Safety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With Adrenoleukodystrophy

• Status: Withdrawn
• Trial end date: 2020-12-01
• Target enrollment: 0
• Participant gender: 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 2
• Accepts Healthy Volunteers?: No

Details

• 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