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Phuturdex › Diseases › Ataxia telangiectasia

Drugs in development · updated 30 October 2026

Ataxia telangiectasia: drugs in development

Rare diseasePrevalence 1-9 / 1 000 000 according to Orphanet · ORPHA:100

As of 30 October 2026 there are 3 active clinical trials (recruiting or ongoing, phases I–III) on 3 drugs for ataxia telangiectasia. 3 are experimental, that is not yet approved in the United States.

3Active trials
3Experimental drugs
1Experimental, in phase III

Summary updated every week · data as of 30 October 2026. In Phuturdex you get the full detail of every drug and an email when an alert is published on the ones you follow.

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Experimental drugs

Not yet approved by the US FDA for any indication.

DrugMost advanced phaseActive trialsReference trial
N-Acetyl-L-LeucineIII1NCT06673056
Nicotinamide ribosideNicotinamide ribonucleosideII1NCT04870866
TCC1727I–II1NCT07371663

Sorted by most advanced phase and number of active trials.

All 3 drugs, with the details

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Broader categories

Source: Phuturdex analysis of ClinicalTrials.gov data (U.S. National Library of Medicine), updated 30 October 2026. Active trials = recruiting or ongoing, phases I–III, with at least one identified drug; phase and sites refer to the trials on this disease. Disease name: Wikidata; NLM MeSH term: D001260 “Ataxia Telangiectasia”. Prevalence: Orphanet.

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