7 completed, 3 active not recruiting, 3 terminated, 2 unknown. 17 Phase 3, 2 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 17 | Completed 7, Active not recruiting 3, Terminated 3, Unknown 2, Not yet recruiting 1, Recruiting 1 |
| Phase 2 | 2 | Completed 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 |
|---|---|---|---|---|---|---|
| Preventive Dendritic Cell Vaccination for Lynch Syndrome Carriers Arm A: DC vaccination, Arm B: Placebo vaccination - Lynch Syndrome | Phase 3 | Not yet recruiting | Radboud University Medical Center | 372 | 2026-10-01 | 2026-05-27 |
| A Pivotal Study of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T) N-Acetyl-L-Leucine, Placebo - Ataxia-Telangiectasia, Ataxia-Telangiectasia (A-T) | Phase 3 | Active not recruiting | IntraBio Inc | 60 | 2025-03-18 | 2025-07-03 |
| Open-Label Extension of EryDex Study IEDAT-04-2022 Dexamethasone sodium phosphate - Ataxia Telangiectasia | Phase 3 | Terminated | Quince Therapeutics S.p.A. | 101 | 2024-12-11 | 2026-05-05 |
| Evaluate the Neurological Effects of EryDex on Subjects With A-T Dexamethasone sodium phosphate, Placebo - Ataxia Telangiectasia | Phase 3 | Completed | Quince Therapeutics S.p.A. | 105 | 2024-06-24 | 2026-04-30 |
| Safety and Efficacy of Metformin for Treatment of Cytopenia in Children and Adolescents With Fanconi Anemia Metformin - Fanconi Anemia | Phase 3 | Unknown | Ain Shams University | 30 | 2022-10-05 | 2024-07-25 |
| PD-1 Antibody for The Prevention of Adenomatous Polyps and Second Primary Tumors in Lynch Syndrome Patients PD-1 Antibody - Lynch Syndrome | Phase 3 | Unknown | Sun Yat-sen University | 260 | 2020-11-01 | 2021-01-20 |
| Extension Treatment Using EryDex System in Patients With AT Who Participated in the ATTeST-IEDAT-02-2015 Study EryDex System - Ataxia Telangiectasia, Genetic Syndrome | Phase 3 | Terminated | Quince Therapeutics S.p.A. | 104 | 2018-06-12 | 2024-10-08 |
| Effect of Chemoprevention by Low-dose Aspirin of New or Recurrent Colorectal Adenomas in Patients With Lynch Syndrome Acetylsalicylic acid lysinate 300 mg, Placebo (for Aspirin 300), Acetylsalicylic acid lysinate 100 mg - Lynch Syndrome | Phase 3 | Active not recruiting | Assistance Publique - Hôpitaux de Paris | 852 | 2017-11-14 | 2025-07-08 |
| Combination Chemotherapy With or Without Atezolizumab in Treating Patients With Stage III Colon Cancer and Deficient DNA Mismatch Repair Atezolizumab, Biospecimen Collection, Computed Tomography - Colon Adenocarcinoma, DNA Repair Disorder | Phase 3 | Active not recruiting | National Cancer Institute (NCI) | 712 | 2017-10-16 | 2026-10-28 |
| EZN-2279 in Patients With ADA-SCID EZN-2279, Adagen - ADA-SCID, Adenosine Deaminase Deficiency | Phase 3 | Completed | Leadiant Biosciences, Inc. | 7 | 2014-01-24 | 2020-04-16 |
| The Cancer of the Pancreas Screening-5 CAPS5)Study Secretin, MRI, Tumor marker gene test with CA19-9 - Pancreas Cancer, Peutz-Jeghers Syndrome (PJS) | Phase 3 | Recruiting | Johns Hopkins University | 9000 | 2014-01-06 | 2026-10-22 |
| LIFSCREEN : Evaluation of Whole Body MRI for Early Detection of Cancers in Subjects With P53 Mutation (Li-Fraumeni Syndrome) whole body MRI - Li Fraumeni Syndrome | Phase 3 | Completed | Gustave Roussy, Cancer Campus, Grand Paris | 107 | 2011-10-27 | 2021-02-25 |
| Intrauterine Levonorgestrel and Observation or Observation Alone in Preventing Atypical Endometrial Hyperplasia and Endometrial Cancer in Women With Hereditary Non-Polyposis Colorectal Cancer or Lynch Syndrome levonorgestrel-releasing intrauterine system, questionnaire administration, observation - Endometrial Cancer, Hereditary Non-polyposis Colon Cancer (hmsh2, hmlh1, hpms1, hpms2) | Phase 3 | Terminated | St George's, University of London | 600 | 2007-07-01 | 2013-07-10 |
| Conjugate Pneumococcal Vaccine in Ataxia Telangiectasia (AT) Conjugated pneumococcal vaccine (Prevenar) - Ataxia Telangiectasia (AT) | Phase 3 | Completed | Institute of Child Health | 30 | 2006-06-01 | 2008-04-11 |
| TBI Dose De-escalation for Fanconi Anemia Cyclophosphamide, Fludarabine, Total Body Irradiation - Fanconi Anemia | Phase 2 / Phase 3 | Completed | Masonic Cancer Center, University of Minnesota | 83 | 2006-05-18 | 2021-11-24 |
| Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant Diseases Fludarabine, Cyclophosphamide, Busulfan - Bone Marrow Failure, Osteopetrosis | Phase 2 / Phase 3 | Completed | Columbia University | 38 | 2002-09-01 | 2024-10-31 |
| Rapid Infusion of Immune Globulin Intravenous (Human) In Primary Immunodeficiency Patients Immune Globulin Intravenous [Human], 10% Caprylate/Chromatography Purified, Dextrose, 5% in Water - Immunologic Deficiency Syndrome, Agammaglobulinemia | Phase 3 | Completed | Grifols Therapeutics LLC | 100 | 2002-08-01 | 2009-09-25 |
Colorectal Neoplasms, Hereditary Nonpolyposis (6) · Ataxia Telangiectasia (5) · Fanconi Anemia (3) · Severe Combined Immunodeficiency (3) · Agammaglobulinemia (1) · Bone Marrow Failure Disorders (1) · Breast Neoplasms (1) · Colonic Neoplasms (1) · Common Variable Immunodeficiency (1) · Dysplastic Nevus Syndrome (1) · Endometrial Neoplasms (1) · Genetic Diseases, Inborn (1) · Hereditary Sensory and Autonomic Neuropathies (1) · Hereditary pancreatitis (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (17) · Metabolic Diseases (17) · Nutritional and Metabolic Diseases (17) · Genetic Diseases, Inborn (16) · Immune System Diseases (8) · Immunologic Deficiency Syndromes (7) · Neoplasms (7) · Neoplastic Syndromes, Hereditary (7) · Primary Immunodeficiency Diseases (7) · Colonic Diseases (6) · Colorectal Neoplasms (6) · Digestive System Diseases (6) · Digestive System Neoplasms (6) · Gastrointestinal Diseases (6)
Cyclophosphamide (2) · dexamethasone 21-phosphate (2) · fludarabine (2) · fludarabine phosphate (2) · Alemtuzumab (1) · Antilymphocyte Serum (1) · Bone Marrow Transplantation (1) · Busulfan (1) · CA-19-9 Antigen (1) · Caprylates (1) · Fluorouracil (1) · Glucose (1) · Heptavalent Pneumococcal Conjugate Vaccine (1) · IB1001 (1) · Immunoglobulins, Intravenous (1)
Quince Therapeutics S.p.A. (3) · Ain Shams University (1) · Assistance Publique - Hôpitaux de Paris (1) · Columbia University (1) · Grifols Therapeutics LLC (1) · Gustave Roussy, Cancer Campus, Grand Paris (1) · Institute of Child Health (1) · IntraBio Inc (1) · Johns Hopkins University (1) · Leadiant Biosciences, Inc. (1) · Masonic Cancer Center, University of Minnesota (1) · National Cancer Institute (NCI) (1) · Radboud University Medical Center (1) · St George's, University of London (1) · Sun Yat-sen University (1)
United States (10) · United Kingdom (6) · Germany (5) · Spain (4) · Italy (3) · Norway (3) · Poland (3) · Switzerland (3) · Denmark (2) · France (2) · Australia (1) · Belgium (1) · Canada (1) · China (1) · Egypt (1)
Dirk Thye, MD (2) · Elie Haddad, MD, PhD (1) · Erwin Gelfand, MD (1) · Frank A Sinicrope (1) · Guenter R Janhofer, MD, PhD (1) · James Garvin, MD, PhD (1) · Margaret L MacMillan, M.D. (1) · Michael Goggins, MD (1) · Peirong Ding, MD, Ph D (1) · Shirley Hodgson, MD (1)
PMID 28772306, PMID 31197699, PMID 31676359, PMID 32887653, PMID 33161160, PMID 34149034, PMID 39917433, PMID 40953502, PMID 41880612, PMID 42309084, PMID 42600624
2018 (1), R01CA176828 (1), U01CA210170 (1), U10CA180821 (1)