Growth Hormone for Osteoporosis Pseudoglioma Syndrome

Not Applicable

Withdrawn

• Conditions: Osteoporosis Pseudoglioma Syndrome

• Registration Number: NCT01614171
• Lead Sponsor: University of Maryland, Baltimore

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 Description

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.

Study Timeline

• Study First Submitted: 2012-06-05
• Study First Submitted QC: 2012-06-05
• Study First Posted: 2012-06-07
• Study Start: 2013-12
• Primary Completion: 2015-12
• Study Completion: 2015-12
• Status Verified: 2017-04
• Last Update Submitted: 2022-03-30
• Last Update Posted: 2022-04-08

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

• Osteoporosis pseudoglioma syndrome
• Age 4 years and above
• not on medication for osteoporosis

Exclusion Criteria

• pregnant
• Age under 4 yrs
• Active malignancy

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Primary Outcome Measures

Name	Time	Method
Bone quality by pQCT	6 months	By pQCT: periosteal circumference, cortical density, trabecular density, section modulus

Secondary Outcome Measures

Name	Time	Method
Body fat percent	6 months	Percent body fat by DXA

Trial Locations

Locations (1)

University of Maryland School of Medicine; 🇺🇸 Baltimore, Maryland, United States