A Clinical Study in Male Pediatric Patients With Cerebral X-linked Adrenoleukodystrophy (Cald) to Assess the Effects of MIN-102 Treatment on Disease Progression Prior to Human Stem Cell Transplant (HSCT)

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Brief Title

A Clinical Study in Male Pediatric Patients With Cerebral X-linked Adrenoleukodystrophy (Cald) to Assess the Effects of MIN-102 Treatment on Disease Progression Prior to Human Stem Cell Transplant (HSCT)

Official Title

An Open-label, Multicenter Study in Male Pediatric Patients With Cerebral X-linked Adrenoleukodystrophy (Cald) to Assess the Effects of MIN-102 Treatment on Disease Progression Prior to Human Stem Cell Transplant (HSCT)

Brief Summary

      An Open-Label, multicenter study in male pediatric patients with cerebral x-linked
      adrenoleukodystrophy (cald) to assess the effects of MIN-102 treatment on disease progression
      prior to human stem cell transplant (HSCT)
    


Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

evaluate whether MIN-102 can halt disease progression of cALD if administered prior to hematopoietic stem-cell transplantation (HSCT), as determined by serial clinical and MRI investigations in pediatric subjects.


Condition

Cerebral Adrenoleukodystrophy

Intervention

MIN-102

Study Arms / Comparison Groups

 MIN-102
Description:  

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

13

Start Date

September 13, 2019

Completion Date

February 28, 2021

Primary Completion Date

November 30, 2020

Eligibility Criteria

        Inclusion Criteria:

          -  Males aged ≥2 and ≤12 years with a diagnosis of X-linked ALD

          -  White matter involvement as determined by cerebral MRI lesions without Gd enhancement
             at baseline (Population 1), or with Gd enhancement at baseline (Population 2).

          -  Major Functional Disabilities (MFD) score of 0.

          -  Baseline Loes score >0 and ≤10

          -  Gadolinium Intensity Score >2

        Exclusion Criteria:

          -  Previous HSCT

          -  Too far progressed inflammatory brain lesions
      

Gender

Male

Ages

2 Years - 12 Years

Accepts Healthy Volunteers

No

Contacts

, +34 93 544 14 66, [email protected]

Location Countries

France

Location Countries

France

Administrative Informations


NCT ID

NCT04528706

Organization ID

MT-2-02


Responsible Party

Sponsor

Study Sponsor

Minoryx Therapeutics, S.L.


Study Sponsor

, , 


Verification Date

July 2020