Growth Hormone for Osteoporosis Pseudoglioma Syndrome

Osteoporosis Pseudoglioma Syndrome Clinical Trial

— GHOPPG  

Official title:

Trial of Growth Hormone for Osteoporosis Pseudoglioma Syndrome

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT01614171
• Other study ID #: HP-00053014
• Status: Withdrawn
• Phase: N/A
• Start date: December 2013
• Est. completion date: December 2015

Study information

• Verified date: April 2017
• Source: University of Maryland, Baltimore
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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 information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. completion date: December 2015
• Est. primary completion date: December 2015
• Accepts healthy volunteers: No
• Gender: All
• Age group: 4 Years and older

Eligibility

Inclusion Criteria:

• Osteoporosis pseudoglioma syndrome

• Age 4 years and above

• not on medication for osteoporosis

Exclusion Criteria:

• pregnant

• Age under 4 yrs

• Active malignancy

Related Conditions & MeSH terms

• Blindness
• Genetic Diseases, X-Linked
• Osteogenesis Imperfecta
• Osteoporosis
• Osteoporosis Pseudoglioma Syndrome
• Spasms, Infantile
• Syndrome

Intervention

Biological:
• Human recombinant growth hormone
growth hormone treatment for 6 months (daily, weight based)

Locations

Country	Name	City	State
United States	University of Maryland School of Medicine	Baltimore	Maryland

Sponsors (2)

Lead Sponsor	Collaborator
University of Maryland, Baltimore	Children's Hospital of Philadelphia

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Bone quality by pQCT	By pQCT: periosteal circumference, cortical density, trabecular density, section modulus	6 months
Secondary	Body fat percent	Percent body fat by DXA	6 months