Overview
Safety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With Adrenoleukodystrophy
Status:
Withdrawn
Withdrawn
Trial end date:
2020-12-01
2020-12-01
Target enrollment:
0
0
Participant gender:
Male
Male
Summary
This is a phase I/II, open label dose escalation study of multiple dose levels of NV1205 with a long-term treatment phase.Phase:
Phase 1/Phase 2Accepts Healthy Volunteers?
NoDetails
Lead Sponsor:
NeuroVia, Inc.
Criteria
Inclusion Criteria:1. Males ≥4 years and <18 years of age
2. CCALD diagnosis confirmed by genetic testing
3. Loes score of >0 and ≤15
4. Patients on VLCFA lowering agents such as Lorenzo's oil must stop the medication and
have high VLCFA levels before enrollment
Exclusion Criteria:
- Significant medical conditions such as heart, thyroid, or liver disease
- HSCT recipients