5) · Alemtuzumab (4) · Antilymphocyte Serum (4) · Whole-Body Irradiation (4) · plerixafor (4) · Methylprednisolone (3) · Mycophenolic Acid (3) · Specimen Handling (3) · pembrolizumab (3)
National Cancer Institute (NCI) (5) · Fred Hutchinson Cancer Center (4) · Bellicum Pharmaceuticals (3) · Children's Hospital Medical Center, Cincinnati (3) · Assistance Publique - Hôpitaux de Paris (2) · Baylor College of Medicine (2) · Boston Children's Hospital (2) · Emory University (2) · Masonic Cancer Center, University of Minnesota (2) · National Institute of Allergy and Infectious Diseases (NIAID) (2) · Sierra Oncology LLC - a GSK company (2) · St. Jude Children's Research Hospital (2) · University of California, San Francisco (2) · University of Minnesota (2) · Agnieszka Czechowicz (1)
United States (45) · Spain (6) · United Kingdom (6) · Canada (4) · France (3) · Germany (2) · Italy (2) · Australia (1) · Brazil (1) · China (1) · Ireland (1) · Israel (1) · Netherlands (1) · Portugal (1) · Puerto Rico (1)
Eduardo Vilar-Sanchez (2) · Hans-Peter Kiem (2) · John E. Wagner, Jr. (2) · Agata Smogorzewska, MD, PhD (1) · Anita Scheuber, MD, PhD (1) · Biljana Horn, MD (1) · Bordignon Claudio, MD (1) · Chantal Lagresle-Peyrou, MD (1) · Christelle Achkar, MD (1) · Christina M Annunziata, M.D. (1) · Claire Booth, Dr (1) · Colin A Sieff, MB.BCh (1)
PMID 25824692, PMID 18218852, PMID 19179314, PMID 19633200, PMID 19652199, PMID 21725047, PMID 22184407, PMID 24296492, PMID 25295500, PMID 27869650, PMID 30261899, PMID 31501599, PMID 32641470, PMID 33974366, PMID 34665631, PMID 34751952, PMID 36378548, PMID 37120671, PMID 37142543, PMID 39642902, PMID 39820512, PMID 40696207, PMID 40859225, PMID 41545594
P30CA015704 (3), P30CA016672 (2), R01HL081499 (2), 2014 (1), 2018 (1), 2019 (1), 2025 (1), N01CN00034 (1), P01HL036444 (1), UG1CA242609 (1), UM1CA081457 (1), UM1CA154967 (1)