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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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EUCTR |
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Last refreshed on:
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25 November 2019 |
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Main ID: |
EUCTR2004-000642-21-IE |
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Date of registration:
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18/06/2004 |
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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, Multinational, Open-Label Extension Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients with Mucopolysaccharidosis VI - Aryplase Phase 3 Open-Label Extension
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Scientific title:
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A Multicenter, Multinational, Open-Label Extension Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients with Mucopolysaccharidosis VI - Aryplase Phase 3 Open-Label Extension |
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Date of first enrolment:
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Target sample size:
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38 |
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Recruitment status: |
Not Recruiting |
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URL:
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https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2004-000642-21 |
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Study type:
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Interventional clinical trial of medicinal product |
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Study design:
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Controlled: yes
Randomised: no
Open: yes
Single blind:
Double blind:
Parallel group:
Cross over:
Other:
If controlled, specify comparator, Other Medicinial Product:
Placebo:
Other:
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Phase:
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Human pharmacology (Phase I):
Therapeutic exploratory (Phase II):
Therapeutic confirmatory - (Phase III): yes
Therapeutic use (Phase IV):
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Countries of recruitment
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Ireland
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Contacts
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Affiliation:
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Key inclusion & exclusion criteria
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Inclusion criteria:
· The patient must sign an informed consent form before any protocol-related procedures can be performed. If the patient is under 18 years of age, a legally authorized guardian must sign the informed consent form. In addition, patients who are under 18 years of age and can understand the consent form may be required to indicate their willingness to participate by signing the minor assent portion of the informed consent, if stipulated by the site’s IRB.
· The patient must have successfully completed Study ASB-03-05, including having received at least 20 of the 24 scheduled weekly infusions and having missed no more than two consecutive infusions.
· A female patient of childbearing potential must have a negative pregnancy test (urine b–human chorionic gonadotropin) at entry (prior to the first infusion). Note: All female patients of childbearing potential and sexually mature male patients must be advised to use a medically accepted method of contraception throughout the study. Female patients of childbearing potential must be willing to undergo periodic pregnancy tests during the course of the study.
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
Exclusion criteria:
· The patient is pregnant or lactating.
· The patient has received an investigational drug (other than rhASB in Study ASB-03-05) within 30 days prior to study enrollment.
· The patient is unwilling or unable to travel to the primary site for periodic assessments.
Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes
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Health Condition(s) or Problem(s) studied
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Mucopolysaccharidosis Type VI (MPS VI; Maroteaux-Lamy Syndrome)
MedDRA version: 6.0
Level: PT
Classification code 10056892
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Intervention(s)
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Product Name: recombinant human N-acetylgalactosamine 4-sulfatase
Product Code: N/A
Pharmaceutical Form: Concentrate for solution for infusion
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Primary Outcome(s)
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Secondary Objective:
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Main Objective: The objective of this study is to evaluate the long-term efficacy and safety of rhASB treatment in patients with MPS VI.
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Primary end point(s): The primary efficacy endpoint, change in the number of metres walked in the 12-minute walk test, will be compared between two treatment groups. Differences between the rhASB/rhASB and placebo/rhASB groups with respect to the change from baseline (beginning of Study ASB-03-05) to Week 48 of the extension study and the change from Week 24 (end of Study ASB-03 05) to Week 48 of the extension study will be tested using analysis of variance models with the number of metres walked at baseline as the only covariate. Similar analyses will be performed for secondary and tertiary efficacy endpoints.
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Secondary ID(s)
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ASB-03-06
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Source(s) of Monetary Support
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Ethics review
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Status:
Approval date:
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Results
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Results available:
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Date Posted:
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Date Completed:
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