Growth Hormone for Osteoporosis Pseudoglioma Syndrome (GHOPPG)

University of Maryland Baltimore (UMB)

Status: Withdrawn

Conditions: Osteoporosis Pseudoglioma Syndrome

Treatments: Biological: Human recombinant growth hormone

Study type: Interventional

Funder types: Other

Identifiers

NCT01614171

HP-00053014

Details and patient eligibility

About

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.

Full 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.

Sex: All

Ages: 4+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

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

Exclusion criteria

• pregnant
• Age under 4 yrs
• Active malignancy

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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0 participants in 1 patient group

Growth hormone therapy

Experimental group

Description:

Growth hormone treatment for 6 months

Treatment:

Biological: Human recombinant growth hormone