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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: |
EUCTR2010-024647-32-IT |
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Date of registration:
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09/05/2011 |
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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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Combined enzyme enhancement therapy (EET) and enzyme replacement therapy (ERT) in patients with Pompe disease - ND
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Scientific title:
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Combined enzyme enhancement therapy (EET) and enzyme replacement therapy (ERT) in patients with Pompe disease - ND |
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Date of first enrolment:
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18/03/2011 |
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Target sample size:
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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:2010-024647-32 |
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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: no
Open: no
Single blind: no
Double blind: no
Parallel group: no
Cross over: no
Other: no
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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Italy
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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 and / or parents / legal tutors signed informed consent -Patients of both sex younger than 65 years - The patient has a diagnosis of Pompe Disease confirmed by analysis of acid a-glucosidase enzyme activity (determined on leukocytes, muscle, or fibroblasts) and molecular analysis of the GAA gene. - The patient is on enzyme replacement therapy (ERT) for at least 6 months -Fertile female with negative BhCG -Use of contraception in fertile male patients until 3 months after the last administration of investigational drug
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 has major congenital anomalies - The patient has comorbidities (excluding symptoms related to Pompe disease) involving organs and systems, which in the opinion of the investigator would preclude their inclusion in the study and potentially reduce their survival -Female patients during pregnancy -Non compliant patients
Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes
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Health Condition(s) or Problem(s) studied
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Patients with Pompe Disease (Type II Glycogenosis) on therapy with Enzyme Replacement Therapy (ERT) for at least 6 months
MedDRA version: 13.1
Level: LLT
Classification code 10036143
Term: Pompe's disease
System Organ Class: 10010331 - Congenital, familial and genetic disorders
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Intervention(s)
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Trade Name: ZAVESCA
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: Miglustat
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 100-
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Primary Outcome(s)
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Primary end point(s): -Increase in the peak of activity of GAA and / or slowing of the decline in levels of activity of GAA -reduction in serum CPK
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Main Objective: Evaluation of efficacy of therapy with Miglustat in co-administration with ERT considering: increase in the peak of activity of acid glucosidase (GAA) e/o slowing of the decline of levels of activity of GAA; decrease in serum levels of CPK
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Secondary Objective: -Evaluation of stabilization and/or improvement in muscular efficiency -Evaluation of safety of Miglustat in patients with Pompe disease in co-administration with ERT
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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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