Maroteaux-Lamy SyndromeNCT02156674
Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome
A study testing Naglazyme® in people with Maroteaux-Lamy Syndrome, run by Masonic Cancer Center, University of Minnesota at 1 site in United States.
Trial summary
This is a single center study in which Naglazyme® will be given weekly for two years in patients with Maroteaux-Lamy syndrome, also known as mucopolysaccharide VI (MPS VI), who have previously been treated with an allogeneic transplant. Read more about this study
- Who is it for?
- Ages 3 years and older
- Conditions the study is for
- Maroteaux-Lamy Syndrome
- What is being tested?
- Naglazyme®
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 1 siteUnited States
- Study dates
- 26 Jan 2016to 1 Dec 2019
- Phase
- Interventional
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 3 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Mucopolysaccharidosis type VI (MPS VI, Maroteaux-Lamy syndrome) treated with a prior allogeneic transplant >2 years previously
- Persons currently receiving Naglazyme may be accepted into the study
- Age > 2 years
- >10% engrafted based on most recent testing
- Willing to commit to traveling to the University of Minnesota every 6 months
- Written informed consent with parent/guardian consent for children < 18 years of age or persons unable to consent with minor assent if appropriate
Who cannot join
- History of cardiac or pulmonary insufficiency or those requiring continuous supplemental oxygen
- Pregnant or breastfeeding
- Any condition that, in the view of the investigator, places the patient at high risk of poor treatment compliance or of not completing the study
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Minnesota Medical Center, Fairview
Minneapolis, Minnesota, 55455
About this study
Official title: Study of Administration of Intravenous Naglazyme® Following Allogeneic Transplantation for Maroteaux-Lamy Syndrome
This is a single center study in which Naglazyme® will be given weekly for two years in patients with Maroteaux-Lamy syndrome, also known as mucopolysaccharide VI (MPS VI), who have previously been treated with an allogeneic transplant.
- Conditions
- Maroteaux-Lamy Syndrome
- Being tested
- Naglazyme®
- Keywords
- Maroteaux-Lamy Syndrome, mucopolysaccharide VI, MPS VI
- Medical subject headings
- Mucopolysaccharidosis VI
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Naglazyme®Drug
1 mg per kg of body weight administered once weekly as an intravenous infusion
| Study group | Role | What they receive |
|---|---|---|
| Naglazyme® | Experimental | weekly Naglazyme® infusion for 2 years Receives: Drug: Naglazyme® |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Urinary Glycosaminoglycan (GAG) Excretion
Change in urinary glycosaminoglycan (GAG) excretion from baseline to 2 years
Measured: Baseline and 2 years
Change in Distance Traveled
Change in distance traveled in 6 minute walk and standard tests of range of motion and mobility. Improvement in distance walked from the start to study to end of the study is reported
Measured: Baseline and 2 years
Change in Neurocognitive Ability
Subjects will undergo full neuropsychological testing annually and abbreviated testing for attention and adaptive skills at the six-month interval visits
Measured: Baseline and 2 years
Secondary outcomes (2)
Number of Participants With Development of Neutralizing Antibodies to Naglazyme Therapy
Development of antibodies including neutralizing antibodies with weekly Naglazyme therapy and the impact of these antibodies on cardiorespiratory and skeletal parameters as measured by the 6--minute walk test and standard tests of range of motion and mobility, and the change in neurocognitive ability.
Anti-rhASB antibodies in patient's sera were tested in the Biomarin Laboratory using an in-house test of ELISA.
Measured: 6 months
Number of Participants With Development of Neutralizing Antibodies to Naglazyme Therapy
Development of antibodies including neutralizing antibodies with weekly Naglazyme therapy and the impact of these antibodies on cardiorespiratory and skeletal parameters as measured by the 6--minute walk test and standard tests of range of motion and mobility, and the change in neurocognitive ability.
Anti-rhASB antibodies in patient's sera were tested in the Biomarin Laboratory using an in-house test of ELISA.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jun 2014 (estimated)
- Study start26 Jan 2016
- Primary completion1 Dec 2019
- Study completion1 Dec 2019
Why it stopped: Slow accrual
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Maroteaux-Lamy SyndromeStudies of the same condition, open and under way.
- More trials run by Masonic Cancer Center, University of MinnesotaThe sponsor's other registered studies.
- 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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Sponsor type
- University, hospital or other
- Collaborators
- BioMarin Pharmaceutical
- Responsible party
- Sponsor
- Investigators
- Elizabeth Braulin, M.D., Principal investigator, University of Minnesota
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02156674
- Sponsor's protocol ID
- 2014LS014
- Other IDs
- MT2014-08R
- Status last verified
- Dec 2020
- First submitted
- 3 Jun 2014
- Last updated
- 23 Dec 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 23 Dec 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study of Administration of Intravenous Naglazyme® Following Allogeneic Transplantation for Maroteaux-Lamy Syndrome
- ClinicalTrials.gov ID
- NCT02156674
- Acronym / sponsor ID
- 2014LS014
- Conditions
- Maroteaux-Lamy Syndrome
- Keywords
- Maroteaux-Lamy Syndrome, mucopolysaccharide VI, MPS VI
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Slow accrual
- Enrolment
- 1 participants (actual)
- Lead sponsor
- Masonic Cancer Center, University of MinnesotaUniversity, hospital or other
- Collaborators
- BioMarin Pharmaceutical
- Study start
- 26 Jan 2016 (actual)
- Primary completion
- 1 Dec 2019 (actual)
- Study completion
- 1 Dec 2019 (actual)
- First posted
- 5 Jun 2014 (estimated)
- Results
- Posted 23 Dec 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a single center study in which Naglazyme® will be given weekly for two years in patients with Maroteaux-Lamy syndrome, also known as mucopolysaccharide VI (MPS VI), who have previously been treated with an allogeneic transplant.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Naglazyme® | Experimental | Drug: Naglazyme®weekly Naglazyme® infusion for 2 years |
Who may qualify
- Ages
- 3 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Mucopolysaccharidosis type VI (MPS VI, Maroteaux-Lamy syndrome) treated with a prior allogeneic transplant >2 years previously
- Persons currently receiving Naglazyme may be accepted into the study
- Age > 2 years
- >10% engrafted based on most recent testing
- Willing to commit to traveling to the University of Minnesota every 6 months
Key exclusion criteria (3 listed)
- History of cardiac or pulmonary insufficiency or those requiring continuous supplemental oxygen
- Pregnant or breastfeeding
- Any condition that, in the view of the investigator, places the patient at high risk of poor treatment compliance or of not completing the study
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 |
|---|---|---|
| Naglazyme®1 mg per kg of body weight administered once weekly as an intravenous infusion | Drug | Naglazyme® |
Primary outcome
Change in Urinary Glycosaminoglycan (GAG) Excretion
Time frame: Baseline and 2 years
Change in urinary glycosaminoglycan (GAG) excretion from baseline to 2 years
Change in Distance Traveled
Time frame: Baseline and 2 years
Change in distance traveled in 6 minute walk and standard tests of range of motion and mobility. Improvement in distance walked from the start to study to end of the study is reported
Change in Neurocognitive Ability
Time frame: Baseline and 2 years
Subjects will undergo full neuropsychological testing annually and abbreviated testing for attention and adaptive skills at the six-month interval visits
2 secondary outcomes
Number of Participants With Development of Neutralizing Antibodies to Naglazyme Therapy
Time frame: 6 months
Number of Participants With Development of Neutralizing Antibodies to Naglazyme Therapy
Time frame: 2 years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Dec 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related trials for Maroteaux-Lamy Syndrome
Active, not recruitingPhase 2
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
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Enrolling by invitationObservational study
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
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Early Check: Expanded Screening in Newborns
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RecruitingObservational study
Registry of Patients Diagnosed With Lysosomal Storage Diseases
Mucopolysaccharidosis I, Mucopolysaccharidosis II
RecruitingPhase 1
PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
MPS I, MPS II
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.
Elizabeth Braulin, M.D. principal investigator
University of Minnesota
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Naglazyme® in people with Maroteaux-Lamy 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 6 inclusion and 3 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 23 Dec 2020. 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.
Are results available for this study?
Yes. Results are posted in the registry (first posted 23 Dec 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Change in Urinary Glycosaminoglycan (GAG) Excretion (time frame: Baseline and 2 years). The study lists 3 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 23 Dec 2020.