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NV1205 in Pediatric Male Subjects With Childhood Cerebral Adrenoleukodystrophy
NCT03196765Phase 2
Key facts
- Status
- Withdrawn
- Start
- Aug 2018
- Primary completion
- Dec 2020
- Countries
- 8
- Sponsor
- NeuroVia
- Modality
- Small Molecules
- Drugs
- sobetirome
- Conditions
- cerebral adrenoleukodystrophy (CALD)
01
Overview
Phase 2, open-label, single-arm trial of sobetirome in cerebral adrenoleukodystrophy (CALD), sponsored by NeuroVia. Primary endpoint: Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 at 2 years. Planned start Aug 2018; status: withdrawn.
02
Design
1 arm- Study design
- Non-randomizedOpen-label
NV1205 Dose Escalation
Oral sobetirome (NV1205) at escalating dose levels
03
Who it enrols
Male patients 4-18 years old with confirmed CCALD diagnosis and Loes score >0 and ≤15. Patients must stop VLCFA lowering agents like Lorenzo's oil. HSCT recipients and those with significant heart, thyroid, or liver disease are excluded.
04
Endpoints
Primary
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Secondary
Area under the curve concentration of NV1205 in plasma
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