Long-Term Safety Extension Study of ACTIMMUNE® (Interferon γ-1b) in Children and Young Adults With Friedreich's Ataxia (STEADFAST)
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ClinicalTrials.gov Identifier: NCT02797080 |
Recruitment Status :
Completed
First Posted : June 13, 2016
Results First Posted : May 1, 2018
Last Update Posted : May 1, 2018
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Condition or disease | Intervention/treatment | Phase |
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Friedreich's Ataxia | Drug: interferon γ-1b | Phase 3 |
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 |

Arm | Intervention/treatment |
---|---|
Experimental: interferon γ-1b
ACTIMMUNE® will be administered 3 times per week (TIW) by subcutaneous (SC) injection.
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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® |
- 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.

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 11 Years to 27 Years (Child, Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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.

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
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 |
Study Director: | Julie Ball, MS | Horizon Pharma Ireland, Ltd., Dublin Ireland |
Documents provided by Horizon Pharma Ireland, Ltd., Dublin Ireland:
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 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
ACTIMMUNE interferon y-1b |
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 |