Trial · NCT06447025
An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia
NCT06447025, Phase 2, Recruiting, Larimar Therapeutics, Inc.
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia. Trials, record NCT06447025. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT06447025
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia
- NCT number
- NCT06447025
- Status
- Recruiting
- Phases
- Phase 2
- Enrollment
- 85 participants, estimated
- Lead sponsor
- Larimar Therapeutics, Inc.
- Conditions
- Friedreich Ataxia
- Interventions
- CTI-1601
- First posted
- 2024-06-06
- Last updated
- 2026-10-27
Source ClinicalTrials.gov
Registered outcomes20
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of subjects with treatment-emergent adverse events (TEAEs) by System Organ Class (SOC), Preferred Term (PT) and Maximum Severity | Up to 24 months | Number of subjects |
| Primary | Change from baseline in electrocardiogram (ECG) parameters including, but not limited to, HR, RR interval, PR interval, QRS duration, QT interval, and QTcF interval | Up to 24 months | Number change in ECG parameters |
| Primary | Change from baseline in left ventricular ejection fraction (LVEF) | Up to 24 months | LVEF indicates the percentage of change in LV volume from diastole to systole that measures how well the left ventricle of the heart pumps blood. |
| Primary | Change from baseline in left ventricular end-diastolic volume (LVEDV) | Up to 24 months | LVEDV is the amount of blood, measured in milliliters (mL), in the heart's left ventricle just before the heart contracts. |
| Primary | Number of subjects with any suicidal ideation or behavior (Categories 1-10) of the Columbia Suicide Severity Rating Scale (C-SSRS) | Up to 24 months | The Columbia Suicide Severity Rating Scale (C-SSRS) is a tool used to assess the occurrence, severity, and frequency of suicidal thoughts and behaviors. A higher score on the C-SSRS generally indicate a worse outcome, as they signify a higher level of suicidal ideation or behavior. |
| Primary | Change from baseline at each collection timepoint in tissue frataxin concentrations normalized to total protein observed in buccal cells collected from cheek swabs and skin cells collected from skin punch biopsies | Up to 24 months | |
| Primary | Change from baseline in motor function as assessed by 9-hole peg test (9-HPT) | Up to 24 months | |
| Primary | Change from baseline in motor function as assessed by the timed 25-foot walk test (T25-FW) | Up to 24 months | |
| Primary | Change from baseline in neurologic function as assessed by the modified Friedreich's Ataxia Rating Scale (mFARS) total score | Up to 24 months | The Modified Friedreich's Ataxia Rating Scale (mFARS) is a modified neurologic scale involving direct subject participation and targets specific areas impacted by Friedreich's ataxia (bulbar, upper limb, lower limb, and upright stability), with scores ranging from 0-67 points, with higher scores indicating a greater level of disability. |
| Primary | Change from baseline in neurologic function as assessed by the upright stability subscale examination of the mFARS | Through study completion, up to 24 months | The Upright Stability Subscale is an assessment of an individual's ability to maintain balance and stability while standing upright. It has a minimum value of 0 and a maximum value of 36. A higher score indicates a better outcome, reflecting greater stability and balance abilities while standing upright. |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 registered outcomesOutcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers9
The papers the trial lists on ClinicalTrials.gov.
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 comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
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/NCT06447025
GET https://conductscience.org/api/trials/v1/NCT06447025/registered