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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02255435




Registration number
NCT02255435
Ethics application status
Date submitted
30/09/2014
Date registered
2/10/2014

Titles & IDs
Public title
RTA 408 Capsules in Patients With Friedreich's Ataxia - MOXIe
Scientific title
A Phase 2 Study of the Safety, Efficacy, and Pharmacodynamics of RTA 408 in the Treatment of Friedreich's Ataxia (MOXIe)
Secondary ID [1] 0 0
2015-002762-23
Secondary ID [2] 0 0
RTA 408-C-1402
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Friedreich Ataxia 0 0
Condition category
Condition code
Neurological 0 0 0 0
Other neurological disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Neurodegenerative diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Experimental: Part 1 Omaveloxolone Capsules 2.5 and 5 mg - omaveloxolone (RTA 408) Capsules, 2.5 mg administered orally one daily for 2 weeks, then 5 mg taken orally once daily for 10 weeks

Experimental: Part 1 Omaveloxolone Capsules 10 mg - omaveloxolone (RTA 408) Capsules, 10 mg administered orally once daily for 12 weeks

Experimental: Part 1 Omaveloxolone Capsules 20 mg - Omaveloxolone (RTA 408) Capsules, 20 mg administered orally once daily for 12 weeks

Experimental: Part 1 Omaveloxolone Capsules 40 mg - Omaveloxolone (RTA 408) Capsules, 40 mg administered orally once daily for 12 weeks

Experimental: Part 1 Omaveloxolone Capsules 80 mg - Omaveloxolone (RTA 408) Capsules, 80 mg administered orally once daily for 12 weeks

Experimental: Part 1 Omaveloxolone Capsules 160 mg - Omaveloxolone (RTA 408) Capsules, 160 mg administered orally once daily for 12 weeks

Experimental: Part 1 Omaveloxolone Capsules 300 mg - Omaveloxolone (RTA 408) Capsules, 300 mg administered orally once daily for 12 weeks

Placebo comparator: Part 1 Placebo Capsules - Placebo capsules administered orally once daily for 12 weeks

Placebo comparator: Part 2 Placebo Capsules - Placebo capsules administered orally once daily for 48 weeks

Experimental: Part 2 Omaveloxolone Capsules 150 mg - Omaveloxolone (RTA 408) Capsules, 150 mg administered orally once daily for 48 weeks
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change From Baseline in Peak Work (in Watts/kg) During Exercise Testing at Week 12 in Part 1
Timepoint [1] 0 0
Baseline through 12 weeks after participant receives the first dose in Part 1.
Primary outcome [2] 0 0
Change in the Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 48 in Part 2
Timepoint [2] 0 0
48 weeks after participant receives the first dose in Part 2
Secondary outcome [1] 0 0
Change in the Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 12 in Part 1
Timepoint [1] 0 0
12 weeks after participant receives the first dose in Part 1

Eligibility
Key inclusion criteria
1. Have genetically confirmed Friedreich's ataxia
2. Have a modified FARS score =20 and =80
3. Be male or female and =16 years of age and =40 years of age
4. Have no changes to exercise regimen within 30 days prior to Study Day 1 and be willing to remain on the same exercise regimen during the 16-week study period
5. Have the ability to complete maximal exercise testing
6. Be able to swallow capsules
Minimum age
16 Years
Maximum age
40 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Have uncontrolled diabetes (HbA1c >11.0%)
2. Have B-type natriuretic peptide value >200 pg/mL
3. Have a history of clinically significant left-sided heart disease and/or clinically significant cardiac disease
4. Have known active fungal, bacterial, and/or viral infection, including human immunodeficiency virus or hepatitis virus (B or C)
5. Have known or suspected active drug or alcohol abuse
6. Have clinically significant abnormalities of clinical hematology or biochemistry, including but not limited to elevations greater than 1.5 times the upper limit of normal of aspartate aminotransferase, or alanine aminotransferase
7. Have any abnormal laboratory test value or serious pre-existing medical condition that, in the opinion of the investigator, would put the patient at risk by study enrollment
8. Have taken any of the following drugs within 7 days prior to Study Day 1 or plan to take any of these drugs during the time of study participation:

1. Sensitive substrates for cytochrome P450 2C8 or 3A4 (e.g., repaglinide, midazolam, sildenafil)
2. Moderate or strong inhibitors or inducers of cytochrome P450 3A4 (e.g., carbamazepine, phenytoin, ciprofloxacin, grapefruit juice)
3. Substrates for p-glycoprotein transporter (e.g., ambrisentan, digoxin)
9. Have participated in any other interventional clinical study within 30 days prior to Study Day 1
10. Have a cognitive impairment that may preclude ability to comply with study procedures
11. Prior participation in a trial with omaveloxolone (RTA 408)

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
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
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
31/01/2015
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/12/2024
Actual
Sample size
Target
Accrual to date
Final
172
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Murdoch Childrens Research Institute - Parkville
Recruitment postcode(s) [1] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Florida
Country [3] 0 0
United States of America
State/province [3] 0 0
Georgia
Country [4] 0 0
United States of America
State/province [4] 0 0
Iowa
Country [5] 0 0
United States of America
State/province [5] 0 0
Ohio
Country [6] 0 0
United States of America
State/province [6] 0 0
Pennsylvania
Country [7] 0 0
Austria
State/province [7] 0 0
Innsbruck
Country [8] 0 0
Italy
State/province [8] 0 0
Milan
Country [9] 0 0
United Kingdom
State/province [9] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Reata, a wholly owned subsidiary of Biogen
Address
Country
Other collaborator category [1] 0 0
Commercial sector/industry
Name [1] 0 0
AbbVie
Address [1] 0 0
Country [1] 0 0
Other collaborator category [2] 0 0
Other
Name [2] 0 0
Friedreich's Ataxia Research Alliance
Address [2] 0 0
Country [2] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
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

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: https://vivli.org/


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT02255435