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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT03196765




Registration number
NCT03196765
Ethics application status
Date submitted
5/05/2017
Date registered
23/06/2017
Date last updated
7/03/2019

Titles & IDs
Public title
Safety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With Adrenoleukodystrophy
Scientific 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)
Secondary ID [1] 0 0
NV1205-009
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
X-Linked Adrenoleukodystrophy 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Other neurological disorders
Metabolic and Endocrine 0 0 0 0
Metabolic disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Sobetirome (NV1205)

Treatment: Drugs: Sobetirome (NV1205)
Once a day oral dose of the study drug
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Timepoint [1] 0 0
2 years
Secondary outcome [1] 0 0
Area under the curve concentration of NV1205 in plasma
Timepoint [1] 0 0
12 weeks

Eligibility
Key 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
Minimum age
4 Years
Maximum age
18 Years
Sex
Males
Can healthy volunteers participate?
No
Key exclusion criteria
- Significant medical conditions such as heart, thyroid, or liver disease

- HSCT recipients

Study design
Purpose of the study
Treatment
Allocation to intervention
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Other
Other design features
Phase
Phase 1/Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Withdrawn
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
1/08/2018
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/12/2020
Actual
Sample size
Target
Accrual to date
Final
0
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Monash Health - Clayton
Recruitment postcode(s) [1] 0 0
3168 - Clayton
Recruitment outside Australia
Country [1] 0 0
Argentina
State/province [1] 0 0
Buenos Aires
Country [2] 0 0
Chile
State/province [2] 0 0
Santiago
Country [3] 0 0
Colombia
State/province [3] 0 0
Bogotá
Country [4] 0 0
France
State/province [4] 0 0
Paris
Country [5] 0 0
Russian Federation
State/province [5] 0 0
Moscow
Country [6] 0 0
Russian Federation
State/province [6] 0 0
Saint Petersburg
Country [7] 0 0
Ukraine
State/province [7] 0 0
Kiev
Country [8] 0 0
United Kingdom
State/province [8] 0 0
London
Country [9] 0 0
United Kingdom
State/province [9] 0 0
Manchester

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
NeuroVia, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT03196765
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
John Henderson, MD
Address 0 0
NeuroVia, Inc.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT03196765