A Double-Blind, Placebo-Controlled, Dose Exploration Study of CTI-1601 in Adult Subjects With Friedreich's Ataxia
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT05579691 |
Recruitment Status :
Completed
First Posted : October 14, 2022
Last Update Posted : February 16, 2024
|
Sponsor:
Larimar Therapeutics, Inc.
Information provided by (Responsible Party):
Larimar Therapeutics, Inc.
Tracking Information | |||||
---|---|---|---|---|---|
First Submitted Date ICMJE | October 5, 2022 | ||||
First Posted Date ICMJE | October 14, 2022 | ||||
Last Update Posted Date | February 16, 2024 | ||||
Actual Study Start Date ICMJE | September 21, 2022 | ||||
Actual Primary Completion Date | December 4, 2023 (Final data collection date for primary outcome measure) | ||||
Current Primary Outcome Measures ICMJE |
Number of Participants with Treatment Emergent Adverse Events [ Time Frame: Through study completion, an average of 93 days ] Overall summary of Participants with Treatment Emergent Adverse Events
|
||||
Original Primary Outcome Measures ICMJE |
Number of Participants with Treatment Emergent Adverse Events [ Time Frame: Through study completion, an average of 86 days ] Overall summary of Participants with Treatment Emergent Adverse Events
|
||||
Change History | |||||
Current Secondary Outcome Measures ICMJE |
|
||||
Original Secondary Outcome Measures ICMJE | Same as current | ||||
Current Other Pre-specified Outcome Measures | Not Provided | ||||
Original Other Pre-specified Outcome Measures | Not Provided | ||||
Descriptive Information | |||||
Brief Title ICMJE | A Double-Blind, Placebo-Controlled, Dose Exploration Study of CTI-1601 in Adult Subjects With Friedreich's Ataxia | ||||
Official Title ICMJE | A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Dose Exploration Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous CTI-1601 in Adult Subjects With Friedreich's Ataxia | ||||
Brief Summary | To evaluate the safety and tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of subcutaneous (SC) administration of CTI-1601 over 28 days in subjects with Friedreich's ataxia (FRDA). | ||||
Detailed Description | This is a double-blind, placebo-controlled, study evaluating two doses (25 mg and 50 mg) of CTI-1601. This study will consist of at least 2 cohorts with 12 to 15 subjects participating in each cohort. Subjects will be dosed once daily (QD) for 14 days followed by dosing every other day (QOD) through Day 28. |
||||
Study Type ICMJE | Interventional | ||||
Study Phase ICMJE | Phase 2 | ||||
Study Design ICMJE | Allocation: Randomized Intervention Model: Parallel Assignment Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) Primary Purpose: Treatment |
||||
Condition ICMJE | Friedreich Ataxia | ||||
Intervention ICMJE |
|
||||
Study Arms ICMJE |
|
||||
Publications * |
|
||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
|||||
Recruitment Information | |||||
Recruitment Status ICMJE | Completed | ||||
Actual Enrollment ICMJE |
28 | ||||
Original Estimated Enrollment ICMJE |
15 | ||||
Actual Study Completion Date ICMJE | December 4, 2023 | ||||
Actual Primary Completion Date | December 4, 2023 (Final data collection date for primary outcome measure) | ||||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria: Subjects are excluded from the study if any of the following exclusion criteria are met:
|
||||
Sex/Gender ICMJE |
|
||||
Ages ICMJE | 18 Years and older (Adult, Older Adult) | ||||
Accepts Healthy Volunteers ICMJE | No | ||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||
Listed Location Countries ICMJE | United States | ||||
Removed Location Countries | |||||
Administrative Information | |||||
NCT Number ICMJE | NCT05579691 | ||||
Other Study ID Numbers ICMJE | CLIN-1601-200 | ||||
Has Data Monitoring Committee | Yes | ||||
U.S. FDA-regulated Product |
|
||||
IPD Sharing Statement ICMJE | Not Provided | ||||
Current Responsible Party | Larimar Therapeutics, Inc. | ||||
Original Responsible Party | Same as current | ||||
Current Study Sponsor ICMJE | Larimar Therapeutics, Inc. | ||||
Original Study Sponsor ICMJE | Same as current | ||||
Collaborators ICMJE | Not Provided | ||||
Investigators ICMJE |
|
||||
PRS Account | Larimar Therapeutics, Inc. | ||||
Verification Date | February 2024 | ||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |