Growth Hormone for Osteoporosis Pseudoglioma Syndrome

NCT ID: NCT01614171

Last Updated: 2022-04-08

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

WITHDRAWN

Clinical Phase

NA

Study Classification

INTERVENTIONAL

Study Start Date

2013-12-31

Study Completion Date

2015-12-31

Brief Summary

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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

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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.

Conditions

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Osteoporosis Pseudoglioma Syndrome

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Growth hormone therapy

Growth hormone treatment for 6 months

Group Type EXPERIMENTAL

Human recombinant growth hormone

Intervention Type BIOLOGICAL

growth hormone treatment for 6 months (daily, weight based)

Interventions

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Human recombinant growth hormone

growth hormone treatment for 6 months (daily, weight based)

Intervention Type BIOLOGICAL

Eligibility Criteria

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Inclusion Criteria

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

Exclusion Criteria

* pregnant
* Age under 4 yrs
* Active malignancy
Minimum Eligible Age

4 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Children's Hospital of Philadelphia

OTHER

Sponsor Role collaborator

University of Maryland, Baltimore

OTHER

Sponsor Role lead

Responsible Party

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Elizabeth Streeten

Associate Professor of Medicine

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Elizabeth A Streeten, MD

Role: PRINCIPAL_INVESTIGATOR

University of Maryland School of Medicine, Division of Endocrinology

Locations

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University of Maryland School of Medicine

Baltimore, Maryland, United States

Site Status

Countries

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United States

Other Identifiers

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HP-00053014

Identifier Type: -

Identifier Source: org_study_id

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