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

NCT02797080 | Completed Phase 3 Results FDA Drug

• 1 other identifier: HZNP-ACT-303
• Study Type: interventional
• Target: 38
• Locations: 1 country
• Sites: 4

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).

Conditions

Friedreich's Ataxia

Keywords

ACTIMMUNE; interferon y-1b

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Treatment-Emergent Adverse Events (AEs), Serious Adverse Events (SAEs), and Discontinuations Due to AEs

  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.

Study Arms (1)

interferon γ-1b

EXPERIMENTAL

ACTIMMUNE® will be administered 3 times per week (TIW) by subcutaneous (SC) injection.

Drug: interferon γ-1b

Interventions

interferon γ-1b DRUG

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.

Also known as: ACTIMMUNE®

interferon γ-1b

Eligibility Criteria

• Age: 11 Years - 27 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Amgen: lead
• Friedreich's Ataxia Research Alliance: collaborator

Study Sites (4)

University of California, Los Angeles Neurology Clinic

Los Angeles, California, 90038, United States

Location

University of Florida Clinical Research Center

Gainesville, Florida, 32610, United States

Location

University of Iowa Children's Hospital

Iowa City, Iowa, 52242, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

MeSH Terms

Conditions

Friedreich Ataxia

Interventions

interferon gamma-1b

Condition Hierarchy (Ancestors)

Spinocerebellar Degenerations; Cerebellar Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Mitochondrial Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Limitations and Caveats

This study was stopped by the Sponsor because the development of ACTIMMUNE® for the treatment of FA was discontinued after Study HZNP-ACT-301 (NCT02415127) failed to meet its primary efficacy endpoint.

Results Point of Contact

• Title: Julie Ball, MS, Executive Director, Clinical Development & Operations
• Organization: Horizon Pharma Ireland, Ltd, Dublin, Ireland

clinicaltrials@horizonpharma.com

(224) 383-3000

Study Officials

• MD

  Amgen

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 2, 2016
• First Posted: June 13, 2016
• Study Start: June 28, 2016
• Primary Completion: March 31, 2017
• Study Completion: March 31, 2017
• Last Updated: December 10, 2024
• Results First Posted: May 1, 2018

Record last verified: 2024-12

Locations

US (4)