A Study to Learn More About the Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich's Ataxia Who Are Prescribed it for the First Time

ClinicalTrials.gov ID NCT06623890.  Last Update Posted 2025-03-04

In this study, researchers will learn more about the safety of BIIB141, also known as omaveloxolone or SKYCLARYS®. This is a drug available for doctors to prescribe for people with Friedreich's Ataxia, also known as FA. This is known as an "observational" study, which collects health information about study participants without changing their medical care. Participants for this study will be found using a group called the Friedreich's Ataxia Global Clinical Consortium (FA GCC). This is a group of study research centers that help provide clinical care for people with FA.

The primary objective of this study is to assess the long-term safety of omaveloxolone as prescribed to participants with FA in the real-world setting, including characterization of all drug-induced liver injury (DILI) and congestive heart failure (CHF) AEs. The secondary objective of this study is to capture the reasons and timing of omaveloxolone treatment interruptions, discontinuations, and drug overdose.

Design Therapeutics Highlights Progress Across Lead GeneTAC® Programs and Reports Fourth Quarter and Full Year 2024 Financial Results

CARLSBAD, Calif., March 10, 2025 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc.. Friedreich Ataxia (FA) Design has initiated dosing in a Phase 1 clinical trial in healthy volunteers in Australia to evaluate the safety and pharmacokinetics (PK) of single ascending doses of DT-216P2 via multiple routes of administration (intravenous infusion, subcutaneous infusion and subcutaneous injection). A Phase 1/2 multiple ascending dose (MAD) clinical trial to assess safety, PK and pharmacodynamics (PD) in FA patients is anticipated to begin in mid-2025. Data based on twelve weeks of DT-216P2 dosing in patients is anticipated in 2026.

Autosomal Recessive Ataxias in Northeast Brazil: A Regional Multicenter Case Series

Camelo-Filho, A.E., da Rosa, R.F., Lima, P.L.G.S.B. et al. Autosomal Recessive Ataxias in Northeast Brazil: A Regional Multicenter Case Series. Cerebellum 24, 59 (2025). Doi:10.1007/s12311-025-01814-1

Patients underwent clinical evaluations, including neurological examinations and functional assessments. Results: Friedreich’s ataxia (FRDA) was the most prevalent diagnosis, accounting for 12 cases (21%), followed by Ataxia-Telangiectasia (A-T) with (N = 9; 15.8%) and Niemann-Pick Disease Type C (NPC) (N = 9; 15.8%). Metabolic disorders, including Cerebrotendinous Xanthomatosis (N = 6;10.5%) were also common causes.