Trial · NCT05168774
FRDA Investigator Initiated Study (IIS) With Elamipretide
NCT05168774, Phase 1, Phase 2, Completed, Children's Hospital of Philadelphia; results first posted 2025-12-12
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. FRDA Investigator Initiated Study (IIS) With Elamipretide. Trials, record NCT05168774. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05168774
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- FRDA Investigator Initiated Study (IIS) With Elamipretide
- NCT number
- NCT05168774
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 20 participants, actual
- Lead sponsor
- Children's Hospital of Philadelphia
- Conditions
- Friedreich Ataxia
- Interventions
- Elamipretide
- First posted
- 2021-12-23
- Last updated
- 2025-12-12
- Results first posted
- 2025-12-12
Source ClinicalTrials.gov
Registered outcomes8
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change in High Contrast Visual Acuity | Baseline to 52 weeks | 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 | Change in Low Contrast Visual Acuity | Baseline to 52 weeks | 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). |
| Secondary | Change in Low Luminescence Visual Activity | Baseline to 52 weeks | 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). |
| Secondary | Change in Retinal Nerve Fiber Layer by Optical Coherence Tomography (OCT) | Baseline to 52 weeks | 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. |
| Secondary | Change in Visual Quality of Life by Visual Functioning Questionnaire (VFQ) | Baseline to 52 weeks | 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. |
| Secondary | Change in Cardiac Strain | Baseline to 36 weeks | 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. |
| Secondary | Change in Cardiac Fibrosis | Baseline to 36 weeks | The change in cardiac fibrosis over time by T1 mapping using late gadolinium enhancement between groups (low dose and high dose). |
| Secondary | Change Cardiac Stroke Volume | Baseline to 36 weeks | 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). |
Source ClinicalTrials.gov
Outcomes8
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change in High Contrast Visual Acuity | Baseline to 52 weeks | 2 subjects in low dose and 1 subject in the high dose did not complete Week 52 study visit | Number of Letters Read on a Vision Board | MEDIAN |
| SECONDARY | Change in Low Contrast Visual Acuity | Baseline to 52 weeks | All subjects in both low dose and high dose were unable to read a single letter on the low contrast vision board | Number of Letters Read on a Vision Board | MEDIAN |
| SECONDARY | Change in Low Luminescence Visual Activity | Baseline to 52 weeks | 2 subjects in low dose and 1 subject in the high dose did not complete Week 52 study visit | Number of Letters Read on a Vision Board | MEDIAN |
| SECONDARY | Change in Retinal Nerve Fiber Layer by Optical Coherence Tomography (OCT) | Baseline to 52 weeks | 6 low-dose and 7 high-dose subjects attempted to complete this procedure. However, due to disease progression (abnormal eye movements) and visual impairment (unable to focus on visual target) subjects were unable to perform the OCT exam in both low dose and high dose, thus resulting in poor quality images that were unable to be analyzed and will never be analyzed in the future. | micron | MEDIAN |
| SECONDARY | Change in Visual Quality of Life by Visual Functioning Questionnaire (VFQ) | Baseline to 52 weeks | 2 subjects in low dose and 1 subject in the high dose did not complete Week 52 study visit | Units on a scale | MEAN |
| SECONDARY | Change in Cardiac Strain | Baseline to 36 weeks | Due to insufficient number of high quality reproducible scans we were unable to conduct a systematic analysis. The speckle tracking needed for this analysis was not done because reproducible scans were unobtainable in individuals with advanced FA due to inability to lie still for sufficient amounts of time and will never be analyzed in the future. | MEAN | |
| SECONDARY | Change in Cardiac Fibrosis | Baseline to 36 weeks | Due to insufficient number of high quality reproducible scans we were unable to conduct a systematic analysis. The T1 mapping using late gadolinium enhancement needed for this analysis was not done because reproducible scans were unobtainable in individuals with advanced FA due to inability to lie still for sufficient amounts of time and will never be analyzed in the future. | ||
| SECONDARY | Change Cardiac Stroke Volume | Baseline to 36 weeks | 2 subjects in low dose and 2 subject in the high dose did not complete cardiac testing at the Week 36 study visit | percentage of change in stroke volume | MEAN |
Source ClinicalTrials.gov
Results by group10
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change in High Contrast Visual Acuity | Low Dose (20-30mg) | 5.5 | Inter-Quartile Range 0 to 6 | Number of Letters Read on a Vision Board | |
| Change in High Contrast Visual Acuity | High Dose (40-60 mg) | 0 | Inter-Quartile Range -5 to 6 | Number of Letters Read on a Vision Board | |
| Change in Low Contrast Visual Acuity | Low Dose (20-30mg) | 0 | Inter-Quartile Range 0 to 0 | Number of Letters Read on a Vision Board | |
| Change in Low Contrast Visual Acuity | High Dose (40-60 mg) | 0 | Inter-Quartile Range 0 to 0 | Number of Letters Read on a Vision Board | |
| Change in Low Luminescence Visual Activity | Low Dose (20-30mg) | 0.5 | Inter-Quartile Range 0 to 2 | Number of Letters Read on a Vision Board | |
| Change in Low Luminescence Visual Activity | High Dose (40-60 mg) | 0 | Inter-Quartile Range 0 to 0 | Number of Letters Read on a Vision Board | |
| Change in Visual Quality of Life by Visual Functioning Questionnaire (VFQ) | Low Dose (20-30mg) | -2.42 | Standard Deviation 3.85 | Units on a scale | |
| Change in Visual Quality of Life by Visual Functioning Questionnaire (VFQ) | High Dose (40-60 mg) | 2.08 | Standard Deviation 5.77 | Units on a scale | |
| Change Cardiac Stroke Volume | Low Dose (20-30mg) | 0.12 | Standard Deviation 0.16 | percentage of change in stroke volume | |
| Change Cardiac Stroke Volume | High Dose (40-60 mg) | -0.02 | Standard Deviation 0.29 | percentage of change in stroke volume |
Source ClinicalTrials.gov
Participant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Low Dose (20-30mg) | 8 | |
| Overall Study | STARTED | High Dose (40-60 mg) | 8 | |
| Overall Study | COMPLETED | Low Dose (20-30mg) | 4 | |
| Overall Study | COMPLETED | High Dose (40-60 mg) | 4 | |
| Overall Study | NOT COMPLETED | Low Dose (20-30mg) | 4 | |
| Overall Study | NOT COMPLETED | High Dose (40-60 mg) | 4 |
Source ClinicalTrials.gov
Adverse events84
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Injection Site Reaction: Pain, Tenderness, or Burning | Skin and subcutaneous tissue disorders | Low Dose (20-30mg) | 3 | 8 | 3 |
| other | Injection Site Reaction: Pain, Tenderness, or Burning | Skin and subcutaneous tissue disorders | High Dose (40-60 mg) | 1 | 8 | 1 |
| other | Injection Site Reaction: Erythema or Redness | Skin and subcutaneous tissue disorders | Low Dose (20-30mg) | 3 | 8 | 3 |
| other | Injection Site Reaction: Erythema or Redness | Skin and subcutaneous tissue disorders | High Dose (40-60 mg) | 6 | 8 | 6 |
| other | Injection Site Reaction: Induration or Swelling | Skin and subcutaneous tissue disorders | Low Dose (20-30mg) | 4 | 8 | 4 |
| other | Injection Site Reaction: Induration or Swelling | Skin and subcutaneous tissue disorders | High Dose (40-60 mg) | 6 | 8 | 6 |
| other | Injection Site Reaction: Pruritus or Itching | Skin and subcutaneous tissue disorders | Low Dose (20-30mg) | 6 | 8 | 6 |
| other | Injection Site Reaction: Pruritus or Itching | Skin and subcutaneous tissue disorders | High Dose (40-60 mg) | 6 | 8 | 6 |
| other | Injection Site Reaction: Urticaria or Hives | Skin and subcutaneous tissue disorders | Low Dose (20-30mg) | 3 | 8 | 3 |
| other | Injection Site Reaction: Urticaria or Hives | Skin and subcutaneous tissue disorders | High Dose (40-60 mg) | 2 | 8 | 2 |
1 to 10 of 84Source ClinicalTrials.gov
See all 84 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Low Dose (20-30mg) | 0 | 8 |
| Total, all-cause mortality | High Dose (40-60 mg) | 0 | 8 |
| Total, other adverse events | Low Dose (20-30mg) | 8 | 8 |
| Total, other adverse events | High Dose (40-60 mg) | 8 | 8 |
| Total, serious adverse events | Low Dose (20-30mg) | 2 | 8 |
| Total, serious adverse events | High Dose (40-60 mg) | 2 | 8 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Low Dose (20-30mg) vs High Dose (40-60 mg): deaths from any cause | Trial comparisons |
| Low Dose (20-30mg) vs High Dose (40-60 mg): serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT05168774
GET https://conductscience.org/api/trials/v1/NCT05168774/events?page=2