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

This study is not yet open for participant recruitment.
Verified October 2017 by NeuroVia, Inc.
Sponsor:
ClinicalTrials.gov Identifier:
NCT03196765
First Posted: June 23, 2017
Last Update Posted: October 18, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Information provided by (Responsible Party):
NeuroVia, Inc.
May 5, 2017
June 23, 2017
October 18, 2017
November 1, 2017
December 1, 2020   (Final data collection date for primary outcome measure)
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 [ Time Frame: 2 years ]
Adverse events
Same as current
Complete list of historical versions of study NCT03196765 on ClinicalTrials.gov Archive Site
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
Same as current
Loes score [ Time Frame: 2 years ]
Percent change from baseline in brain lesions assessed as Loes score will be calculated
Same as current
 
Safety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With Adrenoleukodystrophy
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)
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:

  • 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.

Interventional
Phase 1
Phase 2
Intervention Model: Sequential Assignment
Intervention Model Description:
Sequential dose escalation
Masking: None (Open Label)
Primary Purpose: Treatment
X-Linked Adrenoleukodystrophy
Drug: Sobetirome
Once a day oral dose of the study drug
Other Name: NV1205
Not Provided
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Not yet recruiting
25
December 1, 2020
December 1, 2020   (Final data collection date for primary outcome measure)

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 b. 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
Sexes Eligible for Study: Male
Gender Based Eligibility: Yes
4 Years to 18 Years   (Child, Adult)
No
Contact: Program Director 415-870-6967 patients@neurovia-inc.com
Not Provided
Australia
 
NCT03196765
NV1205-009
Yes
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Plan to Share IPD: No
NeuroVia, Inc.
NeuroVia, Inc.
Not Provided
Study Director: Masoud Mokhtarani, MD NeuroVia, Inc.
NeuroVia, Inc.
October 2017

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