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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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12 December 2020 |
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Main ID: |
NCT01298141 |
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Date of registration:
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15/02/2011 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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A Multicenter Open-Label Treatment Protocol to Observe the Safety of Replagal (Agalsidase Alfa) Enzyme Replacement Therapy in Canadian Patients With Fabry Disease
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Scientific title:
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A Multicenter Open-Label Treatment Protocol to Observe the Safety of Replagal® (Agalsidase Alfa) Enzyme Replacement Therapy in Canadian Patients With Fabry Disease |
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Date of first enrolment:
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August 10, 2011 |
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Target sample size:
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171 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT01298141 |
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Study type:
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Interventional |
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Study design:
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Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 3
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Countries of recruitment
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Canada
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Contacts
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Name:
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Shire Physician |
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Address:
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Telephone:
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Email:
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Affiliation:
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Shire |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
Cohort 1:
1. The patient has a documented diagnosis of Fabry disease.
2. The patient is sufficiently compliant with study activities to participate in this
treatment plan, as judged by the Investigator.
3. The patient must meet current Canadian guidelines for enzyme replacement therapy for
Fabry disease by meeting one of the following criteria:
1. Age-adjusted glomerular filtration rate (GFR) <80 ml/min or a decline in GFR of
>10% which is sustained for 3 months and for which other causes of declining
renal function have been excluded by a nephrologist or any 2 of the following:
- Isolated proteinuria =500 mg/day/1.73 m2 without other cause
- Nephrogenic diabetes insipidus
- Fanconi syndrome
- Hypertension
2. Evidence of cardiac involvement related to Fabry disease including any 2 of the
following:
- Left ventricular (LV) wall thickness >12 mm
- Left ventricular hypertrophy (LVH) by electrocardiogram (ECG); Estes ECG
score must be >5
- Left ventricular mass index (LVMI) by 2D echocardiogram 20% above normal for
age
- Diastolic filling abnormalities by 2D echocardiogram or by other accepted
measures of diastolic filling. E/A ration >2.0 and deceleration time <140
msec
- Increase of LV mass of at least 5 g/m2/year, with three measurements over a
minimum of 12 months
- Increase of left atrium (LA) size on 2D echo at least 10% above normal for
age. In parasternal long axis view (PLAX) >33 mm; in four chamber view >42
mm
- Cardiac conduction and rhythm abnormalities: atrioventricular (AV) block,
short PR interval, left branch bundle block (LBBB), ventricular or atrial
tachyarrhythmias, sinus bradycardia (in the absence of drugs with negative
chronotropic activity)
- Delayed posterolateral left ventricular wall late enhancement on MRI as
evidence of advanced cardiac disease with fibrosis
3. Evidence of neurological involvement related to Fabry disease including 1 of the
following:
- Stroke or transient ischemic attack (TIA) prior to the age of 55 documented
by a neurologist
- Acute onset unilateral hearing loss
- Acut monocular visual loss without other cause
4. Chronic, intractable diarrhea and/or abdominal pain/cramps, refractory to
standard management for at least 6 months.
5. Chronic, intractable neuropathic pain, refractory to analgesics and standard pain
management for at least 6 months.
Cohort 2:
4. Patient must have participated in Study REP001a.
Exclusion Criteria:
1. The patient has experienced an anaphylactic or anaphylactoid reaction or other
infusion-related reaction which, in the opinion of the Investigator, precludes further
treatment with agalsidase alfa or may interfere with the interpretation of the study.
2. The patient is otherwise unsuitable for the study, in the opinion of the Investigator.
3. The patient is enrolled in another clinical study, other than the Canadian Fabry
Disease Initiative (CFDI).
Age minimum:
N/A
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Fabry Disease
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Intervention(s)
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Biological: agalsidase alfa
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Primary Outcome(s)
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Number of Participants Who Reported Positive to Immunoglobulin A (IgA)
[Time Frame: Baseline (within 6 months prior to first dose) up to Week 129]
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Number of Participants With Treatment-emergent Adverse Events (TEAEs)
[Time Frame: From the start of study treatment up to 30 days after the last dose of study drug administration (up to 320 weeks)]
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Number of Participants Who Reported Positive to Neutralizing Antibody (NAb)
[Time Frame: Baseline (within 6 months prior to first dose) up to Week 285]
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Number of Participants Who Reported Positive to Immunoglobulin M (IgM)
[Time Frame: Baseline (within 6 months prior to first dose) up to Week 129]
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Number of Participants Who Reported Positive to Immunoglobulin E (IgE)
[Time Frame: Baseline (within 6 months prior to first dose) up to Week 129]
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Number of Participants Who Reported Positive to Anti-drug Antibody (ADA)
[Time Frame: Baseline (within 6 months prior to first dose) up to Week 285]
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Number of Participants With Infusion-Related Reactions (IRR)
[Time Frame: From the start of study treatment up to 30 days after the last dose of study drug administration (up to 320 weeks)]
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Secondary ID(s)
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HGT-REP-081
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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