Phase 2 N=25 Treatment

Stem Cell Transplant w/Laronidase for Hurler

Mucopolysaccharidosis I · Hurler Syndrome

Bottom Line

View on ClinicalTrials.gov: NCT00176891 ↗

Enrolled (actual)

Serious AEs

27.3%

Results posted

Jul 2019

Primary outcome: Primary: Number of Patients Alive at One Year Post Transplant — 18 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Stem Cell Transplant (Procedure); Laronidase ERT (Drug)
Age: Pediatric
Sex: All
Sponsor: Masonic Cancer Center, University of Minnesota
Primary completion: Nov 2012

Outcome Measures

Outcome	Result	p-value
PRIMARY Number of Patients Alive at One Year Post Transplant	18	—
PRIMARY Number of Patients Requiring Ventilator Support at One Year Post Transplant	6	—
SECONDARY Donor Engraftment	21	—
SECONDARY Patients With Grade III-IV Acute GVHD	1	—
SECONDARY Reduction in Glycosaminoglycans (GAG)	—	—
SECONDARY Toxicity (Adverse Events) Associated With Infusions of Laronidase	—	—
SECONDARY Development of Anti-iduronidase Antibodies in Serum	—	—
SECONDARY Patients With Improvement in Obstructive Apnea (Breathing) by Polysomnography	—	—

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.

Eligibility Criteria

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.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00176891). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.