Administration of IV Laronidase Post Bone Marrow Transplant in Hurler

NCT ID: NCT01173016

Last Updated: 2020-03-20

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 11 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2012-05-29
• Study Completion Date: 2016-03-04

Brief 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.

Detailed Description

This 2-year open-label pilot study of laronidase includes patients (age 5-13 years) who are at least 2 years post-hematopoietic cell transplantation (HCT) and donor engrafted. Outcomes are assessed semi-annually and compared to historic controls. Eligible patients will receive Laronidase as an infusion over several hours once a week at a local site. The dosing of enzyme will be the standard doses recommended by Genzyme.

The findings of this Pilot Study will be used to assess whether a subsequent larger study can be conducted.

Conditions

Hurler Syndrome

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Laronidase After Transplantation

Patients with Mucopolysaccharidosis type IH (MPS I, Hurler syndrome) treated with a prior allogeneic transplant \>2 years previously and treated with Laronidase weekly for 2 years after transplant.

Group Type: EXPERIMENTAL

Laronidase

Intervention Type: DRUG

Laronidase 0.58 mg/kg intravenously (IV) once a week for a maximum of 2 years

Interventions

Laronidase

Laronidase 0.58 mg/kg intravenously (IV) once a week for a maximum of 2 years

Intervention Type: DRUG

Other Intervention Names

Aldurazyme

Eligibility Criteria

Inclusion Criteria

• Mucopolysaccharidosis type IH (MPS I, Hurler syndrome) treated with a prior allogeneic transplant >2 years previously
• Age <14 years old
• >10% engrafted based on recent testing (<4 months prior to enrollment)
• Willing to commit to traveling to the University of Minnesota every 6 months
• Written informed consent prior to the performance of any study related procedures

Exclusion Criteria

• Previous administration of Laronidase enzyme > 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

• Maximum Eligible Age: 14 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Masonic Cancer Center, University of Minnesota

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Paul Orchard, MD

Role: PRINCIPAL_INVESTIGATOR

Masonic Cancer Center, University of Minnesota

Locations

Masonic Cancer Center, University of Minnesota

Minneapolis, Minnesota, United States

Countries

United States

References

Lund TC, Miller WP, Liao AY, Tolar J, Shanley R, Pasquali M, Sando N, Bigger BW, Polgreen LE, Orchard PJ. Post-transplant laronidase augmentation for children with Hurler syndrome: biochemical outcomes. Sci Rep. 2019 Oct 1;9(1):14105. doi: 10.1038/s41598-019-50595-1.

Reference Type: DERIVED

PMID: 31575939 (View on PubMed)

Related Links

https://www.nature.com/articles/s41390-019-0541-2

Related Info

https://www.nature.com/articles/s41598-019-50595-1#citeas

Related Info

Other Identifiers

MT2009-20

Identifier Type: OTHER

Identifier Source: secondary_id

1004M80513

Identifier Type: OTHER

Identifier Source: secondary_id

2009LS090

Identifier Type: -

Identifier Source: org_study_id