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

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ClinicalTrials.gov Identifier: NCT03196765
Recruitment Status : Withdrawn (No funding)
First Posted : June 23, 2017
Last Update Posted : March 7, 2019
Sponsor:
Information provided by (Responsible Party):
NeuroVia, Inc.

Tracking Information
First Submitted Date  ICMJE May 5, 2017
First Posted Date  ICMJE June 23, 2017
Last Update Posted Date March 7, 2019
Estimated Study Start Date  ICMJE August 2018
Estimated Primary Completion Date December 1, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (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  ICMJE Same as current
Change History Complete list of historical versions of study NCT03196765 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (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  ICMJE 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  ICMJE Safety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With Adrenoleukodystrophy
Official Title  ICMJE 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  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Intervention Model: Sequential Assignment
Intervention Model Description:
Sequential dose escalation
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE X-Linked Adrenoleukodystrophy
Intervention  ICMJE Drug: Sobetirome (NV1205)
Once a day oral dose of the study drug
Other Name: NV1205
Study Arms  ICMJE Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Withdrawn
Actual Enrollment  ICMJE
 (submitted: March 6, 2019)
0
Original Estimated Enrollment  ICMJE
 (submitted: June 21, 2017)
25
Estimated Study Completion Date  ICMJE December 1, 2020
Estimated Primary Completion Date December 1, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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  ICMJE
Sexes Eligible for Study: Male
Gender Based Eligibility: Yes
Ages  ICMJE 4 Years to 18 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Argentina,   Australia,   Chile,   Colombia,   France,   Russian Federation,   Ukraine,   United Kingdom
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03196765
Other Study ID Numbers  ICMJE 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  ICMJE
Plan to Share IPD: No
Responsible Party NeuroVia, Inc.
Study Sponsor  ICMJE NeuroVia, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: John Henderson, MD NeuroVia, Inc.
PRS Account NeuroVia, Inc.
Verification Date May 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP