Trial Review

Registering a new trial?

To achieve prospective registration, we recommend submitting your trial for registration at the same time as ethics submission.

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 endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from ClinicalTrials.gov

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