A total of 29 patients have been enrolled in the study protocol and were randomized. Nine patients in group 1, eleven patients in group 2 and nine patients in group 3. The investigators noted a significant increase in fat fraction in all three groups. The mean global increase in fat fraction was 4.35 + 5.51 (p=0.001). All the increase was achieved in the first six months of therapy or 60 u/kg dose every two weeks. In one subsequent months, at 30 u/kg the fat fraction leveled off. Calitriol supplementation had no effect on none demsitry as an adjunct to enzyme replacement therapy. Trabecular bone density measurements and markers of bone turnover at 2 years are pending on the twentynine patients enrolled in the study. Preliminary findings suggest an increase in blood osterocalcin and urinary deoxyprindoline. Supportive of increased bone turnover.

Agency
National Institute of Health (NIH)
Institute
National Center for Research Resources (NCRR)
Type
General Clinical Research Centers Program (M01)
Project #
3M01RR001066-22S3
Application #
6220116
Study Section
Project Start
1998-12-01
Project End
1999-11-30
Budget Start
1998-10-01
Budget End
1999-09-30
Support Year
22
Fiscal Year
1999
Total Cost
Indirect Cost
Name
Massachusetts General Hospital
Department
Type
DUNS #
City
Boston
State
MA
Country
United States
Zip Code
02199
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