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Trial record 4 of 57 for:    Friedreich's Ataxia

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

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ClinicalTrials.gov Identifier: NCT02415127
Recruitment Status : Completed
First Posted : April 14, 2015
Results First Posted : December 8, 2017
Last Update Posted : December 8, 2017
Sponsor:
Collaborator:
Friedreich's Ataxia Research Alliance
Information provided by (Responsible Party):
Horizon Pharma Ireland, Ltd., Dublin Ireland

Study Type: Interventional
Study Design: Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Double (Participant, Investigator);   Primary Purpose: Treatment
Condition: Friedreich's Ataxia
Interventions: Drug: Interferon γ-1b
Drug: Placebo

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

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 γ-1b Subcutaneous (SC) doses of ACTIMMUNE® 3 times a week (TIW) for a total of 26 weeks.
Placebo SC doses of placebo TIW for a total of 26 weeks.

Participant Flow:   Overall Study
    Interferon γ-1b   Placebo
STARTED   47   45 
COMPLETED   46   45 
NOT COMPLETED   1   0 
Adverse Event                1                0 



  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 γ-1b SC doses of ACTIMMUNE® TIW for a total of 26 weeks.
Placebo SC doses of placebo TIW for a total of 26 weeks.
Total Total of all reporting groups

Baseline Measures
   Interferon γ-1b   Placebo   Total 
Overall Participants Analyzed 
[Units: Participants]
 47   45   92 
Age 
[Units: Years]
Mean (Standard Deviation)
 16.5  (4.41)   16.1  (3.75)   16.3  (4.08) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
     
Female      26  55.3%      26  57.8%      52  56.5% 
Male      21  44.7%      19  42.2%      40  43.5% 


  Outcome Measures

1.  Primary:   Change From Baseline to Week 26 in the Friedreich’s Ataxia Rating Scale (FARS)-mNeuro Score   [ Time Frame: Baseline, Week 26 ]

2.  Secondary:   Change From Baseline to Week 26 in Activities of Daily Living (ADL) Score   [ Time Frame: Baseline, Week 26 ]

3.  Secondary:   Change From Baseline at Week 26 in Timed 25-Foot Walk (T25FW)   [ Time Frame: Baseline, Week 26 ]

4.  Secondary:   Number of FARS-mNeuro Responders and Non-Responders at Week 26   [ Time Frame: Week 26 ]

5.  Secondary:   Change From Baseline to Week 26 in Total Friedreich Ataxia Rating Scale Score (FARStot)   [ Time Frame: Baseline, Week 26 ]


  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
No text entered.


  More Information

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.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Julie Ball, Executive Director Clinical Development & Operations
Organization: Horizon Pharma Ireland, Ltd. Dublin, Ireland
e-mail: clinicaltrials@horizonpharma.com



Responsible Party: Horizon Pharma Ireland, Ltd., Dublin Ireland
ClinicalTrials.gov Identifier: NCT02415127     History of Changes
Other Study ID Numbers: HZNP-ACT-301
First Submitted: February 12, 2015
First Posted: April 14, 2015
Results First Submitted: November 6, 2017
Results First Posted: December 8, 2017
Last Update Posted: December 8, 2017