Maroteaux-Lamy Syndrome
Conditions
Keywords
Maroteaux-Lamy Syndrome, mucopolysaccharide VI, MPS VI
Brief 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.
Interventions
DRUGNaglazyme®
1 mg per kg of body weight administered once weekly as an intravenous infusion
Sponsors
BioMarin Pharmaceutical
Masonic Cancer Center, University of Minnesota
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
3 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* 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
Exclusion criteria
* 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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in Urinary Glycosaminoglycan (GAG) Excretion | Baseline and 2 years | Change in urinary glycosaminoglycan (GAG) excretion from baseline to 2 years |
| Change in Distance Traveled | 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 | 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 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Development of Neutralizing Antibodies to Naglazyme Therapy | 6 months | 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. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Naglazyme® weekly Naglazyme® infusion for 2 years
Naglazyme®: 1 mg per kg of body weight administered once weekly as an intravenous infusion | 1 |
| Total | 1 |
Baseline characteristics
| Characteristic | Naglazyme® |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 1 Participants |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 0 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 1 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 1 Participants |
| Region of Enrollment United States | 1 participants |
| Sex: Female, Male Female | 0 Participants |
| Sex: Female, Male Male | 1 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 1 |
| other Total, other adverse events | 0 / 1 |
| serious Total, serious adverse events | 0 / 1 |
Outcome results
Primary
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
Time frame: Baseline and 2 years
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Naglazyme® | Change in Distance Traveled | 136 Foot |
Primary
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
Time frame: Baseline and 2 years
Population: Not Evaluated
Primary
Change in Urinary Glycosaminoglycan (GAG) Excretion
Change in urinary glycosaminoglycan (GAG) excretion from baseline to 2 years
Time frame: Baseline and 2 years
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Naglazyme® | Change in Urinary Glycosaminoglycan (GAG) Excretion | -0.8 mg/mm Cr |
Secondary
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.
Time frame: 6 months
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Naglazyme® | Number of Participants With Development of Neutralizing Antibodies to Naglazyme Therapy | 0 participants |
Secondary
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.
Time frame: 2 years
Population: No data available