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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

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
12 subjects were screened and all were enrolled in the study between September - December 2013.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Interferon Gamma-1b (ACTIMMUNE) 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.

Participant Flow:   Overall Study
    Interferon Gamma-1b (ACTIMMUNE)
STARTED   12 
COMPLETED   10 [1] 
NOT COMPLETED   2 
Physician Decision                2 
[1] Prior to completion, 2 subjects were withdrawn by the PI for failure to adhere to study protocol.



  Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Interferon Gamma-1b (ACTIMMUNE) 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.

Baseline Measures
   Interferon Gamma-1b (ACTIMMUNE) 
Overall Participants Analyzed 
[Units: Participants]
 12 
Age 
[Units: Participants]
 
<=18 years   12 
Between 18 and 65 years   0 
>=65 years   0 
Age 
[Units: Years]
Mean (Full Range)
 12 
 (8 to 17) 
Gender 
[Units: Participants]
 
Female   3 
Male   9 
Ethnicity (NIH/OMB) 
[Units: Participants]
 
Hispanic or Latino   0 
Not Hispanic or Latino   12 
Unknown or Not Reported   0 
Race (NIH/OMB) 
[Units: Participants]
 
American Indian or Alaska Native   0 
Asian   1 
Native Hawaiian or Other Pacific Islander   0 
Black or African American   0 
White   11 
More than one race   0 
Unknown or Not Reported   0 
Region of Enrollment 
[Units: Participants]
 
United States   12 


  Outcome Measures

1.  Primary:   Change in Whole Blood Frataxin Levels   [ Time Frame: Frataxin levels were measured at the beginning and conclusion of treatment (baseline and 12 weeks) ]

2.  Secondary:   Change in Total Friedreich Ataxia Rating Scale (FARS) Score   [ Time Frame: FARS score was calculated at the beginning and conclusion of treatment (baseline and 12 weeks) ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
This study was performed in a small population of children with Friedreich ataxia (n=12) and without a placebo arm.


  More Information

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
e-mail: lynch@email.chop.edu



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