Phase 3
N=92
Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia
Friedreich's Ataxia
Bottom Line
View on ClinicalTrials.gov: NCT02415127 ↗Enrolled (actual)
92
Serious AEs
3.3%
Results posted
Dec 2017
Primary outcome: Primary: Change From Baseline to Week 26 in the Friedreich's Ataxia Rating Scale (FARS)-mNeuro Score — -0.6; -1.0 units on a scale — p=0.5442
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Interferon γ-1b (Drug); Placebo (Drug)
- Age
- Pediatric, Adult · 10+ yrs
- Sex
- All
- Sponsor
- Amgen
- Primary completion
- Nov 2016
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline to Week 26 in the Friedreich's Ataxia Rating Scale (FARS)-mNeuro Score |
-0.6; -1.0 | 0.5442 |
| SECONDARY Change From Baseline to Week 26 in Activities of Daily Living (ADL) Score |
0.64; 0.01 | — |
| SECONDARY Change From Baseline at Week 26 in Timed 25-Foot Walk (T25FW) |
-0.006; -0.003 | — |
| SECONDARY Number of FARS-mNeuro Responders and Non-Responders at Week 26 |
14; 16; 32; 28 | — |
| SECONDARY Change From Baseline to Week 26 in Total Friedreich Ataxia Rating Scale Score (FARStot) |
-0.2; -0.6 | — |
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 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 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.
Data sourced from ClinicalTrials.gov (NCT02415127). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.