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The study aims at identifying the predictive markers after one month of Saizen therapy in Growth Hormone Deficiency (GHD) and Turner Syndrome children.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Turner Syndrome (TS) | Experimental |
| |
| Growth Hormone Deficiency (GHD) | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Saizen | Drug | Subjects with TS will receive SAIZEN® as subcutaneous injection at a dose of 0.050 milligram per kilogram (mg/kg) of body weight per day (within the recommended dosage 0.045-0.050 mg/kg body weight) for a period of 1 month |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Insulin Like Growth Factor-1 Standard Deviation Score (IGF-1 SDS) at Month 1 | IGF-1 SDS was calculated using the Elmlinger reference method. Change in within subject IGF-1 levels (standard deviation scores) at Month 1 from Baseline was assessed. Descriptive statistics were determined for the Baseline and Month 1 assessments, and also for the level of change between these two assessments. If either the Baseline or Month 1 IGF-1 level was missing, then the within-subject change in IGF-1 was assumed to be missing. | Baseline, Month 1 |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Insulin-like Growth Factor Binding Protein - 3 (IGFBP-3) Level at Month 1 | Baseline, Month 1 | |
| Change From Baseline in Fasting Glucose Levels at Month 1 | Baseline, Month 1 |
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Inclusion Criteria:
A) GHD: documented pre-established diagnosis of GHD with a growth hormone (GH) peak response of <10 microgram per liter (mcg/L) with 2 GH stimulation tests, without priming with oestradiol.
B) Turner syndrome: documented pre-established diagnosis by karyotype.
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Medical Responsible | Merck KGaA, Darmstadt, Germany | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Local Medical Information Office | Buenos Aires | Argentina | ||||
| Local Medical Information Office |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 23567489 | Result | Stevens A, Clayton P, Tato L, Yoo HW, Rodriguez-Arnao MD, Skorodok J, Ambler GR, Zignani M, Zieschang J, Della Corte G, Destenaves B, Champigneulle A, Raelson J, Chatelain P. Pharmacogenomics of insulin-like growth factor-I generation during GH treatment in children with GH deficiency or Turner syndrome. Pharmacogenomics J. 2014 Feb;14(1):54-62. doi: 10.1038/tpj.2013.14. Epub 2013 Apr 9. | |
| 34045667 |
| Label | URL |
|---|---|
| Full FDA approved prescribing information can be found here | View source |
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A total of 319 subjects were screened for this trial. Only 1 subject withdrew from the study prior to receiving the treatment due to personal reasons. Overall, 318 subjects were enrolled into the study.
First informed consent date: May 2005. Clinical data cutoff date: Oct 2007, Study completion date: Sep 2007.
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| ID | Title | Description |
|---|---|---|
| FG000 | Turner Syndrome (TS) | Subjects with TS were administered with SAIZEN® as subcutaneous injection at a dose of 0.050 milligram per kilogram (mg/kg) of body weight per day (within the recommended dosage 0.045-0.050 mg/kg body weight) for a period of 1 month. |
| FG001 | Growth Hormone Deficiency (GHD) |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Saizen | Drug | Subjects with GHD will receive SAIZEN® as subcutaneous injection at a dose of 0.035 mg/kg of body weight per day (within the recommended dosage 0.025-0.035 mg/kg body weight) for a period of 1 month. |
|
| Change From Baseline in Fasting Insulin Levels at Month 1 | Baseline, Month 1 |
| Change From Baseline in Homeostasis Model Assessment of Insulin Resistance (HOMA-IR) at Month 1 | HOMA-IR is used to assess insulin resistance and calculated by an empirical mathematical formula based on fasting plasma glucose and fasting plasma insulin levels. HOMA-IR = fasting plasma insulin (picomole/liter [pmol/L]) * fasting plasma glucose (millimole/liter [mmol/L]) divided by 22.5. | Baseline, Month 1 |
| Change From Baseline in Bone Alkaline Phosphatase Levels at Month 1 | Baseline, Month 1 |
| Sydney |
| Australia |
| Local Medical Information Office | Vienna | Austria |
| Local Medical Information Office | Mississauga | Canada |
| Local Medical InformationOffice | Paris | France |
| Local Medical Information Office | Munich | Germany |
| Local Medical Information Office | Rome | Italy |
| Local Medical Information Office | Oslo | Norway |
| Local Medical Information Office | Russia | Russia |
| Local Medical Information Office | Singapore | Singapore |
| Local Medical Information Office | Madrid | Spain |
| Local Medical Information Office | Stockholm | Sweden |
| Local Medical Information Office | Feltham | United Kingdom |
| Derived |
| Stevens A, Murray P, De Leonibus C, Garner T, Koledova E, Ambler G, Kapelari K, Binder G, Maghnie M, Zucchini S, Bashnina E, Skorodok J, Yeste D, Belgorosky A, Siguero JL, Coutant R, Vangsoy-Hansen E, Hagenas L, Dahlgren J, Deal C, Chatelain P, Clayton P. Gene expression signatures predict response to therapy with growth hormone. Pharmacogenomics J. 2021 Oct;21(5):594-607. doi: 10.1038/s41397-021-00237-5. Epub 2021 May 27. |
| 29618660 | Derived | Murray PG, Stevens A, De Leonibus C, Koledova E, Chatelain P, Clayton PE. Transcriptomics and machine learning predict diagnosis and severity of growth hormone deficiency. JCI Insight. 2018 Apr 5;3(7):e93247. doi: 10.1172/jci.insight.93247. eCollection 2018 Apr 5. |
| 26340968 | Derived | Valsesia A, Chatelain P, Stevens A, Peterkova VA, Belgorosky A, Maghnie M, Antoniazzi F, Koledova E, Wojcik J, Farmer P, Destenaves B, Clayton P; PREDICT Investigator group. GH deficiency status combined with GH receptor polymorphism affects response to GH in children. Eur J Endocrinol. 2015 Dec;173(6):777-89. doi: 10.1530/EJE-15-0474. Epub 2015 Sep 4. |
Subjects with GHD were administered with SAIZEN® as subcutaneous injection at a dose of 0.035 mg/kg of body weight per day (within the recommended dosage 0.025-0.035 mg/kg body weight) for a period of 1 month. |
| COMPLETED |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | Turner Syndrome (TS) | Subjects with TS were administered with SAIZEN® as subcutaneous injection at a dose of 0.050 milligram per kilogram (mg/kg) of body weight per day (within the recommended dosage 0.045-0.050 mg/kg body weight) for a period of 1 month. |
| BG001 | Growth Hormone Deficiency (GHD) | Subjects with GHD were administered with SAIZEN® as subcutaneous injection at a dose of 0.035 mg/kg of body weight per day (within the recommended dosage 0.025-0.035 mg/kg body weight) for a period of 1 month. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
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| 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 Insulin Like Growth Factor-1 Standard Deviation Score (IGF-1 SDS) at Month 1 | IGF-1 SDS was calculated using the Elmlinger reference method. Change in within subject IGF-1 levels (standard deviation scores) at Month 1 from Baseline was assessed. Descriptive statistics were determined for the Baseline and Month 1 assessments, and also for the level of change between these two assessments. If either the Baseline or Month 1 IGF-1 level was missing, then the within-subject change in IGF-1 was assumed to be missing. | The Intention to Treat (ITT) population included all subjects who received at least 1 dose of study medication. Here "Overall Number of Subjects Analyzed" signifies those subjects who were evaluable for this outcome measure. | Posted | Mean | Standard Deviation | Standard deviation score (SDS) | Baseline, Month 1 |
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| Secondary | Change From Baseline in Insulin-like Growth Factor Binding Protein - 3 (IGFBP-3) Level at Month 1 | The ITT population included all subjects who received at least 1 dose of study medication. Here "Overall Number of Subjects Analyzed" signifies those subjects who were evaluable for this outcome measure. | Posted | Mean | Standard Deviation | milligram per liter (mg/L) | Baseline, Month 1 |
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| Secondary | Change From Baseline in Fasting Glucose Levels at Month 1 | The ITT population included all subjects who received at least 1 dose of study medication. Here "Overall Number of Subjects Analyzed" signifies those subjects who were evaluable for this outcome measure. | Posted | Mean | Standard Deviation | millimoles per liter (mmol/L) | Baseline, Month 1 |
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| Secondary | Change From Baseline in Fasting Insulin Levels at Month 1 | The ITT population included all subjects who received at least 1 dose of study medication. Here "Overall Number of Subjects Analyzed" signifies those subjects who were evaluable for this outcome measure. | Posted | Mean | Standard Deviation | picomole per liter (pmol/L) | Baseline, Month 1 |
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| Secondary | Change From Baseline in Homeostasis Model Assessment of Insulin Resistance (HOMA-IR) at Month 1 | HOMA-IR is used to assess insulin resistance and calculated by an empirical mathematical formula based on fasting plasma glucose and fasting plasma insulin levels. HOMA-IR = fasting plasma insulin (picomole/liter [pmol/L]) * fasting plasma glucose (millimole/liter [mmol/L]) divided by 22.5. | The ITT population included all subjects who received at least 1 dose of study medication. Here "Overall Number of Subjects Analyzed" signifies those subjects who were evaluable for this outcome measure. | Posted | Mean | Standard Deviation | picomole per liter *millimole per liter | Baseline, Month 1 |
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| Secondary | Change From Baseline in Bone Alkaline Phosphatase Levels at Month 1 | The ITT population included all subjects who received at least 1 dose of study medication. Here "Overall Number of Subjects Analyzed" signifies those subjects who were evaluable for this outcome measure. | Posted | Mean | Standard Deviation | Units per liter (U/L) | Baseline, Month 1 |
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Adverse events were captured from first dose until at least 4 weeks following the last SAIZEN® administration or the post-treatment visit, whichever represented the longer period (up to a maximum of 2 months).
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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 | Turner Syndrome (TS) | Subjects with TS were administered with SAIZEN® as subcutaneous injection at a dose of 0.050 milligram per kilogram (mg/kg) of body weight per day (within the recommended dosage 0.045-0.050 mg/kg body weight) for a period of 1 month. | 0 | 149 | 36 | 149 | ||
| EG001 | Growth Hormone Deficiency (GHD) | Subjects with GHD were administered with SAIZEN® as subcutaneous injection at a dose of 0.035 mg/kg of body weight per day (within the recommended dosage 0.025-0.035 mg/kg body weight) for a period of 1 month. | 1 | 169 | 51 | 169 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Tonsillitis streptococcal | Infections and infestations | MedDRA (11.0) | Non-systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Seasonal allergy | Immune system disorders | MedDRA (11.0) | Non-systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (11.0) | Non-systematic Assessment |
| |
| Injection site haemorrhage | General disorders | MedDRA (11.0) | Non-systematic Assessment |
| |
| Injection site irritation | General disorders | MedDRA (11.0) | Non-systematic Assessment |
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| Fatigue | General disorders | MedDRA (11.0) | Non-systematic Assessment |
| |
| Influenza like illness | General disorders | MedDRA (11.0) | Non-systematic Assessment |
| |
| Injection site anaesthesia | General disorders | MedDRA (11.0) | Non-systematic Assessment |
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| Affect lability | Psychiatric disorders | MedDRA (11.0) | Non-systematic Assessment |
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| Blood thyroid stimulating hormone increased | Investigations | MedDRA (11.0) | Non-systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (11.0) | Non-systematic Assessment |
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| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA (11.0) | Non-systematic Assessment |
| |
| Pharyngolaryngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (11.0) | Non-systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA (11.0) | Non-systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (11.0) | Non-systematic Assessment |
| |
| Rhinitis allergic | Respiratory, thoracic and mediastinal disorders | MedDRA (11.0) | Non-systematic Assessment |
| |
| Throat irritation | Respiratory, thoracic and mediastinal disorders | MedDRA (11.0) | Non-systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (11.0) | Non-systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA (11.0) | Non-systematic Assessment |
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| Somnolence | Nervous system disorders | MedDRA (11.0) | Non-systematic Assessment |
| |
| Conjunctivitis allergic | Eye disorders | MedDRA (11.0) | Non-systematic Assessment |
| |
| Vertigo | Ear and labyrinth disorders | MedDRA (11.0) | Non-systematic Assessment |
| |
| Ear pain | Ear and labyrinth disorders | MedDRA (11.0) | Non-systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (11.0) | Non-systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (11.0) | Non-systematic Assessment |
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| Abdominal pain | Gastrointestinal disorders | MedDRA (11.0) | Non-systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (11.0) | Non-systematic Assessment |
| |
| Enteritis | Gastrointestinal disorders | MedDRA (11.0) | Non-systematic Assessment |
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| Flatulence | Gastrointestinal disorders | MedDRA (11.0) | Non-systematic Assessment |
| |
| Acne | Skin and subcutaneous tissue disorders | MedDRA (11.0) | Non-systematic Assessment |
| |
| Dermatitis atopic | Skin and subcutaneous tissue disorders | MedDRA (11.0) | Non-systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA (11.0) | Non-systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (11.0) | Non-systematic Assessment |
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| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (11.0) | Non-systematic Assessment |
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| Bone pain | Musculoskeletal and connective tissue disorders | MedDRA (11.0) | Non-systematic Assessment |
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| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA (11.0) | Non-systematic Assessment |
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| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (11.0) | Non-systematic Assessment |
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| Precocious puberty | Endocrine disorders | MedDRA (11.0) | Non-systematic Assessment |
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| Nasopharyngitis | Infections and infestations | MedDRA (11.0) | Non-systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (11.0) | Non-systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA (11.0) | Non-systematic Assessment |
| |
| Ear infection | Infections and infestations | MedDRA (11.0) | Non-systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA (11.0) | Non-systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA (11.0) | Non-systematic Assessment |
| |
| Tonsillitis | Infections and infestations | MedDRA (11.0) | Non-systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA (11.0) | Non-systematic Assessment |
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| Bronchitis acute | Infections and infestations | MedDRA (11.0) | Non-systematic Assessment |
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| Conjunctivitis viral | Infections and infestations | MedDRA (11.0) | Non-systematic Assessment |
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| Enterocolitis infectious | Infections and infestations | MedDRA (11.0) | Non-systematic Assessment |
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| Otitis externa | Infections and infestations | MedDRA (11.0) | Non-systematic Assessment |
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| Skin infection | Infections and infestations | MedDRA (11.0) | Non-systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA (11.0) | Non-systematic Assessment |
|
Limitations of the trial were short duration of the study treatment, and relatively small sample size
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Merck KGaA Communication Center | Merck Healthcare, a business of Merck KGaA, Darmstadt, Germany | +49-6151-72-5200 | service@merckgroup.com |
| ID | Term |
|---|---|
| D004393 | Dwarfism, Pituitary |
| D014424 | Turner Syndrome |
| ID | Term |
|---|---|
| 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 |
| D004700 | Endocrine System Diseases |
| 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 |
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| ID | Term |
|---|---|
| D019382 | Human Growth Hormone |
| ID | Term |
|---|---|
| D013006 | Growth Hormone |
| D010908 | Pituitary Hormones, Anterior |
| D010907 | Pituitary Hormones |
| D036361 | Peptide Hormones |
| D006728 | Hormones |
| D006730 | Hormones, Hormone Substitutes, and Hormone Antagonists |
| D010455 | Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |
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