Clinical Trial: Growth Hormone for Osteoporosis Pseudoglioma Syndrome

Study Status: Withdrawn
Recruit Status: Withdrawn
Study Type: Interventional

Official Title: Trial of Growth Hormone for Osteoporosis Pseudoglioma Syndrome

Brief Summary: Osteoporosis pseudoglioma (OPPG) syndrome is a rare autosomal recessive condition of childhood osteoporosis and congenital blindness for which new treatments are needed. We have found that body fat is increased in OPPG and muscle mass is reduced. We hypothesize that growth hormone therapy will improve muscle mass and bone strength in OPPG.

Detailed Summary: Osteoporosis pseudoglioma (OPPG) syndrome is a rare autosomal recessive condition of childhood osteoporosis and congenital blindness for which new treatments are needed. We have found that body fat is increased in OPPG and muscle mass is reduced. We hypothesize that growth hormone therapy will improve muscle mass and bone strength in OPPG.
Sponsor: University of Maryland

Current Primary Outcome: Bone quality by pQCT [ Time Frame: 6 months ]

By pQCT: periosteal circumference, cortical density, trabecular density, section modulus


Original Primary Outcome: Same as current

Current Secondary Outcome: Body fat percent [ Time Frame: 6 months ]

Percent body fat by DXA


Original Secondary Outcome: Same as current

Information By: University of Maryland

Dates:
Date Received: June 5, 2012
Date Started: December 2013
Date Completion: December 2015
Last Updated: April 20, 2017
Last Verified: April 2017