Phase 3
Completed N=86
Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia Study
Source: ClinicalTrials.gov NCT02593773 ↗Enrolled (actual)
86
Serious AEs
4.7%
Results posted
May 2018
Primary outcomePrimary: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Discontinuations Due to AEs — 78; 61; 4; 0 participants
◆ Published Evidence
No publication linked
No peer-reviewed publication reporting this trial's results has been linked yet. This can indicate results are unpublished — a known publication-bias signal. We re-check periodically.
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.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Discontinuations Due to AEs |
78; 61; 4; 0; 1; 1 | — |
| PRIMARY Number of Participants With Positive/Negative Neutralizing Antibody (NAb) and Anti-Drug Antibody (ADA) Tests |
85; 1; 1; 0; 84; 0 | — |
| PRIMARY Change From Baseline to Week 26 in the Friedreich's Ataxia Rating Scale (FARS)-mNeuro Score |
— | — |
| SECONDARY Change From Baseline to Week 26 in Activities of Daily Living (ADL) Score |
— | — |
| SECONDARY Change From Baseline at Week 26 in Timed 25-Foot Walk (T25FW) |
— | — |
| SECONDARY Number of FARS-mNeuro Responders and Non-Responders at Week 26 |
— | — |
| SECONDARY Change From Baseline to Week 26 in Total Friedreich Ataxia Rating Scale Score (FARStot) |
— | — |
Eligibility Criteria
Inclusion Criteria
- Written informed consent and child assent, if applicable.
- Completed 26 weeks of blinded treatment in Study HZNP-ACT-301 (NCT02415127).
- 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 urine pregnancy test result at Baseline/Day 1 (Week 26 of Study HZNP-ACT-301 [NCT02415127]), 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
- Subjects will be ineligible if, in the opinion of the Investigator, they are unlikely to comply with the study protocol or have a concomitant disease or condition that could interfere with the conduct of the study or potentially put the subject at unacceptable risk.
NOTE: Additional inclusion/exclusion criteria may apply.
Data sourced from ClinicalTrials.gov (NCT02593773). 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. Informational only — not medical advice.