Trial Outcomes & Findings for (+) Epicatechin to Treat Friedreich's Ataxia (NCT NCT02660112)

Results Overview

The Friedreich Ataxia Rating Scale (FARS) is made up of a measure of ataxia, an activities of daily living subscale and a neurological subscale. This scale also includes the 8m walk at maximum speed (8MW), the 9-hole peg test (9HPT), PATA rate (assesses speech speed by repetitions of pronouncing "PaTa" ) and low-contrast letter acuity. FARS is made up of a measure of ataxia, an activities of daily living subscale and a neurological subscale. This scale also includes the 8m walk at maximum speed (8MW), the 9-hole peg test (9HPT), PATA rate and low-contrast letter acuity. The scores from the three subscales are added to generate a total score ranging from 0 to 159, with a higher score indicating a greater level of disability.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

10 participants

Primary outcome timeframe

Baseline, 24 weeks

Results posted on

2019-12-05

Participant Flow

Participant milestones

Participant milestones
Measure	(+)-Epicatechin Total daily dose 75mg (+)-Epicatechin; 25mg cap three times per day by mouth for 24 weeks (+)-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)
Overall Study STARTED	10
Overall Study COMPLETED	10
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	(+)-Epicatechin n=10 Participants Total daily dose 75mg (+)-Epicatechin; 25mg cap three times per day by mouth for 24 weeks (+)-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)
Age, Continuous	14.5 years n=10 Participants
Sex: Female, Male Female	4 Participants n=10 Participants
Sex: Female, Male Male	6 Participants n=10 Participants
Region of Enrollment United States	10 participants n=10 Participants

PRIMARY outcome

Timeframe: Baseline, 24 weeks

Population: One subject did not complete week 24 questionnaire

The Friedreich Ataxia Rating Scale (FARS) is made up of a measure of ataxia, an activities of daily living subscale and a neurological subscale. This scale also includes the 8m walk at maximum speed (8MW), the 9-hole peg test (9HPT), PATA rate (assesses speech speed by repetitions of pronouncing "PaTa" ) and low-contrast letter acuity. FARS is made up of a measure of ataxia, an activities of daily living subscale and a neurological subscale. This scale also includes the 8m walk at maximum speed (8MW), the 9-hole peg test (9HPT), PATA rate and low-contrast letter acuity. The scores from the three subscales are added to generate a total score ranging from 0 to 159, with a higher score indicating a greater level of disability.

Outcome measures

Outcome measures
Measure	(+)-Epicatechin n=9 Participants Total daily dose 75mg (+)-Epicatechin; 25mg cap three times per day by mouth for 24 weeks (+)-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)
Change From Baseline in Friedreich Ataxia Rating Scale (FARS) Composite Score	55.0 score on a scale Standard Deviation 18.72

PRIMARY outcome

Timeframe: Baseline, 24 weeks

Left ventricular mass and left ventricular (LV) mass indexed to body surface area estimated by Left Ventricle (LV)cavity dimension and wall thickness at end-diastole. Normal values of LV mass indexed to body surface area are found to be 49-115 gL/m2 in men and 43-95 g/m2 in women.

Outcome measures

Outcome measures
Measure	(+)-Epicatechin n=10 Participants Total daily dose 75mg (+)-Epicatechin; 25mg cap three times per day by mouth for 24 weeks (+)-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)
Change in Ventricular Hypertrophy as Shown on Cardiac MRI	55.9 mL/m2 Standard Deviation 9.49

Adverse Events

(+)-Epicatechin

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	(+)-Epicatechin n=10 participants at risk Total daily dose 75mg (+)-Epicatechin; 25mg cap three times per day by mouth for 24 weeks (+)-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)
Nervous system disorders Transient worsening of migraine	30.0% 3/10 • Adverse Events were collected over a 24 week time period

Additional Information

Ralitza H. Gavrilova, M.D.

Mayo Clinic

Phone: 507-293-1283

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place