X-Linked Adrenoleukodystrophy
Conditions
Keywords
X-ALD
Brief summary
This is a phase I/II, open label dose escalation study of multiple dose levels of NV1205 with a long-term treatment phase.
Detailed description
This study is to evaluate the safety, pharmacokinetics, and efficacy of NV1205 in pediatric patients diagnosed with childhood cerebral adrenoleukodystrophy (CCALD). The study consists of: * Screening period: within 30 days of first dose * Main treatment period of 12 weeks (Part 1- from Screening to Week 12) * Long Term Treatment (LTT) period (Part 2- Week 13 through Week 96) In Part 1, subjects will have an initial 4-week treatment period at the assigned dose and, if no safety concerns are noted, subjects continue for another 8 weeks of extended safety assessment. There will be several cohorts of subjects enrolled. After each Cohort has completed the 4-week initial safety assessment, the safety data will be reviewed by an independent Data Safety Monitoring Board (DSMB) and, subject to DSMB recommendation, Cohort 2 will be enrolled and receive the next dose level. After Cohort 2 has completed 4 weeks of treatment, the DSMB will review all available safety data (Cohorts 1 and 2) and, subject to DSMB recommendation, Cohort 3 will be enrolled and receive the next dose level. Additional Cohorts may be enrolled at the recommendation of the DSMB with an incremental dose increase. In Part 2, subjects will continue to receive treatment in the LTT period of the study.
Interventions
DRUGSobetirome (NV1205)
Once a day oral dose of the study drug
Sponsors
NeuroVia, Inc.
Study design
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Sequential dose escalation
Eligibility
Sex/Gender
MALE
Age
4 Years to 18 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 | 2 years | Adverse events |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Area under the curve concentration of NV1205 in plasma | 12 weeks | A sparse sampling approach will be utilized to estimate a 24-hr area under the curve of NV1205 plasma concentration |
Other
| Measure | Time frame | Description |
|---|---|---|
| Loes score | 2 years | Percent change from baseline in brain lesions assessed as Loes score will be calculated |
Countries
Argentina, Australia, Chile, Colombia, France, Russia, Ukraine, United Kingdom
Outcome results
None listed