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

Completed · Phase 3 · Has a placebo group

Conditions studied: Friedreich's Ataxia

In brief

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.

Key facts

Study ID

NCT02415127

Run by

Amgen

People needed

92

Starts

2015-06-01

Expected to finish

2016-11-01

Last updated by the study team

2024-12-19

Who can join

Age: 10 and older, up to 25. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Written informed consent and child assent, if applicable.
• FA confirmed by genetic testing with two expanded guanine-adenine-adenine (GAA) repeats.
• FA functional stage of >1 to <5 and ability to walk 25 feet with or without an assistive device.
• Male or female subject between the ages of 10 and 25 years, inclusive.
• If female, the subject is not pregnant or lactating or intending to become pregnant during the study, or within 30 days after the last dose of study drug. Female subjects of child-bearing potential must have a negative serum pregnancy test result at Screening, a negative urine pregnancy test result at Baseline, and agree to use a reliable method of contraception throughout the study and for 30 days after the last dose of study drug.

You may not qualify if…

• Any unstable illness that in the investigator's opinion precludes participation in the study.
• Use of any investigational product within 30 days prior to randomization.
• A history of substance abuse.
• Presence of clinically significant cardiac disease (as determined by the investigator based on electrocardiogram [ECG] and echocardiogram results at Screening). Specifically, an ejection fraction of <40% or a prolonged QT interval (>50% of cycle duration) will result in exclusion. If the investigator notes any other clinically significant abnormalities on the ECG or echocardiogram, the subject may be eligible if they are provided clearance from a cardiologist.
• History of hypersensitivity to interferon (IFN)-ɣ or E. coli-derived products.
• Presence of moderate or severe renal disease (estimated creatinine clearance <50 mL/min) or hepatic disease (aspartate aminotransferase [AST] or alanine aminotransferase [ALT] >2x the upper limit of normal) as evidenced by laboratory results at Screening.
• Clinically significant abnormal white blood cell count, hemoglobin, or platelet count as evidenced by laboratory test results at Screening.

Where it is running

• University of California, Los Angeles Neurology Clinic — Los Angeles, California, United States
• University of Florida - Clinical Research Center — Gainesville, Florida, United States
• University of Iowa Children's Hospital — Iowa City, Iowa, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States

Full record on ClinicalTrials.gov

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