(+) Epicatechin to Treat Friedreich's Ataxia

NCT ID: NCT02660112

Last Updated: 2019-12-05

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 10 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2016-09-30
• Study Completion Date: 2018-12-30

Brief Summary

This 24-week study will test the safety and effectiveness of synthetically produced (+) Epicatechin in treating patients who have Friedreich's Ataxia, a neurological disorder.

Conditions

Friedreich's Ataxia

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

(+)-Epicatechin

Total daily dose 75mg (+)-Epicatechin; 25mg cap three times per day by mouth for 24 weeks

Group Type: EXPERIMENTAL

(+)-Epicatechin

Intervention Type: DRUG

25mg (+)-Epicatechin capsules. Starting dose 75mg total daily dose (one 25mg cap taken three times per day). Dose escalation at 12 weeks for non-responders to 150mg total daily dose (two 25mg caps taken three times per day)

Interventions

(+)-Epicatechin

25mg (+)-Epicatechin capsules. Starting dose 75mg total daily dose (one 25mg cap taken three times per day). Dose escalation at 12 weeks for non-responders to 150mg total daily dose (two 25mg caps taken three times per day)

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• Confirmed diagnosis of Friedreich's Ataxia (FA) by Frataxin genetic testing and/or Frataxin enzyme analysis
• Between age 10 and 50 years of age, inclusive
• Body weight of 25 kilograms or higher
• Minimum of one affected organ (cardiac or neurological) system, as evidenced by clinical signs/symptoms
• Disease duration ≤7 years, based on onset date of FA symptoms
• Has no known contraindication to gadolinium contrast such as severe allergy or Glomerular Filtration Rate <30 ml/min/m^2.
• Has no known contraindication to non-contrast Magnetic Resonance Imaging (MRI) evaluation such as pacemaker or magnetically active metal fragments.
• Women of childbearing age must:

• Have a negative pregnancy human chorionic gonadotropin test prior to receiving study drug.
• Agree to use contraception for the duration of the study drug dosing, plus 1 month after completion of the study.

Exclusion Criteria

• Advanced cardiac failure, New York Heart Association (NYHA) Classification Scale-Class IV (advanced stage heart failure)
• Clinically significant comorbidities that may also lead to cardiomyopathy, for example long standing hypertension, familial cardiomyopathy.
• Clinically significant comorbidities that would, in the opinion of the investigators, compromise the interpretation of test results.
• Pregnant, breast-feeding or planning to become pregnant during study timeframe.
• Patients with contraindications to regadenoson, i. e. second- or third-degree atrioventricular (AV) block or sinus node dysfunction. Has received an investigational drug within thirty (30) days of baseline visit.
• Thrombocytopenia (<125 x 10^9/Liter) or prolonged Prothrombin Time/Partial Thromboplastin Time (PT/PTT) at baseline.
• Clinically significant hypotension (systolic blood pressure <90) due to heart failure or other conditions.

• Minimum Eligible Age: 10 Years
• Maximum Eligible Age: 50 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Cardero Therapeutics, Inc.

INDUSTRY

Sponsor Role: collaborator

Ralitza Gavrilova

OTHER

Sponsor Role: lead

Responsible Party

Ralitza Gavrilova

MD, Sponsor-Investigator

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

Ralitza H Gavrilova, MD

Role: PRINCIPAL_INVESTIGATOR

Mayo Clinic

Locations

Mayo Clinic

Rochester, Minnesota, United States

Countries

United States

References

Qureshi MY, Patterson MC, Clark V, Johnson JN, Moutvic MA, Driscoll SW, Kemppainen JL, Huston J 3rd, Anderson JR, Badley AD, Tebben PJ, Wackel P, Oglesbee D, Glockner J, Schreiner G, Dugar S, Touchette JC, Gavrilova RH. Safety and efficacy of (+)-epicatechin in subjects with Friedreich's ataxia: A phase II, open-label, prospective study. J Inherit Metab Dis. 2021 Mar;44(2):502-514. doi: 10.1002/jimd.12285. Epub 2020 Aug 31.

Reference Type: DERIVED

PMID: 32677106 (View on PubMed)

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

15-006845

Identifier Type: -

Identifier Source: org_study_id