Growth Hormone for Osteoporosis Pseudoglioma Syndrome (GHOPPG)

March 30, 2022 updated by: Elizabeth Streeten, University of Maryland, Baltimore

Trial of Growth Hormone for Osteoporosis Pseudoglioma Syndrome

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 Overview

Status

Withdrawn

Conditions

Intervention / Treatment

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

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Maryland
      • Baltimore, Maryland, United States, 21201
        • University of Maryland School of Medicine

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

4 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

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

Exclusion Criteria:

  • pregnant
  • Age under 4 yrs
  • Active malignancy

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / 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)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Bone quality by pQCT
Time Frame: 6 months
By pQCT: periosteal circumference, cortical density, trabecular density, section modulus
6 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Body fat percent
Time Frame: 6 months
Percent body fat by DXA
6 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University of Maryland, Baltimore

Collaborators

Children's Hospital of Philadelphia

Investigators

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

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

December 1, 2013

Primary Completion (Anticipated)

December 1, 2015

Study Completion (Anticipated)

December 1, 2015

Study Registration Dates

First Submitted

June 5, 2012

First Submitted That Met QC Criteria

June 5, 2012

First Posted (Estimate)

June 7, 2012

Study Record Updates

Last Update Posted (Actual)

April 8, 2022

Last Update Submitted That Met QC Criteria

March 30, 2022

Last Verified

April 1, 2017

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • HP-00053014

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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