PseudohypoparathyroidismNCT02411461
Early-onset Obesity and Cognitive Impairment in Children With Pseudohypoparathyroidism
An observational study of Pseudohypoparathyroidism and Albright Hereditary Osteodystrophy, run by Vanderbilt University Medical Center at 1 site in United States.
Trial summary
Pseudohypoparathyroidism type 1a (PHP1a) is a rare disease that causes childhood obesity and learning difficulties. This study will investigate eating behaviors and perform cognitive testing on children with PHP1a. The investigators will compare their results to those of healthy siblings and other obese children. Read more about this study
- Who is it for?
- Ages 6 to 18 yearsHealthy volunteers can take part
- Conditions the study is for
- Pseudohypoparathyroidism, Albright Hereditary Osteodystrophy
- How many people
- 39 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Dec 2014to Jan 2017
- Phase
- Observational
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 6 to 18 years
- All sexes
- Healthy volunteers accepted
- Child, Adult
Study population: Children with pseudohypoparathyroidism type 1a (PHP1a), their healthy siblings and matched obese controls
Who may be able to join
- Clinical diagnosis of PHP1a, sibling of a patient with PHP1a or matched obese control
Who cannot join
- Treatment with appetite-altering drug or initiation of a new weight loss program in the past 3 months
- Type 2 diabetes
- Obesity due to a genetic syndrome, growth hormone deficiency, untreated hypothyroidism, Cushing syndrome or exogenous glucocorticoid administration
- Weight loss greater than 10% over the previous 6 months
- Autism or other significant learning disorder
- Type 2 diabetes
- Other significant medical condition
Trial locations and contacts
1 site in 1 country.
About this study
Pseudohypoparathyroidism type 1a (PHP1a) is a rare disease that causes childhood obesity and learning difficulties. This study will investigate eating behaviors and perform cognitive testing on children with PHP1a. The investigators will compare their results to those of healthy siblings and other obese children.
Read the detailed description
Pseudohypoparathyroidism type 1a (PHP1a) is a genetic disorder that causes early-onset, syndromic obesity and cognitive impairment. This study aims to evaluate eating behaviors, cognition and executive function in children with PHP1a, compared with healthy siblings and matched obese controls.
- Keywords
- Pediatric obesity, pseudohypoparathyroidism, Albright Hereditary Osteodystrophy
- Medical subject headings
- Pseudohypoparathyroidism, Pediatric Obesity
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Pseudohypoparathyroidism type 1a | Study group | |
| Healthy siblings | Control group | |
| Obese patients | Control group |
What is being measured
The primary outcome is the main question the study is designed to answer.
Intelligence Quotient
Intelligence quotient as measured by Kaufman Brief Intelligence Test, 2nd Edition. This scale yields standard scores where the mean is 100 and one standard deviation is 15. Higher scores indicate better function.
Measured: one day
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Apr 2015 (estimated)
- Study start1 Dec 2014
- Primary completionJan 2017
- Study completionJan 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for PseudohypoparathyroidismStudies of the same condition, open and under way.
- More trials run by Vanderbilt University Medical CenterThe 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
- Observational
- Observational model
- Case-control
- Time perspective
- Cross-sectional
- Enrolment
- 39 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vanderbilt University Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Ashley Shoemaker, Vanderbilt University Medical Center
- Investigators
- Ashley Shoemaker, MD, Principal investigator, Vanderbilt University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02411461
- Sponsor's protocol ID
- VR7195
- Other IDs
- 1K23DK101689-01A1
- Status last verified
- Oct 2019
- First submitted
- 25 Feb 2015
- Last updated
- 13 Nov 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- Individual data sharing
- No
Publications
Shoemaker AH, Lomenick JP, Saville BR, Wang W, Buchowski MS, Cone RD. Energy expenditure in obese children with pseudohypoparathyroidism type 1a. Int J Obes (Lond). 2013 Aug;37(8):1147-53. doi: 10.1038/ijo.2012.200. Epub 2012 Dec 11.
PubMed 23229731Wang L, Shoemaker AH. Eating behaviors in obese children with pseudohypoparathyroidism type 1a: a cross-sectional study. Int J Pediatr Endocrinol. 2014;2014(1):21. doi: 10.1186/1687-9856-2014-21. Epub 2014 Oct 15.
PubMed 25337124Landreth H, Malow BA, Shoemaker AH. Increased Prevalence of Sleep Apnea in Children with Pseudohypoparathyroidism Type 1a. Horm Res Paediatr. 2015;84(1):1-5. doi: 10.1159/000381452. Epub 2015 Apr 23.
PubMed 25925491Perez KM, Lee EB, Kahanda S, Duis J, Reyes M, Juppner H, Shoemaker AH. Cognitive and behavioral phenotype of children with pseudohypoparathyroidism type 1A. Am J Med Genet A. 2018 Feb;176(2):283-289. doi: 10.1002/ajmg.a.38534. Epub 2017 Nov 28.
PubMed 29193623
Study at a glance
From the registry record, last updated by the sponsor on 13 Nov 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Early-onset Obesity and Cognitive Impairment in Children With Pseudohypoparathyroidism
- ClinicalTrials.gov ID
- NCT02411461
- Acronym / sponsor ID
- VR7195
- Keywords
- Pediatric obesity, pseudohypoparathyroidism, Albright Hereditary Osteodystrophy
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 39 participants (actual)
- Lead sponsor
- Vanderbilt University Medical CenterUniversity, hospital or other
- Collaborators
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
- Study start
- 1 Dec 2014
- Primary completion
- Jan 2017 (actual)
- Study completion
- Jan 2017 (actual)
- First posted
- 8 Apr 2015 (estimated)
- Results
- Posted 8 Jun 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Pseudohypoparathyroidism type 1a (PHP1a) is a rare disease that causes childhood obesity and learning difficulties. This study will investigate eating behaviors and perform cognitive testing on children with PHP1a. The investigators will compare their results to those of healthy siblings and other obese children.
Detailed description
Pseudohypoparathyroidism type 1a (PHP1a) is a genetic disorder that causes early-onset, syndromic obesity and cognitive impairment. This study aims to evaluate eating behaviors, cognition and executive function in children with PHP1a, compared with healthy siblings and matched obese controls.
Study design
- Observational model
- Case-control
- Time perspective
- Cross-sectional
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Pseudohypoparathyroidism type 1a | – | –Study group |
| Healthy siblings | – | –Control group |
| Obese patients | – | –Control group |
Who may qualify
- Ages
- 6 to 18 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
- Study population
- Children with pseudohypoparathyroidism type 1a (PHP1a), their healthy siblings and matched obese controls
Key inclusion criteria (1 listed)
- Clinical diagnosis of PHP1a, sibling of a patient with PHP1a or matched obese control
Key exclusion criteria (7 listed)
- Treatment with appetite-altering drug or initiation of a new weight loss program in the past 3 months
- Type 2 diabetes
- Obesity due to a genetic syndrome, growth hormone deficiency, untreated hypothyroidism, Cushing syndrome or exogenous glucocorticoid administration
- Weight loss greater than 10% over the previous 6 months
- Autism or other significant learning disorder
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Intelligence Quotient
Time frame: one day
Intelligence quotient as measured by Kaufman Brief Intelligence Test, 2nd Edition. This scale yields standard scores where the mean is 100 and one standard deviation is 15. Higher scores indicate better function.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Nov 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 23229731Shoemaker AH, Lomenick JP, Saville BR, Wang W, Buchowski MS, Cone RD. Energy expenditure in obese children with pseudohypoparathyroidism type 1a. Int J Obes (Lond). 2013 Aug;37(8):1147-53. doi: 10.1038/ijo.2012.200. Epub 2012 Dec 11.
- Background: PubMed 25337124Wang L, Shoemaker AH. Eating behaviors in obese children with pseudohypoparathyroidism type 1a: a cross-sectional study. Int J Pediatr Endocrinol. 2014;2014(1):21. doi: 10.1186/1687-9856-2014-21. Epub 2014 Oct 15.
- Background: PubMed 25925491Landreth H, Malow BA, Shoemaker AH. Increased Prevalence of Sleep Apnea in Children with Pseudohypoparathyroidism Type 1a. Horm Res Paediatr. 2015;84(1):1-5. doi: 10.1159/000381452. Epub 2015 Apr 23.
- Background: PubMed 29193623Perez KM, Lee EB, Kahanda S, Duis J, Reyes M, Juppner H, Shoemaker AH. Cognitive and behavioral phenotype of children with pseudohypoparathyroidism type 1A. Am J Med Genet A. 2018 Feb;176(2):283-289. doi: 10.1002/ajmg.a.38534. Epub 2017 Nov 28.
- Vanderbilt PHP Research Group WebsiteLink provided by the sponsor.
- Individual participant dataSharing planned: No
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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.
Ashley Shoemaker, MD principal investigator
Vanderbilt University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Pseudohypoparathyroidism and Albright Hereditary Osteodystrophy, run by Vanderbilt University Medical Center 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 1 inclusion and 7 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: finished, as of the sponsor's last update on 13 Nov 2019. 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?
Yes. Results are posted in the registry (first posted 8 Jun 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Intelligence Quotient (time frame: one day). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02411461, which also lists 4 linked publications.
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 13 Nov 2019.