An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia
brief 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.
detailed 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 FRDA * Gene expression and select lipids
official title
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