Osteoporosis Pseudoglioma SyndromeGHOPPGNCT01614171
Growth Hormone for Osteoporosis Pseudoglioma Syndrome
A study testing Human recombinant growth hormone in people with Osteoporosis Pseudoglioma Syndrome, run by University of Maryland, Baltimore at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 4 years and older
- Conditions the study is for
- Osteoporosis Pseudoglioma Syndrome
- What is being tested?
- Human recombinant growth hormone
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- Dec 2013to Dec 2015
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Osteoporosis pseudoglioma syndrome
- Age 4 years and above
- not on medication for osteoporosis
Who cannot join
- pregnant
- Age under 4 yrs
- Active malignancy
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Maryland School of Medicine
Baltimore, Maryland, 21201
About this study
Official title: 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.
Read the 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.
- Conditions
- Osteoporosis Pseudoglioma Syndrome
- Being tested
- Human recombinant growth hormone
- Keywords
- osteoporosis pseudoglioma, OPPG, growth hormone, osteoporosis
- Medical subject headings
- Exudative Vitreoretinopathy 4, Osteoporosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Human recombinant growth hormoneBiological
growth hormone treatment for 6 months (daily, weight based)
| Study group | Role | What they receive |
|---|---|---|
| Growth hormone therapy | Experimental | Growth hormone treatment for 6 months Receives: Biological: Human recombinant growth hormone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Bone quality by pQCT
By pQCT: periosteal circumference, cortical density, trabecular density, section modulus
Measured: 6 months
Secondary outcomes (1)
Body fat percent
Percent body fat by DXA
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Jun 2012 (estimated)
- Study startDec 2013
- Primary completionDec 2015 (estimated)
- Study completionDec 2015 (estimated)
Why it stopped: it was never started because it was never funded
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Osteoporosis Pseudoglioma SyndromeStudies of the same condition, open and under way.
- More trials run by University of Maryland, BaltimoreThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Maryland, Baltimore
- Sponsor type
- University, hospital or other
- Collaborators
- Children's Hospital of Philadelphia
- Responsible party
- Principal investigator, Elizabeth Streeten, University of Maryland, Baltimore
- Investigators
- Elizabeth A Streeten, MD, Principal investigator, University of Maryland School of Medicine, Division of Endocrinology
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01614171
- Sponsor's protocol ID
- HP-00053014
- Acronym
- GHOPPG
- Status last verified
- Apr 2017
- First submitted
- 5 Jun 2012
- Last updated
- 8 Apr 2022
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 8 Apr 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Trial of Growth Hormone for Osteoporosis Pseudoglioma Syndrome
- ClinicalTrials.gov ID
- NCT01614171
- Acronym / sponsor ID
- GHOPPG / HP-00053014
- Conditions
- Osteoporosis Pseudoglioma Syndrome
- Keywords
- osteoporosis pseudoglioma, OPPG, growth hormone, osteoporosis
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: it was never started because it was never funded
- Enrolment
- 0 participants (actual)
- Lead sponsor
- University of Maryland, BaltimoreUniversity, hospital or other
- Collaborators
- Children's Hospital of Philadelphia
- Study start
- Dec 2013
- Primary completion
- Dec 2015 (estimated)
- Study completion
- Dec 2015 (estimated)
- First posted
- 7 Jun 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Growth hormone therapy | Experimental | Biological: Human recombinant growth hormoneGrowth hormone treatment for 6 months |
Who may qualify
- Ages
- 4 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Osteoporosis pseudoglioma syndrome
- Age 4 years and above
- not on medication for osteoporosis
Key exclusion criteria (3 listed)
- pregnant
- Age under 4 yrs
- Active malignancy
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Human recombinant growth hormonegrowth hormone treatment for 6 months (daily, weight based) | Biological | Growth hormone therapy |
Primary outcome
Bone quality by pQCT
Time frame: 6 months
By pQCT: periosteal circumference, cortical density, trabecular density, section modulus
1 secondary outcome
Body fat percent
Time frame: 6 months
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Apr 2022.
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Elizabeth A Streeten, MD principal investigator
University of Maryland School of Medicine, Division of Endocrinology
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Human recombinant growth hormone in people with Osteoporosis Pseudoglioma Syndrome, run by University of Maryland, Baltimore at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 8 Apr 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Bone quality by pQCT (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Apr 2022.