Clinical trial NCT02660112: (+) Epicatechin to Treat Friedreich's Ataxia
Official title: A Phase II, Open Label Prospective Single Center Drug Study Evaluating the Safety and Efficacy of (+)-Epicatechin in Subjects With Friedreich's Ataxia
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Friedreich's Ataxia
Lead sponsor: Mayo Clinic
Sponsor class: OTHER
Collaborators: name: Cardero Therapeutics, Inc.
First posted: 2016-01-21
Enrollment: 10
Sex: ALL
Minimum age (years): 10
Maximum age (years): 50
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


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.

Primary outcomes:
measure: Change From Baseline in Friedreich Ataxia Rating Scale (FARS) Composite Score
timeFrame: Baseline, 24 weeks
measure: Change in Ventricular Hypertrophy as Shown on Cardiac MRI
timeFrame: Baseline, 24 weeks
Secondary outcomes:
Other outcomes:
Locations:
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
Study officials:
affiliation: Mayo Clinic
name: Ralitza H Gavrilova, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.