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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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EUCTR |
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Last refreshed on:
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19 March 2012 |
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Main ID: |
EUCTR2007-001453-26-NL |
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Date of registration:
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13/11/2009 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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Natural course, effects of enzyme therapy and health economic aspects in patients with mucopolysaccharidosis type I, II and VI.
Long-term folloe-up of untreated patients and patients receiving commercially available Aldurazyme, Elaprase and Naglazyme.
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Scientific title:
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Natural course, effects of enzyme therapy and health economic aspects in patients with mucopolysaccharidosis type I, II and VI.
Long-term folloe-up of untreated patients and patients receiving commercially available Aldurazyme, Elaprase and Naglazyme. |
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Date of first enrolment:
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02/07/2007 |
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Target sample size:
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50 |
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Recruitment status: |
Authorised-recruitment may be ongoing or finished |
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URL:
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https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2007-001453-26 |
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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:
Open:
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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Countries of recruitment
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Netherlands
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Contacts
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Affiliation:
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Key inclusion & exclusion criteria
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Inclusion criteria:
1. The patient should have a biochemically confirmed deficiency of a-L-iduronidase (MPS I). iduronidate-2-sulfatase (MPS II), or N-acetylgalactosamine-4-sulfatase (MPS VI); or a confirmed mutation in the gene encoding for a-L-iduronidase (MPS I). iduronidate-2-sulfatase (MPS II, or N-acetylgalactosamine-4-sulfatase (MPS VI).
2. The patient has had least one evaluation through which the severity of the disease has been assessed and the urgency of enzyme therapy can be determined.
3. Written informed consent must be obtained from the patient and/or from the patient's parent/guardian if the patient is under 18 years of age.
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:
1. The patient (or parent/legal guardian) is unable or unwilling to comply with the study protocol.
2. The patient has severe neurological involvement as evidenced by:
* total or subtotal absence of cortical activity.
* untreatable seizures
* loss of (almost) all abilities to communicate.
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 I, II and VI.
MedDRA version: 12.0
Level: LLT
Classification code 10028095
Term: Mucopolysaccharidosis IV
MedDRA version: 12.0
Level: LLT
Classification code 10056886
Term: Mucopolysaccharidosis I
MedDRA version: 12.0
Level: PT
Classification code 10056889
Term: Mucopolysaccharidosis II
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Intervention(s)
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Trade Name: Elaprase
Product Name: idursulfase
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: IDURSULFASE
CAS Number: 50936-59-9
Other descriptive name: Elaprase
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 2-
Trade Name: Naglazyme
Product Name: Naglazyme
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: GALSULFASE
CAS Number: 552858-79-4
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 1-
Trade Name: Aldurazyme
Product Name: Aldurazyme
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: LARONIDASE
CAS Number: 210589-09-6
Other descriptive name: Aldurazyme
Concentration unit: IU/ml international unit(s)/millilitre
Concentration type: equal
Concentration number: 100-
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Primary Outcome(s)
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Main Objective: To increase knowledge on the long-term clinical effects of enzyme replacement therapy and to improve the level of care in children and adults with MPS I, II and VI.
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Primary end point(s): Survival
Physical endurance
Joint mobility
Cardiac size and function
Pulmonary function, apnea syndrome and need for respiratory support
Urine GAG levels
Size of liver and spleen
Corneal clouding and eye function
Morphometry of the face
Mental and social functioning
Quality of life
Costs
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Secondary Objective: To increase knowledge in the natural history of MPS I, II and VI;
To obtain data to evaluate continuation of reimbursement by the Dutch health authorities after the initial 3 years;
To assess the economic burden of MPS I, II and VI (inventory of the costs of (medical) care, informal care and loss of productivity) and the effects of enzyme therapy on these costs.
To improve the efficacy of enzyme therapy development of guidelines when to start or stop enzyme therapy;
To safely install enzyme therapy at home.
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Secondary ID(s)
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MPS protocol 08-06-07
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Source(s) of Monetary Support
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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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URL:
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