Mucopolysaccharidosis IPhase 1NCT00215527
Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I
A Phase 1 study testing laronidase in people with Mucopolysaccharidosis I and Lysosomal Storage Diseases, run by Patricia I. Dickson, M.D. at 2 sites in 2 countries.
Trial 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 Read more about this study
- Who is it for?
- Ages 8 years and older
- Conditions the study is for
- Mucopolysaccharidosis I, Lysosomal Storage Diseases, Spinal Cord Compression
- What is being tested?
- laronidase
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 2 sitesin 2 countries
- Study dates
- Nov 2005to Oct 2011
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 8 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 10Show fewer
- 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
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
United States1 site
Los Angeles Biomedical Research Institute at Harbor-UCLA ( LA BioMed )
Torrance, California, 90502
About this study
Official title: A Study of Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis I
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
Read the 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.
- Being tested
- laronidase
- Keywords
- mucopolysaccharidosis, Hurler-Scheie, Scheie, laronidase, spinal cord compression, central nervous system, enzyme replacement therapy, intrathecal, LA Biomed
- Medical subject headings
- Mucopolysaccharidosis I, Lysosomal Storage Diseases, Spinal Cord Compression, Mucopolysaccharidoses
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
laronidaseDrug
Also called Aldurazyme
0.58 mg/ml solution for intravenous injection, dose 1.74 mg intrathecally once per month for four injections.
| Study group | Role | What they receive |
|---|---|---|
| intrathecal laronidase | Experimental | laronidase dose 1.74 mg, route intrathecal, frequency every 30 days, duration three months Receives: Drug: laronidase |
What is being measured
The primary outcome is the main question the study is designed to answer.
safety of intrathecal enzyme treatment by blood and spinal fluid tests each month
Measured: four months
Secondary outcomes (1)
improvement in spinal cord compression due to mucopolysaccharidosis I
Measured: four months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Sep 2005 (estimated)
- Study startNov 2005
- Primary completionOct 2011
- Study completionOct 2011
Why it stopped: Due to slow enrolment.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Mucopolysaccharidosis IStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- Patricia I. Dickson, M.D.
- Collaborators
- The Ryan Foundation, University of California, Los Angeles, FDA Office of Orphan Products Development
- Responsible party
- Sponsor-investigator, Patricia I. Dickson, M.D., Dickson, Patricia I., M.D.
- Investigators
- Patricia I Dickson, M.D., Principal investigator, Los Angeles Biomedical Research Institute at Harbor-UCLA
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00215527
- Sponsor's protocol ID
- MIRC-001
- Other IDs
- 12069-01
- Status last verified
- Feb 2013
- First submitted
- 19 Sep 2005
- Last updated
- 21 Feb 2013
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 21 Feb 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Study of Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis I
- ClinicalTrials.gov ID
- NCT00215527
- Acronym / sponsor ID
- MIRC-001
- Keywords
- mucopolysaccharidosis, Hurler-Scheie, Scheie, laronidase, spinal cord compression, central nervous system, enzyme replacement therapy, intrathecal
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Due to slow enrolment.
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Patricia I. Dickson, M.D.
- Collaborators
- The Ryan Foundation, University of California, Los Angeles, FDA Office of Orphan Products Development
- Study start
- Nov 2005
- Primary completion
- Oct 2011 (actual)
- Study completion
- Oct 2011 (actual)
- First posted
- 22 Sep 2005 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| intrathecal laronidase | Experimental | Drug: laronidaselaronidase dose 1.74 mg, route intrathecal, frequency every 30 days, duration three months |
Who may qualify
- Ages
- 8 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- 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
Key exclusion criteria (10 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| laronidaseAlso: Aldurazyme0.58 mg/ml solution for intravenous injection, dose 1.74 mg intrathecally once per month for four injections. | Drug | intrathecal laronidase |
Primary outcome
safety of intrathecal enzyme treatment by blood and spinal fluid tests each month
Time frame: four months
1 secondary outcome
improvement in spinal cord compression due to mucopolysaccharidosis I
Time frame: four months
Results and safety
The study was stopped early (reason given: Due to slow enrolment.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Feb 2013.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Patricia I Dickson, M.D. principal investigator
Los Angeles Biomedical Research Institute at Harbor-UCLA
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing laronidase in people with Mucopolysaccharidosis I and Lysosomal Storage Diseases, run by Patricia I. Dickson, M.D. at 2 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 21 Feb 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States, Finland. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Due to slow enrolment.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
safety of intrathecal enzyme treatment by blood and spinal fluid tests each month (time frame: four months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Feb 2013.