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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
Date last updated
8/09/2025
Titles & IDs
Public title
A Study to Learn About the Effects and Safety of RTA 408 (Omaveloxolone) in People Aged 16 to 40 With Friedreich's Ataxia
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Scientific title
A Phase 2 Study of the Safety, Efficacy, and Pharmacodynamics of RTA 408 in the Treatment of Friedreich's Ataxia (MOXIe)
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Secondary ID [1]
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2024-517436-22
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Secondary ID [2]
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RTA 408-C-1402
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Friedreich Ataxia
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Condition category
Condition code
Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Neurological
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Neurodegenerative diseases
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Metabolic and Endocrine
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Metabolic disorders
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Intervention/exposure
Study type
Interventional
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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
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Change From Baseline in Peak Work (in Watts/kg) During Exercise Testing at Week 12 in Part 1
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Assessment method [1]
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Peak work attained during maximal exercise testing. Cycle ergometry using a recumbent stationary bicycle was used, and workload was increased incrementally. Peak work is defined as the workload at which patients reach maximal volition (defined as an inability to continue to exercise due to exhaustion).
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Timepoint [1]
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Baseline through 12 weeks after participant receives the first dose in Part 1.
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Primary outcome [2]
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Change in the Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 48 in Part 2
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Assessment method [2]
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The mFARS includes 4 of the 5 sections of the Friedreich's Ataxia Rating Scale (FARS): bulbar (score 0 to 11), upper limb coordination (score 0 to 36), lower limb coordination (score 0 to 16), and upright stability (score 0 to 36). The minimum score is 0 and the maximum score is 99. A lower score indicates better neurological function.
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Timepoint [2]
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48 weeks after participant receives the first dose in Part 2
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Secondary outcome [1]
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Change in the Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 12 in Part 1
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Assessment method [1]
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The mFARS includes 4 of the 5 sections of the Friedreich's Ataxia Rating Scale (FARS): bulbar (score 0 to 11), upper limb coordination (score 0 to 36), lower limb coordination (score 0 to 16), and upright stability (score 0 to 36). The minimum score is 0 and the maximum score is 99. A lower score indicates better neurological function.
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Timepoint [1]
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12 weeks after participant receives the first dose in Part 1
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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
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Minimum age
16
Years
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Maximum age
40
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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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)
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people analysing the results/data
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Active, not recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
31/01/2015
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
31/08/2025
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Actual
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Sample size
Target
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Accrual to date
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Final
172
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Recruitment in Australia
Recruitment state(s)
VIC
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Recruitment hospital [1]
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Murdoch Childrens Research Institute - Parkville
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Recruitment postcode(s) [1]
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3052 - Parkville
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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California
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United States of America
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State/province [2]
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Florida
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Country [3]
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United States of America
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Georgia
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Country [4]
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United States of America
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State/province [4]
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Iowa
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Country [5]
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United States of America
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Ohio
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Country [6]
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United States of America
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State/province [6]
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Pennsylvania
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Country [7]
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Austria
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State/province [7]
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Innsbruck
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Country [8]
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Italy
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State/province [8]
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Milan
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Country [9]
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United Kingdom
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State/province [9]
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London
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Biogen
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Address
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Other collaborator category [1]
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Commercial sector/industry
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Name [1]
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AbbVie
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Address [1]
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Other collaborator category [2]
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Other
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Name [2]
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Friedreich's Ataxia Research Alliance
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Address [2]
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Ethics approval
Ethics application status
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Summary
Brief summary
In this study, researchers are learning more about RTA 408, also known as omaveloxolone, BIIB141, or SKYCLARYS®. The main goal of this study is to learn more about the safety of RTA 408 and how it affects physical effort, movement, coordination, and how participants feel in daily life. The main questions researchers want to answer in this study are: * How much physical effort can a participant produce during a cycling test after 12 weeks of treatment? * How do scores on the modified Friedreich's Ataxia Rating Scale (mFARS) change after 48 weeks? Researchers will use the modified Friedreich's Ataxia Rating Scale (mFARS) to measure how FA affects the nervous system. The mFARS looks at movement ability, balance, coordination, speech, and how well the arms and legs work. They will also use a cycling test to measure physical effort, along with questionnaires to learn how participants feel and function in daily life. Safety will also be tested using physical exams, vital sign checks, echocardiograms (ECHO), electrocardiograms (ECG), and blood and urine tests. The study will be done in 2 main parts, followed by an optional Extension period: * In Part 1, participants will be randomly assigned to take different doses of RTA 408 or a placebo by mouth once a day for 12 weeks. A placebo looks like the study drug but contains no real medicine. * Researchers will compare these doses to decide which one to use in Part 2. * In Part 2, a different group of participants will take either the chosen dose of RTA 408 (150 mg) or placebo once a day for 48 weeks. * Participants who complete Part 1 or Part 2 may be able to join an Extension period, where everyone receives RTA 408. * In the Extension period, participants will continue to receive RTA 408 until the drug becomes commercially available or until they leave the study * Participants in Part 1 will have up to 9 study visits and 2 phone calls. If they do not move onto the Extension period, they will stay in the study for up to 20 weeks. * Participants in Part 2 will have up to 10 study visits and 3 phone calls. If they do not move onto the Extension period, they will stay in the study for up to 61 weeks. * Participants in the Extension period will have 2 visits in the first month, followed by visits every 6 months.
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Trial website
https://clinicaltrials.gov/study/NCT02255435
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Trial related presentations / publications
Lynch DR, Chin MP, Delatycki MB, Subramony SH, Corti M, Hoyle JC, Boesch S, Nachbauer W, Mariotti C, Mathews KD, Giunti P, Wilmot G, Zesiewicz T, Perlman S, Goldsberry A, O'Grady M, Meyer CJ. Safety and Efficacy of Omaveloxolone in Friedreich Ataxia (MOXIe Study). Ann Neurol. 2021 Feb;89(2):212-225. doi: 10.1002/ana.25934. Epub 2020 Nov 5. Lynch DR, Farmer J, Hauser L, Blair IA, Wang QQ, Mesaros C, Snyder N, Boesch S, Chin M, Delatycki MB, Giunti P, Goldsberry A, Hoyle C, McBride MG, Nachbauer W, O'Grady M, Perlman S, Subramony SH, Wilmot GR, Zesiewicz T, Meyer C. Safety, pharmacodynamics, and potential benefit of omaveloxolone in Friedreich ataxia. Ann Clin Transl Neurol. 2018 Nov 10;6(1):15-26. doi: 10.1002/acn3.660. eCollection 2019 Jan.
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Public notes
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Contacts
Principal investigator
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Fax
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Email
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Contact person for public queries
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Fax
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Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
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What data in particular will be shared?
In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/
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When will data be available (start and end dates)?
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Available to whom?
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Available for what types of analyses?
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How or where can data be obtained?
IPD available at link: https://vivli.org/
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What supporting documents are/will be available?
No Supporting Document Provided
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Other Details
Attachment
Study protocol
Study Protocol: Main Protocol
https://cdn.clinicaltrials.gov/large-docs/35/NCT02255435/Prot_000.pdf
Study protocol
Study Protocol: United Kingdom Protocol
https://cdn.clinicaltrials.gov/large-docs/35/NCT02255435/Prot_001.pdf
Statistical analysis plan
Statistical Analysis Plan: Part 1
https://cdn.clinicaltrials.gov/large-docs/35/NCT02255435/SAP_002.pdf
Statistical analysis plan
Statistical Analysis Plan: Part 2
https://cdn.clinicaltrials.gov/large-docs/35/NCT02255435/SAP_003.pdf
Statistical analysis plan
Statistical Analysis Plan: Part 2 Addendum
https://cdn.clinicaltrials.gov/large-docs/35/NCT02255435/SAP_004.pdf
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results are available at
https://clinicaltrials.gov/study/NCT02255435
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