Conditions: Friedreich's Ataxia
Keywords: Interferon gamma
Allocation: RANDOMIZED | Intervention model: PARALLEL
Enrollment: 92
Healthy volunteers: no

Brief summary:
The purpose of this phase 3 randomized, multi-center, double-blind, placebo-controlled study is to evaluate the efficacy and safety of ACTIMMUNE® (interferon-γ 1b) in the treatment of Friedreich's Ataxia (FA) and to evaluate the pharmacokinetic (PK) characteristics of ACTIMMUNE® in FA patients.

Detailed description:
Study with completed results acquired from Horizon in 2024.

Arms:
arm 1: Approximately 45 participants will receive subcutaneous (SC) doses of ACTIMMUNE® 3 times a week (TIW) for a total of 26 weeks. arm 2: Approximately 45 participants will receive SC doses of placebo TIW for a total of 26 weeks.

Interventions: intervention 1: Interferon γ-1b intervention 2: Placebo
Intervention descriptions: intervention 1: The study drug dose is planned to be escalated on a weekly basis over the first 4 weeks of treatment (from 10 µg/m² to 25, 50, and 100 µg/m²). The dose may be reduced, interrupted, or held based on tolerability. By Week 13, all participants are to be on a stable tolerated dose of study drug in order to continue study participation; the dose may not be further increased after week 13, however, it may be reduced on a case-by-case basis to manage drug-related adverse events (AEs). intervention 2: The volume of placebo is planned to correspond with volume of study drug that would be given to the participant if the participant was randomized to the study drug arm.

Protocol document: