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

March 29, 2018 updated by: Horizon Pharma Ireland, Ltd., Dublin Ireland
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).

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Actual)

38

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • California
      • Los Angeles, California, United States, 90038
        • University of California, Los Angeles Neurology Clinic
    • Florida
      • Gainesville, Florida, United States, 32610
        • University of Florida Clinical Research Center
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • University of Iowa Children's Hospital
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Children's Hospital of Philadelphia

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

9 years to 25 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / 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 Names:
  • ACTIMMUNE®

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
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.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Horizon Pharma Ireland, Ltd., Dublin Ireland

Collaborators

Friedreich's Ataxia Research Alliance

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 28, 2016

Primary Completion (Actual)

March 31, 2017

Study Completion (Actual)

March 31, 2017

Study Registration Dates

First Submitted

June 2, 2016

First Submitted That Met QC Criteria

June 7, 2016

First Posted (Estimate)

June 13, 2016

Study Record Updates

Last Update Posted (Actual)

May 1, 2018

Last Update Submitted That Met QC Criteria

March 29, 2018

Last Verified

March 1, 2018

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • HZNP-ACT-303

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

Undecided

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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