An Open-Label Extension Study to Assess the Long-Term Safety, Efficacy, Pharmacodynamics, Pharmacokinetics, and Tolerability of Subcutaneous CTI-1601 in Subjects With Friedreich's Ataxia
Larimar Therapeutics, Inc.
Summary
An open label study designed to evaluate the safety, PK, PD, and clinical effects of long-term daily administration of CTI-1601 enrolling patients with FRDA who have participated in a prior clinical study of CTI-1601 as well as adolescents and adults with FRDA who have not participated in a prior clinical study of CTI-1601.
Description
An open-label study designed to evaluate the long-term safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and clinical effects of subcutaneous (SC) administration of CTI-1601, also known as nomlabofusp, in subjects with Friedreich's ataxia (FRDA). The objectives of this study are: * To evaluate the safety and PK of long-term subcutaneous (SC) administration of CTI-1601 in subjects with FRDA * To evaluate the PD and clinical effects of long-term subcutaneous (SC) administration of CTI-1601 in subjects with FRDA on: * Tissue FXN concentrations * Clinical evaluations of FR…
Eligibility
- Age range
- 12–60 years
- Sex
- All
- Healthy volunteers
- No
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 drug; b) significant AE, defined as Grade 3 or higher according to the Common Terminology Criteria for Adverse Events (CTCAE), version 5.0 (or higher), related to study drug; c) some other event, related to participation in a previous study with CTI-1601, that supports the exclusion of the subject…
Interventions
- DrugCTI-1601
CTI-1601 is a recombinant fusion protein and is intended to deliver human frataxin, the protein deficient in patients with Friedreich's ataxia
Locations (8)
- University of California Los AngelesLos Angeles, California
- Fixel Institute for Neurological Disease, University of Florida HealthGainesville, Florida
- Morsani Center for Advanced Health Care, University of South Florida HealthTampa, Florida
- University of IowaIowa City, Iowa
- Uncommon CuresChevy Chase, Maryland
- Clinilabs Drug Development, Corp.Eatontown, New Jersey