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| ID | Type | Description | Link |
|---|---|---|---|
| 2009-015985-75 | EudraCT Number |
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This study will evaluate safety and clinical outcomes of treatment with Replagal in adult participants with Fabry disease who have completed Study TKT028 (NCT00864851).
HGT-REP-060 was designed to provide participants who participated in TKT028 an additional 52 weeks of treatment with Replagal at the standard dose of 0.2 milligram per kilogram (mg/kg) every other week (EOW) to assess the effect of continued Replagal therapy on long-term safety and clinical outcomes for this participant population.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Replagal 0.2 mg/kg EOW | Experimental | Intravenous, 0.2mg/kg EOW |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Replagal | Biological | 0.2 mg/kg administered intravenously [IV] every other week |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Left Ventricular Mass Indexed to Height (LVMI) | Baseline to 12 months | |
| Safety Evaluations | Baseline to 12 months |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Maximal Oxygen Consumption (VO2max) at Peak Exercise | Baseline to 12 months | |
| Change From Baseline in Distance Walked in 6- Minute Walk Test (6MWT) | Baseline to 12 months | |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Study Director | Takeda | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Iowa Hospitals and Clinics | Iowa City | Iowa | 52242 | United States | ||
| O&O Alpan LLC |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 26185417 | Result | Golan L, Goker-Alpan O, Holida M, Kantola I, Klopotowski M, Kuusisto J, Linhart A, Musial J, Nicholls K, Gonzalez-Rodriguez D, Sharma R, Vujkovac B, Chang P, Wijatyk A. Evaluation of the efficacy and safety of three dosing regimens of agalsidase alfa enzyme replacement therapy in adults with Fabry disease. Drug Des Devel Ther. 2015 Jul 8;9:3435-44. doi: 10.2147/DDDT.S80928. eCollection 2015. |
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Screening details: Thirty-five of the 40 subjects who completed the parent study (Study TKT028 [NCT00864851]) were enrolled in this extension study.
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| ID | Title | Description |
|---|---|---|
| FG000 | Replagal® (0.2 mg/kg) | Replagal 0.2 milligram per kilogram (mg/kg) intravenously (IV), every other week (EOW). |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Replagal® (0.2 mg/kg) | Replagal 0.2 mg/kg IV, EOW |
| 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 in Left Ventricular Mass Indexed to Height (LVMI) | The Intent-to-Treat (ITT) participant population in this study was defined as all participants who provided informed consent and received study drug. Participants who did not have left ventricular hypertrophy were not included in this analysis. | Posted | Mean | Standard Deviation | g/m^2.7 | Baseline to 12 months |
|
|
Baseline to 12 Months
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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 | Replagal® (0.2 mg/kg) | Replagal 0.2 mg/kg IV, EOW |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Acute myocardial infarction | Cardiac disorders | MedDRA (12) | Non-systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA (12) | Non-systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Shire | +1 866 842 5335 | ClinicalTransparency@shire.com |
| ID | Term |
|---|---|
| D000795 | Fabry Disease |
| ID | Term |
|---|---|
| D013106 | Sphingolipidoses |
| D020140 | Lysosomal Storage Diseases, Nervous System |
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
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| ID | Term |
|---|---|
| C000627036 | agalsidase alfa |
| D000519 | alpha-Galactosidase |
| ID | Term |
|---|---|
| D005696 | Galactosidases |
| D006026 | Glycoside Hydrolases |
| D006867 | Hydrolases |
| D004798 | Enzymes |
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| Change From Baseline in the Minnesota Living With Heart Failure Questionnaire (MLHF- Q) |
The MLHF-Q contains 21 questions with answers ranging from 0 (no) to 5 (very much). The final score ( 0 to 105) is the sum of the points for the 21 questions. A higher score indicates a worse quality of life. |
| Baseline to 12 months |
| Change From Baseline in New York Heart Association (NYHA) Functional Class | Class I: No limitation of physical activity. Ordinary physical activity does not cause undue fatigue, palpitation, dyspnea. Class II: Slight limitation of physical activity. Comfortable at rest, but ordinary physical activity results in fatigue, palpitation, or dyspnea. Class III: Marked limitation of physical activity. Comfortable at rest, but less than ordinary activity causes fatigue, palpitation, or dyspnea. Class IV: Unable to carry out any physical activity without discomfort. Symptoms of cardiac insufficiency at rest. If any physical activity is undertaken, discomfort is increased. | Baseline to 12 months |
| Change From Baseline in Plasma Gb3 | Baseline to 12 months |
| Change From Baseline in eGFR | Baseline to 12 months |
| Change From Baseline in Albumin/Creatinine (A/Cr) Ratio | Baseline to 12 months |
| Fairfax |
| Virginia |
| 22030 |
| United States |
| The Royal Melbourne Hospital | Parkville | 3050 | Australia |
| 1st School of Medicine Charles University | Prague | Czechia |
| Turku University Central Hospital | Turku | FI-20520 | Finland |
| Szpital Uniwersytecki w Krakowie | Krakow | 31-066 | Poland |
| Instytut Kardiologii, I Klinika Choroby Wiencowej | Warsaw | Poland |
| General Hospital Slovenj Gradec | Slovenj Gradec | 2380 | Slovenia |
| Salford Royal NHS Foundation Trust | Salford | England | M6 8HD | United Kingdom |
| Years |
|
| Age, Customized | Age at time of consent | Count of Participants | Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Region of Enrollment | Count of Participants | Participants |
|
| Left Ventricular Mass Indexed to Height (LVMI) | Number of participants analyzed for this baseline characteristic was 33 | Mean | Standard Deviation | gram per meter to the power 2.7(g/m^2.7) |
|
| Maximal Oxygen Consumption (VO2max) | Number of participants analyzed for this baseline characteristic was 32 | Mean | Standard Deviation | milliliter/minute/kilogram (mL/min/kg) |
|
| Distance Walked in 6-Minute Walk Test (6MWT) | Number of participants analyzed for this baseline characteristic was 34 | Mean | Standard Deviation | meters |
|
| Minnesota Living with Heart Failure Questionnaire (MLHF-Q) Summary Score | The MLHF-Q contains 21 questions with answers ranging from 0 (no) to 5 (very much). The final score ( 0 to 105) is the sum of the points for the 21 questions. A higher score indicates a worse quality of life. | Mean | Standard Deviation | units on a scale |
|
| New York Heart Association (NYHA) Functional Class | Class I = No limitation of physical activity. Ordinary physical activity does not cause undue fatigue, palpitation, dyspnea Class II = Slight limitation of physical activity. Comfortable at rest, but ordinary physical activity results in fatigue, palpitation, or dyspnea Class III = Marked limitation of physical activity. Comfortable at rest, but less than ordinary activity causes fatigue, palpitation, or dyspnea Class IV = Unable to carry out any physical activity without discomfort. Symptoms of cardiac insufficiency at rest. If any physical activity is undertaken, discomfort is increased | Count of Participants | Participants |
|
| Plasma Gb3 (globotriaosylceramide) | Mean | Standard Deviation | nanomoles per milliliter (nmol/mL) |
|
| Estimated Glomerular Filtration Rate (eGFR) | Mean | Standard Deviation | (mL/min/1.73m^2) |
|
| Albumin-to-Creatinine (A/Cr) Ratio | Number of participants analyzed for this baseline characteristic was 33 | Mean | Standard Deviation | Ratio |
|
|
| Secondary | Change From Baseline in Maximal Oxygen Consumption (VO2max) at Peak Exercise | ITT population. Test was not done or test was not valid for all participants. | Posted | Mean | Standard Deviation | (mL/min/kg) | Baseline to 12 months |
|
|
|
| Secondary | Change From Baseline in Distance Walked in 6- Minute Walk Test (6MWT) | ITT population. Test was not done or test was not valid for all participants. | Posted | Mean | Standard Deviation | meters | Baseline to 12 months |
|
|
|
| Secondary | Change From Baseline in the Minnesota Living With Heart Failure Questionnaire (MLHF- Q) | The MLHF-Q contains 21 questions with answers ranging from 0 (no) to 5 (very much). The final score ( 0 to 105) is the sum of the points for the 21 questions. A higher score indicates a worse quality of life. | ITT population. Number of participants analyzed signifies participants evaluable for this endpoint. | Posted | Mean | Standard Deviation | units on a scale | Baseline to 12 months |
|
|
|
| Secondary | Change From Baseline in New York Heart Association (NYHA) Functional Class | Class I: No limitation of physical activity. Ordinary physical activity does not cause undue fatigue, palpitation, dyspnea. Class II: Slight limitation of physical activity. Comfortable at rest, but ordinary physical activity results in fatigue, palpitation, or dyspnea. Class III: Marked limitation of physical activity. Comfortable at rest, but less than ordinary activity causes fatigue, palpitation, or dyspnea. Class IV: Unable to carry out any physical activity without discomfort. Symptoms of cardiac insufficiency at rest. If any physical activity is undertaken, discomfort is increased. | ITT population. | Posted | Number | participants | Baseline to 12 months |
|
|
|
| Secondary | Change From Baseline in Plasma Gb3 | ITT population. Number of participants analyzed signifies participants evaluable for this endpoint. | Posted | Mean | Standard Deviation | (nmol/mL) | Baseline to 12 months |
|
|
|
| Secondary | Change From Baseline in eGFR | ITT population. Number of participants analyzed signifies participants evaluable for this endpoint. | Posted | Mean | Standard Deviation | (mL/min/1.73m^2) | Baseline to 12 months |
|
|
|
| Secondary | Change From Baseline in Albumin/Creatinine (A/Cr) Ratio | ITT population. Number of participants analyzed signifies participants evaluable for this endpoint. | Posted | Mean | Standard Deviation | Ratio | Baseline to 12 months |
|
|
|
| Primary | Safety Evaluations | ITT population | Posted | Number | participants | Baseline to 12 months |
|
|
|
| 6 |
| 35 |
| 32 |
| 35 |
| Atrial fibrillation | Cardiac disorders | MedDRA (12) | Non-systematic Assessment |
|
| Cardiac failure | Cardiac disorders | MedDRA (12) | Non-systematic Assessment |
|
| Myocardial infarction | Cardiac disorders | MedDRA (12) | Non-systematic Assessment |
|
| Hydrocele | Congenital, familial and genetic disorders | MedDRA (12) | Non-systematic Assessment |
|
| Gastritis | Gastrointestinal disorders | MedDRA (12) | Non-systematic Assessment |
|
| Arteriovenous fistula site complication | Injury, poisoning and procedural complications | MedDRA (12) | Non-systematic Assessment |
|
| Arteriovenous fistula site haemorrhage | Injury, poisoning and procedural complications | MedDRA (12) | Non-systematic Assessment |
|
| Intervertebral disc disorder | Musculoskeletal and connective tissue disorders | MedDRA (12) | Non-systematic Assessment |
|
| Cerebrovascular accident | Nervous system disorders | MedDRA (12) | Non-systematic Assessment |
|
| Iron deficiency anaemia | Blood and lymphatic system disorders | MedDRA (12) | Non-systematic Assessment |
|
| Atrial fibrillation | Cardiac disorders | MedDRA (12) | Non-systematic Assessment |
|
| Dilatation atrial | Cardiac disorders | MedDRA (12) | Non-systematic Assessment |
|
| Left atrial dilatation | Cardiac disorders | MedDRA (12) | Non-systematic Assessment |
|
| Palpitations | Cardiac disorders | MedDRA (12) | Non-systematic Assessment |
|
| Hypothyroidism | Endocrine disorders | MedDRA (12) | Non-systematic Assessment |
|
| Abdominal pain | Gastrointestinal disorders | MedDRA (12) | Non-systematic Assessment |
|
| Abdominal pain upper | Gastrointestinal disorders | MedDRA (12) | Non-systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA (12) | Non-systematic Assessment |
|
| Dyspepsia | Gastrointestinal disorders | MedDRA (12) | Non-systematic Assessment |
|
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA (12) | Non-systematic Assessment |
|
| Nausea | Gastrointestinal disorders | MedDRA (12) | Non-systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA (12) | Non-systematic Assessment |
|
| Chest pain | General disorders | MedDRA (12) | Non-systematic Assessment |
|
| Oedema peripheral | General disorders | MedDRA (12) | Non-systematic Assessment |
|
| Pyrexia | General disorders | MedDRA (12) | Non-systematic Assessment |
|
| Bronchitis | Infections and infestations | MedDRA (12) | Non-systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | MedDRA (12) | Non-systematic Assessment |
|
| Oral herpes | Infections and infestations | MedDRA (12) | Non-systematic Assessment |
|
| Respiratory tract infection | Infections and infestations | MedDRA (12) | Non-systematic Assessment |
|
| Urinary tract infection | Infections and infestations | MedDRA (12) | Non-systematic Assessment |
|
| Blood glucose increased | Investigations | MedDRA (12) | Non-systematic Assessment |
|
| Blood lactate dehydrogenase increased | Investigations | MedDRA (12) | Non-systematic Assessment |
|
| Electrocardiogram QT prolonged | Investigations | MedDRA (12) | Non-systematic Assessment |
|
| Troponin T increased | Investigations | MedDRA (12) | Non-systematic Assessment |
|
| Impaired fasting glucose | Metabolism and nutrition disorders | MedDRA (12) | Non-systematic Assessment |
|
| Vitamin D deficiency | Metabolism and nutrition disorders | MedDRA (12) | Non-systematic Assessment |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (12) | Non-systematic Assessment |
|
| Muscle tightness | Musculoskeletal and connective tissue disorders | MedDRA (12) | Non-systematic Assessment |
|
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA (12) | Non-systematic Assessment |
|
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA (12) | Non-systematic Assessment |
|
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (12) | Non-systematic Assessment |
|
| Dizziness | Nervous system disorders | MedDRA (12) | Non-systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA (12) | Non-systematic Assessment |
|
| Disorientation | Psychiatric disorders | MedDRA (12) | Non-systematic Assessment |
|
| Sleep disorder | Psychiatric disorders | MedDRA (12) | Non-systematic Assessment |
|
| Nephrolithiasis | Renal and urinary disorders | MedDRA (12) | Non-systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (12) | Non-systematic Assessment |
|
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (12) | Non-systematic Assessment |
|
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (12) | Non-systematic Assessment |
|
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA (12) | Non-systematic Assessment |
|
Shire's agreements with investigators vary. All agreements provide Shire the right to embargo communications regarding trial results prior to public release for a period ≤180 days from the time submitted to Shire for review. Shire does not prohibit publication, but can require the removal of confidential information(excluding trial results) and can request postponement of a single-center publication until after disclosure of the trial's multi-center publication.
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D059345 | Cerebral Small Vessel Diseases |
| D002561 | Cerebrovascular Disorders |
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008661 | Metabolism, Inborn Errors |
| D008064 | Lipidoses |
| D008052 | Lipid Metabolism, Inborn Errors |
| D016464 | Lysosomal Storage Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D052439 | Lipid Metabolism Disorders |
| D045762 |
| Enzymes and Coenzymes |
| Title | Measurements |
|---|---|
|