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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

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ClinicalTrials.gov Identifier: NCT01419249
Recruitment Status : Completed
First Posted : August 18, 2011
Results First Posted : January 16, 2014
Last Update Posted : January 16, 2014
Sponsor:
Collaborator:
Merck Serono S.A., Geneva
Information provided by (Responsible Party):
Merck KGaA, Darmstadt, Germany

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: None (Open Label)
Conditions Idiopathic Growth Hormone Deficiency
Turner Syndrome
Intervention Other: Blood sampling
Enrollment 458
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Idiopathic Growth Hormone Deficiency (IGHD) Cohort Turner Syndrome (TS) Cohort
Hide Arm/Group Description 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. 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.
Period Title: Overall Study
Started 318 140
Completed 318 140
Not Completed 0 0
Arm/Group Title Idiopathic Growth Hormone Deficiency (IGHD) Cohort Turner Syndrome (TS) Cohort Total
Hide Arm/Group Description 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. 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 of all reporting groups
Overall Number of Baseline Participants 293 132 425
Hide Baseline Analysis Population Description
Full Analysis Set (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.
Age, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 293 participants 132 participants 425 participants
less than (<) 8 years 197 95 292
Between 8 to 12 years 84 31 115
greater than (>) 12 years 12 6 18
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 293 participants 132 participants 425 participants
Female
85
  29.0%
132
 100.0%
217
  51.1%
Male
208
  71.0%
0
   0.0%
208
  48.9%
1.Primary Outcome
Title Change From Baseline in Height at Year 1
Hide Description 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.
Time Frame Baseline and Year 1
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Idiopathic Growth Hormone Deficiency (IGHD) Cohort Turner Syndrome (TS) Cohort
Hide Arm/Group Description:
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.
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 Number of Participants Analyzed 293 132
Mean (Standard Deviation)
Unit of Measure: centimeter
Baseline 103.6  (18.1) 103.5  (16.3)
Change at Year 1 9.8  (2.7) 8.6  (2.0)
2.Primary Outcome
Title Change From Baseline in Height Standard Deviation Score (SDS) at Year 1
Hide Description 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.
Time Frame Baseline and Year 1
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Idiopathic Growth Hormone Deficiency (IGHD) Cohort Turner Syndrome (TS) Cohort
Hide Arm/Group Description:
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.
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 Number of Participants Analyzed 293 132
Mean (Standard Deviation)
Unit of Measure: standard deviation score
Baseline -2.60  (1.06) -2.17  (1.03)
Change at Year 1 0.98  (0.67) 0.71  (0.48)
3.Primary Outcome
Title Height Velocity Standard Deviation Score (SDS) at Year 1
Hide Description 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.
Time Frame Year 1
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Idiopathic Growth Hormone Deficiency (IGHD) Cohort Turner Syndrome (TS) Cohort
Hide Arm/Group Description:
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.
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 Number of Participants Analyzed 293 132
Mean (Standard Deviation)
Unit of Measure: standard deviation score
4.18  (2.90) 2.59  (1.92)
4.Secondary Outcome
Title Evaluation of the Contribution of Validated Genetic Markers to the Amplitude of First Year Growth Response to r-hGH Therapy in IGHD Children Using Growth Hormone Deficiency Kabi-Pharmacia International Growth Study (GHD KIGS) Predictive Model
Hide Description 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.
Time Frame Year 1
Hide Outcome Measure Data
Hide Analysis Population Description
No genetic markers were identified during the study therefore, the data for this outcome measure was not analyzed.
Arm/Group Title Idiopathic Growth Hormone Deficiency (IGHD) Cohort
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
5.Secondary Outcome
Title Evaluation of the Contribution of Validated Genetic Markers to the Amplitude of First Year Growth Response to r-hGH Therapy in TS Girls Using Turner Syndrome Kabi-Pharmacia International Growth Study (TS KIGS) Predictive Model
Hide Description 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.
Time Frame Year 1
Hide Outcome Measure Data
Hide Analysis Population Description
No genetic markers were identified during the study therefore, the data for this outcome measure was not analyzed.
Arm/Group Title Turner Syndrome (TS) Cohort
Hide Arm/Group Description:
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 Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
Time Frame [Not Specified]
Adverse Event Reporting Description As it is a retrospective study, only serious adverse events which were considered by the investigator to be at least possibly related to the conduct of the trial were collected.
 
Arm/Group Title Idiopathic Growth Hormone Deficiency (IGHD) Cohort Turner Syndrome (TS) Cohort
Hide Arm/Group Description 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. 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.
All-Cause Mortality
Idiopathic Growth Hormone Deficiency (IGHD) Cohort Turner Syndrome (TS) Cohort
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Idiopathic Growth Hormone Deficiency (IGHD) Cohort Turner Syndrome (TS) Cohort
Affected / at Risk (%) Affected / at Risk (%)
Total   0/293 (0.00%)   0/132 (0.00%) 
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Idiopathic Growth Hormone Deficiency (IGHD) Cohort Turner Syndrome (TS) Cohort
Affected / at Risk (%) Affected / at Risk (%)
Total   0/0   0/0 
No genetic markers were identified, therefore data for the secondary outcome measures was not analyzed.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
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.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Merck KGaA Communication Center
Organization: Merck Serono, a division of Merck KGaA
Phone: +49-6151-72-5200
EMail: service@merckgroup.com
Layout table for additonal information
Responsible Party: Merck KGaA, Darmstadt, Germany
ClinicalTrials.gov Identifier: NCT01419249     History of Changes
Other Study ID Numbers: EMR 200104_010
First Submitted: August 16, 2011
First Posted: August 18, 2011
Results First Submitted: November 26, 2013
Results First Posted: January 16, 2014
Last Update Posted: January 16, 2014