Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I
This study has been terminated.
(Due to slow enrolment.)
Sponsor:
Patricia I. Dickson, M.D.
Collaborators:
Ryan Foundation for MPS Children
University of California, Los Angeles
Information provided by (Responsible Party):
Patricia I. Dickson, M.D., Dickson, Patricia I., M.D.
ClinicalTrials.gov Identifier:
NCT00215527
First received: September 19, 2005
Last updated: February 20, 2013
Last verified: February 2013
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Purpose
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
| Condition | Intervention | Phase |
|---|---|---|
|
Mucopolysaccharidosis I Lysosomal Storage Diseases Spinal Cord Compression |
Drug: laronidase |
Phase 1 |
| Study Type: | Interventional |
| Study Design: | Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | A Study of Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis I |
Resource links provided by NLM:
Further study details as provided by Dickson, Patricia I., M.D.:
Primary Outcome Measures:
- safety of intrathecal enzyme treatment by blood and spinal fluid tests each month [ Time Frame: four months ] [ Designated as safety issue: Yes ]
Secondary Outcome Measures:
- improvement in spinal cord compression due to mucopolysaccharidosis I [ Time Frame: four months ] [ Designated as safety issue: No ]
| Enrollment: | 4 |
| Study Start Date: | November 2005 |
| Study Completion Date: | October 2011 |
| Primary Completion Date: | October 2011 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Experimental: intrathecal laronidase
laronidase dose 1.74 mg, route intrathecal, frequency every 30 days, duration three months
|
Drug: laronidase
0.58 mg/ml solution for intravenous injection, dose 1.74 mg intrathecally once per month for four injections.
Other Name: Aldurazyme
|
Detailed Description:
Enzyme replacement therapy (ERT) has been developed for mucopolysaccharidosis I (MPS I), a lysosomal storage disorder. ERT helps many physical ailments due to the disease, but does not treat the central nervous system, due to inability to cross the blood brain barrier. Our purpose is to test delivery of ERT to the spinal fluid via intrathecal injection in patients with MPS I. In this pilot study, we will use recombinant human α-L-iduronidase administered intrathecally once per month for four months to individuals with the Hurler-Scheie and Scheie forms of MPS I and spinal cord compression. If successful, intrathecal delivery could represent a practical, straightforward method of treating central nervous system disease due to lysosomal storage.
Eligibility| Ages Eligible for Study: | 8 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- 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
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
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00215527
Locations
| United States, California | |
| Los Angeles Biomedical Research Institute at Harbor-UCLA ( LA BioMed ) | |
| Torrance, California, United States, 90502 | |
| Finland | |
| Helsinki University Central Hospital | |
| Helsinki, Finland, FI-00014 | |
Sponsors and Collaborators
Patricia I. Dickson, M.D.
Ryan Foundation for MPS Children
University of California, Los Angeles
Investigators
| Principal Investigator: | Patricia I Dickson, M.D. | Los Angeles Biomedical Research Institute at Harbor-UCLA |
More Information
No publications provided
| Responsible Party: | Patricia I. Dickson, M.D., Associate Professor of Pediatrics, Dickson, Patricia I., M.D. |
| ClinicalTrials.gov Identifier: | NCT00215527 History of Changes |
| Other Study ID Numbers: | MIRC-001, 12069-01 |
| Study First Received: | September 19, 2005 |
| Last Updated: | February 20, 2013 |
| Health Authority: | United States: Food and Drug Administration |
Keywords provided by Dickson, Patricia I., M.D.:
|
mucopolysaccharidosis Hurler-Scheie Scheie laronidase spinal cord compression |
central nervous system enzyme replacement therapy intrathecal LA Biomed |
Additional relevant MeSH terms:
|
Mucopolysaccharidosis I Mucopolysaccharidoses Spinal Cord Compression Lysosomal Storage Diseases Metabolic Diseases Carbohydrate Metabolism, Inborn Errors Metabolism, Inborn Errors Genetic Diseases, Inborn |
Mucinoses Connective Tissue Diseases Spinal Cord Diseases Central Nervous System Diseases Nervous System Diseases Spinal Cord Injuries Wounds and Injuries |
ClinicalTrials.gov processed this record on May 19, 2013