Trial Outcomes & Findings for Long-Term Growth and Skeletal Effects of Early Growth Hormone Treatment in Turner Syndrome (NCT NCT00266656)

Last Updated: 2017-03-31

Results Overview

SDS reports the number of standard deviations from the mean for age and sex for an individual measurement (normal range is -2 to +2 SDS). Height SDS is derived by subtracting the population mean from individual's height value and then dividing that difference by the population standard deviation. Greater height SDS values indicate greater height.

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

69 participants

Primary outcome timeframe

Baseline through End of Study (10 years)

Results posted on

2017-03-31

Participant Flow

To be included in this study, participants had to be females with karyotype-proven Turner syndrome who were previously randomized in Study B9R-US-GDFG (NCT00406926).

Participant milestones

Participant milestones
Measure	Early Treated Humatrope administered according to investigator's clinical practice and guided by the approved package insert. Humatrope administered in B9R-US-GDFG (NCT00406926).	Early Untreated Humatrope administered according to investigator's clinical practice and guided by the approved package insert. Control: Humatrope was not administered in B9R-US-GDFG (NCT00406926).
Overall Study STARTED	36	33
Overall Study COMPLETED	22	20
Overall Study NOT COMPLETED	14	13

Reasons for withdrawal

Reasons for withdrawal
Measure	Early Treated Humatrope administered according to investigator's clinical practice and guided by the approved package insert. Humatrope administered in B9R-US-GDFG (NCT00406926).	Early Untreated Humatrope administered according to investigator's clinical practice and guided by the approved package insert. Control: Humatrope was not administered in B9R-US-GDFG (NCT00406926).
Overall Study Death	1	0
Overall Study Lost to Follow-up	4	1
Overall Study Withdrawal by Subject	4	2
Overall Study Did Not Reach Final Height	4	8
Overall Study Parent/Caregiver Decision	1	2

Baseline Characteristics

Long-Term Growth and Skeletal Effects of Early Growth Hormone Treatment in Turner Syndrome

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Early Treated n=36 Participants Early treated n=36	Early Untreated n=33 Participants Early untreated n=33	Total n=69 Participants Total of all reporting groups
Age, Continuous	8.29 years STANDARD_DEVIATION 1.22 • n=5 Participants	8.41 years STANDARD_DEVIATION 1.30 • n=7 Participants	8.35 years STANDARD_DEVIATION 1.25 • n=5 Participants
Sex: Female, Male Female	36 Participants n=5 Participants	33 Participants n=7 Participants	69 Participants n=5 Participants
Sex: Female, Male Male	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Height	124.34 centimeter STANDARD_DEVIATION 10.08 • n=5 Participants	121.06 centimeter STANDARD_DEVIATION 9.76 • n=7 Participants	122.77 centimeter STANDARD_DEVIATION 9.99 • n=5 Participants
Height Standard Deviation Score (SDS)	-0.84 Standard Deviation Score STANDARD_DEVIATION 1.22 • n=5 Participants	-1.48 Standard Deviation Score STANDARD_DEVIATION 1.23 • n=7 Participants	-1.15 Standard Deviation Score STANDARD_DEVIATION 1.26 • n=5 Participants
Bone Age	8.73 years STANDARD_DEVIATION 1.55 • n=5 Participants	8.34 years STANDARD_DEVIATION 1.57 • n=7 Participants	8.54 years STANDARD_DEVIATION 1.56 • n=5 Participants

PRIMARY outcome

Timeframe: Baseline through End of Study (10 years)

Population: All participants who achieved Near Adult Height (NAH). NAH is defined as first height measured when height velocity is less than or equal to 2.0 centimeter per year over the preceding year (in the absence of growth-impairing process such as hypothyroidism or inflammatory bowel disease), or bone age greater than or equal to14.5 years.

Outcome measures

Outcome measures
Measure	Early Treated n=25 Participants Humatrope administered according to investigator's clinical practice and guided by the approved package insert. Humatrope administered in B9R-US-GDFG (NCT00406926).	Early Untreated n=26 Participants Humatrope administered according to investigator's clinical practice and guided by the approved package insert. Control: Humatrope was not administered in B9R-US-GDFG (NCT00406926).
Most Mature Height Standard Deviation Score (SDS)	-1.37 standard deviation score Standard Deviation 1.09	-1.60 standard deviation score Standard Deviation 1.21

SECONDARY outcome

Timeframe: Age 10, Age 13, Age 16

Population: All participants who had a baseline visit and at least one post-baseline visit.

Outcome measures

Outcome measures
Measure	Early Treated n=35 Participants Humatrope administered according to investigator's clinical practice and guided by the approved package insert. Humatrope administered in B9R-US-GDFG (NCT00406926).	Early Untreated n=32 Participants Humatrope administered according to investigator's clinical practice and guided by the approved package insert. Control: Humatrope was not administered in B9R-US-GDFG (NCT00406926).
Height SDS at Various Ages Age 10 (n=32, 30)	-0.66 Standard deviation score Standard Deviation 1.16	-1.28 Standard deviation score Standard Deviation 1.17
Height SDS at Various Ages Age 13 (n=29, 29)	-1.29 Standard deviation score Standard Deviation 1.24	-1.87 Standard deviation score Standard Deviation 1.16
Height SDS at Various Ages Age 16 (n=18, 18)	-1.66 Standard deviation score Standard Deviation 1.11	-1.69 Standard deviation score Standard Deviation 1.30

SECONDARY outcome

Timeframe: Baseline through End of Study (10 years)

Population: All participants who had a baseline visit and at least one post-baseline visit.

The Tanner 2 breast development is the age at first evidence of breast development.

Outcome measures

Outcome measures
Measure	Early Treated n=35 Participants Humatrope administered according to investigator's clinical practice and guided by the approved package insert. Humatrope administered in B9R-US-GDFG (NCT00406926).	Early Untreated n=32 Participants Humatrope administered according to investigator's clinical practice and guided by the approved package insert. Control: Humatrope was not administered in B9R-US-GDFG (NCT00406926).
Age at Attainment of Tanner 2 Breast Development	11.80 years Standard Error 0.26	12.14 years Standard Error 0.31

SECONDARY outcome

Timeframe: Baseline through End of Study (10 years)

Bone age was measured by standard radiograph, x-ray at baseline and annually for 10 years or until attainment of height velocity less than or equal to 1.0 centimeter per year (cm/year) and bone age greater or equal to 15 years.