Intrathecal Enzyme Replacement Therapy for Spinal Cord Co... | NCT00215527

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

NCT00215527

Sponsor

Patricia I. Dickson, M.D.

Status

Terminated

Last Update Posted

Feb 21, 2013Estimated

Enrollment

4Actual

Phase

Phase 1

Conditions

Mucopolysaccharidosis I
Lysosomal Storage Diseases
Spinal Cord Compression

Interventions

laronidase

Countries

United States
Finland

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

ID	Type	Description	Link
12069-01	Other Identifier	Los Angeles Biomedical Research Institute at Harbor-UCLA

Status Module

Record Verification Date

Feb 2013

Overall Recruitment Status or Expanded Access Status

Terminated

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Due to slow enrolment.

Expanded Access Info

Start Date

Nov 2005

Primary Completion Date

Oct 2011Actual

Completion Date

Oct 2011Actual

First Submitted Date

Sep 19, 2005

First Submission Date that Met QC Criteria

Sep 19, 2005

First Posted Date

Sep 22, 2005Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Feb 20, 2013

Last Update Posted Date

Feb 21, 2013Estimated

Name	Class
The Ryan Foundation	OTHER
University of California, Los Angeles	OTHER
FDA Office of Orphan Products Development	FED

Oversight Module

Has Data Monitoring Committee (DMC)

Yes

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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

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.

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 1

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

4Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
intrathecal laronidase	Experimental	laronidase dose 1.74 mg, route intrathecal, frequency every 30 days, duration three months	Drug: laronidase

Interventions

Name	Type	Description	Arm Group Labels	Other Names
laronidase	Drug	0.58 mg/ml solution for intravenous injection, dose 1.74 mg intrathecally once per month for four injections.	intrathecal laronidase

Measure	Description	Time Frame
safety of intrathecal enzyme treatment by blood and spinal fluid tests each month		four months

Measure	Description	Time Frame
improvement in spinal cord compression due to mucopolysaccharidosis I		four months

Eligibility Module

Eligibility 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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

8 Years

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Patricia I Dickson, M.D.	Los Angeles Biomedical Research Institute at Harbor-UCLA	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Los Angeles Biomedical Research Institute at Harbor-UCLA ( LA BioMed )		Torrance	California	90502	United States

Condition Browse Module

MeSH Terms

ID	Term
D008059	Mucopolysaccharidosis I
D016464	Lysosomal Storage Diseases
D013117	Spinal Cord Compression
D009083	Mucopolysaccharidoses

Ancestor Terms

ID	Term
D002239	Carbohydrate Metabolism, Inborn Errors
D008661	Metabolism, Inborn Errors
D030342	Genetic Diseases, Inborn
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Browse Leaves

Not provided

Browse Branches

Not provided

ID	Term
D006026	Glycoside Hydrolases
D006867	Hydrolases
D004798	Enzymes
D045762	Enzymes and Coenzymes