Clinical trial NCT02415127: Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Friedreich's Ataxia
Keywords: Interferon gamma
Lead sponsor: Amgen
Sponsor class: INDUSTRY
Collaborators: name: Friedreich's Ataxia Research Alliance
First posted: 2015-04-14
Enrollment: 92
Sex: ALL
Minimum age (years): 10
Maximum age (years): 25
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

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.

Primary outcomes:
measure: Change From Baseline to Week 26 in the Friedreich's Ataxia Rating Scale (FARS)-mNeuro Score
timeFrame: Baseline, Week 26
Secondary outcomes:
measure: Change From Baseline to Week 26 in Activities of Daily Living (ADL) Score
timeFrame: Baseline, Week 26
measure: Change From Baseline at Week 26 in Timed 25-Foot Walk (T25FW)
timeFrame: Baseline, Week 26
measure: Number of FARS-mNeuro Responders and Non-Responders at Week 26
timeFrame: Week 26
measure: Change From Baseline to Week 26 in Total Friedreich Ataxia Rating Scale Score (FARStot)
timeFrame: Baseline, Week 26
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: University of California, Los Angeles Neurology Clinic
state: California
city: Gainesville
country: United States
facility: University of Florida - Clinical Research Center
state: Florida
city: Iowa City
country: United States
facility: University of Iowa Children's Hospital
state: Iowa
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
Study officials:
affiliation: Amgen
name: MD
role: STUDY_DIRECTOR
References: