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

• ClinicalTrials.gov Identifier: NCT03196765
• Recruitment Status: Withdrawn
• First Posted: June 23, 2017
• Last Update Posted: March 7, 2019
• Sponsor: NeuroVia, Inc.
• Information provided by (Responsible Party): NeuroVia, Inc.

Tracking Information

• First Submitted Date: May 5, 2017
• First Posted Date: June 23, 2017
• Last Update Posted Date: March 7, 2019
• Estimated Study Start Date: August 2018
• Estimated Primary Completion Date: December 1, 2020 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: June 21, 2017)

Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 [ Time Frame: 2 years ]

Adverse events

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: June 21, 2017)

Area under the curve concentration of NV1205 in plasma [ Time Frame: 12 weeks ]

A sparse sampling approach will be utilized to estimate a 24-hr area under the curve of NV1205 plasma concentration

• Original Secondary Outcome Measures: Same as current

Current Other Pre-specified Outcome Measures (submitted: June 21, 2017)

Loes score [ Time Frame: 2 years ]

Percent change from baseline in brain lesions assessed as Loes score will be calculated

• Original Other Pre-specified Outcome Measures: Same as current

Descriptive Information

• Brief Title: Safety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With Adrenoleukodystrophy
• Official Title: Phase I/II, Open-Label, Dose Escalation Study of the Safety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With Childhood Cerebral Adrenoleukodystrophy (CCALD)
• 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.

• Study Type: Interventional
• Study Phase: Phase 1; Phase 2

Study Design

Intervention Model: Sequential Assignment
Intervention Model Description:

Sequential dose escalation

Masking: None (Open Label)
Primary Purpose: Treatment

• Condition: X-Linked Adrenoleukodystrophy

Intervention

Drug: Sobetirome (NV1205)

Once a day oral dose of the study drug

Other Name: NV1205

• Study Arms: Not Provided
• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Withdrawn
• Actual Enrollment (submitted: March 6, 2019): 0
• Original Estimated Enrollment (submitted: June 21, 2017): 25
• Estimated Study Completion Date: December 1, 2020
• Estimated Primary Completion Date: December 1, 2020 (Final data collection date for primary outcome measure)

Eligibility 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

Sex/Gender

• Sexes Eligible for Study: Male
• Gender Based Eligibility: Yes

• Ages: 4 Years to 18 Years (Child, Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Argentina, Australia, Chile, Colombia, France, Russian Federation, Ukraine, United Kingdom

Administrative Information

• NCT Number: NCT03196765
• Other Study ID Numbers: NV1205-009
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

• Responsible Party: NeuroVia, Inc.
• Study Sponsor: NeuroVia, Inc.
• Collaborators: Not Provided

Investigators

• Study Director: John Henderson, MD; NeuroVia, Inc.

• PRS Account: NeuroVia, Inc.
• Verification Date: May 2018