A Randomized, Double-Blind, Placebo-Controlled Study of NIDO-361 in Patients with SBMA
Research type
Research Study
Full title
A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of NIDO-361 in Patients with Spinal and Bulbar Muscular Atrophy (SBMA)
IRAS ID
1008791
Contact name
Vissia Viglietta
Contact email
Sponsor organisation
Nido Biosciences, Inc.
Research summary
Spinal and Bulbar Muscular Atrophy (SBMA) is a genetic disorder that causes progressive weakening and wasting of the muscles, particularly the arms and legs.
Despite the substantial decline on quality of life this progressive disease causes, currently there are no curative treatments.
This study will investigate a new medicine, NIDO-361, which has the potential to reverse the effects of muscle weakening that SBMA causes.
Patients with SBMA have a mutated androgen receptor gene which in turn creates a mutated androgen receptor protein. This leads to toxic increased function of the protein and ultimately to disease progression.
NIDO-361 works by binding to the mutated androgen receptor protein, which allows it to resume its normal function.Participants taking part in this trial will receive either 100 mg of NIDO-361 or a placebo, which they will take orally, every day for 12 months. The placebo will look like the study drug but will not contain any active drug and will be used to determine if taking the study drug is better than taking nothing at all. Neither the participant nor the study doctor will know whether the participant has been assigned to receive the study drug or the placebo. Participant will have a 50% chance of receiving the study drug ad a 50% chance of receiving the placebo.
The main objectives of this study are to find out if the study drug can help restore muscle volume and to determine the safety of the study drug in patients with SBMA.
Approximately 54 participants will be enrolled in this study. For each participants the study will last for approximately 13.5 months. This is composed of a 28-day screening period, a 12-month treatment period, and a 2-week follow-up period.
REC name
East of England - Cambridge East Research Ethics Committee
REC reference
23/EE/0237
Date of REC Opinion
14 Nov 2023
REC opinion
Further Information Favourable Opinion