A Phase 2 Study of the Safety and Efficacy of RTA 408 in Patients withFriedreich's Ataxia

Phase 1

• Conditions: Friedreich's Ataxia; MedDRA version: 20.0Level: PTClassification code 10017374Term: Friedreich's ataxiaSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2015-002762-23-IT
• Lead Sponsor: REATA PHARMACEUTICALS, INC.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-01-29
• Last Update Posted: 5 April 2021

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 172

Inclusion Criteria

Part 1 and Part 2
Patients must:
1. Have genetically confirmed Friedreich's ataxia
2. Have an mFARS score =20 and =80. The average of the two
mFARS values collected at Screening and Day 1 visits must fall within
the allowable range, and they must be within 4.5 points of each other
3. Be male or female and =16 years of age and =40 years of age
4. Have no changes to their exercise regimen within 30 days prior to
Study Day 1 and be willing to remain on the same exercise regimen
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during the 16-week study period
5. Have the ability to complete maximal exercise testing
6. Have adequate kidney function defined as an estimated glomerular
filtration rate (eGFR) =60 mL/min/1.73 m2 using the Modification of
Diet in Renal Disease (MDRD) 4-variable formula
7. Have a left ventricular ejection fraction =40% (based on
echocardiogram performed at Screening Visit or within 90 days prior to
Screening Visit)
8. Be able to swallow capsules
9. Be willing and able to cooperate with all aspects of the protocol
10. Be willing to practice medically acceptable methods of birth control
(Section 9.7.2)
11. Provide written informed consent for study participation, approved
by the appropriate Institutional Review Board (IRB)
Extension eligibility:
Patients must complete 12 weeks of treatment in Part 1 or 48 weeks of
treatment in Part 2, have no major protocol deviations, and meet
inclusion criteria as follows:
1. Have adequate kidney function defined as an estimated glomerular
filtration rate (eGFR) = 60 mL/min/1.73 m2 using the Modification of
Diet in Renal Disease (MDRD) 4-variable formula
2. Have a left ventricular ejection fraction = 40% (based on
echocardiogram performed at Screening Visit or within 90 days prior to
Screening Visit)
3. Be able to swallow capsules
4. Be willing and able to cooperate with all aspects of the extension
5. Be willing to practice medically acceptable methods of birth control
6. Provide written informed consent for study participation, approved by
the appropriate Institutional Review Board (IRB)
7. Have been enrolled in Part 1 or Part 2 and completed assessments
through the follow-up visit with no major protocol deviations.
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 134
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Part 1/ Part 2 Patients must not:1.Have uncontrolled diabetes (HbA1c >11.0%) 2.Have BNP level >200 pg/mL 3. Have a history of clinically significant left-sided heart disease and/or clinically significant cardiac disease, with the exception of mild to moderate cardiomyopathy associated with Friedreich's ataxia 4. Have known active fungal, bacterial, and/or viral infection, including human immunodeficiency virus (HIV) or hepatitis virus (B or C) 5. Have known or suspected active drug or alcohol abuse, as per investigator judgment 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 (ULN) of AST, or ALT. Levels above this threshold are allowable if attributable to muscle injury 7. Have any abnormal laboratory test value or clinically significant preexisting 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: 9. Have a history of clinically significant liver disease (e.g., fibrosis, cirrhosis, hepatitis), or has, at screening, clinically relevant deviations in laboratory tests including any one of the following: a. ALT and/or AST > 1.5-fold ULN, b. bilirubin > 1.2-fold ULN, c. ALP > 2-fold ULN, d. albumin < lower limit of normal (LLN) 10. Have participated in any other interventional clinical study within 30 days prior to Study Day 1 11. Have a cognitive impairment that may reclude ability to comply with study procedures 12. Be unable to comply with the requirements of the study protocol or be unsuitable for the study for any reason, in the opinion of the investigator 13. Have used antioxidant supplements, including but not limited to idebenone, coenzyme Q10, nicotinamide, and vitamin E above the recommended daily allowance, within 14 days prior to Study Day 1, or plan to take any of these supplements during the time of study participation 14. Have previously documented mitochondrial respiratory chain disease 15. Have a history of thromboembolic events within the past 5 years 16. Have taken anticoagulant therapy within 30 days prior to Study Day 1 17. Have scheduled surgical treatment for scoliosis or foot deformity during the study 18. Have had significant suicidal ideation within 1 month prior to Screening Visit as per investigator judgment or any history of suicide attempts 19. Be pregnant or breastfeeding 20. Prior participation in a trial with RTA 408 Extension eligibility: Patients must not: • Have uncontrolled diabetes (HbA1c > 11.0%) • Have B-type natriuretic peptide (BNP) level > 200 pg/mL • Have a history of clinically significant left-sided heart disease and/or clinically significant cardiac disease, with the exception of mild to moderate cardiomyopathy associated with Friedreich's ataxia • Have a history of clinically significant liver disease or has, at screening, clinically relevant deviations in laboratory tests • Have a cognitive impairment that may preclude ability to comply with study procedures • Be unable to comply with the requirements of the study protocol or be unsuitable for the study for any reason, in the opinion of the investigator • Have a history of thromboembolic events within the past 5 years • Have taken anticoagulant therapy within 30 days prior to Extension Day 1 • Be pregnant or br

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified