Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
CTI-1601 · 4 trials · 1 indication
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...
CTI-1601 is an investigational therapy being developed for Friedreich Ataxia, a rare inherited disease that affects the nervous system and heart. It is currently in Phase 2 clinical development and has not been approved by the FDA.
CTI-1601 targets FXN, the protein that is deficient in Friedreich Ataxia. It is a monoclonal antibody designed to address the underlying cause of the disease by targeting this specific protein.
CTI-1601 is being developed by Larimar Therapeutics, Inc., a biopharmaceutical company traded on the Nasdaq under the ticker symbol LRMR.
CTI-1601 is currently in Phase 2 clinical development. It has completed Phase 1 and Phase 2 trials and is now being studied in an open-label Phase 2 trial. It is investigational and not yet FDA approved.
CTI-1601 has been studied in four clinical trials. Completed trials include NCT04176991, NCT04519567, and NCT05579691. The ongoing trial is NCT06447025, an open-label Phase 2 study in Friedreich Ataxia patients aged 2 years and older.
CTI-1601 is the primary name for this investigational therapy. No alternative names have been reported for this drug in clinical trial records.