Back to resultsSafety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With Adrenoleukodystrophy
Primary Purpose
X-Linked Adrenoleukodystrophy
Status
Withdrawn
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
Sobetirome (NV1205)
Sponsored by
About this trial
This is an interventional treatment trial for X-Linked Adrenoleukodystrophy focused on measuring X-ALD
Eligibility Criteria
Inclusion Criteria:
- Males ≥4 years and <18 years of age
- CCALD diagnosis confirmed by genetic testing
- Loes score of >0 and ≤15
- 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
Sites / Locations
- Hospital Austral
- Hospital General de ninos Pedro de Elizalde
- Monash Health
- Hospital Clínico San Borja Arriarán
- Hospital Dr. Luis Calvo Mackenna
- Fundacion Cardioinfantil
- Hôpital Bicêtre - Paris Sud
- Endocrinology Research Center
- Moscow Morozov's Children Clinical Hospital
- Saint Petersburg State Pediatric Medical University
- National Children's Specialized Hospital 'OKHMATDET'
- Great Ormond Street Hospital for Children
- Manchester Children's Hospital
Outcomes
Primary Outcome Measures
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Adverse events
Secondary Outcome Measures
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
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT03196765
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)
Study Type
Interventional
2. Study Status
Record Verification Date
May 2018
Overall Recruitment Status
Withdrawn
Why Stopped
No funding
Study Start Date
August 2018 (Anticipated)
Primary Completion Date
December 1, 2020 (Anticipated)
Study Completion Date
December 1, 2020 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
NeuroVia, Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
X-Linked Adrenoleukodystrophy
Keywords
X-ALD
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Sequential Assignment
Model Description
Sequential dose escalation
Masking
None (Open Label)
Enrollment
0 (Actual)
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
Sobetirome (NV1205)
Other Intervention Name(s)
NV1205
Intervention Description
Once a day oral dose of the study drug
Primary Outcome Measure Information:
Title
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Description
Adverse events
Time Frame
2 years
Secondary Outcome Measure Information:
Title
Area under the curve concentration of NV1205 in plasma
Description
A sparse sampling approach will be utilized to estimate a 24-hr area under the curve of NV1205 plasma concentration
Time Frame
12 weeks
Other Pre-specified Outcome Measures:
Title
Loes score
Description
Percent change from baseline in brain lesions assessed as Loes score will be calculated
Time Frame
2 years
10. Eligibility
Sex
Male
Gender Based
Yes
Minimum Age & Unit of Time
4 Years
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Males ≥4 years and <18 years of age
CCALD diagnosis confirmed by genetic testing
Loes score of >0 and ≤15
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
John Henderson, MD
Organizational Affiliation
NeuroVia, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
Hospital Austral
City
Buenos Aires
Country
Argentina
Facility Name
Hospital General de ninos Pedro de Elizalde
City
Buenos Aires
Country
Argentina
Facility Name
Monash Health
City
Clayton
State/Province
Victoria
ZIP/Postal Code
3168
Country
Australia
Facility Name
Hospital Clínico San Borja Arriarán
City
Santiago
Country
Chile
Facility Name
Hospital Dr. Luis Calvo Mackenna
City
Santiago
Country
Chile
Facility Name
Fundacion Cardioinfantil
City
Bogotá
Country
Colombia
Facility Name
Hôpital Bicêtre - Paris Sud
City
Paris
Country
France
Facility Name
Endocrinology Research Center
City
Moscow
Country
Russian Federation
Facility Name
Moscow Morozov's Children Clinical Hospital
City
Moscow
Country
Russian Federation
Facility Name
Saint Petersburg State Pediatric Medical University
City
Saint Petersburg
Country
Russian Federation
Facility Name
National Children's Specialized Hospital 'OKHMATDET'
City
Kiev
Country
Ukraine
Facility Name
Great Ormond Street Hospital for Children
City
London
Country
United Kingdom
Facility Name
Manchester Children's Hospital
City
Manchester
Country
United Kingdom
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Safety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With Adrenoleukodystrophy
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