Jackson-Weiss syndrome |
JWS |
Clinical Trial: MPS I Registry
This study is currently recruiting patients.
Verified by Genzyme September 2005
|
Purpose
| Condition | Intervention | Phase |
|---|---|---|
| Mucopolysaccharidosis I Hurler''''s Syndrome Hurler-Scheie Syndrome Scheie Syndrome | Procedure: Aldurazyme (Recombinant Human Alpha-L-Iduronidase) | Phase IV |
MedlinePlus related topics: Metabolic Disorders
Study Type: Observational
Study Design: Natural History, Longitudinal, Case Control, Retrospective/Prospective Study
Study start: October 2003
Last follow-up: October 2018
Eligibility
Inclusion Criteria:
- All patients with a confirmed diagnosis of MPS I are eligible for inclusion:
confirmed diagnosis is defined as: documented biochemical evidence of a deficiency in a-L-iduronidase enzyme activity and/or mutation and/or mutations(s) in the gene coding for a-L-iduronidase, or measurable clinical signs and symptoms of MPS I
- For all patients there should be a completed patient authorization form
Exclusion Criteria:
- no exclusion criteria in this Registry
Location and Contact Information
Medical Information 617-252-7832 medinfo@genzyme.com
California
Children''''s Hospital Los Angeles, Los Angeles, California, 90027, United States; Recruiting
Minnesota
Gene Therapy Center, Minneapolis, Minnesota, 55455, United States; Recruiting
More Information
Last Updated: September 2, 2005
Record first received: September 2, 2005
ClinicalTrials.gov Identifier: NCT00144794
Health Authority: United States: Food and Drug Administration
ClinicalTrials.gov processed this record on 2005-09-06
Resources
- Genetics Home Reference: Jackson-Weiss syndrome (Genetics Home Reference)

Not Signed In -


