Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I

NCT00215527 | Terminated Phase 1 DMC

• 2 other identifiers: MIRC-00112069-01
• Study Type: interventional
• Target: 4
• Locations: 2 countries
• Sites: 2

Brief Summary

The investigators are studying the use of enzyme replacement therapy into the spinal fluid for treatment of spinal cord compression in the Hurler-Scheie and Scheie forms of mucopolysaccharidosis I (MPS I). Funding source -- FDA OOPD

Conditions

Mucopolysaccharidosis I; Lysosomal Storage Diseases; Spinal Cord Compression

Keywords

mucopolysaccharidosis; Hurler-Scheie; Scheie; laronidase; spinal cord compression; central nervous system; enzyme replacement therapy; intrathecal; LA Biomed

Outcome Measures

Primary Outcomes (1)

• safety of intrathecal enzyme treatment by blood and spinal fluid tests each month

  four months

Secondary Outcomes (1)

• improvement in spinal cord compression due to mucopolysaccharidosis I

  four months

Study Arms (1)

intrathecal laronidase

EXPERIMENTAL

laronidase dose 1.74 mg, route intrathecal, frequency every 30 days, duration three months

Drug: laronidase

Interventions

laronidase DRUG

0.58 mg/ml solution for intravenous injection, dose 1.74 mg intrathecally once per month for four injections.

Also known as: Aldurazyme

intrathecal laronidase

Eligibility Criteria

• Age: 8 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Hurler-Scheie,Scheie form of MPS I, of Hurler 2 years after hematopoietic stem cell transplantation
• Spinal cord compression
• Age greater than 8 years
• Able to provide legal informed consent
• Aware of clinical treatment option of observation without treatment or surgical decompression
• Negative urine pregnancy test at screening (non-sterile females of child-bearing potential only)
• Currently using two acceptable methods of birth control (non-sterile females of child-bearing potential who are sexually active only)
• Willing and able to comply with study procedures

You may not qualify if:

• Severe (Hurler) form of MPS I
• Desires surgical or medical treatment of spinal cord compression
• Spinal cord compression that warrants immediate surgical intervention
• Pregnancy or lactation
• Hematopoietic stem cell transplantation within 2 years of study enrollment
• Receipt of an investigational drug within 30 days of enrollment
• Infusion reactions to laronidase that required medical intervention, prophylaxis, or altered enzyme administration
• Significant anti-iduronidase antibody titer
• Recent initiation of intravenous laronidase (within past 6 months)
• Presence of cervical subluxation or similar external pathology as the major cause of cord compression symptoms for which surgical intervention should be immediately undertaken

Sponsors & Collaborators

• Patricia I. Dickson, M.D.: lead
• The Ryan Foundation: collaborator
• University of California, Los Angeles: collaborator
• FDA Office of Orphan Products Development: collaborator

Study Sites (2)

Los Angeles Biomedical Research Institute at Harbor-UCLA ( LA BioMed )

Torrance, California, 90502, United States

Location

Helsinki University Central Hospital

Helsinki, FI-00014, Finland

Location

MeSH Terms

Conditions

Mucopolysaccharidosis I; Lysosomal Storage Diseases; Spinal Cord Compression; Mucopolysaccharidoses

Interventions

Iduronidase

Condition Hierarchy (Ancestors)

Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Mucinoses; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Spinal Cord Injuries; Wounds and Injuries

Intervention Hierarchy (Ancestors)

Glycoside Hydrolases; Hydrolases; Enzymes; Enzymes and Coenzymes

Study Officials

• Patricia I Dickson, M.D.

  Los Angeles Biomedical Research Institute at Harbor-UCLA

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDIV
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Associate Professor of Pediatrics

Study Record Dates

• First Submitted: September 19, 2005
• First Posted: September 22, 2005
• Study Start: November 1, 2005
• Primary Completion: October 1, 2011
• Study Completion: October 1, 2011
• Last Updated: February 21, 2013

Record last verified: 2013-02

Locations

FI (1); US (1)