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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)
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| MIN-102 | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| MIN-102 | Drug | Once-daily dosing with a volume specified by the pharmacokineteic specialist to achive the desired plasma exposure. Min-102 Oral suspension, strength 15mg/ml. |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Hernan Amartino | Bueno Aires | Buenos Aires | B16641NZ | Argentina | ||
| CHU Kremlin Bicêtre |
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| Paris |
| France |
| Universitätsmedizin Göttingen Georg-August-Universität | Göttingen | Germany |
| Universitätsklinikum Hamburg-Eppendorf Klinik und Poliklinik für Kinder- und Jugendmedizin | Hamburg | Germany |
| Hospital Sant Joan de Déu | Barcelona | Spain |