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Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT05168774
Other study ID # 20-018049
Secondary ID SPIFA-101
Status Active, not recruiting
Phase Phase 1/Phase 2
First received
Last updated
Start date March 3, 2022
Est. completion date December 31, 2024

Study information

Verified date August 2023
Source Children's Hospital of Philadelphia
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

To evaluate the safety, tolerability, and activity of Elamipretide in treating vision loss in Friedreich Ataxia (FRDA).


Description:

To evaluate the effect of high dose (40-60mg) versus low dose (20-30mg) Elamipretide on high contrast visual acuity in FRDA compared to baseline at 52 weeks with the option to extend for an additional 52 weeks if there are objective signs of clinical improvement on primary or secondary endpoints. The interim analysis will be based on data from a 36-week visit. For subjects worse than 20/800 at study start, they will be followed using low vision alternatives only.


Recruitment information / eligibility

Status Active, not recruiting
Enrollment 18
Est. completion date December 31, 2024
Est. primary completion date July 31, 2024
Accepts healthy volunteers No
Gender All
Age group 16 Years and older
Eligibility Inclusion Criteria: 1. Genetically confirmed FRDA (point mutations allowed). 2. Age >16 years. 3. Disease onset before 18 years of age. 4. If female, the subject is not pregnant or lactating or intending to become pregnant before, during, or within 30 days after the last dose of study drug. Female subjects of child-bearing potential must have a negative serum pregnancy test result at Screening, a negative urine pregnancy test result at Baseline. 5. All subjects must agree to use a reliable method of contraception throughout the study and for 30 days after the last dose of study drug. Male subjects should not father a baby during the study or for at least 30 days after the last dose of study drug. 6. All concomitant medications (including over-the-counter medications), vitamins, and supplements must be at stable doses for 30 days prior to study entry and kept stable throughout the study to the best of their ability. 7. Visual acuity (VA) worse than 20/40 (binocular) on the basis of FRDA. Must not be correctable by refraction, or subjects must have sufficient physical exam findings of optic neuropathy (funduscopic, visual fields, or retinal ganglion cell loss) to justify the primary diagnosis of FRDA related optic neuropathy Or 8. Ejection Fraction (EF) less than 50% at last evaluation (within 1 year before screening), with a history consistent with cardiomyopathy from FRDA, and VA 20/25- 20/40. Exclusion Criteria: 1. Any unstable illness that in the investigator's opinion precludes participation in the study. 2. Use of any investigational product within 30 days prior to Screening. 3. A history of substance abuse. 4. Diagnosis of active HIV or Hepatitis B or C infection. 5. Presence of severe renal disease (eGFR <30 mL/min) or hepatic disease [aspartate aminotransferase (AST) or alanine aminotransferase (ALT) >2x the upper limit of normal] as evidenced by laboratory results at Screening. 6. Clinically significant abnormal white blood cell count (ANC <1500), hemoglobin (< 9.0 gm/dL), or platelet count (100 K or >500 K) as evidenced by laboratory test results at Screening. 7. Any other active cause of optic neuropathy (Vitamin B12 deficiency, Vitamin E deficiency, etc.) or cardiac disease 8. EF less than 35% at last echocardiographic evaluation 9. Uncontrolled arrhythmia 10. Current use of any systemic chronic immunosuppressive drugs 11. Current use of Metformin

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
Elamipretide
Elamipretide is a tetra peptide with limited blood brain barrier penetration being developed for use in a variety of mitochondrial disorders, including FRDA, mitochondrial myopathy and Barth Syndrome.

Locations

Country Name City State
United States Children's Hospital of Philadelphia - Neurology Philadelphia Pennsylvania

Sponsors (3)

Lead Sponsor Collaborator
Children's Hospital of Philadelphia Friedreich's Ataxia Research Alliance, Stealth BioTherapeutics Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary Change in High Contrast Visual Acuity Change in High Contrast Visual Acuity will be measured by assessing the differences in the number of letters read (binocular) on the ETDRS High Contrast Visual Acuity Chart between groups (low dose and high dose). Baseline to 52 weeks
Secondary Change in Low Contrast Visual Acuity Change in Low Contrast Visual Acuity will be measured by assessing the differences in the number of letters read (binocular) on the ETDRS Low Contrast Visual Acuity Chart between groups (low dose and high dose). Baseline to 52 weeks
Secondary Change in Low Luminescence Visual Activity Change in Low Luminescence Visual Acuity will be measured by assessing the difference in the number of letters read (binocular) on the ETDRS High Contrast Visual Acuity Chart with Low Luminescence Filter between groups (low dose and high dose). Baseline to 52 weeks
Secondary Change in retinal nerve fiber layer by Optical Coherence Tomography (OCT) The change in thickness of the retinal nerve fiber layer between groups (low dose and high dose) will be measured using the OCT, a non-invasive imaging test that uses light waves to take cross-section pictures of the retina. Baseline to 52 weeks
Secondary Change in visual quality of life by Visual Functioning Questionnaire (VFQ) The VFQ is a 25 item patient reported outcome on visual symptomology to assess change in patient self-report of visual ability over time compared to baseline between groups (low dose and high dose). Baseline to 52 weeks
Secondary Change in Cardiac Strain The change in cardiac strain (dL/L) in each dimension per cardiac cycle between groups (low dose and high dose) is measured by speckle tracking on imaging Baseline to 36 weeks
Secondary Change in Cardiac Fibrosis The change in cardiac fibrosis over time by T1 mapping using late gadolinium enhancement between groups (low dose and high dose). Baseline to 36 weeks
Secondary Change Cardiac Stroke Volume The change in stroke volume will be calculated by Ejection Fraction x Ventricular Volume x Pulse Rate, over time between groups (low dose and high dose). Baseline to 36 weeks
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