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

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

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Recruitment Details

Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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Pre-Assignment Details

Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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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

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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.
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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

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1. Primary:

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

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2. Secondary:

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

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3. Secondary:

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

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4. Secondary:

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

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5. Secondary:

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

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  Serious Adverse Events

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  Other Adverse Events

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  Limitations and Caveats

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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
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  More Information

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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: 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. 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. Other disclosure agreement that restricts the right of the PI to discuss or publish trial results after the trial is completed.; Restriction Description: 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, 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