NCT04528706

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)

Trial Health

47
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
13

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Sep 2019

Typical duration for phase_2

Geographic Reach
4 countries

5 active sites

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

September 13, 2019

Completed
11 months until next milestone

First Submitted

Initial submission to the registry

July 31, 2020

Completed
27 days until next milestone

First Posted

Study publicly available on registry

August 27, 2020

Completed
3.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 31, 2023

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 31, 2023

Completed
Last Updated

July 18, 2023

Status Verified

January 1, 2023

Enrollment Period

4.3 years

First QC Date

July 31, 2020

Last Update Submit

July 17, 2023

Conditions

Cerebral Adrenoleukodystrophy

Outcome Measures

Primary Outcomes (2)

  • 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.

    6 months- 2 years

  • "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

    at 24 weeks and 96 weeks

Study Arms (1)

MIN-102

EXPERIMENTAL
Drug: MIN-102

Interventions

MIN-102DRUG

Once-daily dosing with a volume specified by the pharmacokineteic specialist to achive the desired plasma exposure. Min-102 Oral suspension, strength 15mg/ml.

MIN-102

Eligibility Criteria

Age2 Years - 12 Years
Sexmale
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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

You may not qualify if:

  • Previous HSCT
  • Too far progressed inflammatory brain lesions

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (5)

Hernan Amartino

Bueno Aires, Buenos Aires, B16641NZ, Argentina

Location

CHU Kremlin Bicêtre

Paris, France

Location

Universitätsmedizin Göttingen Georg-August-Universität

Göttingen, Germany

Location

Universitätsklinikum Hamburg-Eppendorf Klinik und Poliklinik für Kinder- und Jugendmedizin

Hamburg, Germany

Location

Hospital Sant Joan de Déu

Barcelona, Spain

Location

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 31, 2020

First Posted

August 27, 2020

Study Start

September 13, 2019

Primary Completion

December 31, 2023

Study Completion

December 31, 2023

Last Updated

July 18, 2023

Record last verified: 2023-01

Locations

FR(1)AR(1)ES(1)DE(2)