UnknownPhase 2

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)

Argentina, France, Germany13 participantsStarted 2019-09-13

Plain-language 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)

Who can participate

Age range2 Years – 12 Years

SexMALE

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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

What they're measuring

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.

Timeframe: 6 months- 2 years

"arrested disease" will be assessed at 24 weeks and at 96 weeks

Timeframe: at 24 weeks and 96 weeks

Trial details

NCT IDNCT04528706

SponsorMinoryx Therapeutics, S.L.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2023-12-31

View on ClinicalTrials.gov More Cerebral Adrenoleukodystrophy trials