Growth Hormone for Osteoporosis Pseudoglioma Syndrome | NCT01614171

Studies
Growth Hormone for Osteoporosis Pseudoglioma Syndrome

NCT01614171

Sponsor

University of Maryland, Baltimore

Status

Withdrawn

Last Update Posted

Apr 8, 2022Actual

Enrollment

0Actual

Phase

Not Applicable

Conditions

Osteoporosis Pseudoglioma Syndrome

Interventions

Human recombinant growth hormone

Countries

United States

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Apr 2017

Overall Recruitment Status or Expanded Access Status

Withdrawn

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

it was never started because it was never funded

Expanded Access Info

Start Date

Dec 2013

Primary Completion Date

Dec 2015Estimated

Completion Date

Dec 2015Estimated

First Submitted Date

Jun 5, 2012

First Submission Date that Met QC Criteria

Jun 5, 2012

First Posted Date

Jun 7, 2012Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Mar 30, 2022

Last Update Posted Date

Apr 8, 2022Actual

Sponsor/Collaborators Module

Responsible Party, by Official Title

Elizabeth Streeten, Associate Professor of Medicine, University of Maryland, BaltimorePrincipal Investigator

Lead Sponsor

University of Maryland, BaltimoreOTHER

Collaborators

Name	Class
Children's Hospital of Philadelphia	OTHER

Oversight Module

Has Data Monitoring Committee (DMC)

Yes

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Not Applicable

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

0Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Growth hormone therapy	Experimental	Growth hormone treatment for 6 months	Biological: Human recombinant growth hormone

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Human recombinant growth hormone	Biological	growth hormone treatment for 6 months (daily, weight based)	Growth hormone therapy

Outcomes Module

Primary Outcomes

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

Secondary Outcomes

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

Other Outcomes

Not provided

Eligibility Module

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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

4 Years

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Elizabeth A Streeten, MD	University of Maryland School of Medicine, Division of Endocrinology	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
University of Maryland School of Medicine		Baltimore	Maryland	21201	United States

Condition Browse Module

MeSH Terms

ID	Term
C566619	Exudative Vitreoretinopathy 4
D010024	Osteoporosis

Ancestor Terms

ID	Term
D001851	Bone Diseases, Metabolic
D001847	Bone Diseases
D009140	Musculoskeletal Diseases
D008659	Metabolic Diseases

Browse Leaves

Not provided

Browse Branches

Not provided