Effect of hypoxia on FRDA patients
Research type
Research Study
Full title
A preliminary investigation into the impacts of normobaric hypoxia on neuromuscular and cardiac function in Friedreich’s Ataxia.
IRAS ID
229069
Contact name
Hugh Montgomery
Contact email
Sponsor organisation
University College London
Clinicaltrials.gov Identifier
Z6364106/2019/10/117, UCL Data Protection
Duration of Study in the UK
0 years, 4 months, 1 days
Research summary
In this open-label pilot, we shall explore the effects of controlled hypoxia on individuals diagnosed with FRDA. Using established, safe protocols, our participants will be exposed to progressively lower levels of oxygen in a normobaric hypoxic chamber over the course of five days. Subjects will be studied in the week prior to, and as soon as feasible after, hypoxic exposure. If an effect of hypoxic exposure is identified, we may repeat the studies at 3 months to identify a washout effect. The investigations have been chosen with the primary aim of demonstrating a beneficial effect on mitochondrial function in vivo in clinically affected tissues, with exploratory investigations (i) seeking a signal for an associated clinically relevant improvement in neuromuscular function and metabolic profile (urine and blood) and (ii) assessing related changes in in vitro measures of mitochondrial function. This is the first such trial of hypoxia in FRDA patients which could, should our data prove positive, pave the way to the first effective disease-modifying therapy this incurable condition.
REC name
London - Westminster Research Ethics Committee
REC reference
20/LO/0227
Date of REC Opinion
18 May 2020
REC opinion
Further Information Favourable Opinion