search
Back to results

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

Primary Purpose

Adrenoleukodystrophy

Status
Unknown status
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
MIN-102
Placebos
Sponsored by
Minoryx Therapeutics, S.L.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Adrenoleukodystrophy

Eligibility Criteria

18 Years - 65 Years (Adult, Older Adult)MaleDoes not accept healthy volunteers

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.

Sites / Locations

  • Stanford University Medical Center
  • Kennedy Krieger Institute
  • Massachusetts General Hospital
  • Hospital de la Pitié-Salpêtrière
  • Universitat Leipzig Klinik and Poliklinik für Neurologie
  • Institute of Genomic Medicine and Rare Disorders
  • Instituto Neurologico Carlo Besta
  • Academish Medisch Centrum
  • Hospital Universitari Vall d'Hebrón
  • National hospital for Neurology and Neurosurgery Charles Dent Metabolic Unit)

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

Placebo Comparator

Arm Label

Active

Placebo

Arm Description

Outcomes

Primary Outcome Measures

To evaluate the efficacy of MIN-102 on the progression of adrenomyeloneuropathy (AMN) in male patients as determined by a motor function test.

Secondary Outcome Measures

To evaluate the efficacy of MIN-102 in terms of patient reported outcomes.
SSPROM (Severity Score System for Progressive Myelopathy )
EDSS (Expanded Disability Status Scale )
Quality of life scales (Euroqol)
Incidence of cerebral inflammatory lesions

Full Information

First Posted
July 24, 2017
Last Updated
October 29, 2021
Sponsor
Minoryx Therapeutics, S.L.
search

1. Study Identification

Unique Protocol Identification Number
NCT03231878
Brief Title
A Clinical Study to Evaluate the Efficacy and Safety of MIN-102 (IMP) in Male AMN Patients.
Acronym
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
Study Type
Interventional

2. Study Status

Record Verification Date
November 2020
Overall Recruitment Status
Unknown status
Study Start Date
December 8, 2017 (Actual)
Primary Completion Date
June 25, 2021 (Actual)
Study Completion Date
June 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Minoryx Therapeutics, S.L.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No

5. 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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Adrenoleukodystrophy

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigator
Allocation
Randomized
Enrollment
105 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Active
Arm Type
Active Comparator
Arm Title
Placebo
Arm Type
Placebo Comparator
Intervention Type
Drug
Intervention Name(s)
MIN-102
Intervention Description
MIN-102 treatment
Intervention Type
Drug
Intervention Name(s)
Placebos
Intervention Description
Placebo
Primary Outcome Measure Information:
Title
To evaluate the efficacy of MIN-102 on the progression of adrenomyeloneuropathy (AMN) in male patients as determined by a motor function test.
Time Frame
in 96 weeks
Secondary Outcome Measure Information:
Title
To evaluate the efficacy of MIN-102 in terms of patient reported outcomes.
Time Frame
in 96 weeks
Title
SSPROM (Severity Score System for Progressive Myelopathy )
Time Frame
in 96 weeks
Title
EDSS (Expanded Disability Status Scale )
Time Frame
in 96 weeks
Title
Quality of life scales (Euroqol)
Time Frame
in 96 weeks
Title
Incidence of cerebral inflammatory lesions
Time Frame
in 96 weeks

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Facility Information:
Facility Name
Stanford University Medical Center
City
Stanford
State/Province
California
ZIP/Postal Code
94304
Country
United States
Facility Name
Kennedy Krieger Institute
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21205
Country
United States
Facility Name
Massachusetts General Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Facility Name
Hospital de la Pitié-Salpêtrière
City
Paris
Country
France
Facility Name
Universitat Leipzig Klinik and Poliklinik für Neurologie
City
Leipzig
Country
Germany
Facility Name
Institute of Genomic Medicine and Rare Disorders
City
Budapest
Country
Hungary
Facility Name
Instituto Neurologico Carlo Besta
City
Milano
Country
Italy
Facility Name
Academish Medisch Centrum
City
Amsterdam
Country
Netherlands
Facility Name
Hospital Universitari Vall d'Hebrón
City
Barcelona
Country
Spain
Facility Name
National hospital for Neurology and Neurosurgery Charles Dent Metabolic Unit)
City
London
Country
United Kingdom

12. IPD Sharing Statement

Learn more about this trial

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

We'll reach out to this number within 24 hrs