Trial · NCT02415127

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

NCT02415127, Phase 3, Completed, Amgen; results first posted 2017-12-08

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia. Trials, record NCT02415127. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02415127

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia
NCT number: NCT02415127
Status: Completed
Phases: Phase 3
Enrollment: 92 participants, actual
Lead sponsor: Amgen
Conditions: Friedreich's Ataxia
Interventions: Interferon γ-1b, Placebo
First posted: 2015-04-14
Last updated: 2024-12-19
Results first posted: 2017-12-08

Source ClinicalTrials.gov

Registered outcomes5

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Change From Baseline to Week 26 in the Friedreich's Ataxia Rating Scale (FARS)-mNeuro Score	Baseline, Week 26	The FARS assessment includes neurological signs that specifically reflect neural substrates affected in FA. Based on a neurological examination, bulbar, upper limb, lower limb, peripheral nerve, and upright stability/gait functions were assessed. The FARS-mNeuro score excludes the peripheral nervous system subscale score and the facial and tongue atrophy and fasciculations from the bulbar subscale score. Scores range from 0 (normal) to 93 (most impairment). A negative change from baseline is an improvement.
Secondary	Change From Baseline to Week 26 in Activities of Daily Living (ADL) Score	Baseline, Week 26	Participants and/or their caregivers rated 9 areas of daily living skills (speech, swallowing, cutting food and handling utensils, dressing, personal hygiene, falling, walking, quality of sitting position, and bladder function) on a 5-point scale (0=normal, 4=greatest loss of function) with allowable increments of 0.5 if the participant or caregiver strongly felt that a task falls between 2 scores. ADL scores can range from 0 (normal) to 36 (greatest loss of function). A negative change from baseline indicates improvement.
Secondary	Change From Baseline at Week 26 in Timed 25-Foot Walk (T25FW)	Baseline, Week 26	The T25FW is a quantitative measure of lower extremity function. Participants are directed to 1 end of a clearly marked 25-foot course and instructed to walk 25 feet as quickly as possible, but safely. The task is immediately administered again by having the participant walk back the same distance, and the score for the test is the average of the 2 walks (after reciprocal transformation). Participants may use assistive devices when performing this task, with the same assistive device used at each assessment. A negative change from Baseline indicates improvement.
Secondary	Number of FARS-mNeuro Responders and Non-Responders at Week 26	Week 26	A participant was considered a responder if they had an improvement (decrease) of at least 3 points from Baseline at Week 26 for the FARS-mNeuro score. The FARS assessment includes neurological signs that specifically reflect neural substrates affected in FA. Based on a neurological examination, bulbar, upper limb, lower limb, peripheral nerve, and upright stability/gait functions were assessed. The FARS-mNeuro score excludes the peripheral nervous system subscale score and the facial and tongue atrophy and fasciculations from the bulbar subscale score. Scores range from 0 (normal) to 93 (most impairment).
Secondary	Change From Baseline to Week 26 in Total Friedreich Ataxia Rating Scale Score (FARStot)	Baseline, Week 26	The FARS assessment includes neurological signs that specifically reflect neural substrates affected in FA. Based on a neurological examination, bulbar, upper limb, lower limb, peripheral nerve, and upright stability/gait functions are assessed. FARStot scores range from 0 (normal) to 125 (most impairment). A negative change from baseline indicates improvement.

Source ClinicalTrials.gov

Outcomes5

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Change From Baseline to Week 26 in the Friedreich's Ataxia Rating Scale (FARS)-mNeuro Score	Baseline, Week 26	Intent-to-Treat (ITT) Population: All randomized participants with a valid baseline (including Screening) and at least 1 valid post baseline measurement in the primary efficacy outcome (FARS-mNeuro) and at Week 26. A valid FARS-mNeuro score was defined as no missing values in the questionnaire.	units on a scale	MEAN
SECONDARY	Change From Baseline to Week 26 in Activities of Daily Living (ADL) Score	Baseline, Week 26	ITT Population: All randomized participants with a valid baseline (including Screening) and at least 1 valid post baseline measurement in the primary efficacy outcome (FARS-mNeuro) and ADL data at Baseline and Week 26. A valid FARS-mNeuro score was defined as no missing values in the questionnaire.	units on a scale	MEAN
SECONDARY	Change From Baseline at Week 26 in Timed 25-Foot Walk (T25FW)	Baseline, Week 26	ITT Population: All randomized participants with a valid baseline (including Screening) and at least 1 valid post baseline measurement in the primary efficacy outcome (FARS-mNeuro), and T25FW data at Baseline and Week 26. A valid FARS-mNeuro score was defined as no missing values in the questionnaire.	1/seconds	MEAN
SECONDARY	Number of FARS-mNeuro Responders and Non-Responders at Week 26	Week 26	ITT Population: All randomized participants with a valid baseline (including Screening) and at least 1 valid post baseline measurement in the primary efficacy outcome (FARS-mNeuro) and data at Week 26. A valid FARS-mNeuro score was defined as no missing values in the questionnaire.	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Change From Baseline to Week 26 in Total Friedreich Ataxia Rating Scale Score (FARStot)	Baseline, Week 26	ITT Population: All randomized participants with a valid baseline (including Screening) and at least 1 valid post baseline measurement in the primary efficacy outcome (FARS-mNeuro) and FARStot data at Baseline and Week 26. A valid FARS-mNeuro score was defined as no missing values in the questionnaire.	units on a scale	MEAN

Source ClinicalTrials.gov

Results by group12

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
Change From Baseline to Week 26 in the Friedreich's Ataxia Rating Scale (FARS)-mNeuro Score	Interferon γ-1b		-0.6	Standard Deviation 4.61	units on a scale
Change From Baseline to Week 26 in the Friedreich's Ataxia Rating Scale (FARS)-mNeuro Score	Placebo		-1.0	Standard Deviation 4.41	units on a scale
Change From Baseline to Week 26 in Activities of Daily Living (ADL) Score	Interferon γ-1b		0.64	Standard Deviation 2.938	units on a scale
Change From Baseline to Week 26 in Activities of Daily Living (ADL) Score	Placebo		0.01	Standard Deviation 2.595	units on a scale
Change From Baseline at Week 26 in Timed 25-Foot Walk (T25FW)	Interferon γ-1b		-0.006	Standard Deviation 0.0247	1/seconds
Change From Baseline at Week 26 in Timed 25-Foot Walk (T25FW)	Placebo		-0.003	Standard Deviation 0.0181	1/seconds
Number of FARS-mNeuro Responders and Non-Responders at Week 26	Interferon γ-1b	Responder	14		Participants
Number of FARS-mNeuro Responders and Non-Responders at Week 26	Placebo	Responder	16		Participants
Number of FARS-mNeuro Responders and Non-Responders at Week 26	Interferon γ-1b	Non-responder	32		Participants
Number of FARS-mNeuro Responders and Non-Responders at Week 26	Placebo	Non-responder	28		Participants

1 to 10 of 12Source ClinicalTrials.gov

See all 12 results by group

Participant flow6

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Interferon γ-1b	47
Overall Study	STARTED	Placebo	45
Overall Study	COMPLETED	Interferon γ-1b	46
Overall Study	COMPLETED	Placebo	45
Overall Study	NOT COMPLETED	Interferon γ-1b	1
Overall Study	NOT COMPLETED	Placebo	0

Source ClinicalTrials.gov

Adverse events72

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk
other	Neutropenia	Blood and lymphatic system disorders	Interferon γ-1b	7	47
other	Neutropenia	Blood and lymphatic system disorders	Placebo	1	45
other	Abdominal pain upper	Gastrointestinal disorders	Interferon γ-1b	3	47
other	Abdominal pain upper	Gastrointestinal disorders	Placebo	5	45
other	Constipation	Gastrointestinal disorders	Interferon γ-1b	1	47
other	Constipation	Gastrointestinal disorders	Placebo	3	45
other	Diarrhoea	Gastrointestinal disorders	Interferon γ-1b	3	47
other	Diarrhoea	Gastrointestinal disorders	Placebo	2	45
other	Nausea	Gastrointestinal disorders	Interferon γ-1b	18	47
other	Nausea	Gastrointestinal disorders	Placebo	11	45

1 to 10 of 72Source ClinicalTrials.gov

See all 72 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Interferon γ-1b
Total, all-cause mortality	Placebo
Total, other adverse events	Interferon γ-1b	45	47
Total, other adverse events	Placebo	37	45
Total, serious adverse events	Interferon γ-1b	1	47
Total, serious adverse events	Placebo	2	45

Source ClinicalTrials.gov

Papers0

The papers the trial lists on ClinicalTrials.gov.

None in this release.

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons1

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison
Interferon γ-1b vs Placebo: serious adverse events

Source Trial comparisons

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT02415127

GET https://conductscience.org/api/trials/v1/NCT02415127/events?page=2

API reference For AI assistants