Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia (STEADFAST)
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ClinicalTrials.gov Identifier: NCT02415127 |
Recruitment Status :
Completed
First Posted : April 14, 2015
Results First Posted : December 8, 2017
Last Update Posted : December 8, 2017
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Sponsor:
Horizon Pharma Ireland, Ltd., Dublin Ireland
Collaborator:
Friedreich's Ataxia Research Alliance
Information provided by (Responsible Party):
Horizon Pharma Ireland, Ltd., Dublin Ireland
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Tracking Information | ||||
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First Submitted Date ICMJE | February 12, 2015 | |||
First Posted Date ICMJE | April 14, 2015 | |||
Results First Submitted Date ICMJE | November 6, 2017 | |||
Results First Posted Date ICMJE | December 8, 2017 | |||
Last Update Posted Date | December 8, 2017 | |||
Study Start Date ICMJE | June 2015 | |||
Actual Primary Completion Date | November 2016 (Final data collection date for primary outcome measure) | |||
Current Primary Outcome Measures ICMJE |
Change From Baseline to Week 26 in the Friedreich's Ataxia Rating Scale (FARS)-mNeuro Score [ Time Frame: Baseline, Week 26 ] The FARS assessment includes neurological signs that specifically reflect neural substrates affected in FA. Based on a neurological examination, bulbar, upper limb, lower limb, peripheral nerve, and upright stability/gait functions were assessed. The FARS-mNeuro score excludes the peripheral nervous system subscale score and the facial and tongue atrophy and fasciculations from the bulbar subscale score. Scores range from 0 (normal) to 93 (most impairment). A negative change from baseline is an improvement.
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Original Primary Outcome Measures ICMJE |
Change in neurological outcome measured by Friedreich's Ataxia Rating Scale (FARS) [ Time Frame: Baseline, Week 13, Week 26 ] To evaluate the effect of ACTIMMUNE versus placebo on the change from Baseline to Week 26 in neurological outcome as measured by FARS excluding the peripheral nervous system subscale score and the facial and tongue atrophy and fasciculations from the bulbar subscale score (FARS-mNeuro score).
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Change History | ||||
Current Secondary Outcome Measures ICMJE |
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Original Secondary Outcome Measures ICMJE |
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Current Other Pre-specified Outcome Measures | Not Provided | |||
Original Other Pre-specified Outcome Measures |
Pharmacokinetic profile analysis of ACTIMMUNE [ Time Frame: Week 4, Week 13, and Week 26 ] PK profile will be characterized. Approximately 40 patients will remain confined overnight at week 4 for serial blood collections. All patients will contribute a single blood sample at weeks 13 and 26 for PK analysis.
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Descriptive Information | ||||
Brief Title ICMJE | Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia | |||
Official Title ICMJE | 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 | |||
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 | Not Provided | |||
Study Type ICMJE | Interventional | |||
Study Phase ICMJE | Phase 3 | |||
Study Design ICMJE | Allocation: Randomized Intervention Model: Parallel Assignment Masking: Double (Participant, Investigator) Primary Purpose: Treatment |
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Condition ICMJE | Friedreich's Ataxia | |||
Intervention ICMJE |
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Study Arms ICMJE |
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Publications * | Not Provided | |||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | ||||
Recruitment Status ICMJE | Completed | |||
Actual Enrollment ICMJE |
92 | |||
Original Estimated Enrollment ICMJE |
90 | |||
Actual Study Completion Date ICMJE | November 2016 | |||
Actual Primary Completion Date | November 2016 (Final data collection date for primary outcome measure) | |||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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Sex/Gender ICMJE |
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Ages ICMJE | 10 Years to 25 Years (Child, Adult) | |||
Accepts Healthy Volunteers ICMJE | No | |||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | |||
Listed Location Countries ICMJE | United States | |||
Removed Location Countries | ||||
Administrative Information | ||||
NCT Number ICMJE | NCT02415127 | |||
Other Study ID Numbers ICMJE | HZNP-ACT-301 | |||
Has Data Monitoring Committee | Yes | |||
U.S. FDA-regulated Product |
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IPD Sharing Statement ICMJE | Not Provided | |||
Current Responsible Party | Horizon Pharma Ireland, Ltd., Dublin Ireland | |||
Original Responsible Party | Same as current | |||
Current Study Sponsor ICMJE | Horizon Pharma Ireland, Ltd., Dublin Ireland | |||
Original Study Sponsor ICMJE | Same as current | |||
Collaborators ICMJE | Friedreich's Ataxia Research Alliance | |||
Investigators ICMJE |
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PRS Account | Horizon Pharma Ireland, Ltd., Dublin Ireland | |||
Verification Date | December 2017 | |||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |