Protocolised follow-up of Pompe patients receiving enzyme replacement therapy on a compassionate use basis
| ISRCTN | ISRCTN72578000 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN72578000 |
| Secondary identifying numbers | N/A |
- Submission date
- 23/02/2007
- Registration date
- 23/02/2007
- Last edited
- 01/07/2016
- Recruitment status
- Recruiting
- Overall study status
- Ongoing
- Condition category
- Nutritional, Metabolic, Endocrine
Prospectively registered
Protocol
Statistical analysis plan
Results
Individual participant data
Record updated in last year
Plain English summary of protocol
Not provided at time of registration
Contact information
Dr A van der Ploeg
Scientific
Scientific
Erasmus Medical Centre
Sophia Children's Hospital
PO Box 2060
Rotterdam
3000 CB
Netherlands
| Phone | +31 (0)10 463 7044 |
|---|---|
| a.vanderploeg@erasmusmc.nl |
Study information
| Study design | Protocolised follow up of parallel group trial |
|---|---|
| Primary study design | Observational |
| Secondary study design | Single-centre |
| Study setting(s) | Other |
| Study type | Treatment |
| Participant information sheet | Not available in web format, please use contact details to request a participant information sheet |
| Scientific title | Protocolised follow-up of Pompe patients receiving enzyme replacement therapy on a compassionate use basis |
| Study objectives | 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(s) | Ethics approval received from the local medical ethics committee |
| Health condition(s) or problem(s) studied | Pompe Disease |
| Intervention | Enzyme replacement therapy |
| Intervention type | Drug |
| Pharmaceutical study type(s) | |
| Phase | Not Specified |
| Drug / device / biological / vaccine name(s) | Recombinant human alpha glucosidase |
| Primary outcome measure | 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 study start date | 01/01/1999 |
| Completion date | 01/01/2050 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Not Specified |
| Sex | Not Specified |
| Target number of participants | 12 |
| Key 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 |
| Key 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 |
| Date of first enrolment | 01/01/1999 |
| Date of final enrolment | 01/01/2050 |
Locations
Countries of recruitment
- Netherlands
Study participating centre
Erasmus Medical Centre
Rotterdam
3000 CB
Netherlands
3000 CB
Netherlands
Sponsor information
Erasmus Medical Centre (Netherlands)
Hospital/treatment centre
Hospital/treatment centre
Sophia Children's Hospital
Dr. Molewaterplein 60
Rotterdam
3015 GJ
Netherlands
| Website | http://www.erasmusmc.nl/ |
|---|---|
"ROR" |
https://ror.org/018906e22 |
Funders
Funder type
Industry
Genzyme Corporation (Netherlands)
No information available
Results and Publications
| Intention to publish date | |
|---|---|
| Individual participant data (IPD) Intention to share | No |
| IPD sharing plan summary | Not provided at time of registration |
| Publication and dissemination plan | Not provided at time of registration |
| IPD sharing plan |
