Back to resultsGrowth Hormone for Osteoporosis Pseudoglioma Syndrome (GHOPPG)
Primary Purpose
Osteoporosis Pseudoglioma Syndrome
Status
Withdrawn
Phase
Not Applicable
Locations
United States
Study Type
Interventional
Intervention
Human recombinant growth hormone
Sponsored by
About this trial
This is an interventional treatment trial for Osteoporosis Pseudoglioma Syndrome focused on measuring osteoporosis pseudoglioma, OPPG, growth hormone, osteoporosis
Eligibility 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
Sites / Locations
- University of Maryland School of Medicine
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Growth hormone therapy
Arm Description
Growth hormone treatment for 6 months
Outcomes
Primary Outcome Measures
Bone quality by pQCT
By pQCT: periosteal circumference, cortical density, trabecular density, section modulus
Secondary Outcome Measures
Body fat percent
Percent body fat by DXA
Full Information
NCT ID
NCT01614171
First Posted
June 5, 2012
Last Updated
March 30, 2022
Sponsor
University of Maryland, Baltimore
Collaborators
Children's Hospital of Philadelphia
1. Study Identification
Unique Protocol Identification Number
NCT01614171
Brief Title
Growth Hormone for Osteoporosis Pseudoglioma Syndrome
Acronym
GHOPPG
Official Title
Trial of Growth Hormone for Osteoporosis Pseudoglioma Syndrome
Study Type
Interventional
2. Study Status
Record Verification Date
April 2017
Overall Recruitment Status
Withdrawn
Why Stopped
it was never started because it was never funded
Study Start Date
December 2013 (undefined)
Primary Completion Date
December 2015 (Anticipated)
Study Completion Date
December 2015 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
University of Maryland, Baltimore
Collaborators
Children's Hospital of Philadelphia
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
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.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Osteoporosis Pseudoglioma Syndrome
Keywords
osteoporosis pseudoglioma, OPPG, growth hormone, osteoporosis
7. Study Design
Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
0 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Growth hormone therapy
Arm Type
Experimental
Arm Description
Growth hormone treatment for 6 months
Intervention Type
Biological
Intervention Name(s)
Human recombinant growth hormone
Intervention Description
growth hormone treatment for 6 months (daily, weight based)
Primary Outcome Measure Information:
Title
Bone quality by pQCT
Description
By pQCT: periosteal circumference, cortical density, trabecular density, section modulus
Time Frame
6 months
Secondary Outcome Measure Information:
Title
Body fat percent
Description
Percent body fat by DXA
Time Frame
6 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
4 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Elizabeth A Streeten, MD
Organizational Affiliation
University of Maryland School of Medicine, Division of Endocrinology
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of Maryland School of Medicine
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21201
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Growth Hormone for Osteoporosis Pseudoglioma Syndrome
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