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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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8 October 2021 |
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Main ID: |
EUCTR2005-003512-30-FR |
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Date of registration:
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26/03/2007 |
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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 Phase 4 Multi-center, Multi-national, Open-label, Randomized, Two Dose Level Study of Naglazyme (galsulfase) in Infants with Maroteaux-Lamy Syndrome (MPS VI) - N/A
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Scientific title:
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A Phase 4 Multi-center, Multi-national, Open-label, Randomized, Two Dose Level Study of Naglazyme (galsulfase) in Infants with Maroteaux-Lamy Syndrome (MPS VI) - N/A |
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Date of first enrolment:
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05/04/2007 |
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Target sample size:
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4 |
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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:2005-003512-30 |
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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: no
Randomised: yes
Open: yes
Single blind: no
Double blind: no
Parallel group: no
Cross over: no
Other: no
If controlled, specify comparator, Other Medicinial Product: no
Placebo: no
Other: no
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Phase:
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Human pharmacology (Phase I): no
Therapeutic exploratory (Phase II): no
Therapeutic confirmatory - (Phase III): no
Therapeutic use (Phase IV): yes
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Countries of recruitment
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France
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Portugal
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Contacts
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Key inclusion & exclusion criteria
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Inclusion criteria:
· In the case of subjects under the age of 18, provide written assent (if required) and written informed consent by a parent or legal guardian after the nature of the study has been explained and prior to any research-related procedures;
· Willing and able to comply with all study procedures;
· Equal to or greater than 36 weeks estimated gestational age by physical exam at birth;
· Has a diagnosis of MPS VI based on a documented prenatal diagnosis or fibroblast or leukocyte N-acetylgalactosamine 4-sulfatase (ASB) enzyme activity level of less than 10% of the lower limit of the normal range of the measuring laboratory;
· Is less than one year of age;
· Has no evidence of skeletal dysplasia based on physical exam.
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) no
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:
· Perceived to be unreliable or unavailable for study participation or, if under the age of 18, have parents or legal guardians who are perceived to be unreliable or unavailable;
· Use of any investigational drug within 30 days prior to screening, or requirement for any investigational agent prior to completion of all scheduled study assessments;
· Concurrent disease or condition that would interfere with study participation or safety (i.e., has previously undergone hematopoietic stem cell transplantation such as bone marrow or cord blood transplantation, or major organ transplantation);
· Any condition that, in the view of the PI, renders the subject at high risk from treatment compliance and/or completing the study;
· Has known hypersensitivity to Naglazyme;
· Has previously received Naglazyme.
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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Trade Name: Naglazyme
Product Name: Naglazyme
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: galsulfase
CAS Number: 552858-79-4
Other descriptive name: recombinant human Arysulfatase B, recombinant human N-acetylgalactosamine 4-sulfatase
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 1.0-
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Primary Outcome(s)
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Secondary Objective: The secondary objective of the study is to evaluate the efficacy of the two dose levels of Naglazyme in preventing several measures of disease progression in infants under the age of one year who have MPS VI by monitoring urinary GAGs, gross and fine motor function, cardiac function, vision, hearing, and use of health resources.
The safety objective of the study is to evaluate the safety of two dose levels of Naglazyme in infants under the age of one year who have MPS VI.
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Main Objective: The primary objective of the study is to evaluate the efficacy of two dose levels of Naglazyme in preventing the progression of skeletal dysplasia in infants under the age of one year who have MPS VI by monitoring physical appearance, x-ray of the skeletal system and growth.
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Primary end point(s): The primary efficacy endpoint will be assessed by changes in dysmorphic features as assessed by photographs, changes in skeletal dysplasia as assessed by skeletal survey, and growth as measured by height, weight and head circumference. Other potential benefits of treatment will be periodically assessed by evaluation of gross and fine motor skills as part of the Denver II Development Test, cardiac evaluation by echocardiography, complete eye and visual exam, hearing test by audiometry, Health Resource Utilization Questionnaire, and urinary GAGs.
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Source(s) of Monetary Support
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Ethics review
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Status: Approved
Approval date: 22/03/2007
Contact:
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