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

Sponsor
Minoryx Therapeutics, S.L. (Industry)
Overall Status
Active, not recruiting
CT.gov ID
NCT03231878
Collaborator
(none)
105
Enrollment
10
Locations
2
Arms
65.7
Anticipated Duration (Months)
10.5
Patients Per Site
0.2
Patients Per Site Per Month

Study Details

Study Description

Brief 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).

Condition or Disease Intervention/Treatment Phase
Phase 2/Phase 3

Study Design

Study Type:
Interventional
Anticipated Enrollment :
105 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
Triple (Participant, Care Provider, Investigator)
Primary Purpose:
Treatment
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
Actual Study Start Date :
Dec 8, 2017
Actual Primary Completion Date :
Jun 25, 2021
Anticipated Study Completion Date :
Jun 1, 2023

Arms and Interventions

Arm Intervention/Treatment
Active Comparator: Active

Drug: MIN-102
MIN-102 treatment
Placebo Comparator: Placebo

Drug: Placebos
Placebo

Outcome Measures

Primary Outcome Measures

  1. 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 Outcome Measures

  1. To evaluate the efficacy of MIN-102 in terms of patient reported outcomes. [in 96 weeks]

  2. SSPROM (Severity Score System for Progressive Myelopathy ) [in 96 weeks]

  3. EDSS (Expanded Disability Status Scale ) [in 96 weeks]

  4. Quality of life scales (Euroqol) [in 96 weeks]

  5. Incidence of cerebral inflammatory lesions [in 96 weeks]

Eligibility Criteria

Criteria

Ages Eligible for Study:
18 Years to 65 Years
Sexes Eligible for Study:
Male
Accepts Healthy Volunteers:
No
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.

Contacts and Locations

Locations

Site City State Country Postal Code
1 Stanford University Medical Center Stanford California United States 94304
2 Kennedy Krieger Institute Baltimore Maryland United States 21205
3 Massachusetts General Hospital Boston Massachusetts United States 02114
4 Hospital de la Pitié-Salpêtrière Paris France
5 Universitat Leipzig Klinik and Poliklinik für Neurologie Leipzig Germany
6 Institute of Genomic Medicine and Rare Disorders Budapest Hungary
7 Instituto Neurologico Carlo Besta Milano Italy
8 Academish Medisch Centrum Amsterdam Netherlands
9 Hospital Universitari Vall d'Hebrón Barcelona Spain
10 National hospital for Neurology and Neurosurgery Charles Dent Metabolic Unit) London United Kingdom

Sponsors and Collaborators

  • Minoryx Therapeutics, S.L.

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
Minoryx Therapeutics, S.L.
ClinicalTrials.gov Identifier:
NCT03231878
Other Study ID Numbers:
  • MT-2-01
First Posted:
Jul 27, 2017
Last Update Posted:
Nov 1, 2021
Last Verified:
Nov 1, 2020
Studies a U.S. FDA-regulated Drug Product:
Yes
Studies a U.S. FDA-regulated Device Product:
No
Additional relevant MeSH terms:
Adrenoleukodystrophy

Study Results

No Results Posted as of Nov 1, 2021