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| Name | Class |
|---|---|
| Merck Serono S.A., Geneva | INDUSTRY |
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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).
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Retrospective cohort | Other |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Blood sampling | Other | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 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. | 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. | Year 1 |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Gilles Della Corte | Merck Serono S.A. , Geneva, Switzerland | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Hospital de Niños Ricardo Gutiérrez | Buenos Aires | Argentina | ||||
| Hospital de Pediatria Garrahan |
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| ID | Title | Description |
|---|---|---|
| FG000 | 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. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| 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. | Year 1 |
| Buenos Aires |
| Argentina |
| Hospital de Niños de la Santisima Trinidad | Córdoba | Argentina |
| University of Calgary - Alberta Children's Hospital | Calgary | Canada |
| CHU Sainte Justine Montréal | Montreal | Canada |
| Centre Hospitalier Universitaire de Sherbrooke - Fleurimont | Sherbrooke | Canada |
| British Columbia Children's Hospital | Vancouver | Canada |
| Fakultní nemocnice Brno | Brno | Czechia |
| University Hospital Hradec Kralove | Hradec Králové | Czechia |
| Faculty Hospital | Olomouc | Czechia |
| University Hospital Praha Motol | Prague | Czechia |
| Centre d'Endocrinologie Pédiatrique | Bordeaux | France |
| CHU Bordeaux - Hopital pédiatrique Pellegrin | Bordeaux | France |
| Hôpital Femme-Mère-Enfant | Bron | France |
| University of Cologne Children's Hospital | Cologne | Germany |
| University Children's Hospital | München | Germany |
| University of Bari Aldo Moro | Bari | Italy |
| Ospedale Microcitemico di Cagliari | Cagliari | Italy |
| Centro di Endocrinologia e Diabetologia Pediatrica | Catania | Italy |
| Istituto Giannina Gaslini - Clinica Pediatrica | Genova | Italy |
| Hospital 12 de Octubre | Madrid | Spain |
| Hospital Infantil Universitario Niño Jesús | Madrid | Spain |
| Hospital Universitario Gregorio Maran | Madrid | Spain |
| Hospital Clínico Universitario de Santiago de Compostela | Santiago de Compostela | Spain |
| Hospital Miguel Servet | Zaragoza | Spain |
| Queen Silvia Children's Hospital | Gothenburg | Sweden |
| Faculty of Health Sciences, Linkping University | Linköping | Sweden |
| Karolinska University Hospital Campus Solna | Stockholm | Sweden |
| Birmingham Children's Hospital | Birmingham | United Kingdom |
| Royal Manchester Children's Hospital | Manchester | United Kingdom |
| Sheffield Children's Hospital | Sheffield | United Kingdom |
| FG001 | 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. |
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| NOT COMPLETED |
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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.
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| ID | Title | Description |
|---|---|---|
| BG000 | 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. |
| BG001 | 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. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Customized | Number | participants |
| ||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | 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. | Posted | Mean | Standard Deviation | centimeter | Baseline and Year 1 |
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| Secondary | 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. | No genetic markers were identified during the study therefore, the data for this outcome measure was not analyzed. | Posted | Year 1 |
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| Secondary | 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. | No genetic markers were identified during the study therefore, the data for this outcome measure was not analyzed. | Posted | Year 1 |
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| Primary | 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. | 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. | Posted | Mean | Standard Deviation | standard deviation score | Baseline and Year 1 |
| |||||||||||||||||||||||||||||||||||||
| Primary | 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. | 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. | Posted | Mean | Standard Deviation | standard deviation score | Year 1 |
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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.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 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. | 0 | 293 | 0 | 0 | ||
| EG001 | 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. | 0 | 132 | 0 | 0 |
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No genetic markers were identified, therefore data for the secondary outcome measures was not analyzed.
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.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Merck KGaA Communication Center | Merck Serono, a division of Merck KGaA | +49-6151-72-5200 | service@merckgroup.com |
| ID | Term |
|---|---|
| D014424 | Turner Syndrome |
| D004393 | Dwarfism, Pituitary |
| ID | Term |
|---|---|
| D006059 | Gonadal Dysgenesis |
| D012734 | Disorders of Sex Development |
| D014564 | Urogenital Abnormalities |
| D052776 | Female Urogenital Diseases |
| D005261 | Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
| D058533 | Sex Chromosome Disorders of Sex Development |
| D052801 | Male Urogenital Diseases |
| D006330 | Heart Defects, Congenital |
| D018376 | Cardiovascular Abnormalities |
| D002318 | Cardiovascular Diseases |
| D006331 | Heart Diseases |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D025064 | Sex Chromosome Disorders |
| D025063 | Chromosome Disorders |
| D030342 | Genetic Diseases, Inborn |
| D006058 | Gonadal Disorders |
| D004700 | Endocrine System Diseases |
| D004392 | Dwarfism |
| D001848 | Bone Diseases, Developmental |
| D001847 | Bone Diseases |
| D009140 | Musculoskeletal Diseases |
| D001849 | Bone Diseases, Endocrine |
| D007018 | Hypopituitarism |
| D010900 | Pituitary Diseases |
| D007027 | Hypothalamic Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
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| ID | Term |
|---|---|
| D001800 | Blood Specimen Collection |
| ID | Term |
|---|---|
| D013048 | Specimen Handling |
| D019411 | Clinical Laboratory Techniques |
| D019937 | Diagnostic Techniques and Procedures |
| D003933 | Diagnosis |
| D011677 | Punctures |
| D013514 | Surgical Procedures, Operative |
| D008919 | Investigative Techniques |
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| greater than (>) 12 years |
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| Male |
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