17 completed, 11 unknown, 4 recruiting, 4 terminated. 10 Phase 2, 9 Not applicable, 5 Phase 3, 4 Phase 1, 2 Phase 4.
Follow Ataxia Telangiectasia: new trials by email Literature Review of Ataxia Telangiectasia trials Research Report on Ataxia Telangiectasia
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 |
|---|---|---|---|---|---|---|
| Biomarkers for Babies and Young Children With Ataxia Telangiectasia Ataxia Telangiectasia | Not yet recruiting | University of Nottingham | 56 | 2026-09-01 | 2026-07-16 | |
| 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 |
| 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 |
| 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 |
| 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 |
| Ataxia-telangiectasia: Treating Mitochondrial Dysfunction With Nicotinamide Riboside Nicotinamide riboside - Ataxia Telangiectasia | Not applicable | Unknown | The University of Queensland | 10 | 2024-05-01 | 2024-04-18 |
| Home-based Complex Intervention for Children With Ataxia Telangiectasia Whole-body exercise and respiratory exercise - Ataxia Telangiectasia in Children, Ataxia Telangiectasia | Not applicable | Unknown | University of Plymouth | 40 | 2023-06-01 | 2023-11-29 |
| Whole Body MRI for Cancer Surveillance in A-T Magnetic Resonance Imaging and an optional blood test - Ataxia Telangiectasia | Completed | University of Nottingham | 32 | 2022-10-17 | 2024-02-07 | |
| The Pancreas Interception Center (PIC) for Early Detection, Prevention, and Novel Therapeutics Data collection - Pancreatic Ductal Adenocarcinoma, Pancreatic Cyst | Active not recruiting | H. Lee Moffitt Cancer Center and Research Institute | 317 | 2022-10-06 | 2026-09-15 | |
| 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 |
| Ataxia-telangiectasia: Treating Mitochondrial Dysfunction With a Novel Form of Anaplerosis Triheptanoin - Ataxia Telangiectasia | Phase 2 | Completed | The University of Queensland | 30 | 2022-03-15 | 2023-07-20 |
| MBM-01 (Tempol) for the Treatment of Ataxia Telangiectasia MBM-01 - Ataxia Telangiectasia Louis-Bar, Ataxia Telangiectasia in Children | Phase 2 | Unknown | Matrix Biomed, Inc. | 20 | 2021-07-01 | 2021-05-17 |
| Videoocular Assessment of Eye Movement Activity in an Ataxia Telangiectasia Ataxia Telangiectasia, Dysmetria | Completed | Federal Research Institute of Pediatric Hematology, Oncology and Immunology | 5 | 2021-03-15 | 2022-07-22 | |
| Neurofilament Light- Chain in Ataxia Telangiectasia blood withdrawal - Ataxia Telangiectasia | Unknown | Johann Wolfgang Goethe University Hospital | 40 | 2020-02-01 | 2020-10-30 | |
| N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T) IB1001 - Ataxia Telangiectasia | Phase 2 | Terminated | IntraBio Inc | 17 | 2020-01-08 | 2026-03-25 |
| 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 |
| Treatment of Leukemia and Lymphoma in Children With Ataxia Telangiectasia Ataxia Telangiectasia, Leukemia | Unknown | Rabin Medical Center | 250 | 2019-07-28 | 2021-10-01 | |
| NAD Supplementation to Prevent Progressive Neurological Disease in Ataxia Telangiectasia Nicotinamide ribonucleoside - Ataxia Telangiectasia | Phase 2 | Active not recruiting | University Hospital, Akershus | 13 | 2019-06-05 | 2022-08-17 |
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Ataxia (7) · Telangiectasis (4) · Hereditary Sensory and Autonomic Neuropathies (3) · Immunologic Deficiency Syndromes (3) · Infections (3) · Cerebellar Ataxia (2) · Colorectal Neoplasms, Hereditary Nonpolyposis (2) · Genetic Diseases, Inborn (2) · Nerve Degeneration (2) · Peutz-Jeghers Syndrome (2) · Retinitis Pigmentosa (2) · Spinocerebellar Ataxias (2) · 3-Methylglutaconic Aciduria, Type V (1) · Achalasia Addisonianism Alacrimia syndrome (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (42) · Nutritional and Metabolic Diseases (42) · Brain Diseases (41) · Cardiovascular Diseases (41) · Central Nervous System Diseases (41) · Cerebellar Diseases (41) · DNA Repair-Deficiency Disorders (41) · Dyskinesias (41) · Immune System Diseases (41) · Metabolic Diseases (41) · Nervous System Diseases (41) · Neurocutaneous Syndromes (41) · Neurologic Manifestations (41) · Primary Immunodeficiency Diseases (41) · Vascular Diseases (41)
Blood Specimen Collection (3) · acetylleucine (3) · nicotinamide-beta-riboside (3) · Heptavalent Pneumococcal Conjugate Vaccine (2) · IB1001 (2) · dexamethasone 21-phosphate (2) · Asparaginase (1) · BAY 1895344 (1) · Baclofen (1) · Breathing Exercises (1) · Buprenorphine (1) · Butyric Acid (1) · Caspofungin (1) · Clonidine (1) · Cyclophosphamide (1)
Johann Wolfgang Goethe University Hospital (6) · Quince Therapeutics S.p.A. (4) · IntraBio Inc (3) · University of Nottingham (3) · Johns Hopkins University (2) · Sheba Medical Center (2) · The University of Queensland (2) · Bayer (1) · Beijing Tide Pharmaceutical Co., Ltd (1) · BioDelivery Sciences International (1) · European Organisation for Research and Treatment of Cancer - EORTC (1) · Federal Research Institute of Pediatric Hematology, Oncology and Immunology (1) · Grace Therapeutics Inc. (1) · H. Lee Moffitt Cancer Center and Research Institute (1) · Hadassah Medical Organization (1)
United States (16) · Germany (11) · United Kingdom (11) · Spain (6) · Israel (5) · Italy (5) · Switzerland (5) · Norway (4) · Australia (3) · Poland (3) · Belgium (2) · Denmark (2) · India (2) · Slovakia (2) · Canada (1)
Stefan Zielen, Prof. Dr. (5) · David Coman, MBBS FRACP (2) · Dirk Thye, MD (2) · Alexander F. Karelin, PhD (1) · Benjamin E Reubinoff, MD, PhD (1) · Christelle Achkar, MD (1) · Claudio Viscoli, MD (1) · Ewan Pearson, MD PhD (1) · Guenter R Janhofer, MD, PhD (1) · Hilde L Nilsen (1) · Janice Faulknor, MD (1) · John T. Sandlund, MD (1) · Kent D McKelvey, MD (1) · Luca Benatti, MD (1) · Michel Willemsen, Prof. (1)
PMID 34477998, PMID 40739200, PMID 16763896, PMID 16934683, PMID 19841031, PMID 22144182, PMID 22875082, PMID 22927201, PMID 24236946, PMID 25656498, PMID 26433381, PMID 26547562, PMID 26645295, PMID 26851119, PMID 28871440, PMID 28969699, PMID 29110179, PMID 30586396, PMID 30796268, PMID 30948626, PMID 33437944, PMID 33482890, PMID 34349180, PMID 34620022, PMID 39917433, PMID 42309084, PMID 42600624
2018 (2)