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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
Sponsor:
Collaborator:
Friedreich's Ataxia Research Alliance
Information provided by (Responsible Party):
Horizon Pharma Ireland, Ltd., Dublin Ireland

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Friedreich's Ataxia
Intervention Drug: interferon γ-1b
Enrollment 38
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Interferon γ-1b
Hide Arm/Group Description Subcutaneous (SC) ACTIMMUNE® 3 times a week (TIW) at an individualized dose.
Period Title: Overall Study
Started 38
Completed 36
Not Completed 2
Reason Not Completed
Lost to Follow-up             1
Withdrawal by Subject             1
Arm/Group Title Interferon γ-1b
Hide Arm/Group Description SC ACTIMMUNE® TIW at an individualized dose
Overall Number of Baseline Participants 38
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 38 participants
16.4  (3.66)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 38 participants
Female
21
  55.3%
Male
17
  44.7%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 38 participants
Hispanic or Latino
1
   2.6%
Not Hispanic or Latino
37
  97.4%
Unknown or Not Reported
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 38 participants
American Indian or Alaska Native
0
   0.0%
Asian
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
0
   0.0%
White
37
  97.4%
More than one race
0
   0.0%
Unknown or Not Reported
1
   2.6%
1.Primary Outcome
Title Number of Participants With Treatment-Emergent Adverse Events (AEs), Serious Adverse Events (SAEs), and Discontinuations Due to AEs
Hide Description 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.
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.
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population, defined as all participants who received at least 1 dose of study drug after the Baseline Visit for Study HZNP-ACT-303.
Arm/Group Title Interferon γ-1b
Hide Arm/Group Description:
SC ACTIMMUNE® TIW at an individualized dose
Overall Number of Participants Analyzed 38
Measure Type: Number
Unit of Measure: participants
≥ 1 TEAE 21
≥ 1 Related TEAE 7
≥ 1 SAE 0
≥ 1 Related SAE 0
≥ 1 TEAE Leading to Discontinuation 0
≥ 1 TEAE Leading to Death 0
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.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Interferon γ-1b
Hide Arm/Group Description SC ACTIMMUNE® TIW at an individualized dose
All-Cause Mortality
Interferon γ-1b
Affected / at Risk (%)
Total   0/38 (0.00%) 
Show Serious Adverse Events Hide Serious Adverse Events
Interferon γ-1b
Affected / at Risk (%)
Total   0/38 (0.00%) 
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Interferon γ-1b
Affected / at Risk (%)
Total   17/38 (44.74%) 
Blood and lymphatic system disorders   
Neutropenia  1  2/38 (5.26%) 
Gastrointestinal disorders   
Vomiting  1  2/38 (5.26%) 
General disorders   
Fatigue  1  3/38 (7.89%) 
Infections and infestations   
Influenza  1  3/38 (7.89%) 
Nasopharyngitis  1  2/38 (5.26%) 
Upper respiratory tract infection  1  2/38 (5.26%) 
Nervous system disorders   
Headache  1  4/38 (10.53%) 
Skin and subcutaneous tissue disorders   
Skin discolouration  1  2/38 (5.26%) 
1
Term from vocabulary, MedDRA 16.1
Indicates events were collected by systematic assessment
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.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Horizon requests that any Investigator/institution that plans on presenting or publishing results provide written notification of their request a minimum of 60 days prior to presentation or publication. The Sponsor may request that Confidential Information be deleted and/or the publication be postponed in order to protect the Sponsors’ Intellectual Property rights.
Results Point of Contact
Name/Title: Julie Ball, MS, Executive Director, Clinical Development & Operations
Organization: Horizon Pharma Ireland, Ltd, Dublin, Ireland
Phone: (224) 383-3000
Responsible Party: Horizon Pharma Ireland, Ltd., Dublin Ireland
ClinicalTrials.gov Identifier: NCT02797080     History of Changes
Other Study ID Numbers: HZNP-ACT-303
First Submitted: June 2, 2016
First Posted: June 13, 2016
Results First Submitted: March 29, 2018
Results First Posted: May 1, 2018
Last Update Posted: May 1, 2018