Contact information
Type
Scientific
Primary contact
Dr A van der Ploeg
ORCID ID
Contact details
Erasmus Medical Centre
Sophia Children's Hospital
PO Box 2060
Rotterdam
3000 CB
Netherlands
+31 (0)10 463 7044
a.vanderploeg@erasmusmc.nl
Additional identifiers
EudraCT number
ClinicalTrials.gov number
Protocol/serial number
N/A
Study information
Scientific title
Protocolised follow-up of Pompe patients receiving enzyme replacement therapy on a compassionate use basis
Acronym
Study hypothesis
Enzyme therapy with recombinant human alpha glucosidase results in:
1. Prolonged survival
2. Improvement or stabilisation of cardiac hypertrophy and function
3. Improvement or stabilisation of pulmonary function
4. Improvement or stabilisation of muscle function and strength
Ethics approval
Ethics approval received from the local medical ethics committee
Study design
Protocolised follow up of parallel group trial
Primary study design
Observational
Secondary study design
Single-centre
Trial setting
Other
Trial type
Treatment
Patient information sheet
Not available in web format, please use contact details to request a participant information sheet
Condition
Pompe Disease
Intervention
Enzyme replacement therapy
Intervention type
Drug
Phase
Not Specified
Drug names
Recombinant human alpha glucosidase
Primary outcome measures
1. Infantile: Survival
2. Late-onset: Improvement and/or stabilisation of muscle function
Secondary outcome measures
1. Infantile:
a. improvement of cardiac hypertrophy and function
b. achievement of motor milestones
2. Late-onset:
a. improvement and/or stabilisation of pulmonary function
b. improvement of quality of life
Overall trial start date
01/01/1999
Overall trial end date
01/01/2050
Reason abandoned
Eligibility
Participant inclusion criteria
1. Confirmed diagnosis of Pompe Disease
2. Infantile-onset:
2.1. Age less than one year
2.2. Delayed motor milestones, and/or
2.3. Hypertrophic cardiomyopathy
3. Late-onset:
3.1. 24 hour/day artificial ventilation
3.2. Wheelchair bound
3.3. Previously enrolled in AGLU 1202 study
Participant type
Patient
Age group
Not Specified
Gender
Not Specified
Target number of participants
12
Participant exclusion criteria
1. Infantile-onset:
1.1. congenital abnormalities
1.2. allergy to food and/or proteins
1.3. ventilator dependency
2. Late-onset:
2.1. developmental delays not explained by Pompe's Disease
2.2. allergies
2.3. severe co-morbidity
Recruitment start date
01/01/1999
Recruitment end date
01/01/2050
Locations
Countries of recruitment
Netherlands
Trial participating centre
Erasmus Medical Centre
Rotterdam
3000 CB
Netherlands
Sponsor information
Organisation
Erasmus Medical Centre (Netherlands)
Sponsor details
Sophia Children's Hospital
Dr. Molewaterplein 60
Rotterdam
3015 GJ
Netherlands
Sponsor type
Hospital/treatment centre
Website
Funders
Funder type
Industry
Funder name
Genzyme Corporation (Netherlands)
Alternative name(s)
Funding Body Type
Funding Body Subtype
Location
Results and Publications
Publication and dissemination plan
Not provided at time of registration
Intention to publish date
Participant level data
Not provided at time of registration
Results - basic reporting
Publication summary