Phase 2
Completed N=25
Stem Cell Transplant w/Laronidase for Hurler
Mucopolysaccharidosis I · Hurler Syndrome
Source: ClinicalTrials.gov NCT00176891 ↗
Enrolled (actual)
25
Serious AEs
27.3%
Results posted
Jul 2019
Primary outcomePrimary: Number of Patients Alive at One Year Post Transplant — 18 Participants
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.
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 |
— | — |
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.
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. Informational only — not medical advice.