Ataxia Telangiectasia clinical trials
The registry lists 42 studies for Ataxia Telangiectasia across more than 10 countries. 7 are recruiting or about to start. 35 of them are mainly about Ataxia Telangiectasia (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-06.
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Who runs them
Johann Wolfgang Goethe University Hospital (6) · Quince Therapeutics S.p.A. (4) · University of Nottingham (3) · IntraBio Inc (3) · The University of Queensland (2) · Sheba Medical Center (2) · Johns Hopkins University (2) · Sara Elva Espinosa Padilla, MD (1)
What is being tested here
Drugs under test in Ataxia Telangiectasia studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
| Drug | Open | Phase 3 | All |
|---|---|---|---|
| Nicotinamide-beta-riboside | 1 | 0 | 2 |
| Dexamethasone | 0 | 2 | 3 |
| Dexamethasone phosphate | 0 | 2 | 2 |
| Levacetylleucine | 0 | 1 | 1 |
| Sirolimus | 0 | 0 | 1 |
| BAY 1895344 | 0 | 0 | 1 |
| Triheptanoin | 0 | 0 | 1 |
| Niacinamide | 0 | 0 | 1 |
| Niacin | 0 | 0 | 1 |
| Baclofen | 0 | 0 | 1 |
Drug classes tested here Corticosteroids 3
Coming up
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
The study
- Jul 2027The Pancreas Interception Center (PIC) for Early Detection, Prevention, and Novel Therapeutics · H. Lee Moffitt Cancer Center and Research Institute
Who these studies take
Of the 5 Ataxia Telangiectasia studies recruiting now, 3 (60%) set no upper age limit and 2 (40%) stop at 75 or younger. 3 (60%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 1 (20%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 3 (60%) | 3 (60%) |
| 18 | 3 (60%) | 3 (60%) |
| 30 | 3 (60%) | 3 (60%) |
| 40 | 3 (60%) | 3 (60%) |
| 50 | 3 (60%) | 3 (60%) |
| 65 | 3 (60%) | 3 (60%) |
| 75 | 3 (60%) | 3 (60%) |
| 85 | 3 (60%) | 3 (60%) |
Across all 42 studies, recruiting or not: 48% stop at 75 or younger and 48% at 65; 79% take children; 0% are for women only and 0% for men only; 21% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
What changed recently
From the registry's own updates since Oct 14, 2026.
Results posted1
- Whole Body MRI for Cancer Surveillance in A-TNov 6, 2026NCT05252819
Studies
Recruiting first, then most recently updated. Showing 42 of 42. search them.
- Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at SanfordRecruitingNCT017931681 country
- Biomarkers for Babies and Young Children With Ataxia TelangiectasiaNot yet recruitingNCT07709728
- Safety and Efficacy of Mutation-targeted Precision Genetic Therapy for Ataxia-Telangiectasia (A-T)RecruitingNCT07215416Phase 1/21 country
- An Phase Ib/II Clinical Trial of TCC1727 Combination Therapy in Advanced Solid TumorsRecruitingNCT07371663Phase 1/21 country
- Development of IPS from Donated Somatic Cells of Patients with Neurological DiseasesRecruitingNCT008747831 country
- Ataxia-telangiectasia: Treating Mitochondrial Dysfunction With Nicotinamide RibosideNot yet recruitingNCT06324877Not applicable1 country
- Home-based Complex Intervention for Children With Ataxia TelangiectasiaRecruitingNCT05692622Not applicable1 country
- Whole Body MRI for Cancer Surveillance in A-TCompletedNCT052528191 country
- A Pivotal Study of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T)Active, not recruitingNCT06673056Phase 36 countries
- Expanded Access Program (EAP) of Levacetylleucine for Ataxia-Telangiectasia (A-T)Approved for marketingNCT07380165
- The Pancreas Interception Center (PIC) for Early Detection, Prevention, and Novel TherapeuticsActive, not recruitingNCT056925961 country
- Sirolimus Treatment in Pediatric Patients With Ataxia-TelangiectasiaCompletedNCT07820579Phase 21 country
- Open-Label Extension of EryDex Study IEDAT-04-2022TerminatedNCT06664853Phase 39 countries
- Evaluate the Neurological Effects of EryDex on Subjects With A-TCompletedNCT06193200Phase 39 countries
- N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T)TerminatedNCT03759678Phase 24 countries
- Extension Treatment Using EryDex System in Patients With AT Who Participated in the ATTeST-IEDAT-02-2015 StudyTerminatedNCT03563053Phase 311 countries
- 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 DrugCompletedNCT04095273Phase 15 countries
- Ataxia-telangiectasia: Treating Mitochondrial Dysfunction With a Novel Form of AnaplerosisCompletedNCT04513002Phase 21 country
- Comparative Bioavailability of Betamethasone Oral Solution Metered Spray (GTX-102) in Healthy SubjectsStatus unknownNCT05531890Phase 11 country
- NAD Supplementation to Prevent Progressive Neurological Disease in Ataxia TelangiectasiaActive, not recruitingNCT04870866Phase 21 country
- Effects of Vitamin B3 in Patients With Ataxia TelangiectasiaCompletedNCT03962114Phase 21 country
- Videoocular Assessment of Eye Movement Activity in an Ataxia TelangiectasiaCompletedNCT054713101 country
- A National Retrospective Population Based Cohort Study of the Natural History of Ataxia TelangiectasiaStatus unknownNCT049917011 country
- Treatment of Leukemia and Lymphoma in Children With Ataxia TelangiectasiaStatus unknownNCT040371891 country
- Oxidative Stress, Low Grade Inflammation, Tissue Breakdown and Biomarkers in Cerebrospinal Fluid of A-TCompletedNCT02285348Not applicable1 country
- MBM-01 (Tempol) for the Treatment of Ataxia TelangiectasiaStatus unknownNCT04887311Phase 21 country
- Neurofilament Light- Chain in Ataxia TelangiectasiaStatus unknownNCT046055231 country
- Pancreatic Cancer Screening of High-Risk Individuals in ArkansasWithdrawnNCT02309632Not applicable1 country
- Cell-Based Approaches For Modeling and Treating Ataxia-TelangiectasiaTerminatedNCT02246491Not applicable1 country
- Baclofen Treatment of Ataxia TelangiectasiaCompletedNCT00640003Early Phase 11 country
- Response of Individuals With Ataxia-Telangiectasia to Metformin and PioglitazoneCompletedNCT02733679Phase 41 country
- Susceptibility to Infections in Ataxia TelangiectasiaCompletedNCT02345135Not applicable
- Susceptibility to Infections, Tumor Risk and Liver Disease in Patients With Ataxia TelangiectasiaStatus unknownNCT03357978Not applicable1 country
- Evaluation of the Tolerability of Switching Subjects on Chronic ATC Opioid Therapy to Buprenorphine HCl Buccal FilmCompletedNCT01871285Phase 21 country
- Study for Treatment of Cancer in Children With Ataxia-telangiectasiaCompletedNCT00187057Not applicable1 country
- Body Composition and Hormonal Status in Ataxia TelangiectasiaCompletedNCT02345200Not applicable
- International Ataxia Rating Scale in Younger PatientsCompletedNCT019428504 countries
- Caspofungin Acetate in Treating Aspergillosis in Patients With Hematologic Cancer or in Patients Who Have Undergone a Stem Cell TransplantCompletedNCT00110045Phase 27 countries
- Status of Growth Hormone/ Insulin-like Growth Factor-1 (GH/IGF-1) Axis and Growth Failure in Ataxia Telangiectasia (AT)Status unknownNCT01052623Phase 41 country
- Immunogenicity of Pneumococcal Vaccines in Ataxia-telangiectasia PatientsStatus unknownNCT010754381 country
- The Validity of Forced Expiratory Maneuvers in Ataxia Telangiectasia Studied LongitudinallyStatus unknownNCT009518861 country
- Conjugate Pneumococcal Vaccine in Ataxia Telangiectasia (AT)CompletedNCT00656409Phase 31 country