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| Name | Class |
|---|---|
| Friedreich's Ataxia Research Alliance | OTHER |
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The purpose of this phase 3 randomized, multi-center, double-blind, placebo-controlled study is to evaluate the efficacy and safety of ACTIMMUNE® (interferon-γ 1b) in the treatment of Friedreich's Ataxia (FA) and to evaluate the pharmacokinetic (PK) characteristics of ACTIMMUNE® in FA patients.
Study with completed results acquired from Horizon in 2024.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Interferon γ-1b | Experimental | Approximately 45 participants will receive subcutaneous (SC) doses of ACTIMMUNE® 3 times a week (TIW) for a total of 26 weeks. |
|
| Placebo | Placebo Comparator | Approximately 45 participants will receive SC doses of placebo TIW for a total of 26 weeks. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Interferon γ-1b | Drug | The study drug dose is planned to be escalated on a weekly basis over the first 4 weeks of treatment (from 10 µg/m² to 25, 50, and 100 µg/m²). The dose may be reduced, interrupted, or held based on tolerability. By Week 13, all participants are to be on a stable tolerated dose of study drug in order to continue study participation; the dose may not be further increased after week 13, however, it may be reduced on a case-by-case basis to manage drug-related adverse events (AEs). |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline to Week 26 in the Friedreich's Ataxia Rating Scale (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). A negative change from baseline is an improvement. | Baseline, Week 26 |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline to Week 26 in Activities of Daily Living (ADL) Score | 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. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| MD | Amgen | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of California, Los Angeles Neurology Clinic | Los Angeles | California | 90038 | United States | ||
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| ID | Title | Description |
|---|---|---|
| FG000 | Interferon γ-1b | Subcutaneous (SC) doses of ACTIMMUNE® 3 times a week (TIW) for a total of 26 weeks. |
| FG001 | Placebo | SC doses of placebo TIW for a total of 26 weeks. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Interferon γ-1b | SC doses of ACTIMMUNE® TIW for a total of 26 weeks. |
| BG001 | Placebo | SC doses of placebo TIW for a total of 26 weeks. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Change From Baseline to Week 26 in the Friedreich's Ataxia Rating Scale (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). A negative change from baseline is an improvement. | 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. | Posted | Mean | Standard Deviation | units on a scale | Baseline, Week 26 |
|
Treatment emergent adverse events (TEAEs) are presented. Adverse events (AEs) occurring or worsening after the dose on Day 1 through the end of the study (Week 26) were considered TEAEs. Serious TEAEs were collected through 2 weeks after study discontinuation (Week 28).
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Interferon γ-1b | SC doses of ACTIMMUNE® TIW for a total of 26 weeks. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Chest pain | General disorders | MedDRA 16.1 | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Neutropenia | Blood and lymphatic system disorders | MedDRA 16.1 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Julie Ball, Executive Director Clinical Development & Operations | Horizon Pharma Ireland, Ltd. Dublin, Ireland | clinicaltrials@horizonpharma.com |
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| ID | Term |
|---|---|
| D005621 | Friedreich Ataxia |
| ID | Term |
|---|---|
| D013132 | Spinocerebellar Degenerations |
| D002526 | Cerebellar Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
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| ID | Term |
|---|---|
| C554125 | interferon gamma-1b |
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|
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| Placebo | Drug | The volume of placebo is planned to correspond with volume of study drug that would be given to the participant if the participant was randomized to the study drug arm. |
|
| Baseline, Week 26 |
| Change From Baseline at Week 26 in Timed 25-Foot Walk (T25FW) | 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. | Baseline, Week 26 |
| Number of FARS-mNeuro Responders and Non-Responders at 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). | Week 26 |
| Change From Baseline to Week 26 in Total Friedreich Ataxia Rating Scale Score (FARStot) | 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. | Baseline, Week 26 |
| University of Florida - Clinical Research Center |
| Gainesville |
| Florida |
| 32603 |
| United States |
| University of Iowa Children's Hospital | Iowa City | Iowa | 52242 | United States |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| BG002 | Total | Total of all reporting groups |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
SC doses of ACTIMMUNE® TIW for a total of 26 weeks. |
| OG001 | Placebo | SC doses of placebo TIW for a total of 26 weeks. |
|
|
|
| Secondary | Change From Baseline to Week 26 in Activities of Daily Living (ADL) Score | 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. | 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. | Posted | Mean | Standard Deviation | units on a scale | Baseline, Week 26 |
|
|
|
| Secondary | Change From Baseline at Week 26 in Timed 25-Foot Walk (T25FW) | 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. | 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. | Posted | Mean | Standard Deviation | 1/seconds | Baseline, Week 26 |
|
|
|
| Secondary | Number of FARS-mNeuro Responders and Non-Responders at 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). | 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. | Posted | Count of Participants | Participants | Week 26 |
|
|
|
| Secondary | Change From Baseline to Week 26 in Total Friedreich Ataxia Rating Scale Score (FARStot) | 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. | 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. | Posted | Mean | Standard Deviation | units on a scale | Baseline, Week 26 |
|
|
|
| 1 |
| 47 |
| 45 |
| 47 |
| EG001 | Placebo | SC doses of placebo TIW for a total of 26 weeks. | 2 | 45 | 37 | 45 |
| Pneumonia | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
|
| Dehydration | Metabolism and nutrition disorders | MedDRA 16.1 | Systematic Assessment |
|
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 16.1 | Systematic Assessment |
|
| Constipation | Gastrointestinal disorders | MedDRA 16.1 | Systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA 16.1 | Systematic Assessment |
|
| Nausea | Gastrointestinal disorders | MedDRA 16.1 | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA 16.1 | Systematic Assessment |
|
| Chills | General disorders | MedDRA 16.1 | Systematic Assessment |
|
| Fatigue | General disorders | MedDRA 16.1 | Systematic Assessment |
|
| Injection site bruising | General disorders | MedDRA 16.1 | Systematic Assessment |
|
| Injection site erythema | General disorders | MedDRA 16.1 | Systematic Assessment |
|
| Injection site pain | General disorders | MedDRA 16.1 | Systematic Assessment |
|
| Injection site pruritus | General disorders | MedDRA 16.1 | Systematic Assessment |
|
| Pain | General disorders | MedDRA 16.1 | Systematic Assessment |
|
| Pyrexia | General disorders | MedDRA 16.1 | Systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
|
| Upper respiratory tract infection | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
|
| Urinary tract infection | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
|
| Excoriation | Injury, poisoning and procedural complications | MedDRA 16.1 | Systematic Assessment |
|
| Fall | Injury, poisoning and procedural complications | MedDRA 16.1 | Systematic Assessment |
|
| Joint injury | Injury, poisoning and procedural complications | MedDRA 16.1 | Systematic Assessment |
|
| Laceration | Injury, poisoning and procedural complications | MedDRA 16.1 | Systematic Assessment |
|
| Ligament sprain | Injury, poisoning and procedural complications | MedDRA 16.1 | Systematic Assessment |
|
| Neutrophil count decreased | Investigations | MedDRA 16.1 | Systematic Assessment |
|
| White blood cell count decreased | Investigations | MedDRA 16.1 | Systematic Assessment |
|
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 16.1 | Systematic Assessment |
|
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA 16.1 | Systematic Assessment |
|
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 16.1 | Systematic Assessment |
|
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 16.1 | Systematic Assessment |
|
| Dizziness | Nervous system disorders | MedDRA 16.1 | Systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA 16.1 | Systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 16.1 | Systematic Assessment |
|
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 16.1 | Systematic Assessment |
|
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 16.1 | Systematic Assessment |
|
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 .
| D009422 | Nervous System Diseases |
| D013118 | Spinal Cord Diseases |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D028361 | Mitochondrial Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |