Trial · NCT02593773
Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia Study
NCT02593773, Phase 3, Completed, Amgen; results first posted 2018-05-18
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia Study. Trials, record NCT02593773. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02593773
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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 Study
- NCT number
- NCT02593773
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 86 participants, actual
- Lead sponsor
- Amgen
- Conditions
- Friedreich's Ataxia
- Interventions
- Interferon γ-1b
- First posted
- 2015-11-02
- Last updated
- 2024-12-19
- Results first posted
- 2018-05-18
- Limitations and caveats
This study was terminated early by the Sponsor because the development of ACTIMMUNE® for the treatment of Friedreich's Ataxia was discontinued after Study HZNP-ACT-301 (NCT02415127) failed to meet its primary efficacy endpoint.
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Discontinuations Due to AEs | Baseline/Day 1 (Week 26 of Study HZNP-ACT-301 [NCT02415127]) through Week 28 (follow-up safety visit) | An adverse event (AE) is any untoward medical occurrence, whether or not the event is considered related to the investigational product. A TEAE is any adverse change from the subject's baseline condition, including any laboratory test value abnormality judged as clinically significant by the investigator, that occurs on or after the date of the first dose of study drug administered at home and throughout the duration of the clinical study, whether the adverse event is considered related to the treatment or not. An SAE is an AE that results in death, is life-threatening, results in persistent or significant disability or incapacity, inpatient hospitalization or prolongation of an existing hospitalization, is a congenital anomaly or birth defect, or other medically important event. |
| Primary | Number of Participants With Positive/Negative Neutralizing Antibody (NAb) and Anti-Drug Antibody (ADA) Tests | Baseline/Day 1 (Week 26 of Study HZNP-ACT-301 [NCT02415127]), Week 4, Week 13, Week 26, and Week 28 (follow-up safety visit) | NAb testing only for those participants with a positive ADA test. Baseline is defined as the last non-missing measurement/assessment on the date of Week 26 Visit from study HZNP-ACT-301 (NCT02415127). If this measurement was missing or otherwise unavailable, it was the last non-missing measurement/assessment on or prior to first dose in this study. If the participant discontinued the study, then premature withdrawal assessments were mapped to the nearest scheduled visit based on schedule of the assessment and the study day. If the mapped visit was already available then the visit was mapped to the next schedule visit. Last on study assessment is the last non-missing post-baseline assessment for each participant. |
| Primary | Change From Baseline to Week 26 in the Friedreich's Ataxia Rating Scale (FARS)-mNeuro Score | From Baseline to Week 26 for all participants and from Baseline of HZNP-ACT-301 (NCT02415127)to Week 26 of HZNP-ACT-302 (52-week treatment duration) for participants receiving active treatment in both studies. | 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 | From Baseline to Week 26 for all participants and from Baseline of HZNP-ACT-301 (NCT02415127)to Week 26 of HZNP-ACT-302 (52-week treatment duration) for participants receiving active treatment in both studies. | 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) | From Baseline to Week 26 for all participants and from Baseline of HZNP-ACT-301 (NCT02415127)to Week 26 of HZNP-ACT-302 (52-week treatment duration) for participants receiving active treatment in both studies. | 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) | From Baseline to Week 26 for all participants and from Baseline of HZNP-ACT-301 (NCT02415127)to Week 26 of HZNP-ACT-302 (52-week treatment duration) for participants receiving active treatment in both studies. | 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
Outcomes7
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Discontinuations Due to AEs | Baseline/Day 1 (Week 26 of Study HZNP-ACT-301 [NCT02415127]) through Week 28 (follow-up safety visit) | Safety Population, defined as all participants who received at least 1 dose of open-label study drug after the Baseline Visit. | participants | NUMBER |
| PRIMARY | Number of Participants With Positive/Negative Neutralizing Antibody (NAb) and Anti-Drug Antibody (ADA) Tests | Baseline/Day 1 (Week 26 of Study HZNP-ACT-301 [NCT02415127]), Week 4, Week 13, Week 26, and Week 28 (follow-up safety visit) | Safety Population, defined as all participants who received at least 1 dose of open-label study drug after the Baseline Visit. Participants with an assessment at each time point are presented. | participants | NUMBER |
| PRIMARY | Change From Baseline to Week 26 in the Friedreich's Ataxia Rating Scale (FARS)-mNeuro Score | From Baseline to Week 26 for all participants and from Baseline of HZNP-ACT-301 (NCT02415127)to Week 26 of HZNP-ACT-302 (52-week treatment duration) for participants receiving active treatment in both studies. | Because the Sponsor discontinued the development of ACTIMMUNE® for the treatment of Friedreich's Ataxia, the planned analyses of the efficacy endpoints were not conducted. Any raw data collected for this endpoint exist as by-subject listings only, and are unanalyzed per protocol. | ||
| SECONDARY | Change From Baseline to Week 26 in Activities of Daily Living (ADL) Score | From Baseline to Week 26 for all participants and from Baseline of HZNP-ACT-301 (NCT02415127)to Week 26 of HZNP-ACT-302 (52-week treatment duration) for participants receiving active treatment in both studies. | Because the Sponsor discontinued the development of ACTIMMUNE® for the treatment of Friedreich's Ataxia, the planned analyses of the efficacy endpoints were not conducted. Any raw data collected for this endpoint exist as by-subject listings only, and are unanalyzed per protocol. | ||
| SECONDARY | Change From Baseline at Week 26 in Timed 25-Foot Walk (T25FW) | From Baseline to Week 26 for all participants and from Baseline of HZNP-ACT-301 (NCT02415127)to Week 26 of HZNP-ACT-302 (52-week treatment duration) for participants receiving active treatment in both studies. | Because the Sponsor discontinued the development of ACTIMMUNE® for the treatment of Friedreich's Ataxia, the planned analyses of the efficacy endpoints were not conducted. Any raw data collected for this endpoint exist as by-subject listings only, and are unanalyzed per protocol. | ||
| SECONDARY | Number of FARS-mNeuro Responders and Non-Responders at Week 26 | Week 26 | Because the Sponsor discontinued the development of ACTIMMUNE® for the treatment of Friedreich's Ataxia, the planned analyses of the efficacy endpoints were not conducted. Any raw data collected for this endpoint exist as by-subject listings only, and are unanalyzed per protocol. | ||
| SECONDARY | Change From Baseline to Week 26 in Total Friedreich Ataxia Rating Scale Score (FARStot) | From Baseline to Week 26 for all participants and from Baseline of HZNP-ACT-301 (NCT02415127)to Week 26 of HZNP-ACT-302 (52-week treatment duration) for participants receiving active treatment in both studies. | Because the Sponsor discontinued the development of ACTIMMUNE® for the treatment of Friedreich's Ataxia, the planned analyses of the efficacy endpoints were not conducted. Any raw data collected for this endpoint exist as by-subject listings only, and are unanalyzed per protocol. |
Source ClinicalTrials.gov
Results by group20
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Discontinuations Due to AEs | Interferon γ-1b | ≥ 1 TEAE | 78 | participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Discontinuations Due to AEs | Interferon γ-1b | ≥ 1 Related TEAE | 61 | participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Discontinuations Due to AEs | Interferon γ-1b | ≥ 1 SAE | 4 | participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Discontinuations Due to AEs | Interferon γ-1b | ≥ 1 Related SAE | 0 | participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Discontinuations Due to AEs | Interferon γ-1b | ≥ 1 TEAE Leading to Discontinuation | 1 | participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Discontinuations Due to AEs | Interferon γ-1b | ≥ 1 TEAE Leading to Death | 1 | participants | |
| Number of Participants With Positive/Negative Neutralizing Antibody (NAb) and Anti-Drug Antibody (ADA) Tests | Interferon γ-1b | Baseline ADA = negative | 85 | participants | |
| Number of Participants With Positive/Negative Neutralizing Antibody (NAb) and Anti-Drug Antibody (ADA) Tests | Interferon γ-1b | Baseline ADA = positive | 1 | participants | |
| Number of Participants With Positive/Negative Neutralizing Antibody (NAb) and Anti-Drug Antibody (ADA) Tests | Interferon γ-1b | Baseline NAb = negative | 1 | participants | |
| Number of Participants With Positive/Negative Neutralizing Antibody (NAb) and Anti-Drug Antibody (ADA) Tests | Interferon γ-1b | Baseline NAb = positive | 0 | participants |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Interferon γ-1b | 86 | |
| Overall Study | COMPLETED | Interferon γ-1b | 51 | |
| Overall Study | NOT COMPLETED | Interferon γ-1b | 35 |
Source ClinicalTrials.gov
Adverse events27
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 | Events |
|---|---|---|---|---|---|---|
| other | Neutropenia | Blood and lymphatic system disorders | Interferon γ-1b | 18 | 86 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Interferon γ-1b | 5 | 86 | |
| other | Nausea | Gastrointestinal disorders | Interferon γ-1b | 13 | 86 | |
| other | Vomiting | Gastrointestinal disorders | Interferon γ-1b | 13 | 86 | |
| other | Chills | General disorders | Interferon γ-1b | 5 | 86 | |
| other | Fatigue | General disorders | Interferon γ-1b | 7 | 86 | |
| other | Pain | General disorders | Interferon γ-1b | 7 | 86 | |
| other | Pyrexia | General disorders | Interferon γ-1b | 15 | 86 | |
| other | Gastroenteritis viral | Infections and infestations | Interferon γ-1b | 5 | 86 | |
| other | Nasopharyngitis | Infections and infestations | Interferon γ-1b | 7 | 86 |
1 to 10 of 27Source ClinicalTrials.gov
See all 27 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Interferon γ-1b | 1 | 86 |
| Total, other adverse events | Interferon γ-1b | 66 | 86 |
| Total, serious adverse events | Interferon γ-1b | 4 | 86 |
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 comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
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.
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/NCT02593773
GET https://conductscience.org/api/trials/v1/NCT02593773/events?page=2