Condition category
Nutritional, Metabolic, Endocrine
Date applied
23/02/2007
Date assigned
23/02/2007
Last edited
01/07/2016
Prospective/Retrospective
Retrospectively registered
Overall trial status
Ongoing
Recruitment status
Recruiting

Plain English Summary

Not provided at time of registration

Trial website

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

http://www.erasmusmc.nl/

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

Publication citations

Additional files

Editorial Notes