Trial Outcomes & Findings for First Year Growth Response Associated Genetic Markers Validation Phase IV Open-label Study in Growth Hormone Deficient and Turner Syndrome Pre-pubertal Children: the PREDICT Pharmacogenetics Validation Study (NCT NCT01419249)
NCT ID: NCT01419249
Last Updated: 2014-01-16
Results Overview
Change from baseline in height at year 1 was one of the growth parameter to assess the first year growth response to r-hGH treatment.
COMPLETED
PHASE4
458 participants
Baseline and Year 1
2014-01-16
Participant Flow
Participant milestones
| Measure |
Idiopathic Growth Hormone Deficiency (IGHD) Cohort
Participants with pre-established diagnosis of IGHD and were treated with recombinant human growth hormone (r-hGH) therapy for at least 1 year were observed in this retrospective cohort study wherein blood sampling was performed for genotyping of the various genetic markers along with collection of retrospective data relative to the r-hGH treatment.
|
Turner Syndrome (TS) Cohort
Participants with pre-established diagnosis of TS and were treated with r-hGH therapy for at least 1 year were observed in this retrospective cohort study wherein blood sampling was performed for genotyping of the various genetic markers along with collection of retrospective data relative to the r-hGH treatment.
|
|---|---|---|
|
Overall Study
STARTED
|
318
|
140
|
|
Overall Study
COMPLETED
|
318
|
140
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
First Year Growth Response Associated Genetic Markers Validation Phase IV Open-label Study in Growth Hormone Deficient and Turner Syndrome Pre-pubertal Children: the PREDICT Pharmacogenetics Validation Study
Baseline characteristics by cohort
| Measure |
Idiopathic Growth Hormone Deficiency (IGHD) Cohort
n=293 Participants
Participants with pre-established diagnosis of IGHD and were treated with recombinant human growth hormone (r-hGH) therapy for at least 1 year were observed in this retrospective cohort study wherein blood sampling was performed for genotyping of the various genetic markers along with collection of retrospective data relative to the r-hGH treatment.
|
Turner Syndrome (TS) Cohort
n=132 Participants
Participants with pre-established diagnosis of TS and were treated with r-hGH therapy for at least 1 year were observed in this retrospective cohort study wherein blood sampling was performed for genotyping of the various genetic markers along with collection of retrospective data relative to the r-hGH treatment.
|
Total
n=425 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Customized
less than (<) 8 years
|
197 participants
n=93 Participants
|
95 participants
n=4 Participants
|
292 participants
n=27 Participants
|
|
Age, Customized
Between 8 to 12 years
|
84 participants
n=93 Participants
|
31 participants
n=4 Participants
|
115 participants
n=27 Participants
|
|
Age, Customized
greater than (>) 12 years
|
12 participants
n=93 Participants
|
6 participants
n=4 Participants
|
18 participants
n=27 Participants
|
|
Sex: Female, Male
Female
|
85 Participants
n=93 Participants
|
132 Participants
n=4 Participants
|
217 Participants
n=27 Participants
|
|
Sex: Female, Male
Male
|
208 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
208 Participants
n=27 Participants
|
PRIMARY outcome
Timeframe: Baseline and Year 1Population: FAS population included all the participants who had provided informed consent and had non-missing height at start (defined as within one month prior to treatment start date) and at 1 year (+/- 120 days) of r-hGH treatment and had pharmacogenomics data available.
Change from baseline in height at year 1 was one of the growth parameter to assess the first year growth response to r-hGH treatment.
Outcome measures
| Measure |
Idiopathic Growth Hormone Deficiency (IGHD) Cohort
n=293 Participants
Participants with pre-established diagnosis of IGHD and were treated with recombinant human growth hormone (r-hGH) therapy for at least 1 year were observed in this retrospective cohort study wherein blood sampling was performed for genotyping of the various genetic markers along with collection of retrospective data relative to the r-hGH treatment.
|
Turner Syndrome (TS) Cohort
n=132 Participants
Participants with pre-established diagnosis of TS and were treated with r-hGH therapy for at least 1 year were observed in this retrospective cohort study wherein blood sampling was performed for genotyping of the various genetic markers along with collection of retrospective data relative to the r-hGH treatment.
|
|---|---|---|
|
Change From Baseline in Height at Year 1
Baseline
|
103.6 centimeter
Standard Deviation 18.1
|
103.5 centimeter
Standard Deviation 16.3
|
|
Change From Baseline in Height at Year 1
Change at Year 1
|
9.8 centimeter
Standard Deviation 2.7
|
8.6 centimeter
Standard Deviation 2.0
|
PRIMARY outcome
Timeframe: Baseline and Year 1Population: FAS population included all the participants who had provided informed consent and had non-missing height at start (defined as within one month prior to treatment start date) and at 1 year (+/- 120 days) of r-hGH treatment and had pharmacogenomics data available.
Height SDS was calculated as height minus reference mean height divided by standard deviation of the reference population. Height SDS reflects the height relative to a reference population of the same age and gender. Change from baseline in height SDS at Year 1 was one of the growth parameter to assess the first year growth response to r-hGH treatment.
Outcome measures
| Measure |
Idiopathic Growth Hormone Deficiency (IGHD) Cohort
n=293 Participants
Participants with pre-established diagnosis of IGHD and were treated with recombinant human growth hormone (r-hGH) therapy for at least 1 year were observed in this retrospective cohort study wherein blood sampling was performed for genotyping of the various genetic markers along with collection of retrospective data relative to the r-hGH treatment.
|
Turner Syndrome (TS) Cohort
n=132 Participants
Participants with pre-established diagnosis of TS and were treated with r-hGH therapy for at least 1 year were observed in this retrospective cohort study wherein blood sampling was performed for genotyping of the various genetic markers along with collection of retrospective data relative to the r-hGH treatment.
|
|---|---|---|
|
Change From Baseline in Height Standard Deviation Score (SDS) at Year 1
Baseline
|
-2.60 standard deviation score
Standard Deviation 1.06
|
-2.17 standard deviation score
Standard Deviation 1.03
|
|
Change From Baseline in Height Standard Deviation Score (SDS) at Year 1
Change at Year 1
|
0.98 standard deviation score
Standard Deviation 0.67
|
0.71 standard deviation score
Standard Deviation 0.48
|
PRIMARY outcome
Timeframe: Year 1Population: FAS population included all the participants who had provided informed consent and had non-missing height at start (defined as within one month prior to treatment start date) and at 1 year (+/- 120 days) of r-hGH treatment and had pharmacogenomics data available.
Height velocity SDS was calculated as height velocity minus reference mean height velocity divided by standard deviation of the reference population. Height velocity SDS reflects the height velocity relative to a reference population of the same age and gender. Height velocity SDS at Year 1 was one of the growth parameter to assess the first year growth response to r-hGH treatment.
Outcome measures
| Measure |
Idiopathic Growth Hormone Deficiency (IGHD) Cohort
n=293 Participants
Participants with pre-established diagnosis of IGHD and were treated with recombinant human growth hormone (r-hGH) therapy for at least 1 year were observed in this retrospective cohort study wherein blood sampling was performed for genotyping of the various genetic markers along with collection of retrospective data relative to the r-hGH treatment.
|
Turner Syndrome (TS) Cohort
n=132 Participants
Participants with pre-established diagnosis of TS and were treated with r-hGH therapy for at least 1 year were observed in this retrospective cohort study wherein blood sampling was performed for genotyping of the various genetic markers along with collection of retrospective data relative to the r-hGH treatment.
|
|---|---|---|
|
Height Velocity Standard Deviation Score (SDS) at Year 1
|
4.18 standard deviation score
Standard Deviation 2.90
|
2.59 standard deviation score
Standard Deviation 1.92
|
SECONDARY outcome
Timeframe: Year 1Population: No genetic markers were identified during the study therefore, the data for this outcome measure was not analyzed.
GHD KIGS predictive model includes various clinical, auxological and biological markers which are as follows: maximum growth hormone (GH) response to provocation test; age at onset of therapy; birth weight SDS; average GH dose received during the first year of r-hGH therapy; height SDS at start of therapy; the difference between the pre-treatment height SDS of the subject and the mid parental height SDS; and weight SDS at start of therapy.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Year 1Population: No genetic markers were identified during the study therefore, the data for this outcome measure was not analyzed.
TS KIGS predictive model includes various clinical, auxological and biological markers which are as follows: maximum GH response to provocation test; age at onset of therapy; birth weight SDS; average GH dose received during the first year of r-hGH therapy; height SDS at start of therapy; the difference between the pre-treatment height SDS of the subject and the mid parental height SDS; and weight SDS at start of therapy.
Outcome measures
Outcome data not reported
Adverse Events
Idiopathic Growth Hormone Deficiency (IGHD) Cohort
Turner Syndrome (TS) Cohort
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Merck KGaA Communication Center
Merck Serono, a division of Merck KGaA
Results disclosure agreements
- Principal investigator is a sponsor employee Sponsor does not object to trial results publication by Investigator. Investigator will provide sponsor proposed publication/disclosure for review before submission. In multi-center trial, Investigator agree on first publication to be joint involving all sites. Investigator can decline to be part of joint publication. If joint manuscript has not submitted for publication within 12 months of trial completion/termination at all sites, Investigator can publish separately, subject to this agreement.
- Publication restrictions are in place
Restriction type: OTHER