TerminatedNot Applicable

Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome

Stopped: Research Cancelled

United States20 participantsStarted 2012-03-28

Plain-language summary

This is a standard of care treatment guideline for patients with the diagnosis of mucopolysaccharidosis type IH (MPS I, Hurler syndrome) who are being considered as candidates for first hematopoietic stem cell transplantation (HSCT) according to a University of Minnesota myeloablative HSCT protocol.

Who can participate

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Diagnosis of mucopolysaccharidosis type IH (MPS I, Hurler syndrome) and being considered as a candidate for first transplant according to a University of Minnesota myeloablative hematopoietic stem cell transplant (HSCT) protocol Exclusion Criteria: * No prior therapy with laronidase enzyme replacement therapy (ERT)

What they're measuring

Overall Survival

Timeframe: At 1 Year

Trial details

NCT IDNCT01572636

SponsorMasonic Cancer Center, University of Minnesota

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2018-05-01

View on ClinicalTrials.gov More Mucopolysaccharidosis Type IH trials