Turner SyndromeNCT01367834
Effect of Growth Hormone on Early Brain Development in Girls With Turner Syndrome
A study testing somatotropin in people with Turner Syndrome, run by Rebecca Knickmeyer Santelli, PhD at 1 site in United States.
Trial summary
The purpose of this research study is to learn about brain development in very young girls with Turner syndrome (TS) and the effect that growth hormone (GH) therapy has on early brain development. Read more about this study
- Who is it for?
- Ages 11 to 13 months, women only
- Conditions the study is for
- Turner Syndrome
- What is being tested?
- somatotropin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 17 participants
- Where
- 1 siteUnited States
- Study dates
- May 2010to May 2014
- Phase
- Interventional
- 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 11 to 13 months
- Women only
- No healthy volunteers
- Child
Who may be able to join
- Diagnosis of Turner syndrome
- Less than the 50th percentile for length for the general female population
Who cannot join
- Prior Growth Hormone (GH) therapy
- Diabetes
- Allergy to metacresol (a preservative in the GH liquid that is injected)
- Contraindications for Magnetic Resonance Imaging (MRI) (such as metal in the body)
- Part of a Y chromosome in child's karyotype
- Parent/guardian is not willing for child to be randomized to be in the treatment group (receives Growth Hormone injections for one year) or the control group (receives no Growth Hormone during the study)
- Parent/guardian is not willing for child to have some of her developmental testing digitally recorded for scoring
Trial locations and contacts
1 site in 1 country.
United States1 site
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27599
About this study
The purpose of this research study is to learn about brain development in very young girls with Turner syndrome (TS) and the effect that growth hormone (GH) therapy has on early brain development.
Read the detailed description
Studies in older children and adults have found that IQ (intelligence quotient) in individuals with Turner syndrome (TS) and the general population are similar. However, many individuals with TS have a nonverbal learning disability which may cause problems with imagining and working with objects in space (for example: building blocks, working puzzles, copying designs, driving a car) as well as problems in planning, paying attention, and getting along with people their own age. Studies of brain structure have found changes in the size and function of specific areas of the brain that are involved in these nonverbal processes. However, there are no data published on brain structure or function in girls with Turner Syndrome in the first few years of life when brains are learning and growing most rapidly.
In a recent study, very young, short girls with Turner Syndrome (TS)(averaging 2 years in age) were treated with Growth Hormone (GH) and almost reached an average height after 2 years. The risks of GH for the young girls in that study appeared to be the same as those for older girls. Therefore, it is now recommended that GH therapy be considered as soon as a child with TS has growth failure. Growth failure often occurs during infancy; therefore, more children with TS are now receiving GH therapy as young as 12 months of age. In this study, the investigators wish to start understanding what effect GH has on learning and brain growth when given between the ages of 12 and 24 months. In this study, the girls with TS will have developmental studies, a physical examination, magnetic resonance imaging (MRI), and blood drawn at one and two years of age. Some of the girls in this study will receive GH from 12 until 24 months of life, while others will not. Brain growth and development will be compared between those who have been treated with GH and those who have not. This is a pilot study in which the investigators will gather data to design a larger study that can answer these questions.
- Conditions
- Turner Syndrome
- Being tested
- somatotropin
- Keywords
- Turner Syndrome, growth hormone, brain development
- Medical subject headings
- Turner Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
somatotropinDrug
Also called Genotropin, Growth hormone
Subjects will receive 5 mg somatotropin (growth hormone) pens with cartridges. Subcutaneous injections are to be given every evening around bedtime. Dosing regimen: 50 mcg/kg/day to be adjusted at 4 month intervals to the closest 0.1 mg. Subjects will be given 12 months of treatment (from 12 to 24 months of life). Subjects will visit their pediatrician or pediatric endocrinologist at 4 and 8 months of life.
| Study group | Role | What they receive |
|---|---|---|
| Growth Hormone | Experimental | Subjects in the somatotropin (growth hormone, GH) arm will receive GH injections from 12-24 months of life. Receives: Drug: somatotropin |
| Control | No intervention | Subjects will receive no GH or placebo. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Total Brain Volume
Percent change in total brain volume as determined by magnetic resonance imaging (MRI)
Measured: Change in volume from 12 months of age scan in 24 months of age scan
Secondary outcomes (8)
Volume of Brain Lobes (Occipital)
Percent change in volumes of occipital lobes as determined by MRI.
Measured: Change in volume from 12 months of age scan in 24 months of age scan
White Matter Tracts (SLF)
Change in the fractional anisotropy (FA) of white matter tracts using Diffusion Tensor Imaging (DTI); superior longitudinal fasciculus
Measured: Change in FA from 12 months of age scan in 24 months of age scan
Volume of Brain Lobes (Central)
Percent change in volumes of central brain region (precentral gyrus, postcentral gyrus, rolandic operculum) as determined by MRI.
Measured: Change in volume from 12 months of age scan in 24 months of age scan
Volume of Brain Lobes (Frontal)
Percent change in volumes of frontal lobes as determined by MRI.
Measured: Change in volume from 12 months of age scan in 24 months of age scan
Volume of Brain Lobes (Temporal)
Percent change in volumes of temporal lobes as determined by MRI.
Measured: Change in volume from 12 months of age scan in 24 months of age scan
Volume of Brain Lobes (Parietal)
Percent change in volumes of parietal lobes as determined by MRI.
Measured: Change in volume from 12 months of age scan in 24 months of age scan
Volume of Brain Lobes (Limbic)
Percent change in volumes of parietal lobes as determined by MRI.
Measured: Change in volume from 12 months of age scan in 24 months of age scan
Volume of Brain Lobes (Insular Cortex)
Percent change in volumes of parietal lobes as determined by MRI.
Measured: Change in volume from 12 months of age scan in 24 months of age scan
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Jun 2011 (estimated)
- Study startMay 2010
- Primary completionMay 2014
- Study completionMay 2014
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.
- Other trials for Turner SyndromeStudies of the same condition, open and under way.
- 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
- Basic science
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 17 participants (actual)
Sponsor and investigators
- Lead sponsor
- Rebecca Knickmeyer Santelli, PhD
- Sponsor type
- University, hospital or other
- Collaborators
- Pfizer
- Responsible party
- Sponsor-investigator, Rebecca Knickmeyer Santelli, PhD, University of North Carolina, Chapel Hill
- Investigators
- Rebecca Knickmeyer, Principal investigator, University of North Carolina, Chapel Hill
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01367834
- Sponsor's protocol ID
- 09-2171
- Status last verified
- Feb 2017
- First submitted
- 2 Jun 2011
- Last updated
- 24 Mar 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Mar 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effect of Growth Hormone on Early Brain Development in Girls With Turner Syndrome
- ClinicalTrials.gov ID
- NCT01367834
- Acronym / sponsor ID
- 09-2171
- Conditions
- Turner Syndrome
- Keywords
- Turner Syndrome, growth hormone, brain development
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 17 participants (actual)
- Lead sponsor
- Rebecca Knickmeyer Santelli, PhDUniversity, hospital or other
- Collaborators
- Pfizer
- Study start
- May 2010
- Primary completion
- May 2014 (actual)
- Study completion
- May 2014 (actual)
- First posted
- 7 Jun 2011 (estimated)
- Results
- Posted 24 Mar 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this research study is to learn about brain development in very young girls with Turner syndrome (TS) and the effect that growth hormone (GH) therapy has on early brain development.
Detailed description
Studies in older children and adults have found that IQ (intelligence quotient) in individuals with Turner syndrome (TS) and the general population are similar. However, many individuals with TS have a nonverbal learning disability which may cause problems with imagining and working with objects in space (for example: building blocks, working puzzles, copying designs, driving a car) as well as problems in planning, paying attention, and getting along with people their own age. Studies of brain structure have found changes in the size and function of specific areas of the brain that are involved in these nonverbal processes. However, there are no data published on brain structure or function in girls with Turner Syndrome in the first few years of life when brains are learning and growing most rapidly.
In a recent study, very young, short girls with Turner Syndrome (TS)(averaging 2 years in age) were treated with Growth Hormone (GH) and almost reached an average height after 2 years. The risks of GH for the young girls in that study appeared to be the same as those for older girls. Therefore, it is now recommended that GH therapy be considered as soon as a child with TS has growth failure. Growth failure often occurs during infancy; therefore, more children with TS are now receiving GH therapy as young as 12 months of age. In this study, the investigators wish to start understanding what effect GH has on learning and brain growth when given between the ages of 12 and 24 months. In this study, the girls with TS will have developmental studies, a physical examination, magnetic resonance imaging (MRI), and blood drawn at one and two years of age. Some of the girls in this study will receive GH from 12 until 24 months of life, while others will not. Brain growth and development will be compared between those who have been treated with GH and those who have not. This is a pilot study in which the investigators will gather data to design a larger study that can answer these questions.
Study design
- Primary purpose
- Basic science
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Growth Hormone | Experimental | Drug: somatotropinSubjects in the somatotropin (growth hormone, GH) arm will receive GH injections from 12-24 months of life. |
| Control | No intervention | –Subjects will receive no GH or placebo. |
Who may qualify
- Ages
- 11 to 13 months
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (2 listed)
- Diagnosis of Turner syndrome
- Less than the 50th percentile for length for the general female population
Key exclusion criteria (7 listed)
- Prior Growth Hormone (GH) therapy
- Diabetes
- Allergy to metacresol (a preservative in the GH liquid that is injected)
- Contraindications for Magnetic Resonance Imaging (MRI) (such as metal in the body)
- Part of a Y chromosome in child's karyotype
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 |
|---|---|---|
| somatotropinAlso: Genotropin, Growth hormoneSubjects will receive 5 mg somatotropin (growth hormone) pens with cartridges. Subcutaneous injections are to be given every evening around bedtime. Dosing regimen: 50 mcg/kg/day to be adjusted at 4 month intervals to the closest 0.1 mg. Subjects will be given 12 months of treatment (from 12 to 24 months of life). Subjects will visit their pediatrician or pediatric endocrinologist at 4 and 8 months of life. | Drug | Growth Hormone |
Primary outcome
Total Brain Volume
Time frame: Change in volume from 12 months of age scan in 24 months of age scan
Percent change in total brain volume as determined by magnetic resonance imaging (MRI)
8 secondary outcomes
Volume of Brain Lobes (Occipital)
Time frame: Change in volume from 12 months of age scan in 24 months of age scan
White Matter Tracts (SLF)
Time frame: Change in FA from 12 months of age scan in 24 months of age scan
Volume of Brain Lobes (Central)
Time frame: Change in volume from 12 months of age scan in 24 months of age scan
Volume of Brain Lobes (Frontal)
Time frame: Change in volume from 12 months of age scan in 24 months of age scan
Volume of Brain Lobes (Temporal)
Time frame: Change in volume from 12 months of age scan in 24 months of age scan
Volume of Brain Lobes (Parietal)
Time frame: Change in volume from 12 months of age scan in 24 months of age scan
Volume of Brain Lobes (Limbic)
Time frame: Change in volume from 12 months of age scan in 24 months of age scan
Volume of Brain Lobes (Insular Cortex)
Time frame: Change in volume from 12 months of age scan in 24 months of age scan
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Mar 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- 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.
Rebecca Knickmeyer principal investigator
University of North Carolina, Chapel Hill
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing somatotropin in people with Turner Syndrome, run by Rebecca Knickmeyer Santelli, PhD 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 2 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 24 Mar 2017. 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 24 Mar 2017). 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?
Total Brain Volume (time frame: Change in volume from 12 months of age scan in 24 months of age scan). 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 24 Mar 2017.