A Phase 1/2 interventional study of Sobetirome (NV1205) in X-Linked Adrenoleukodystrophy, sponsored by NeuroVia, Inc.. Withdrawn at 13 sites in 8 countries. Open to male participants aged 4 Years to 18 Years. Per ClinicalTrials.gov, last updated 2019-03-07.
Sponsored by NeuroVia, Inc. · Phase 1/2, Interventional, and Treatment
This is a phase I/II, open label dose escalation study of multiple dose levels of NV1205 with a long-term treatment phase.
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:
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.
Exclusion Criteria:
Once a day oral dose of the study drug
Also known as: NV1205
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Adverse events
Time frame: 2 years
Area under the curve concentration of NV1205 in plasma
A sparse sampling approach will be utilized to estimate a 24-hr area under the curve of NV1205 plasma concentration
Time frame: 12 weeks
Loes score
Percent change from baseline in brain lesions assessed as Loes score will be calculated
Time frame: 2 years
Plan to share: No
No publications or documents are linked to this record.
This study is withdrawn, as verified in May 2018. You cannot join it, but the record below documents what was studied.
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NeuroVia, Inc.