Idiopathic Growth Hormone DeficiencyPhase 4NCT01419249
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
A Phase 4 study testing Blood sampling in people with Idiopathic Growth Hormone Deficiency and Turner Syndrome, run by Merck KGaA, Darmstadt, Germany at 31 sites in 9 countries.
Trial summary
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). Read more about this study
- Who is it for?
- Ages Up to 18 years
- Conditions the study is for
- Idiopathic Growth Hormone Deficiency, Turner Syndrome
- What is being tested?
- Blood sampling
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 458 participants
- Where
- 31 sitesin 9 countries
- Study dates
- Sep 2011to Oct 2012
- Phase
- Phase 4
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
31 sites in 9 countries.
31 sites in 9 countries
Spain5 sites
Hospital 12 de Octubre
Madrid
Hospital Infantil Universitario Niño Jesús
Madrid
Hospital Universitario Gregorio Maran
Madrid
Hospital Clínico Universitario de Santiago de Compostela
Santiago de Compostela
Hospital Miguel Servet
Zaragoza
Canada4 sites
University of Calgary - Alberta Children's Hospital
Calgary
CHU Sainte Justine Montréal
Montreal
Centre Hospitalier Universitaire de Sherbrooke - Fleurimont
Sherbrooke
British Columbia Children's Hospital
Vancouver
Czechia4 sites
Fakultní nemocnice Brno
Brno
University Hospital Hradec Kralove
Hradec Králové
Faculty Hospital
Olomouc
University Hospital Praha Motol
Prague
Italy4 sites
University of Bari Aldo Moro
Bari
Ospedale Microcitemico di Cagliari
Cagliari
Centro di Endocrinologia e Diabetologia Pediatrica
Catania
Istituto Giannina Gaslini - Clinica Pediatrica
Genova
Argentina3 sites
Hospital de Niños Ricardo Gutiérrez
Buenos Aires
Hospital de Pediatria Garrahan
Buenos Aires
Hospital de Niños de la Santisima Trinidad
Córdoba
France3 sites
Centre d'Endocrinologie Pédiatrique
Bordeaux
CHU Bordeaux - Hopital pédiatrique Pellegrin
Bordeaux
Hôpital Femme-Mère-Enfant
Bron
Sweden3 sites
Queen Silvia Children's Hospital
Gothenburg
Faculty of Health Sciences, Linkping University
Linköping
Karolinska University Hospital Campus Solna
Stockholm
United Kingdom3 sites
Birmingham Children's Hospital
Birmingham
Royal Manchester Children's Hospital
Manchester
Sheffield Children's Hospital
Sheffield
Germany2 sites
University of Cologne Children's Hospital
Cologne
University Children's Hospital
München
About this study
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).
Read the detailed description
This study is an open-label, interventional, retrospective, multicenter, international study, single-arm, non-randomized, and non-controlled study. The subject's trial participation includes a single visit. During the visit, subjects who give consent to participate in the trial will undergo blood sampling for genetic markers testing and retrospective data will be collected relative to the first year of the subject's r-hGH treatment.
The r-hGH treatment followed by the subject is indicated the pediatric population, therefore most of the subjects included into the trial will be below 18 years old.
This study is a non-investigational medicinal product (IMP) trial therefore no drug product data is provided.
- Being tested
- Blood sampling
- Keywords
- Growth Hormone Deficiency, Turner syndrome, Pharmacogenetics, Recombinant Human Growth Hormone therapy
- Medical subject headings
- Turner Syndrome, Dwarfism, Pituitary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Blood samplingOther
Subjects with pre-established diagnosis of IGHD and TS and were treated with r-hGH therapy for 1 year, will be observed in this retrospective cohort study wherein blood sampling will performed for genotyping of the various genetic markers along with collection of retrospective data relative to the r-hGH treatment.
| Study group | Role | What they receive |
|---|---|---|
| Retrospective cohort | Other | Not described Receives: Other: Blood sampling |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in Height at Year 1
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.
Measured: Baseline and Year 1
Change From Baseline in Height Standard Deviation Score (SDS) at Year 1
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.
Measured: Baseline and Year 1
Height Velocity Standard Deviation Score (SDS) at Year 1
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.
Measured: Year 1
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
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.
Measured: 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
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.
Measured: Year 1
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Aug 2011 (estimated)
- Study startSep 2011
- Primary completionOct 2012
- Study completionOct 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Idiopathic Growth Hormone DeficiencyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 458 participants (actual)
Sponsor and investigators
- Lead sponsor
- Merck KGaA, Darmstadt, Germany
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Merck Serono S.A., Geneva
- Responsible party
- Sponsor
- Investigators
- Gilles Della Corte, Study director, Merck Serono S.A. , Geneva, Switzerland
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01419249
- Sponsor's protocol ID
- EMR 200104_010
- Status last verified
- Nov 2013
- First submitted
- 16 Aug 2011
- Last updated
- 16 Jan 2014
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Jan 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT01419249
- Acronym / sponsor ID
- EMR 200104_010
- Keywords
- Growth Hormone Deficiency, Turner syndrome, Pharmacogenetics, Recombinant Human Growth Hormone therapy
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 458 participants (actual)
- Lead sponsor
- Merck KGaA, Darmstadt, GermanyIndustry (pharma, biotech, devices)
- Collaborators
- Merck Serono S.A., Geneva
- Study start
- Sep 2011
- Primary completion
- Oct 2012 (actual)
- Study completion
- Oct 2012 (actual)
- First posted
- 18 Aug 2011 (estimated)
- Results
- Posted 16 Jan 2014
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Detailed description
This study is an open-label, interventional, retrospective, multicenter, international study, single-arm, non-randomized, and non-controlled study. The subject's trial participation includes a single visit. During the visit, subjects who give consent to participate in the trial will undergo blood sampling for genetic markers testing and retrospective data will be collected relative to the first year of the subject's r-hGH treatment.
The r-hGH treatment followed by the subject is indicated the pediatric population, therefore most of the subjects included into the trial will be below 18 years old.
This study is a non-investigational medicinal product (IMP) trial therefore no drug product data is provided.
Study design
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Retrospective cohort | Other | Other: Blood sampling |
Who may qualify
- Ages
- Up to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (3 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Blood samplingSubjects with pre-established diagnosis of IGHD and TS and were treated with r-hGH therapy for 1 year, will be observed in this retrospective cohort study wherein blood sampling will performed for genotyping of the various genetic markers along with collection of retrospective data relative to the r-hGH treatment. | Other | Retrospective cohort |
Primary outcome
Change From Baseline in Height at Year 1
Time frame: Baseline and Year 1
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
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
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.
2 secondary outcomes
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
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
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Jan 2014.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Gilles Della Corte study director
Merck Serono S.A. , Geneva, Switzerland
31 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Blood sampling in people with Idiopathic Growth Hormone Deficiency and Turner Syndrome, run by Merck KGaA, Darmstadt, Germany at 31 sites in 9 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 16 Jan 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 31 sites in Spain, Canada, Czechia, Italy, Argentina and 4 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 16 Jan 2014). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change From Baseline in Height at Year 1 (time frame: Baseline and Year 1). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Jan 2014.