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Long-Term Safety Extension Study of ACTIMMUNE® (Interferon γ-1b) in Children and Young Adults With Friedreich's Ataxia (STEADFAST)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02797080
Recruitment Status : Completed
First Posted : June 13, 2016
Results First Posted : May 1, 2018
Last Update Posted : May 1, 2018
Sponsor:
Collaborator:
Friedreich's Ataxia Research Alliance
Information provided by (Responsible Party):
Horizon Pharma Ireland, Ltd., Dublin Ireland

Study Description
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Brief Summary:
The purpose of this long term extension study is to evaluate the long-term safety of ACTIMMUNE® (interferon-γ 1b) in participants with Friedreich's Ataxia (FA).

Condition or disease Intervention/treatment Phase
Friedreich's Ataxia Drug: interferon γ-1b Phase 3

Detailed Description:
This is a multi-center, open-label, long-term safety extension study of ACTIMMUNE® in the treatment of FA in children and young adults. Participants who complete 26 weeks of treatment and the Week 28 Follow-Up Visit in HZNP-ACT-302 (NCT02593773) will be eligible to enter this long-term safety extension protocol. The Day 1 Visit of this study (HZNP-ACT-303) occurs on the same day as the Week 28 Follow-Up Visit for HZNP-ACT-302 (NCT02593773). Participants will be required to return for clinic visits at least every 6 months. The treatment duration is open-ended, and treatment will continue until ACTIMMUNE® is commercially available for the treatment of FA in the United States or until the Sponsor decides not to continue development for the treatment of FA.
Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 38 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Long-Term Safety Extension Study of ACTIMMUNE® (Interferon γ-1b) in Children and Young Adults With Friedreich's Ataxia
Actual Study Start Date : June 28, 2016
Actual Primary Completion Date : March 31, 2017
Actual Study Completion Date : March 31, 2017

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Arms and Interventions
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Arm Intervention/treatment
Experimental: interferon γ-1b
ACTIMMUNE® will be administered 3 times per week (TIW) by subcutaneous (SC) injection.
 Drug: interferon γ-1b
ACTIMMUNE® will be administered three times per week by subcutaneous injection. The initial dose will be individualized for each participant and will be determined by the investigator, provided that the initial dose does not exceed the maximum tolerated dose in HZNP-ACT-302 (NCT02593773). The investigator may subsequently adjust the dose for any participant if deemed clinically appropriate, provided that the dose does not exceed 100 μg/m².
Other Name: ACTIMMUNE®


Outcome Measures
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Primary Outcome Measures :
  1. Number of Participants With Treatment-Emergent Adverse Events (AEs), Serious Adverse Events (SAEs), and Discontinuations Due to AEs [ Time Frame: Baseline/Day 1 (Week 28 Follow-Up Visit for Study HZNP-ACT-302 ([NCT02593773]) through end of study; mean (SD) duration of treatment was 99.2 (58.48) days. ]
    An adverse event (AE) is any untoward medical occurrence, whether or not the event is considered related to the investigational product. A TEAE is any adverse change from the subject's baseline condition, including any laboratory test value abnormality judged as clinically significant by the investigator, that occurs on or after the date of the first dose of study drug administered at home and throughout the duration of the clinical study, whether the adverse event is considered related to the treatment or not. A serious AE (SAE) is an AE that results in death, is life-threatening, results in persistent or significant disability or incapacity, inpatient hospitalization or prolongation of an existing hospitalization, is a congenital anomaly or birth defect, or other medically important event.


Eligibility Criteria
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Ages Eligible for Study:   11 Years to 27 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Written informed consent and child assent, if applicable.
  • Completed 26 weeks of treatment and the Week 28 Follow-Up visit in Study HZNP-ACT-302 (NCT02593773).
  • 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 Week 26 of Study HZNP-ACT-302 (NCT02593773) 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:

  • If in the opinion of the Investigator, patients have a concomitant disease or condition that could interfere with the conduct of the study or potentially put the subject at unacceptable risk, the subject will be excluded from the study.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02797080


Locations
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United States, California
University of California, Los Angeles Neurology Clinic
Los Angeles, California, United States, 90038
United States, Florida
University of Florida Clinical Research Center
Gainesville, Florida, United States, 32610
United States, Iowa
University of Iowa Children's Hospital
Iowa City, Iowa, United States, 52242
United States, Pennsylvania
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Sponsors and Collaborators
Horizon Pharma Ireland, Ltd., Dublin Ireland
Friedreich's Ataxia Research Alliance
Investigators
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Study Director: Julie Ball, MS Horizon Pharma Ireland, Ltd., Dublin Ireland
  Study Documents (Full-Text)

Documents provided by Horizon Pharma Ireland, Ltd., Dublin Ireland:
Study Protocol  [PDF] April 5, 2016
Statistical Analysis Plan  [PDF] June 14, 2017

More Information
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Responsible Party: Horizon Pharma Ireland, Ltd., Dublin Ireland
ClinicalTrials.gov Identifier: NCT02797080    
Other Study ID Numbers: HZNP-ACT-303
First Posted: June 13, 2016    Key Record Dates
Results First Posted: May 1, 2018
Last Update Posted: May 1, 2018
Last Verified: March 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: Yes
Keywords provided by Horizon Pharma Ireland, Ltd., Dublin Ireland:
ACTIMMUNE
interferon y-1b
Additional relevant MeSH terms:
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Ataxia
Cerebellar Ataxia
Friedreich Ataxia
Interferons
Interferon-gamma
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
 Spinocerebellar Degenerations
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Mitochondrial Diseases
Metabolic Diseases
Antineoplastic Agents
Antiviral Agents
Anti-Infective Agents