NCT02593773

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

Multicenter, Safety and Efficacy, Open-Label Extension Study of ACTIMMUNE® (Interferon γ-1b) in Children and Young Adults With Friedreich's Ataxia

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 86 (actual)
• Sponsor: Amgen · Industry
• Sex: All
• Age: 10 Years – 26 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this phase 3 multi-center, open-label extension study is to evaluate the long-term safety of ACTIMMUNE® (interferon-γ 1b) in participants with Friedreich's Ataxia.

Detailed description

Participants who complete 26 weeks of blinded treatment in HZNP-ACT-301 (NCT02415127), will be eligible to enter this 6-month study. All participants will receive ACTIMMUNE® 3 times a week (TIW) for 26 weeks. In order to maintain the study blind in HZNP-ACT-301 (NCT02415127), all participants in this open-label extension study will undergo ACTIMMUNE® titration, regardless if they received ACTIMMUNE® or placebo in HZNP-ACT-301 (NCT02415127). The Week 26 Visit from HZNP-ACT-301 (NCT02415127) will serve as the Baseline Visit (Day 1) for this study. During the treatment period, additional clinic visits are scheduled at Weeks 4, 13, and 26; in between clinic visits, participants (and/or caregivers) will be monitored via emails/phone calls on a weekly basis until participants reach their maximum tolerated dose, and on a monthly basis thereafter. 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).

Timeline

• Start date: 2015-12-25
• Primary completion: 2017-03-21
• Completion: 2017-03-21
• First posted: 2015-11-02
• Last updated: 2024-12-19
• Results posted: 2018-05-18

Locations

4 sites across 1 country: United States

Regulatory

• FDA-regulated drug study