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Trial Outcomes & Findings for Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome (NCT NCT02156674)

NCT ID: NCT02156674

Last Updated: 2020-12-23

Results Overview

Change in urinary glycosaminoglycan (GAG) excretion from baseline to 2 years

Recruitment status

TERMINATED

Study phase

NA

Target enrollment

1 participants

Primary outcome timeframe

Baseline and 2 years

Results posted on

2020-12-23

Participant Flow

Participant milestones

Participant milestones
Measure
Naglazyme®
weekly Naglazyme® infusion for 2 years Naglazyme®: 1 mg per kg of body weight administered once weekly as an intravenous infusion
Overall Study
STARTED
1
Overall Study
COMPLETED
1
Overall Study
NOT COMPLETED
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Naglazyme®
n=1 Participants
weekly Naglazyme® infusion for 2 years Naglazyme®: 1 mg per kg of body weight administered once weekly as an intravenous infusion
Age, Categorical
<=18 years
0 Participants
n=5 Participants
Age, Categorical
Between 18 and 65 years
1 Participants
n=5 Participants
Age, Categorical
>=65 years
0 Participants
n=5 Participants
Sex: Female, Male
Female
0 Participants
n=5 Participants
Sex: Female, Male
Male
1 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
0 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
0 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
1 Participants
n=5 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=5 Participants
Race (NIH/OMB)
Asian
0 Participants
n=5 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=5 Participants
Race (NIH/OMB)
Black or African American
0 Participants
n=5 Participants
Race (NIH/OMB)
White
1 Participants
n=5 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=5 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants
Region of Enrollment
United States
1 participants
n=5 Participants

PRIMARY outcome

Timeframe: Baseline and 2 years

Change in urinary glycosaminoglycan (GAG) excretion from baseline to 2 years

Outcome measures

Outcome measures
Measure
Naglazyme®
n=1 Participants
weekly Naglazyme® infusion for 2 years Naglazyme®: 1 mg per kg of body weight administered once weekly as an intravenous infusion
Change in Urinary Glycosaminoglycan (GAG) Excretion
-0.8 mg/mm Cr

PRIMARY outcome

Timeframe: 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

Outcome measures

Outcome measures
Measure
Naglazyme®
n=1 Participants
weekly Naglazyme® infusion for 2 years Naglazyme®: 1 mg per kg of body weight administered once weekly as an intravenous infusion
Change in Distance Traveled
136 Foot

PRIMARY outcome

Timeframe: Baseline and 2 years

Population: Not Evaluated

Subjects will undergo full neuropsychological testing annually and abbreviated testing for attention and adaptive skills at the six-month interval visits

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure
Naglazyme®
n=1 Participants
weekly Naglazyme® infusion for 2 years Naglazyme®: 1 mg per kg of body weight administered once weekly as an intravenous infusion
Number of Participants With Development of Neutralizing Antibodies to Naglazyme Therapy
0 participants

SECONDARY outcome

Timeframe: 2 years

Population: No data available

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.

Outcome measures

Outcome data not reported

Adverse Events

Naglazyme®

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Elizabeth Braunlin MD, PhD

Masonic Cancer Center, University of Minnesota

Phone: 612 626 2755

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place