Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia

NCT02415127 | Completed Phase 3 Results DMC FDA Drug

• 1 other identifier: HZNP-ACT-301
• Study Type: interventional
• Target: 92
• Locations: 1 country
• Sites: 4

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.

Conditions

Friedreich's Ataxia

Keywords

Interferon gamma

Outcome Measures

Primary Outcomes (1)

• Change From Baseline to Week 26 in the Friedreich's Ataxia Rating Scale (FARS)-mNeuro Score

  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.

  Baseline, Week 26

Secondary Outcomes (4)

• Change From Baseline to Week 26 in Activities of Daily Living (ADL) Score

  Baseline, Week 26

• Change From Baseline at Week 26 in Timed 25-Foot Walk (T25FW)

  Baseline, Week 26

• Number of FARS-mNeuro Responders and Non-Responders at Week 26

  Week 26

• Change From Baseline to Week 26 in Total Friedreich Ataxia Rating Scale Score (FARStot)

  Baseline, Week 26

Study Arms (2)

Interferon γ-1b

EXPERIMENTAL

Approximately 45 participants will receive subcutaneous (SC) doses of ACTIMMUNE® 3 times a week (TIW) for a total of 26 weeks.

Drug: Interferon γ-1b

Placebo

PLACEBO COMPARATOR

Approximately 45 participants will receive SC doses of placebo TIW for a total of 26 weeks.

Drug: Placebo

Interventions

Interferon γ-1b DRUG

The study drug dose is planned to be escalated on a weekly basis over the first 4 weeks of treatment (from 10 µg/m² to 25, 50, and 100 µg/m²). The dose may be reduced, interrupted, or held based on tolerability. By Week 13, all participants are to be on a stable tolerated dose of study drug in order to continue study participation; the dose may not be further increased after week 13, however, it may be reduced on a case-by-case basis to manage drug-related adverse events (AEs).

Also known as: ACTIMMUNE®

Interferon γ-1b

Placebo DRUG

The volume of placebo is planned to correspond with volume of study drug that would be given to the participant if the participant was randomized to the study drug arm.

Placebo

Eligibility Criteria

• Age: 10 Years - 25 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.
• FA confirmed by genetic testing with two expanded guanine-adenine-adenine (GAA) repeats.
• FA functional stage of \>1 to \<5 and ability to walk 25 feet with or without an assistive device.
• Male or female subject between the ages of 10 and 25 years, inclusive.
• 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 serum pregnancy test result at Screening, a negative urine pregnancy test result at Baseline, 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:

• Any unstable illness that in the investigator's opinion precludes participation in the study.
• Use of any investigational product within 30 days prior to randomization.
• A history of substance abuse.
• History of hypersensitivity to interferon (IFN)-ɣ or E. coli-derived products.
• Presence of moderate or severe renal disease (estimated creatinine clearance \<50 mL/min) or hepatic disease (aspartate aminotransferase \[AST\] or alanine aminotransferase \[ALT\] \>2x the upper limit of normal) as evidenced by laboratory results at Screening.
• Clinically significant abnormal white blood cell count, hemoglobin, or platelet count as evidenced by laboratory test results at Screening.

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, 32603, 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

Results Point of Contact

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

clinicaltrials@horizonpharma.com

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: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 12, 2015
• First Posted: April 14, 2015
• Study Start: June 1, 2015
• Primary Completion: November 1, 2016
• Study Completion: November 1, 2016
• Last Updated: December 19, 2024
• Results First Posted: December 8, 2017

Record last verified: 2024-11

Locations

US (4)