Trial · Interventional

CompletedPhase 4Results postedRegistry data

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

NCTNCT01419249Other idEMR 200104_010

Open at ClinicalTrials.govAsk about this trial

This trial does not test a peptide that PepBank tracks.

Measures HGH (somatotropin), does not give it. It appears in an outcome measure.

Overview

Status: Completed
Phase: Phase 4
Enrollment: 458 participants (actual)
Sponsor: Merck KGaA, Darmstadt, Germany · Industry
Design: Not randomized · Open label (no masking)
Countries: 9 countries
Argentina, Canada, Czechia, France, Germany, Italy, Spain, Sweden, United Kingdom
Start: Sept 2011
Primary completion: Oct 2012
Completion: Oct 2012
First posted: 18 Aug 2011 (estimated)
Last update posted: 16 Jan 2014 (estimated)
Conditions: Idiopathic Growth Hormone Deficiency · Turner Syndrome

Timeline

1 trial · 1 completed · 1 with posted results

Completed
Ongoing
Not yet recruiting
Stopped
Estimated dates (est.)
Results posted

201120122013

Ph 4
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
NCT01419249
Sept 2011 to Oct 2012

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

PREDICT Validation is a validation pharmacogenetic trial. The purpose of this study is to confirm that some genes can be used to predict how well a subject diagnosed with idiopathic growth hormone deficiency (IGHD) or turner syndrome (TS) will respond to a treatment with recombinant human growth hormone (r-hGH).

Interventions

Blood samplingOther

Arms1

Other
Retrospective cohort

Primary outcomes3

Outcomes the sponsor listed as primary.

Change From Baseline in Height at Year 1
Time frame · Baseline and Year 1
Registry 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.
Change From Baseline in Height Standard Deviation Score (SDS) at Year 1
Time frame · Baseline and Year 1
Registry 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.
Height Velocity Standard Deviation Score (SDS) at Year 1
Time frame · Year 1
Registry 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.

Secondary outcomes(2)

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
Time frame · Year 1
Registry 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.
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
Time frame · Year 1
Registry 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.

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

Primary outcome results

Primary outcomeMean· Baseline and Year 1

Change From Baseline in Height at Year 1

Group	Value (centimeter)	Spread
Idiopathic Growth Hormone Deficiency (IGHD) Cohort	103.6	18.1
Turner Syndrome (TS) Cohort	103.5	16.3

Primary outcomeMean· Baseline and Year 1

Change From Baseline in Height Standard Deviation Score (SDS) at Year 1

Group	Value (standard deviation score)	Spread
Idiopathic Growth Hormone Deficiency (IGHD) Cohort	-2.60	1.06
Turner Syndrome (TS) Cohort	-2.17	1.03

Primary outcomeMean· Year 1

Height Velocity Standard Deviation Score (SDS) at Year 1

Group	Value (standard deviation score)	Spread
Idiopathic Growth Hormone Deficiency (IGHD) Cohort	4.18	2.90
Turner Syndrome (TS) Cohort	2.59	1.92

Secondary and other outcome results(2)

Secondary outcome· Year 1

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

Group	Value
Idiopathic Growth Hormone Deficiency (IGHD) Cohort	not posted

Secondary outcome· Year 1

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

Group	Value
Turner Syndrome (TS) Cohort	not posted

Adverse events, as posted

Adverse events as posted: participants affected out of participants at risk, by group
Group	Deaths	Serious events	Other events
Idiopathic Growth Hormone Deficiency (IGHD) CohortParticipants 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.	–	0 / 293	0 / 0
Turner Syndrome (TS) CohortParticipants 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.	–	0 / 132	0 / 0

Each cell reads participants affected / participants at risk, as the registry posted them.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies0

Studies that cite this trial.

No study cites this trial.

Eligibility

Who could join, as posted.

Sex: All
Age: up to 18 Years
Healthy volunteers: Not accepted

Inclusion and exclusion criteria, as posted

Inclusion Criteria: * Pre-established diagnosis of IGHD or TS based on classical criteria with at least 1 year of r-hGH therapy and with Tanner stage 1 at treatment start * Retrospective availability of a complete set of clinical, auxological and biological parameters necessary for building the predictive model * Other protocol defined inclusion criteria could apply Exclusion Criteria: * Acquired growth hormone deficiency (GHD) * Any drug or disease that could affect growth during the first year of r-hGH treatment * Other protocol defined exclusion criteria could apply

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