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Trial · Interventional

CompletedPhase 1/2Results postedRegistry data

FRDA Investigator Initiated Study (IIS) With Elamipretide

Short name ELViS-FA

NCTNCT05168774 (opens ClinicalTrials.gov in a new tab)Other id20-018049Other idSPIFA-101

Overview

Status
Completed
Phase
Phase 1/2
Enrollment
20 participants (actual)
Sponsor
Children's Hospital of Philadelphia · Other
Design
Randomized · Open label (no masking)
Countries
1 country

United States

Start
3 Mar 2022
Primary completion
25 Jun 2024
Completion
25 Jul 2024
First posted
23 Dec 2021
Last update posted
12 Dec 2025
Conditions
Friedreich Ataxia

Timeline

1 trial · 1 completed · 1 with posted results

  • Completed
  • Ongoing
  • Not yet recruiting
  • Stopped
  • Estimated dates (est.)
  • Results posted
2022202320242025
  1. Ph 1/2

    ELViS-FA

    NCT05168774

    3 Mar 2022 to 25 Jul 2024

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

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

Interventions

  • ElamipretideDrug

Arms2

  • Experimental

    Low Dose (20-30mg)

    Subjects will receive daily subcutaneous (SC) dosing of Elamipretide (20-30 mg) for 52 weeks

  • Experimental

    High Dose (40-60 mg)

    Subjects will receive daily subcutaneous (SC) dosing of Elamipretide (40-60 mg) for 52 weeks

Primary outcomes1

Outcomes the sponsor listed as primary.

  1. Change in High Contrast Visual Acuity

    Time frame · Baseline to 52 weeks

    Registry description

    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).

Secondary outcomes(7)
  1. Change in Low Contrast Visual Acuity

    Time frame · Baseline to 52 weeks

    Registry description

    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).

  2. Change in Low Luminescence Visual Activity

    Time frame · Baseline to 52 weeks

    Registry description

    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).

  3. Change in Retinal Nerve Fiber Layer by Optical Coherence Tomography (OCT)

    Time frame · Baseline to 52 weeks

    Registry description

    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.

  4. Change in Visual Quality of Life by Visual Functioning Questionnaire (VFQ)

    Time frame · Baseline to 52 weeks

    Registry description

    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). The VFQ-25 consists of a base set of 25 vision-targeted questions representing 11 vision-related constructs, plus an additional single-item general health rating question. Each item is then converted to a 0 to 100 scale. The summary statistic is the difference in mean values from Baseline to Week 52. A negative value represents a worsening over time and a positive value represents improvement.

  5. Change in Cardiac Strain

    Time frame · Baseline to 36 weeks

    Registry description

    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.

  6. Change in Cardiac Fibrosis

    Time frame · Baseline to 36 weeks

    Registry description

    The change in cardiac fibrosis over time by T1 mapping using late gadolinium enhancement between groups (low dose and high dose).

  7. Change Cardiac Stroke Volume

    Time frame · Baseline to 36 weeks

    Registry description

    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).

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

Primary outcome results

Primary outcomeMedian· Baseline to 52 weeks

Change in High Contrast Visual Acuity

GroupValue (Number of Letters Read on a Vision Board)
Low Dose (20-30mg)5.5
High Dose (40-60 mg)0
Secondary and other outcome results(7)
Secondary outcomeMedian· Baseline to 52 weeks

Change in Low Contrast Visual Acuity

GroupValue (Number of Letters Read on a Vision Board)
Low Dose (20-30mg)0
High Dose (40-60 mg)0
Secondary outcomeMedian· Baseline to 52 weeks

Change in Low Luminescence Visual Activity

GroupValue (Number of Letters Read on a Vision Board)
Low Dose (20-30mg)0.5
High Dose (40-60 mg)0
Secondary outcomeMedian· Baseline to 52 weeks

Change in Retinal Nerve Fiber Layer by Optical Coherence Tomography (OCT)

GroupValue (micron)
Low Dose (20-30mg)not posted
High Dose (40-60 mg)not posted
Secondary outcomeMean· Baseline to 52 weeks

Change in Visual Quality of Life by Visual Functioning Questionnaire (VFQ)

GroupValue (Units on a scale)Spread
Low Dose (20-30mg)-2.423.85
High Dose (40-60 mg)2.085.77
Secondary outcomeMean· Baseline to 36 weeks

Change in Cardiac Strain

GroupValue
Low Dose (20-30mg)not posted
High Dose (40-60 mg)not posted
Secondary outcome· Baseline to 36 weeks

Change in Cardiac Fibrosis

GroupValue
Low Dose (20-30mg)not posted
High Dose (40-60 mg)not posted
Secondary outcomeMean· Baseline to 36 weeks

Change Cardiac Stroke Volume

GroupValue (percentage of change in stroke volume)Spread
Low Dose (20-30mg)0.120.16
High Dose (40-60 mg)-0.020.29

Adverse events, as posted

Adverse events as posted: participants affected out of participants at risk, by group
GroupDeathsSerious eventsOther events
Low Dose (20-30mg)Subjects will receive daily subcutaneous (SC) dosing of Elamipretide (20-30 mg) for 52 weeks 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.0 / 82 / 88 / 8
High Dose (40-60 mg)Subjects will receive daily subcutaneous (SC) dosing of Elamipretide (40-60 mg) for 52 weeks 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.0 / 82 / 88 / 8

Each cell reads participants affected / participants at risk, as the registry posted them.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies0

Studies that cite this trial.

No study cites this trial.

Eligibility

Who could join, as posted.

Sex
All
Age
from 16 Years
Healthy volunteers
Not accepted
Inclusion and exclusion criteria, as posted
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

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