Phase 1
Completed N=11
Administration of IV Laronidase Post Bone Marrow Transplant in Hurler
Hurler Syndrome
Source: ClinicalTrials.gov NCT01173016 ↗
Enrolled (actual)
11
Serious AEs
9.1%
Results posted
Mar 2020
Primary outcomePrimary: Percentage of Adherence to the Scheduled Weekly Infusion by the Participants — 99 percentage
Summary
This is a single center pilot study in which Laronidase will be given weekly for two years in patients with Hurler syndrome, also known as mucopolysaccharide IH (MPS I, Hurler syndrome), that have previously been treated with an allogeneic transplant.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Adherence to the Scheduled Weekly Infusion by the Participants |
99 | — |
| PRIMARY Number of Participants Experiencing Severe Adverse Events |
1 | — |
| SECONDARY Changes in Growth Velocity |
-1.1 | — |
| SECONDARY Change in Muscle Strength |
1.1 | — |
| SECONDARY Change in Peak Heart Rate to Monitor "Fitness" |
23 | — |
| SECONDARY Number of Participants Showing Improvements in Joint Range of Motion (ROM) |
4; 3; 5 | — |
| SECONDARY Shortening Fraction to Determine Systolic Cardiac Function |
38 | — |
| SECONDARY Number of Participants With Changes in Cardiac Echo Structural Parameters |
7; 1; 5; 3 | — |
| SECONDARY Correlation of 6 Minute Walk Test With Anti-laronidase Antibody + Status |
-14 | 0.038 sig |
Eligibility Criteria
Inclusion Criteria
- Mucopolysaccharidosis type IH (MPS I, Hurler syndrome) treated with a prior allogeneic transplant >2 years previously
- Age 10% engrafted based on recent testing ( 3 months post transplantation
- Anticipated survival less than 2 years
- History of cardiac or pulmonary insufficiency, including an ejection fraction (EF) < 40% or those requiring continuous supplemental oxygen
Data sourced from ClinicalTrials.gov (NCT01173016). 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.