A Clinical Study to Evaluate the Efficacy and Safety of MIN-102 (IMP) in Male AMN Patients.

Adrenoleukodystrophy Clinical Trial

— Advance  

Official title:

A Randomized, Double-blind, Placebo-controlled, Multinational, Multicenter Study With Open-label Treatment Extension to Assess the Effect of MIN-102 (IMP) on the Progression of Adrenomyeloneuropathy in Male Patients With X-linked Adrenoleukodystrophy

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03231878
• Other study ID #: MT-2-01
• Status: Active, not recruiting
• Phase: Phase 2/Phase 3
• Start date: December 8, 2017
• Est. completion date: June 2025

Study information

• Verified date: November 2023
• Source: Minoryx Therapeutics, S.L.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This is a Phase II/III, randomized, double-blind, placebo-controlled, multicenter, two parallel-group study in male patients with the AMN phenotype of X-linked adrenoleukodystrophy (X-ALD) to assess the efficacy and safety of MIN-102 treatment. Study sites will consist of specialist referral centers experienced in the management of adrenoleukodystrophy (ALD).

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 105
• Est. completion date: June 2025
• Est. primary completion date: June 25, 2021
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 18 Years to 65 Years

Eligibility

Inclusion Criteria:

• Male and between 18-65 years of age.

• Diagnosed with X-linked adrenoleukodystrophy (X-ALD) based on elevated VLCFA and genetic testing.

• Clinical evidence of spinal cord involvement.

Exclusion Criteria:

• Any other chronic neurological disease with signs of spastic paraplegia, such as hereditary spastic paraplegia, multiple sclerosis, etc.

• Presence of inflammatory (Gd-enhancing) MRI lesions or any abnormality other than those mentioned in the inclusion criteria.

• Known type 1 or type 2 diabetes.

• Known intolerance to pioglitazone or any other thiazolidinedione.

• Taking or have taken honokiol, pioglitazone or other thiazolidinediones within the 6 months prior to screening.

• Previous bone marrow transplantation.

• Previous or current history of cancer (other than treated basal cell carcinoma).

• Previous or current history of congestive heart failure.

Related Conditions & MeSH terms

• Adrenoleukodystrophy

Intervention

Drug:
• MIN-102
MIN-102 treatment
• Placebos
Placebo

Locations

Country	Name	City	State
France	Hospital de la Pitié-Salpêtrière	Paris
Germany	Universitat Leipzig Klinik and Poliklinik für Neurologie	Leipzig
Hungary	Institute of Genomic Medicine and Rare Disorders	Budapest
Italy	Instituto Neurologico Carlo Besta	Milano
Netherlands	Academish Medisch Centrum	Amsterdam
Spain	Hospital Universitari Vall d'Hebrón	Barcelona
United Kingdom	National hospital for Neurology and Neurosurgery Charles Dent Metabolic Unit)	London
United States	Kennedy Krieger Institute	Baltimore	Maryland
United States	Massachusetts General Hospital	Boston	Massachusetts
United States	Stanford University Medical Center	Stanford	California

Sponsors (1)

Lead Sponsor	Collaborator
Minoryx Therapeutics, S.L.

Countries where clinical trial is conducted

United States,  France,  Germany,  Hungary,  Italy,  Netherlands,  Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	To evaluate the efficacy of MIN-102 on the progression of adrenomyeloneuropathy (AMN) in male patients as determined by a motor function test.		in 96 weeks
Secondary	To evaluate the efficacy of MIN-102 in terms of patient reported outcomes.		in 96 weeks
Secondary	SSPROM (Severity Score System for Progressive Myelopathy )		in 96 weeks
Secondary	EDSS (Expanded Disability Status Scale )		in 96 weeks
Secondary	Quality of life scales (Euroqol)		in 96 weeks
Secondary	Incidence of cerebral inflammatory lesions		in 96 weeks