Extension Study of Intrathecal Enzyme Replacement Therapy for MPS I

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00786968
• Date of registration: 17/06/2008
• Prospective Registration: No
• Primary sponsor: Patricia I. Dickson, M.D.
• Public title: Extension Study of Intrathecal Enzyme Replacement Therapy for MPS I
• Scientific title: An Extension Study of Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis I
• Date of first enrolment: January 2008
• Target sample size: 3
• Recruitment status: Terminated
• URL: http://clinicaltrials.gov/show/NCT00786968
• Study type: Interventional
• Study design: Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 1

Countries of recruitment

Finland, United States

Contacts

• Name: Patricia I Dickson, MD
• Affiliation: Los Angeles Biomedical Research Institute at Harbor-UCLA

Key inclusion & exclusion criteria

Inclusion Criteria:

- Hurler-Scheie, Scheie form of MPS I, or Hurler 2 years after hematopoietic stem cell
transplantation

- Spinal cord compression

- Has received intrathecal laronidase previously with good response and no
significant safety concerns

- 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 (nonsterile females of child-bearing
potential who are sexually active only)

Exclusion Criteria:

- 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



Age minimum: 8 Years
Age maximum: N/A
Gender: Both

Health Condition(s) or Problem(s) studied

Lysosomal Storage Disease

Mucopolysaccharidosis I

Scheie Syndrome

Hurler-Scheie Syndrome

Spinal Cord Compression

Intervention(s)

Drug: laronidase

Primary Outcome(s)

safety of intrathecal enzyme treatment by blood and spinal fluid tests; improvement or stabilization in neurologic signs and symptoms of spinal cord compression [Time Frame: 1 year]

Secondary ID(s)

MIRC-001-01

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

The Ryan Foundation for MPS Children