Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia
Condition: Friedreich's Ataxia
MeSH terms: Ataxia; Cerebellar Ataxia; Friedreich Ataxia
Intervention: Interferon γ-1b; Placebo
Intervention description: 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).; 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.
Intervention MeSH terms: Interferons; Interferon-gamma
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Double (Participant, Investigator)
Enrollment: 92.0
Sex: All
Minimum age: 10 Years
Maximum age: 25 Years
Healthy volunteers: No
Keywords: Interferon gamma

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.
    

Eligibility criteria:

        Inclusion Criteria:

          -  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.

        Exclusion Criteria:

          -  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.