5 completed, 2 recruiting, 1 active not recruiting, 1 terminated. 10 Phase 2, 2 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 10 | Completed 5, Recruiting 2, Active not recruiting 1, Terminated 1, Unknown 1 |
| Phase 1 | 2 | Recruiting 2 |
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 |
|---|---|---|---|---|---|---|
| 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 |
| 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 |
| 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 |
| N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T) IB1001 - Ataxia Telangiectasia | Phase 2 | Terminated | IntraBio Inc | 17 | 2020-01-08 | 2026-03-25 |
| 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 |
| Effects of Vitamin B3 in Patients With Ataxia Telangiectasia Vitamin B3 - Ataxia Telangiectasia, ATM Gene Mutation | Phase 2 | Completed | Radboud University Medical Center | 24 | 2019-03-18 | 2022-07-25 |
| Evaluation of the Tolerability of Switching Subjects on Chronic ATC Opioid Therapy to Buprenorphine HCl Buccal Film Buprenorphine, Placebo film, Oxycodone - Pain | Phase 2 | Completed | BioDelivery Sciences International | 39 | 2013-06-01 | 2017-02-27 |
| Caspofungin Acetate in Treating Aspergillosis in Patients With Hematologic Cancer or in Patients Who Have Undergone a Stem Cell Transplant caspofungin acetate - Cancer | Phase 2 | Completed | European Organisation for Research and Treatment of Cancer - EORTC | 171 | 2005-02-01 | 2012-09-24 |
Ataxia (1) · Blast Crisis (1) · Breast Neoplasms (1) · Burkitt Lymphoma (1) · Carcinoma, Non-Small-Cell Lung (1) · Carcinoma, Ovarian Epithelial (1) · Congenital Abnormalities (1) · Endometrial Neoplasms (1) · Hereditary Sensory and Autonomic Neuropathies (1) · Hodgkin Disease (1) · Immunoblastic Lymphadenopathy (1) · Immunoglobulin Light-chain Amyloidosis (1) · Infections (1) · Intraocular Lymphoma (1)
Brain Diseases (10) · Cardiovascular Diseases (10) · Central Nervous System Diseases (10) · Cerebellar Ataxia (10) · Cerebellar Diseases (10) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (10) · DNA Repair-Deficiency Disorders (10) · Dyskinesias (10) · Genetic Diseases, Inborn (10) · Immune System Diseases (10) · Immunologic Deficiency Syndromes (10) · Metabolic Diseases (10) · Nervous System Diseases (10) · Neurocutaneous Syndromes (10) · Neurologic Manifestations (10)
nicotinamide-beta-riboside (2) · Buprenorphine (1) · Caspofungin (1) · IB1001 (1) · Morphine (1) · Niacinamide (1) · Oxycodone (1) · Sirolimus (1) · acetylleucine (1) · tempol (1) · triheptanoin (1)
Beijing Tide Pharmaceutical Co., Ltd (1) · BioDelivery Sciences International (1) · European Organisation for Research and Treatment of Cancer - EORTC (1) · IntraBio Inc (1) · Matrix Biomed, Inc. (1) · Radboud University Medical Center (1) · Sara Elva Espinosa Padilla, MD (1) · The University of Queensland (1) · Timothy Yu (1) · University Hospital, Akershus (1)
United States (4) · Germany (2) · Australia (1) · Belgium (1) · China (1) · France (1) · Italy (1) · Mexico (1) · Netherlands (1) · Norway (1) · Slovakia (1) · Spain (1) · Switzerland (1) · Turkey (Türkiye) (1) · United Kingdom (1)
Christelle Achkar, MD (1) · Claudio Viscoli, MD (1) · David Coman, MBBS FRACP (1) · Hilde L Nilsen (1) · Michel Willemsen, Prof. (1) · Sara Espinosa Padilla, MD (1) · Timothy Yu, MD, PhD (1) · Todd Kirby, PhD (1)
PMID 16763896, PMID 16934683, PMID 19841031, PMID 22144182, PMID 22875082, PMID 22927201, PMID 24236946, PMID 25656498, PMID 26433381, PMID 26547562, PMID 26851119, PMID 28871440, PMID 28969699, PMID 29110179, PMID 30586396, PMID 30796268, PMID 30948626, PMID 33437944, PMID 33482890, PMID 34349180, PMID 34620022, PMID 40739200