Back to resultsA 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) (NEXUS)
Primary Purpose
Cerebral Adrenoleukodystrophy
Status
Active
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
MIN-102
Sponsored by
About this trial
This is an interventional treatment trial for Cerebral Adrenoleukodystrophy
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
Sites / Locations
- Hernan Amartino
- CHU Kremlin Bicêtre
- Universitätsmedizin Göttingen Georg-August-Universität
- Universitätsklinikum Hamburg-Eppendorf Klinik und Poliklinik für Kinder- und Jugendmedizin
- Hospital Sant Joan de Déu
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
MIN-102
Arm Description
Outcomes
Primary Outcome Measures
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.
"arrested disease" will be assessed at 24 weeks and at 96 weeks
arrested disease is defined using: Cahnge in NFS from Baseline, Free of MFD and lack of lesion progression on MRI
Secondary Outcome Measures
Full Information
NCT ID
NCT04528706
First Posted
July 31, 2020
Last Updated
July 17, 2023
Sponsor
Minoryx Therapeutics, S.L.
1. Study Identification
Unique Protocol Identification Number
NCT04528706
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)
Acronym
NEXUS
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)
Study Type
Interventional
2. Study Status
Record Verification Date
January 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
September 13, 2019 (Actual)
Primary Completion Date
December 31, 2023 (Anticipated)
Study Completion Date
December 31, 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
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
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)
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cerebral Adrenoleukodystrophy
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
13 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
MIN-102
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
MIN-102
Intervention Description
Once-daily dosing with a volume specified by the pharmacokineteic specialist to achive the desired plasma exposure. Min-102 Oral suspension, strength 15mg/ml.
Primary Outcome Measure Information:
Title
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.
Time Frame
6 months- 2 years
Title
"arrested disease" will be assessed at 24 weeks and at 96 weeks
Description
arrested disease is defined using: Cahnge in NFS from Baseline, Free of MFD and lack of lesion progression on MRI
Time Frame
at 24 weeks and 96 weeks
10. Eligibility
Sex
Male
Minimum Age & Unit of Time
2 Years
Maximum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
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
Facility Information:
Facility Name
Hernan Amartino
City
Bueno Aires
State/Province
Buenos Aires
ZIP/Postal Code
B16641NZ
Country
Argentina
Facility Name
CHU Kremlin Bicêtre
City
Paris
Country
France
Facility Name
Universitätsmedizin Göttingen Georg-August-Universität
City
Göttingen
Country
Germany
Facility Name
Universitätsklinikum Hamburg-Eppendorf Klinik und Poliklinik für Kinder- und Jugendmedizin
City
Hamburg
Country
Germany
Facility Name
Hospital Sant Joan de Déu
City
Barcelona
Country
Spain
12. IPD Sharing Statement
Learn more about this trial
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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