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Trial record 2 of 2 for:    "norrie disease"

Growth Hormone for Osteoporosis Pseudoglioma Syndrome (GHOPPG)

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ClinicalTrials.gov Identifier: NCT01614171
Recruitment Status : Withdrawn (it was never started because it was never funded)
First Posted : June 7, 2012
Last Update Posted : April 21, 2017
Sponsor:
Collaborator:
Children's Hospital of Philadelphia
Information provided by (Responsible Party):
Elizabeth Streeten, University of Maryland

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.

Condition or disease Intervention/treatment Phase
Osteoporosis Pseudoglioma Syndrome Biological: Human recombinant growth hormone Not Applicable

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.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Trial of Growth Hormone for Osteoporosis Pseudoglioma Syndrome
Study Start Date : December 2013
Estimated Primary Completion Date : December 2015
Estimated Study Completion Date : December 2015


Arm Intervention/treatment
Experimental: Growth hormone therapy
Growth hormone treatment for 6 months
Biological: Human recombinant growth hormone
growth hormone treatment for 6 months (daily, weight based)




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


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



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Ages Eligible for Study:   4 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01614171


Locations
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
Investigators
Principal Investigator: Elizabeth A Streeten, MD University of Maryland School of Medicine, Division of Endocrinology

Responsible Party: Elizabeth Streeten, Associate Professor of Medicine, University of Maryland
ClinicalTrials.gov Identifier: NCT01614171     History of Changes
Other Study ID Numbers: HP-00053014
First Posted: June 7, 2012    Key Record Dates
Last Update Posted: April 21, 2017
Last Verified: April 2017

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

Additional relevant MeSH terms:
Syndrome
Osteoporosis
Osteogenesis Imperfecta
Blindness
Spasms, Infantile
Genetic Diseases, X-Linked
Disease
Pathologic Processes
Bone Diseases, Metabolic
Bone Diseases
Musculoskeletal Diseases
Metabolic Diseases
Osteochondrodysplasias
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
Epilepsy
Brain Diseases
Central Nervous System Diseases
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs