Administration of IV Laronidase Post Bone Marrow Transplant in Hurler

NCT01173016 | Completed Phase 1 Results DMC

• 3 other identifiers: 2009LS090MT2009-201004M80513
• Study Type: interventional
• Target: 11
• Locations: 1 country
• Sites: 1

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.

Conditions

Hurler Syndrome

Keywords

mucopolysaccharide IH (MPS IH)

Outcome Measures

Primary Outcomes (2)

• Percentage of Adherence to the Scheduled Weekly Infusion by the Participants

  To determine the feasibility of giving weekly Laronidase for 2 years in patients with Hurler syndrome after allogeneic transplantation, compliance throughout the study with drug administration, the percentage of adherence to the scheduled weekly infusion for each participant is measured.

  24 months

• Number of Participants Experiencing Severe Adverse Events

  Number of participants experiencing severe adverse events that occur after administration with Laronidase to determine the feasibility of giving weekly Laronidase

  24 months

Secondary Outcomes (7)

• Changes in Growth Velocity

  Baseline, Month 24

• Change in Muscle Strength

  Assessed from baseline every 6 months through 2 years; change between baseline and 24 months reported.If baseline and/or 24-month data were not available, the longest interval between measurements was reported, with a minimum requirement of 12 months

• Change in Peak Heart Rate to Monitor "Fitness"

  Assessed from baseline every 6 months through 2 years; change between baseline and 24 months reported.If baseline and/or 24-month data were not available, the longest interval between measurements was reported, with a minimum requirement of 12 months

• Number of Participants Showing Improvements in Joint Range of Motion (ROM)

  Assessed from baseline every 6 months through 2 years; change between baseline and 24 months reported.If baseline and/or 24-month data were not available, the longest interval between measurements was reported, with a minimum requirement of 12 months

• Shortening Fraction to Determine Systolic Cardiac Function

  Baseline and month 24

Study Arms (1)

Laronidase After Transplantation

EXPERIMENTAL

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.

Drug: Laronidase

Interventions

Laronidase DRUG

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

Also known as: Aldurazyme

Laronidase After Transplantation

Eligibility Criteria

• Age: Up to 14 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Masonic Cancer Center, University of Minnesota: lead

Study Sites (1)

Masonic Cancer Center, University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

Related Publications (1)

• 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.

  PMID: 31575939 DERIVED

Related Links

• Related Info
• Related Info

MeSH Terms

Conditions

Mucopolysaccharidosis I

Interventions

Iduronidase

Condition Hierarchy (Ancestors)

Mucopolysaccharidoses; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lysosomal Storage Diseases; Mucinoses; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Glycoside Hydrolases; Hydrolases; Enzymes; Enzymes and Coenzymes

Limitations and Caveats

All listed serious and non-serious adverse events are infusion related except for a seizure that was thought to be unrelated to the study drug.

Results Point of Contact

• Title: Dr. Paul Orchard
• Organization: Masonic Cancer Center, University of Minnesota

orcha001@umn.edu

612-626-2313

Study Officials

• Paul Orchard, MD

  Masonic Cancer Center, University of Minnesota

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 28, 2010
• First Posted: July 30, 2010
• Study Start: May 29, 2012
• Primary Completion: March 4, 2016
• Study Completion: March 4, 2016
• Last Updated: March 20, 2020
• Results First Posted: March 20, 2020

Record last verified: 2020-03

Locations

US (1)