Effect of MIN-102 in Male Patients with Adrenomyeloneuropathy
Research type
Research Study
Full title
A Randomized, Double-Blind, Placebo-Controlled, Multinational, Multicenter Study With Open-Label Treatment Extension to Assess The Effect Of MIN-102 On The Progression Of Adrenomyeloneuropathy In Male Patients With X-Linked Adrenomyeloneuropathy
IRAS ID
228718
Contact name
Robin Henry Lachmann
Contact email
Sponsor organisation
Minoryx Therapeutics S.L.
Eudract number
2017-000748-16
Duration of Study in the UK
3 years, 0 months, 1 days
Research summary
Adrenoleukodystrophy (ALD) is a rare, inherited disorder, seen mainly in males, that affects the brain, nerves and adrenal glands. The gene mutation in ALD results in impaired breakdown of very-long chain fatty acids (VLCFA), which then accumulate in tissues. This leads to a number of different disease manifestations. In boys with ALD, the build-up of VLCFA the brain leads to inflammation and destruction of the myelin protective sheath, which insulates the nerves (leukodystrophy). This form of ALD is a rapidly progressive and life-threatening neurodegenerative disorder which normally starts in childhood. However, not all patients with mutations in the ALD gene develop leukodystrophy. Most men and some women will instead develop adrenomyeloneuropathy (AMN) progressive gait disturbance, due to stiffness and weakness of the legs, impaired sensory function, incontinence and impotence. This is also a progressive disease, and most patients will end up in a wheelchair, although this takes many years. Any patient with an ALD mutation can develop adrenal failure (Addison’s disease) and require steroid hormone replacement.
The target population of this trial are adult male patients aged 18-65 with AMN. The study will evaluate the effectiveness of the drug MIN-102 in limiting the progression of AMN. MIN-102 will be evaluated by comparing it to placebo (a solution that looks like the real drug but contains no active ingredient). Part 1 is “randomised, double blind”: this means that participants will be assigned by chance to receive either the study drug or placebo. Participants will have a two in three chance of receiving the study drug and a one in three chance of receiving the placebo. Neither the participant nor the study doctor will know which group has been assigned.
The study will take place at approximately 10 sites around the world, enrolling 105 participants. Participation in the study will last between 100 and 104 weeks; up to 4 weeks in the screening period, 96 weeks of treatment and 4 weeks of follow-up. The main outcome measures will look at patient’s balance and their walking. They will also undergo a range of safety assessments, imaging and blood and urine tests. When participants complete the 96 weeks of treatment they will be invited to participate in an open-label extension study in which all participants will receive MIN-102.REC name
East of England - Cambridge South Research Ethics Committee
REC reference
17/EE/0303
Date of REC Opinion
14 Sep 2017
REC opinion
Further Information Favourable Opinion