CompletedPhase 2

Stem Cell Transplant w/Laronidase for Hurler

United States25 participantsStarted 2004-03

Plain-language summary

The investigators hypothesize that weekly infusions of Laronidase ERT for 10-12 weeks prior to transplant and 8 weeks following transplant will result in a reduction of glycosaminoglycans (GAG) burden that is associated with decreased complications following transplant.

Who can participate

Age range7 Years

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: * Patients with the diagnosis of mucopolysaccharidosis type IH (MPS I, Hurler syndrome) who are candidates for first hematopoietic stem cell transplant (HSCT) according to a University of Minnesota myeloablative HSCT protocol. Exclusion Criteria: * Not being considered for University of Minnesota myeloablative HSCT protocol. * Previous administration of laronidase enzyme * Second or subsequent HSCT.

What they're measuring

Number of Patients Alive at One Year Post Transplant

Timeframe: one year

Number of Patients Requiring Ventilator Support at One Year Post Transplant

Timeframe: one year

Trial details

NCT IDNCT00176891

SponsorMasonic Cancer Center, University of Minnesota

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2012-11

Results submitted2017-04-06

View on ClinicalTrials.gov More Mucopolysaccharidosis I trials