Trial Outcomes & Findings for Effect of Growth Hormone on Early Brain Development in Girls With Turner Syndrome (NCT NCT01367834)

NCT ID: NCT01367834

Last Updated: 2017-03-24

Results Overview

Percent change in total brain volume as determined by magnetic resonance imaging (MRI)

Recruitment status

COMPLETED

Study phase

NA

Target enrollment

17 participants

Primary outcome timeframe

Change in volume from 12 months of age scan in 24 months of age scan

Results posted on

2017-03-24

Participant Flow

Subjects were recruited locally in the UNC\_Chapel Hill pediatric endocrinology clinic and nationally through the Turner Syndrome Society, Turner Syndrome family internet groups, and advertisements on our UNC research website.

Participant milestones

Participant milestones
Measure	Growth Hormone Subjects in the growth hormone (GH) arm will receive GH injections from 12-24 months of life. somatotropin: 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.	Control Subjects will receive no GH or placebo.
Overall Study STARTED	11	6
Overall Study COMPLETED	9	3
Overall Study NOT COMPLETED	2	3

Reasons for withdrawal

Reasons for withdrawal
Measure	Growth Hormone Subjects in the growth hormone (GH) arm will receive GH injections from 12-24 months of life. somatotropin: 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.	Control Subjects will receive no GH or placebo.
Overall Study Withdrawal by Subject	2	2
Overall Study Study was ended.	0	1

Baseline Characteristics

Effect of Growth Hormone on Early Brain Development in Girls With Turner Syndrome

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Growth Hormone n=11 Participants Subjects in the growth hormone (GH) arm will receive GH injections from 12-24 months of life. somatotropin: 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.	Control n=6 Participants Subjects will receive no GH or placebo.	Total n=17 Participants Total of all reporting groups
Age, Categorical <=18 years	11 Participants n=5 Participants	6 Participants n=7 Participants	17 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Continuous	13.12 months STANDARD_DEVIATION 0.82 • n=5 Participants	12.73 months STANDARD_DEVIATION 0.50 • n=7 Participants	12.96 months STANDARD_DEVIATION 0.73 • n=5 Participants
Sex: Female, Male Female	11 Participants n=5 Participants	6 Participants n=7 Participants	17 Participants n=5 Participants
Sex: Female, Male Male	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Region of Enrollment United States	11 participants n=5 Participants	6 participants n=7 Participants	17 participants n=5 Participants

PRIMARY outcome

Timeframe: Change in volume from 12 months of age scan in 24 months of age scan

Population: Note that only subjects with usable MRI data are included in this analysis (this is a subset of all children completing the protocol).

Percent change in total brain volume as determined by magnetic resonance imaging (MRI)

Outcome measures

Outcome measures
Measure	Growth Hormone n=5 Participants Subjects in the growth hormone (GH) arm will receive GH injections from 12-24 months of life.	Control n=1 Participants No intervention
Total Brain Volume	18.22 percentage change Standard Deviation 1.99	19.78 percentage change Standard Deviation NA N of 1

SECONDARY outcome

Timeframe: Change in volume from 12 months of age scan in 24 months of age scan

Population: Note that only subjects with usable MRI data are included in this analysis (this is a subset of all children completing the protocol).

Percent change in volumes of occipital lobes as determined by MRI.

Outcome measures

Outcome measures
Measure	Growth Hormone n=5 Participants Subjects in the growth hormone (GH) arm will receive GH injections from 12-24 months of life.	Control n=1 Participants No intervention
Volume of Brain Lobes (Occipital)	14.83 percentage change Standard Deviation 2.21	12.16 percentage change Standard Deviation NA N = 1

SECONDARY outcome

Timeframe: Change in FA from 12 months of age scan in 24 months of age scan

Population: Note that only subjects with usable MRI data are included in this analysis (this is a subset of all children completing the protocol).

Change in the fractional anisotropy (FA) of white matter tracts using Diffusion Tensor Imaging (DTI); superior longitudinal fasciculus

Outcome measures

Outcome measures
Measure	Growth Hormone n=4 Participants Subjects in the growth hormone (GH) arm will receive GH injections from 12-24 months of life.	Control n=1 Participants No intervention
White Matter Tracts (SLF)	15.12 percentage change Standard Deviation 5.01	16.35 percentage change Standard Deviation NA N = 1

SECONDARY outcome

Timeframe: Change in volume from 12 months of age scan in 24 months of age scan

Population: Note that only subjects with usable MRI data are included in this analysis (this is a subset of all children completing the protocol).

Percent change in volumes of central brain region (precentral gyrus, postcentral gyrus, rolandic operculum) as determined by MRI.

Outcome measures

Outcome measures
Measure	Growth Hormone n=5 Participants Subjects in the growth hormone (GH) arm will receive GH injections from 12-24 months of life.	Control n=1 Participants No intervention
Volume of Brain Lobes (Central)	17.63 percentage change Standard Deviation 2.13	10.23 percentage change Standard Deviation NA N = 1

SECONDARY outcome

Timeframe: Change in volume from 12 months of age scan in 24 months of age scan

Population: Note that only subjects with usable MRI data are included in this analysis (this is a subset of all children completing the protocol).

Percent change in volumes of frontal lobes as determined by MRI.

Outcome measures

Outcome measures
Measure	Growth Hormone n=5 Participants Subjects in the growth hormone (GH) arm will receive GH injections from 12-24 months of life.	Control n=1 Participants No intervention
Volume of Brain Lobes (Frontal)	15.43 percentage change Standard Deviation 1.84	14.38 percentage change Standard Deviation NA N = 1

SECONDARY outcome

Timeframe: Change in volume from 12 months of age scan in 24 months of age scan

Population: Note that only subjects with usable MRI data are included in this analysis (this is a subset of all children completing the protocol).

Percent change in volumes of temporal lobes as determined by MRI.

Outcome measures

Outcome measures
Measure	Growth Hormone n=5 Participants Subjects in the growth hormone (GH) arm will receive GH injections from 12-24 months of life.	Control n=1 Participants No intervention
Volume of Brain Lobes (Temporal)	10.79 percentage change Standard Deviation 3.68	5.08 percentage change Standard Deviation NA N = 1

SECONDARY outcome

Timeframe: Change in volume from 12 months of age scan in 24 months of age scan

Population: Note that only subjects with usable MRI data are included in this analysis (this is a subset of all children completing the protocol).

Percent change in volumes of parietal lobes as determined by MRI.

Outcome measures

Outcome measures
Measure	Growth Hormone n=5 Participants Subjects in the growth hormone (GH) arm will receive GH injections from 12-24 months of life.	Control n=1 Participants No intervention
Volume of Brain Lobes (Parietal)	18.86 percentage change Standard Deviation 5.01	11.95 percentage change Standard Deviation NA N = 1

SECONDARY outcome

Timeframe: Change in volume from 12 months of age scan in 24 months of age scan

Population: Note that only subjects with usable MRI data are included in this analysis (this is a subset of all children completing the protocol).

Percent change in volumes of parietal lobes as determined by MRI.

Outcome measures

Outcome measures
Measure	Growth Hormone n=5 Participants Subjects in the growth hormone (GH) arm will receive GH injections from 12-24 months of life.	Control n=1 Participants No intervention
Volume of Brain Lobes (Limbic)	15.43 percentage change Standard Deviation 2.99	22.71 percentage change Standard Deviation NA N = 1

SECONDARY outcome

Timeframe: Change in volume from 12 months of age scan in 24 months of age scan

Population: Note that only subjects with usable MRI data are included in this analysis (this is a subset of all children completing the protocol).

Percent change in volumes of parietal lobes as determined by MRI.

Outcome measures

Outcome measures
Measure	Growth Hormone n=5 Participants Subjects in the growth hormone (GH) arm will receive GH injections from 12-24 months of life.	Control n=1 Participants No intervention
Volume of Brain Lobes (Insular Cortex)	15.79 percentage change Standard Deviation 2.11	19.78 percentage change Standard Deviation NA N = 1

Adverse Events

Growth Hormone

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Control

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Rebecca Knickmeyer Santelli

UNC Chapel Hill

Phone: 9199662216

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60