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Growth Hormone for Osteoporosis Pseudoglioma Syndrome

Not Applicable
Withdrawn
Conditions
Osteoporosis Pseudoglioma Syndrome
Interventions
Biological: Human recombinant growth hormone
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.

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WITHDRAWN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
Arm && Interventions
GroupInterventionDescription
Growth hormone therapyHuman recombinant growth hormoneGrowth hormone treatment for 6 months
Primary Outcome Measures
NameTimeMethod
Bone quality by pQCT6 months

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

Secondary Outcome Measures
NameTimeMethod
Body fat percent6 months

Percent body fat by DXA

Trial Locations

Locations (1)

University of Maryland School of Medicine

🇺🇸

Baltimore, Maryland, United States

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