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Growth Hormone for Osteoporosis Pseudoglioma Syndrome (GHOPPG)

The recruitment status of this study is unknown. The completion date has passed and the status has not been verified in more than two years.
Verified January 2013 by University of Maryland.
Recruitment status was:  Not yet recruiting
Children's Hospital of Philadelphia
Information provided by (Responsible Party):
Elizabeth Streeten, University of Maryland Identifier:
First received: June 5, 2012
Last updated: January 14, 2013
Last verified: January 2013
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.

Condition Intervention
Osteoporosis Pseudoglioma Syndrome
Biological: Human recombinant growth hormone

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Trial of Growth Hormone for Osteoporosis Pseudoglioma Syndrome

Resource links provided by NLM:

Further study details as provided by University of Maryland:

Primary Outcome Measures:
  • Bone quality by pQCT [ Time Frame: 6 months ]
    By pQCT: periosteal circumference, cortical density, trabecular density, section modulus

Secondary Outcome Measures:
  • Body fat percent [ Time Frame: 6 months ]
    Percent body fat by DXA

Estimated Enrollment: 10
Study Start Date: December 2013
Estimated Study Completion Date: December 2015
Estimated Primary Completion Date: December 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Growth hormone therapy
Growth hormone treatment for 6 months
Biological: Human recombinant growth hormone
growth hormone treatment for 6 months (daily, weight based)


Ages Eligible for Study:   4 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

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

Exclusion Criteria:

  • pregnant
  • Age under 4 yrs
  • Active malignancy
  Contacts and Locations
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Please refer to this study by its identifier: NCT01614171

United States, Maryland
University of Maryland School of Medicine
Baltimore, Maryland, United States, 21201
Sponsors and Collaborators
University of Maryland
Children's Hospital of Philadelphia
Principal Investigator: Elizabeth A Streeten, MD University of Maryland School of Medicine, Division of Endocrinology
  More Information

Responsible Party: Elizabeth Streeten, Associate Professor of Medicine, University of Maryland Identifier: NCT01614171     History of Changes
Other Study ID Numbers: HP-00053014 
Study First Received: June 5, 2012
Last Updated: January 14, 2013

Keywords provided by University of Maryland:
osteoporosis pseudoglioma
growth hormone

Additional relevant MeSH terms:
Osteogenesis Imperfecta
Spasms, Infantile
Genetic Diseases, X-Linked
Pathologic Processes
Bone Diseases, Metabolic
Bone Diseases
Musculoskeletal Diseases
Bone Diseases, Developmental
Genetic Diseases, Inborn
Collagen Diseases
Connective Tissue Diseases
Vision Disorders
Sensation Disorders
Neurologic Manifestations
Nervous System Diseases
Eye Diseases
Signs and Symptoms
Epilepsy, Generalized
Brain Diseases
Central Nervous System Diseases
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs processed this record on February 20, 2017