| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06447025 | An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia | PHASE2 | RECRUITING | 85 | — | — | Jan 25, 2024 | Jan 1, 2027 | May 13, 2026 | 8 | United States |
| NCT05579691 | A Double-Blind, Placebo-Controlled, Dose Exploration Study of CTI-1601 in Adult Subjects With Friedreich's Ataxia | PHASE2 | COMPLETED | 28 | — | — | Sep 21, 2022 | Dec 4, 2023 | Nov 29, 2024 | 1 | United States |
| NCT04519567 | Multiple Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia | PHASE1 | COMPLETED | 27 | — | — | Jul 31, 2020 | Mar 16, 2021 | Jun 30, 2021 | 1 | United States |
| NCT04176991 | Single Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia | PHASE1 | COMPLETED | 28 | — | — | Dec 11, 2019 | Oct 31, 2020 | Nov 12, 2020 | 1 | United States |
Number of subjects
Number change in ECG parameters
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.
LVEDV is the amount of blood, measured in milliliters (mL), in the heart's left ventricle just before the heart contracts.
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.
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.
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.
The FARS\_ADL, scored 0 to 36, is a subscale of FARS assessing a subject's ability to complete activities of daily living. A higher score indicates a greater level of disability. The FARS\_ADL questionnaire will be performed at the timepoints indicated in protocol.
The Modified Fatigue Impact Scale (MFIS) is a revised form of the Fatigue Impact Scale based on items derived from interviews with MS patients concerning how fatigue impacts their lives. This instrument provides an assessment of the effects of fatigue in terms of physical, cognitive, and psychosocial functioning. Participants rate on a 5-point scale, with 0 = 'Never' to 4 = 'Almost always' their agreement with 21 statements. Total score (0-84) and subscales for physical (0-36), cognitive (0-40) and psychosocial functioning (0-8). The 5-item version is scored (0-20). Higher numbers indicate greater fatigue. The MFIS will be performed at the timepoints indicated in protocol.
The Patient Global Impression of Change (PGI-C) reflects a patient's assessment about the efficacy of treatment. PGIC is a 7 point scale depicting a patient's rating of overall improvement. Patients rate their change as "very much improved," "much improved," "minimally improved," "no change," "minimally worse," "much worse," or "very much worse." The PGI-C will be performed at the timepoints indicated in protocol.
The Clinical Global Impression of Change (CGI-C) is an assessment to measure change in clinical status (symptoms and functional ability) of the subject's condition from baseline with study drug. CGI-C scores range from 1 (very much improved) through to 7 (very much worse). The CGI-C will be performed at the timepoints indicated in protocol.
Overall summary of Participants with Treatment Emergent Adverse Events
Overall summary of Participants with Treatment Emergent Adverse Events by System Organ Classification (MedDRA version 23.0)
Overall summary of the Participants with Treatment Emergent Adverse Events
Overall summary of Participants with Treatment Emergent Adverse Events by System Organ Classification (MedDRA version 22.0)
| Arm | Type | Description |
|---|---|---|
| CTI-1601 | EXPERIMENTAL | Once daily subcutaneous injection of 50 mg CTI-1601 in subjects ≥ 18 years of age or a weight-based dose of 0.8 mg/kg up to a maximum of 50 mg in subjects ≥ 2 to 17 years of age. |
| CTI-160l | EXPERIMENTAL | CTI-1601 is a recombinant fusion protein and is intended to deliver human frataxin, the protein deficient in Friedreich's ataxia |
| Placebo | PLACEBO_COMPARATOR | Placebo Comparator |
| Name | Type | Description |
|---|---|---|
| CTI-1601 | DRUG | CTI-1601 is a recombinant fusion protein and is intended to deliver human frataxin, the protein deficient in patients with Friedreich's ataxia |
| Placebo | OTHER | Placebo Comparator |
Inclusion Criteria: * Subjects with FRDA who have or have not previously completed participation in a study of CTI-1601 are eligible to participate in this study unless the subject experienced one or more of the following in a previous CTI-1601 study: a) serious adverse event (SAE) related to study...