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Interferon Gamma-1b in Friedreich Ataxia (FRDA)

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ClinicalTrials.gov Identifier: NCT01965327
Recruitment Status : Completed
First Posted : October 18, 2013
Results First Posted : April 30, 2015
Last Update Posted : May 15, 2015
Sponsor:
Collaborators:
Friedreich's Ataxia Research Alliance
Vidara Therapeutics Research Ltd
Information provided by (Responsible Party):
Children's Hospital of Philadelphia

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Friedreich Ataxia
Intervention Drug: Interferon Gamma-1b
Enrollment 12
Recruitment Details 12 subjects were screened and all were enrolled in the study between September - December 2013.
Pre-assignment Details  
Arm/Group Title Interferon Gamma-1b (ACTIMMUNE)
Hide Arm/Group Description All individuals in this study were treated with interferon gamma-1b (ACTIMMUNE). Doses were administered via subcutaneous injections three times per week for 12 weeks. Dose-escalation schedule was completed by all subjects as follows: For the first two weeks subjects took10 mcg/m2 of interferon gamma-1b (IFN-g-1b), then the dose was escalated to 25 mcg/m2 of IFN-g-1b for weeks three and four of the study, finally, the dose was escalated to 50 mcg/m2 of IFN-gamma-1b for the last eight weeks of the study, which is the current dose approved by the FDA for children.
Period Title: Overall Study
Started 12
Completed 10 [1]
Not Completed 2
Reason Not Completed
Physician Decision             2
[1]
Prior to completion, 2 subjects were withdrawn by the PI for failure to adhere to study protocol.
Arm/Group Title Interferon Gamma-1b (ACTIMMUNE)
Hide Arm/Group Description All individuals in this study were treated with interferon gamma-1b (ACTIMMUNE). Doses were administered via subcutaneous injections three times per week for 12 weeks. Dose-escalation schedule was completed by all subjects as follows: For the first two weeks subjects took10 mcg/m2 of interferon gamma-1b (IFN-g-1b), then the dose was escalated to 25 mcg/m2 of IFN-g-1b for weeks three and four of the study, finally, the dose was escalated to 50 mcg/m2 of IFN-gamma-1b for the last eight weeks of the study, which is the current dose approved by the FDA for children.
Overall Number of Baseline Participants 12
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 12 participants
<=18 years
12
 100.0%
Between 18 and 65 years
0
   0.0%
>=65 years
0
   0.0%
Age, Continuous  
Mean (Full Range)
Unit of measure:  Years
Number Analyzed 12 participants
12
(8 to 17)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 12 participants
Female
3
  25.0%
Male
9
  75.0%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 12 participants
Hispanic or Latino
0
   0.0%
Not Hispanic or Latino
12
 100.0%
Unknown or Not Reported
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 12 participants
American Indian or Alaska Native
0
   0.0%
Asian
1
   8.3%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
0
   0.0%
White
11
  91.7%
More than one race
0
   0.0%
Unknown or Not Reported
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 12 participants
12
1.Primary Outcome
Title Change in Whole Blood Frataxin Levels
Hide Description Assessment of the change in whole blood frataxin levels as assessed by lateral flow assay using an immunoassay for frataxin. Frataxin levels in the blood were measured at each study visit. Change in frataxin level at the end of treatment (week 12) relative to frataxin level at baseline was analyzed.
Time Frame Frataxin levels were measured at the beginning and conclusion of treatment (baseline and 12 weeks)
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
The primary analysis was based on an intent-to-treat approach including all subjects who had baseline frataxin blood levels collected.
Arm/Group Title Interferon Gamma-1b (ACTIMMUNE)
Hide Arm/Group Description:
All individuals in this study were treated with interferon gamma-1b (ACTIMMUNE). Doses were administered via subcutaneous injections three times per week for 12 weeks. Dose-escalation schedule was completed by all subjects as follows: For the first two weeks subjects took10 mcg/m2 of interferon gamma-1b (IFN-g-1b), then the dose was escalated to 25 mcg/m2 of IFN-g-1b for weeks three and four of the study, finally, the dose was escalated to 50 mcg/m2 of IFN-gamma-1b for the last eight weeks of the study, which is the current dose approved by the FDA for children.
Overall Number of Participants Analyzed 12
Mean (Standard Deviation)
Unit of Measure: percentage of baseline frataxin level
-1.5  (1.9)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Interferon Gamma-1b (ACTIMMUNE)
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.027
Comments [Not Specified]
Method t-test, 2 sided
Comments Missing data were imputed by last observation carried forward.
2.Secondary Outcome
Title Change in Total Friedreich Ataxia Rating Scale (FARS) Score
Hide Description The Friedreich Ataxia Rating Scale (FARS) is neurological rating scale specifically developed and validated for FRDA. The FARS includes assessments of stance, gait, upper and lower limb coordination, speech, proprioception and strength. In addition to the standard neurological examination, the FARS contains three quantitative performance measures and a component that assesses activities of daily living (ADL). Quantitative performance measures include the nine-hole peg test, and a timed 25-foot walk. FARS scores correlate significantly with functional disability, activities of daily living scores and disease duration. The scores from the three subscales are added to generate a total score ranging from 0 to 159, with a higher score indicating a greater level of disability.
Time Frame FARS score was calculated at the beginning and conclusion of treatment (baseline and 12 weeks)
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
The analysis was based on an intent-to-treat approach and included all subjects with baseline FARS assessment.
Arm/Group Title Interferon Gamma-1b (ACTIMMUNE)
Hide Arm/Group Description:
All individuals in this study were treated with interferon gamma-1b (ACTIMMUNE). Doses were administered via subcutaneous injections three times per week for 12 weeks. Dose-escalation schedule was completed by all subjects as follows: For the first two weeks subjects took10 mcg/m2 of interferon gamma-1b (IFN-g-1b), then the dose was escalated to 25 mcg/m2 of IFN-g-1b for weeks three and four of the study, finally, the dose was escalated to 50 mcg/m2 of IFN-gamma-1b for the last eight weeks of the study, which is the current dose approved by the FDA for children.
Overall Number of Participants Analyzed 12
Mean (Standard Deviation)
Unit of Measure: units on a scale
-4.98  (3.6)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Interferon Gamma-1b (ACTIMMUNE)
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0078
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Time Frame 16 weeks
Adverse Event Reporting Description All subjects entered into the study (12) were included in the safety analysis. The frequencies of adverse events were summarized by type, body system, severity and relationship to study drug.
 
Arm/Group Title Interferon Gamma-1b (ACTIMMUNE)
Hide Arm/Group Description All individuals in this study were treated with interferon gamma-1b (ACTIMMUNE). Doses were administered via subcutaneous injections three times per week for 12 weeks. Dose-escalation schedule was completed by all subjects as follows: For the first two weeks subjects took10 mcg/m2 of interferon gamma-1b (IFN-g-1b), then the dose was escalated to 25 mcg/m2 of IFN-g-1b for weeks three and four of the study, finally, the dose was escalated to 50 mcg/m2 of IFN-gamma-1b for the last eight weeks of the study, which is the current dose approved by the FDA for children.
All-Cause Mortality
Interferon Gamma-1b (ACTIMMUNE)
Affected / at Risk (%)
Total   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Interferon Gamma-1b (ACTIMMUNE)
Affected / at Risk (%)
Total   0/12 (0.00%) 
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Interferon Gamma-1b (ACTIMMUNE)
Affected / at Risk (%)
Total   11/12 (91.67%) 
Blood and lymphatic system disorders   
Decreased Platelet Count   1/12 (8.33%) 
Gastrointestinal disorders   
Absominal Pain   2/12 (16.67%) 
Diarrhea   1/12 (8.33%) 
Nausea   7/12 (58.33%) 
Vomiting   4/12 (33.33%) 
General disorders   
Chills   1/12 (8.33%) 
Fatigue   3/12 (25.00%) 
Pyrexia   2/12 (16.67%) 
Decreased Appetite   1/12 (8.33%) 
Myalgia   4/12 (33.33%) 
Headache   3/12 (25.00%) 
Injection Site Bruising   4/12 (33.33%) 
Injection Site Dermititis   1/12 (8.33%) 
Injection Site Discoloration   1/12 (8.33%) 
Injection Site Edema   1/12 (8.33%) 
Infections and infestations   
Influenza like illness   6/12 (50.00%) 
Investigations   
Elevated transaminases   1/12 (8.33%) 
Injection Site Pain   3/12 (25.00%) 
Respiratory, thoracic and mediastinal disorders   
Epistaxis   1/12 (8.33%) 
Sinus Congestion   1/12 (8.33%) 
Skin and subcutaneous tissue disorders   
Contusion   1/12 (8.33%) 
Indicates events were collected by systematic assessment
This study was performed in a small population of children with Friedreich ataxia (n=12) and without a placebo arm.
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: David Lynch, MD, PhD
Organization: Children's Hospital of Philadelphia
Phone: 215-590-1719
Responsible Party: Children's Hospital of Philadelphia
ClinicalTrials.gov Identifier: NCT01965327     History of Changes
Other Study ID Numbers: 13-010121
13-010121 ( Other Identifier: Children's Hospital of Philadelphia )
First Submitted: August 27, 2013
First Posted: October 18, 2013
Results First Submitted: April 15, 2015
Results First Posted: April 30, 2015
Last Update Posted: May 15, 2015