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Growth Hormone for Osteoporosis Pseudoglioma Syndrome (GHOPPG)

This study is not yet open for participant recruitment.
Verified January 2013 by University of Maryland
Sponsor:
Collaborator:
Children's Hospital of Philadelphia
Information provided by (Responsible Party):
Elizabeth Streeten, University of Maryland
ClinicalTrials.gov Identifier:
NCT01614171
First received: June 5, 2012
Last updated: January 14, 2013
Last verified: January 2013
History of Changes
  Purpose

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: Endpoint Classification: Safety/Efficacy Study
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:

MedlinePlus related topics: Osteoporosis
Drug Information available for: Somatropin
U.S. FDA Resources

Further study details as provided by University of Maryland:

Primary Outcome Measures:
  • Bone quality by pQCT [ Time Frame: 6 months ] [ Designated as safety issue: No ]
    By pQCT: periosteal circumference, cortical density, trabecular density, section modulus


Secondary Outcome Measures:
  • Body fat percent [ Time Frame: 6 months ] [ Designated as safety issue: No ]
    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)
Other Name: human recombinant growth hormone

  Eligibility

Ages Eligible for Study:   4 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
Please refer to this study by its ClinicalTrials.gov identifier: NCT01614171

Contacts
Contact: Elizabeth A Streeten, MD 410-328-6219 estreete@medicine.umaryland.edu
Contact: Manige Konig, MD 410-328-1623 mkonig@medicine.umaryland.edu

Locations
United States, Maryland
University of Maryland School of Medicine Not yet recruiting
Baltimore, Maryland, United States, 21201
Contact: Elizabeth A Streeten, MD     410-328-6219     estreete@medicine.umaryland.edu    
Principal Investigator: Elizabeth A Streeten, MD            
Sponsors and Collaborators
University of Maryland
Children's Hospital of Philadelphia
Investigators
Principal Investigator: Elizabeth A Streeten, MD University of Maryland School of Medicine, Division of Endocrinology
  More Information

No publications provided

Responsible Party: Elizabeth Streeten, Associate Professor of Medicine, University of Maryland
ClinicalTrials.gov Identifier: NCT01614171     History of Changes
Other Study ID Numbers: HP-00053014
Study First Received: June 5, 2012
Last Updated: January 14, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by University of Maryland:
osteoporosis pseudoglioma
OPPG
growth hormone
osteoporosis

Additional relevant MeSH terms:
Osteoporosis
Blindness
Spasms, Infantile
Osteogenesis Imperfecta
Bone Diseases, Metabolic
Bone Diseases
Musculoskeletal Diseases
Vision Disorders
Sensation Disorders
Neurologic Manifestations
Nervous System Diseases
Eye Diseases
Signs and Symptoms
 Epilepsy, Generalized
Epilepsy
Brain Diseases
Central Nervous System Diseases
Osteochondrodysplasias
Bone Diseases, Developmental
Genetic Diseases, Inborn
Collagen Diseases
Connective Tissue Diseases
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Pharmacologic Actions

ClinicalTrials.gov processed this record on May 22, 2013