Hereditary cerebellar ataxia
MONDO:0100310Cerebellar ataxia that is transmitted from parent to child.
Also known as: cerebellar hereditary ataxia, hereditary cerebellar ataxia
132 clinical trials for this condition and its sub-types.
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Broader categories
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Experimental Friedreich's ataxia drug tested in kids – but trial halted early
Disease control Stopped earlyThis early-stage trial tested a drug called nomlabofusp (CTI-1601) in 18 adolescents and children with Friedreich's ataxia, a rare genetic disease that affects movement and coordination. The goal was to check safety and how the body processes the drug. However, the study was term…
Phase 1 • Sponsor: Larimar Therapeutics, Inc. • Aim: Disease control
Last updated Jul 12, 2026 00:00 UTC
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Continued EryDex treatment studied in rare neurological disorder
Disease control Stopped earlyThis study offered continued treatment with EryDex to 101 people with ataxia telangiectasia (A-T) who had finished a previous trial. The main goal was to monitor safety, including side effects and serious events. The study was terminated early, and it did not aim to cure the dise…
Phase 3 • Sponsor: Quince Therapeutics S.p.A. • Aim: Disease control
Last updated Jun 27, 2026 12:29 UTC
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Experimental drug shows promise for rare genetic disorder
Disease control Stopped earlyThis Phase II trial tested a drug called N-Acetyl-L-Leucine (IB1001) in 17 people with Ataxia-Telangiectasia, a rare genetic disease that affects movement and immunity. The study aimed to see if the drug could improve symptoms and slow the disease over time. The trial was termina…
Phase 2 • Sponsor: IntraBio Inc • Aim: Disease control
Last updated Jun 27, 2026 12:00 UTC
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Virus therapy fails to advance in colorectal cancer trial
Disease control Stopped earlyThis study tested a new approach using a virus that attacks cancer cells (oncolytic immunotherapy) along with two standard drugs (atezolizumab and bevacizumab) in people with advanced colorectal cancer that had stopped responding to other treatments. The trial was stopped early a…
Phase 2 • Sponsor: Replimune, Inc. • Aim: Disease control
Last updated Jun 27, 2026 09:01 UTC
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Experimental cell therapy targets deadly childhood brain cancer
Disease control Stopped earlyThis early-phase trial tested a new immunotherapy approach for children with DIPG, a rare and aggressive brain stem tumor. After standard radiation and chemotherapy, patients received special vaccines and immune cells designed to attack the tumor. The study was small (11 particip…
Phase 1 • Sponsor: University of Florida • Aim: Disease control
Last updated Jun 27, 2026 07:55 UTC