NCT02415127

Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia

Randomized, Multicenter, Double-Blind, Placebo-Controlled, Efficacy, Safety, and Pharmacokinetic Study of ACTIMMUNE® (Interferon γ-1b) in Children and Young Adults With Friedreich's Ataxia

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 92 (actual)
• Sponsor: Amgen · Industry
• Sex: All
• Age: 10 Years – 25 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Friedreich's Ataxia

Interventions

Type	Name	Description
DRUG	Interferon γ-1b	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).
DRUG	Placebo	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.

Timeline

• Start date: 2015-06-01
• Primary completion: 2016-11-01
• Completion: 2016-11-01
• First posted: 2015-04-14
• Last updated: 2024-12-19
• Results posted: 2017-12-08

Locations

4 sites across 1 country: United States

Regulatory

• FDA-regulated drug study