Mucopolysaccharidosis IPhase 4NCT00144768
A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated Patients
A Phase 4 study testing laronidase in people with Mucopolysaccharidosis I and Hurler's Syndrome, run by Genzyme, a Sanofi Company at 3 sites in United States.
Trial summary
The purpose of this study is to determine whether the development of antibodies to laronidase in patients with MPS I receiving Aldurazyme® impairs the clearance of GAG substrate. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Mucopolysaccharidosis I, Hurler's Syndrome, Hurler-Scheie Syndrome
- What is being tested?
- laronidase
- Type of treatment
- Injection or infusion
- How many people
- 25 participants
- Where
- 3 sitesUnited States
- Study dates
- Jul 2004to May 2007
- Phase
- Phase 4
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Have a documented diagnosis of MPS I confirmed by measurable signs and symptoms and deficient α-L-iduronidase activity (<10% of the lower limit of normal).
- For a patients receiving Aldurazyme therapy prior to study entry: Have available as baseline data the results of urinary GAG levels and IgG antibody titers collected prior to the patient's first Aldurazyme infusion.
- Provide signed, written informed consent prior to any protocol-related procedures being performed. Consent of a legally authorized guardian(s) is (are) required for patients under 18 years.
Who cannot join
- Have previously received Aldurazyme without the collection of baseline samples as specified.
- Have a suspected hypersensitivity to Aldurazyme or a know hypersensitivity to components of infusion solution.
- Have received a Hematopoietic Stem Cell Transplantation, injection fibroblast therapy, or major organ transplant.
- Are receiving chronic immunosuppressant therapy.
- Have a medical condition, serious intercurrent illness, or other extenuating circumstances that may interfere with study compliance including all prescribed evaluations and follow-up activities.
- Are pregnant or lactating
- Have received investigational drug within 30 days prior to study enrollment
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Connecticut Children's Medical Center
Hartford, Connecticut, 06106
Childrens Hospital Los Angeles
Los Angles, California, 90027
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, 53226
About this study
Official title: A Multicenter, Multinational, Open-Label Study of Anti-Laronidase Antibody Formation and Urinary GAG Levels in Patients With Mucopolysaccharidosis I (MPS I) Being Treated With Aldurazyme® (Laronidase).
The purpose of this study is to determine whether the development of antibodies to laronidase in patients with MPS I receiving Aldurazyme® impairs the clearance of GAG substrate.
- Being tested
- laronidase
- Medical subject headings
- Mucopolysaccharidosis I
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
laronidaseDrug
Also called Recombinant Human Alpha-L-Iduronidase, Aldurazyme®
dose of 0.58mg/kg body weight IV every week
What is being measured
The primary outcome is the main question the study is designed to answer.
Urinary GAG (glycosaminoglycans)
Measured: Up to 4 years
Immunogenicity Testing
Measured: Up to 4 years
Secondary outcomes (1)
Safety
Measured: Up to 4 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Sep 2005 (estimated)
- Study startJul 2004
- Primary completionMay 2007
- Study completionMay 2007
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 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 25 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Genzyme, a Sanofi Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- BioMarin/Genzyme LLC
- Responsible party
- Sponsor
- Investigators
- Medical Monitor, Study director, Genzyme, a Sanofi Company
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00144768
- Sponsor's protocol ID
- ALID02003
- Status last verified
- Apr 2013
- First submitted
- 2 Sep 2005
- Last updated
- 5 May 2014
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 May 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Multinational, Open-Label Study of Anti-Laronidase Antibody Formation and Urinary GAG Levels in Patients With Mucopolysaccharidosis I (MPS I) Being Treated With Aldurazyme® (Laronidase).
- ClinicalTrials.gov ID
- NCT00144768
- Acronym / sponsor ID
- ALID02003
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 25 participants (estimated)
- Lead sponsor
- Genzyme, a Sanofi CompanyIndustry (pharma, biotech, devices)
- Collaborators
- BioMarin/Genzyme LLC
- Study start
- Jul 2004
- Primary completion
- May 2007 (actual)
- Study completion
- May 2007 (actual)
- First posted
- 5 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 purpose of this study is to determine whether the development of antibodies to laronidase in patients with MPS I receiving Aldurazyme® impairs the clearance of GAG substrate.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Have a documented diagnosis of MPS I confirmed by measurable signs and symptoms and deficient α-L-iduronidase activity (<10% of the lower limit of normal).
- For a patients receiving Aldurazyme therapy prior to study entry: Have available as baseline data the results of urinary GAG levels and IgG antibody titers collected prior to the patient's first Aldurazyme infusion.
- Provide signed, written informed consent prior to any protocol-related procedures being performed. Consent of a legally authorized guardian(s) is (are) required for patients under 18 years.
Key exclusion criteria (7 listed)
- Have previously received Aldurazyme without the collection of baseline samples as specified.
- Have a suspected hypersensitivity to Aldurazyme or a know hypersensitivity to components of infusion solution.
- Have received a Hematopoietic Stem Cell Transplantation, injection fibroblast therapy, or major organ transplant.
- Are receiving chronic immunosuppressant therapy.
- Have a medical condition, serious intercurrent illness, or other extenuating circumstances that may interfere with study compliance including all prescribed evaluations and follow-up activities.
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: Recombinant Human Alpha-L-Iduronidase, Aldurazyme®dose of 0.58mg/kg body weight IV every week | Drug | – |
Primary outcome
Urinary GAG (glycosaminoglycans)
Time frame: Up to 4 years
Immunogenicity Testing
Time frame: Up to 4 years
1 secondary outcome
Safety
Time frame: Up to 4 years
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 5 May 2014.
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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.
Medical Monitor study director
Genzyme, a Sanofi Company
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing laronidase in people with Mucopolysaccharidosis I and Hurler's Syndrome, run by Genzyme, a Sanofi Company at 3 sites 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 3 inclusion and 7 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 5 May 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
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?
Urinary GAG (glycosaminoglycans) (time frame: Up to 4 years). The study lists 2 primary outcomes in all. 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 5 May 2014.