Hurler SyndromePhase 1NCT00638547
Intrathecal Enzyme Replacement for Hurler Syndrome
A Phase 1 study testing IRT Laronidase in people with Hurler Syndrome, run by Masonic Cancer Center, University of Minnesota at 1 site in United States.
Trial summary
This protocol will examine whether the enzyme alpha-L-iduronidase (Laronidase), delivered into the spinal fluid of patients with Hurler syndrome at intervals before and after bone marrow transplant, is a safe and effective approach to slow the neurologic degeneration seen in Hurler patients undergoing transplantation. Read more about this study
- Who is it for?
- Ages 6 months to 3 years
- Conditions the study is for
- Hurler Syndrome
- What is being tested?
- IRT Laronidase
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 26 participants
- Where
- 1 siteUnited States
- Study dates
- 2 Jan 2008to 18 Nov 2018
- 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 6 months to 3 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Patients with a diagnosis of MPS IH (Hurler syndrome) are candidates for this protocol if they are being considered for hematopoietic stem cell transplantation according the University of Minnesota guidelines.
Who cannot join
- Patients are less than 6 months old, or older than 3 years of age.
- There is a history of clinically-severe hypersensitivity to Laronidase.
- There is a contraindication for repeated lumbar puncture.
- The family is not willing to undergo the necessary procedures and evaluations inherent in the study.
- Consent has not been signed for participation in the 2004-09 study of intravenous Laronidase administration.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Minnesota, Fairview
Minneapolis, Minnesota, 55455
About this study
Official title: Intrathecal Enzyme Replacement Therapy For Patients With Mucopolysaccharidosis Type I (Hurler Syndrome)
This protocol will examine whether the enzyme alpha-L-iduronidase (Laronidase), delivered into the spinal fluid of patients with Hurler syndrome at intervals before and after bone marrow transplant, is a safe and effective approach to slow the neurologic degeneration seen in Hurler patients undergoing transplantation.
Read the detailed description
Subjects will receive an infusion of Laronidase into his/her spinal fluid approximately 12 weeks before, 2 weeks before, 100 days after and 6 months after transplant. This procedure is done by lumbar puncture (also called a "spinal tap").
- Conditions
- Hurler Syndrome
- Being tested
- IRT Laronidase
- Keywords
- Hurler Syndrome, mucopolysaccharidosis type I, Iduronidase deficiency
- Medical subject headings
- Mucopolysaccharidosis I
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IRT LaronidaseDrug
Also called Aldurazyme
Laronidase belongs to a class of drugs called enzyme replacement therapies or ERT that provides people with sufficient quantities of an important enzyme that they cannot create on their own. The main ingredient in laronidase is a protein that is identical to a naturally occurring form of the human enzyme alpha-L-iduronidase. Laronidase replaces the missing enzyme alpha-L-iduronidase and restores sufficient enzyme activity to break down GAG buildup.
Subjects will receive an infusion of Laronidase into his/her spinal fluid approximately 12 weeks before, 2 weeks before, 100 days after and 6 months after transplant. This procedure is done by lumbar puncture
| Study group | Role | What they receive |
|---|---|---|
| Intent-to-Treat | Experimental | All patients who have received at least one dose of Laronidase. Receives: Drug: IRT Laronidase |
What is being measured
The primary outcome is the main question the study is designed to answer.
To demonstrate the efficacy of intrathecally delivering alpha-L-iduronidase in patients with mucopolysaccharidosis type I in decreasing neurodevelopmental deterioration
Measured: 1 year
Secondary outcomes (4)
To determine the safety and toxicity of intrathecally delivering alpha-L-iduronidase in patients with mucopolysaccharidosis type I
Measured: 1 year
To determine brain changes with magnetic resonance imaging
Measured: 1 and 2 years
To determine neurocognitive changes present in patients with Hurler syndrome
Measured: 6, 12, and 24 months
To determine cerebral spinal fluid levels of glycosaminoglycans, cytokines and antibodies to Laronidase at baseline and at each point CSF is obtained
Measured: through 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Mar 2008 (estimated)
- Study start2 Jan 2008
- Primary completion18 Feb 2017
- Study completion18 Nov 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hurler SyndromeStudies 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
- 26 participants (actual)
Sponsor and investigators
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Paul Orchard, MD, Principal investigator, University of Minnesota Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00638547
- Sponsor's protocol ID
- MT2007-10
- Other IDs
- 0707M11762
- Status last verified
- Nov 2018
- First submitted
- 11 Mar 2008
- Last updated
- 3 Dec 2018
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 3 Dec 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Intrathecal Enzyme Replacement Therapy For Patients With Mucopolysaccharidosis Type I (Hurler Syndrome)
- ClinicalTrials.gov ID
- NCT00638547
- Acronym / sponsor ID
- MT2007-10
- Conditions
- Hurler Syndrome
- Keywords
- Hurler Syndrome, mucopolysaccharidosis type I, Iduronidase deficiency
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 26 participants (actual)
- Lead sponsor
- Masonic Cancer Center, University of MinnesotaUniversity, hospital or other
- Study start
- 2 Jan 2008 (actual)
- Primary completion
- 18 Feb 2017 (actual)
- Study completion
- 18 Nov 2018 (actual)
- First posted
- 19 Mar 2008 (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.
This protocol will examine whether the enzyme alpha-L-iduronidase (Laronidase), delivered into the spinal fluid of patients with Hurler syndrome at intervals before and after bone marrow transplant, is a safe and effective approach to slow the neurologic degeneration seen in Hurler patients undergoing transplantation.
Detailed description
Subjects will receive an infusion of Laronidase into his/her spinal fluid approximately 12 weeks before, 2 weeks before, 100 days after and 6 months after transplant. This procedure is done by lumbar puncture (also called a "spinal tap").
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Intent-to-Treat | Experimental | Drug: IRT LaronidaseAll patients who have received at least one dose of Laronidase. |
Who may qualify
- Ages
- 6 months to 3 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (1 listed)
- Patients with a diagnosis of MPS IH (Hurler syndrome) are candidates for this protocol if they are being considered for hematopoietic stem cell transplantation according the University of Minnesota guidelines.
Key exclusion criteria (5 listed)
- Patients are less than 6 months old, or older than 3 years of age.
- There is a history of clinically-severe hypersensitivity to Laronidase.
- There is a contraindication for repeated lumbar puncture.
- The family is not willing to undergo the necessary procedures and evaluations inherent in the study.
- Consent has not been signed for participation in the 2004-09 study of intravenous Laronidase administration.
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 |
|---|---|---|
| IRT LaronidaseAlso: AldurazymeLaronidase belongs to a class of drugs called enzyme replacement therapies or ERT that provides people with sufficient quantities of an important enzyme that they cannot create on their own. The main ingredient in laronidase is a protein that is identical to a naturally occurring form of the human enzyme alpha-L-iduronidase. Laronidase replaces the missing enzyme alpha-L-iduronidase and restores sufficient enzyme activity to break down GAG buildup. Subjects will receive an infusion of Laronidase into his/her spinal fluid approximately 12 weeks before, 2 weeks before, 100 days after and 6 months after transplant. This procedure is done by lumbar puncture | Drug | Intent-to-Treat |
Primary outcome
To demonstrate the efficacy of intrathecally delivering alpha-L-iduronidase in patients with mucopolysaccharidosis type I in decreasing neurodevelopmental deterioration
Time frame: 1 year
4 secondary outcomes
To determine the safety and toxicity of intrathecally delivering alpha-L-iduronidase in patients with mucopolysaccharidosis type I
Time frame: 1 year
To determine brain changes with magnetic resonance imaging
Time frame: 1 and 2 years
To determine neurocognitive changes present in patients with Hurler syndrome
Time frame: 6, 12, and 24 months
To determine cerebral spinal fluid levels of glycosaminoglycans, cytokines and antibodies to Laronidase at baseline and at each point CSF is obtained
Time frame: through 1 year
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Dec 2018.
Related trials for Hurler Syndrome
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ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I
Mucopolysaccharidosis IH/S, Mucopolysaccharidosis IS
Active, not recruitingPhase 1/2
Gene Therapy With Modified Autologous Hematopoietic Stem Cells for the Treatment of Patients With Mucopolysaccharidosis Type I, Hurler Variant
Mucopolysaccharidosis IH
Enrolling by invitationObservational study
Long Term Follow-Up for RGX-111
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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.
Paul Orchard, MD principal investigator
University of Minnesota Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing IRT Laronidase in people with Hurler Syndrome, run by Masonic Cancer Center, University of Minnesota at 1 site in United States.
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 1 inclusion and 5 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: finished, as of the sponsor's last update on 3 Dec 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
To demonstrate the efficacy of intrathecally delivering alpha-L-iduronidase in patients with mucopolysaccharidosis type I in decreasing neurodevelopmental deterioration (time frame: 1 year). 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 3 Dec 2018.