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Also known as CTI-1601
Nomlabofusp · 5 trials · 2 indications
Number - The USS has a score range from 0 to 36. Lower scores mean less impairment and higher scores mean greater neurological impairment.
Number - The mFARS has a total score range from 0 to 93. Lower scores mean less impairment and higher scores mean greater neurological impairment.
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 |
|---|---|---|
| Nomlabofusp | EXPERIMENTAL | Daily subcutaneous injection of nomlabofusp for 72 weeks |
| Placebo | PLACEBO_COMPARATOR | Daily subcutaneous injection of placebo for 72 weeks |
| 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 |
| Name | Type | Description |
|---|---|---|
| Nomlabofusp | DRUG | Nomlabofusp is a recombinant fusion protein provided in a sterile, preservative-free buffered solution for subcutaneous injection intended to deliver human frataxin, the protein deficient in Friedreich's ataxia. |
| Placebo | DRUG | The placebo is a sterile, preservative-free, clear liquid for subcutaneous injection. |
| 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 |
Key Inclusion Criteria: Subjects who meet all of the following criteria are potentially eligible for study participation: 1. Subject must provide genetically confirmed FRDA diagnosis report and is homozygous for GAA repeat expansions documented on the genetic diagnostic report, with repeat sizing ...
Nomlabofusp is an investigational therapy being developed for Friedreich ataxia, a rare inherited neurological disorder. It is a monoclonal antibody designed to target the FXN protein. Larimar Therapeutics, Inc. (ticker LRMR) is developing the drug, which is currently in Phase 3 clinical development and has received several FDA designations including Breakthrough Therapy and Orphan Drug.
Nomlabofusp is being developed as a treatment for Friedreich ataxia, a rare genetic disease that affects the nervous system and movement. It is an investigational product and has not been approved for commercial use. Clinical studies are evaluating its efficacy and safety in patients with Friedreich ataxia, including pediatric and adult populations.
Nomlabofusp targets the FXN protein, which is associated with Friedreich ataxia. As a monoclonal antibody, it is designed to bind to this target. The drug is being studied for its potential to address the underlying protein deficiency in Friedreich ataxia, though it remains investigational and its clinical benefit has not been established.
Nomlabofusp is being developed by Larimar Therapeutics, Inc., a biopharmaceutical company that trades under the ticker symbol LRMR. Larimar is focused on developing treatments for rare diseases, and nomlabofusp is its lead investigational candidate for Friedreich ataxia.
Nomlabofusp is in Phase 3 clinical development. A Phase 3 trial, NCT07778836, is currently recruiting participants with Friedreich ataxia. The drug has not been approved by the FDA and remains investigational. It has received FDA designations including Breakthrough Therapy, Orphan Drug, Rare Pediatric Disease, and Fast Track.
Nomlabofusp has been studied in multiple clinical trials. Active trials include a Phase 3 study (NCT07778836) and a Phase 2 open-label study (NCT06447025). Completed trials include a Phase 2 double-blind, placebo-controlled study (NCT05579691) and a Phase 1 multiple ascending dose study (NCT04519567). These trials enrolled a total of 318 participants.
Yes, nomlabofusp is also known as CTI-1601. Both names refer to the same investigational monoclonal antibody developed by Larimar Therapeutics for Friedreich ataxia. Earlier clinical trials used the CTI-1601 designation, while the current Phase 3 program uses the name nomlabofusp.