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The objective of this study is to evaluate the efficacy and safety of recombinant human growth hormone (r-hGH) treatment in girls with Turner Syndrome under the age of 4 years. After 4 years of treatment, height in these girls will be compared with an historical control group of untreated girls with Turner Syndrome, matched for age and height at baseline.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| r-hGH | Experimental | Participants (girls) will receive r-hGH as a subcutaneous injection administered by a parent in the evening. During Years 1-2, the dose of r-hGH received will depend on participants' baseline height standard deviation score (SDS) relative to the general population standard: participants with a height SDS of -2 standard deviation (SD) or lower will receive 0.05 milligrams per kilogram (mg/kg) per day r-hGH and those with a height SDS between -1 and -2 SD will receive 0.035 mg/kg per day r-hGH. After 2 years of treatment, all participants will receive a fixed dose of 0.05 mg/kg per day for a further 2 years. |
|
| Historical Control | No Intervention | This arm will include matching (age and height) historical control participants (girls) with turner syndrome, who were born between 1961 and 1990 and were untreated. |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| r-hGH | Drug | Subcutaneous administration. |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| Height SDS at Year 4 | 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. | Year 4 |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Abnormal Glycated Hemoglobin (HbA1c) Levels | HbA1c develops when hemoglobin, a protein within red blood cells that carries oxygen throughout the body, joins with glucose in the blood, becoming glycated. The higher the level of glucose in the blood, the higher the level of HbA1c is detectable on red blood cells. The normal range for HbA1c is 4 percent (%) to 5.9%. Number of participants, who had abnormal HbA1c levels any time during the assessment, were reported. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Medical Responsible | Merck KGaA, Darmstadt, Germany | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Hôpital Robert Debré | Paris | 75935 | France |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 21398400 | Result | Linglart A, Cabrol S, Berlier P, Stuckens C, Wagner K, de Kerdanet M, Limoni C, Carel JC, Chaussain JL; French Collaborative Young Turner Study Group. Growth hormone treatment before the age of 4 years prevents short stature in young girls with Turner syndrome. Eur J Endocrinol. 2011 Jun;164(6):891-7. doi: 10.1530/EJE-10-1048. Epub 2011 Mar 11. |
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| ID | Title | Description |
|---|---|---|
| FG000 | Recombinant Human Growth Hormone (r-hGH) | Participants (girls) received r-hGH (Saizen®) as a subcutaneous injection administered by a parent in the evening. During Years 1-2, the dose of r-hGH received depended on participants' baseline height standard deviation score (SDS) relative to the general population standard: participants with a height SDS of -2 standard deviation (SD) or lower received 0.05 milligrams per kilogram (mg/kg) per day r-hGH and those with a height SDS between -1 and -2 SD received 0.035 mg/kg per day r-hGH. After 2 years of treatment, all participants received a fixed dose of 0.05 mg/kg per day for a further 2 years. |
| FG001 | Historical Control | This arm included matching (age and height) historical control participants (girls) with turner syndrome, who were born between 1961 and 1990 and were untreated. |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
All Participants.
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| ID | Title | Description |
|---|---|---|
| BG000 | r-hGH | Participants (girls) received r-hGH as a subcutaneous injection administered by a parent in the evening. During Years 1-2, the dose of r-hGH received depended on participants' baseline height SDS relative to the general population standard: participants with a height SDS of -2 SD or lower received 0.05 mg/kg per day r-hGH and those with a height SDS between -1 and -2 SD received 0.035 mg/kg per day r-hGH. After 2 years of treatment, all participants received a fixed dose of 0.05 mg/kg per day for a further 2 years. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| 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 | Height SDS at Year 4 | 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. | All treated participants from r-hGH arm and all participants from Historical Control arm. | Posted | Mean | Standard Deviation | standard deviation score | Year 4 |
|
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Adverse events were collected only from participants treated with r-hGH. No adverse event data collection occurred in Historical Control arm.
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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 | r-hGH | Participants (girls) received r-hGH as a subcutaneous injection administered by a parent in the evening. During Years 1-2, the dose of r-hGH received depended on participants' baseline height SDS relative to the general population standard: participants with a height SDS of -2 SD or lower received 0.05 mg/kg per day r-hGH and those with a height SDS between -1 and -2 SD received 0.035 mg/kg per day r-hGH. After 2 years of treatment, all participants received a fixed dose of 0.05 mg/kg per day for a further 2 years. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Urethral reflux | Vascular disorders | No Coding Applied | Non-systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Infection | Infections and infestations | No Coding Applied | Non-systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Merck KGaA Communication Center, | Merck Healthcare, a business of Merck KGaA, Darmstadt, Germany | 496151725200 | service@merckgroup.com |
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| ID | Term |
|---|---|
| D014424 | Turner Syndrome |
| ID | Term |
|---|---|
| D006059 | Gonadal Dysgenesis |
| D012734 | Disorders of Sex Development |
| D014564 | Urogenital Abnormalities |
| D052776 | Female Urogenital Diseases |
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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 |
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| Baseline up to Year 2 |
| Difference Between Bone Age (BA) and Chronological Age (CA) (BA-CA) | BA was determined using left wrist and hand X-ray. CA was determined using the date of birth. Difference of BA and CA (BA-CA) was reported. | Baseline, Year 1, Year 2 |
| Number of Participants With Anti r-hGH Antibodies | Baseline up to Year 2 |
| Number of Participants With Abnormal Insulin-Like Growth Factor 1 (IGF1) Levels | The normal range for IGF1 levels is 45 to 117 nanogram per milliliter (ng/mL) for girls aged less than (<) 3 years and 80 to 236 ng/mL for girls aged 3 to 6 years. Values outside the normal range were considered abnormal. Number of participants, who had abnormal IGF1 levels any time during the assessment, were reported. | Baseline up to Year 2 |
| Number of Participants Who Reached Normal Height at Year 4 | Participants with normal height were those who attained a height which was within +/- 2 height SDS of reference population standard. 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. | Year 4 |
| BG001 | Historical Control | This arm included matching (age and height) historical control participants (girls) with turner syndrome, who were born between 1961 and 1990 and were untreated. |
| BG002 | Total | Total of all reporting groups |
| Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| OG001 | Historical Control | This arm included matching (age and height) historical control participants (girls) with turner syndrome, who were born between 1961 and 1990 and were untreated. |
|
|
| Secondary | Number of Participants With Abnormal Glycated Hemoglobin (HbA1c) Levels | HbA1c develops when hemoglobin, a protein within red blood cells that carries oxygen throughout the body, joins with glucose in the blood, becoming glycated. The higher the level of glucose in the blood, the higher the level of HbA1c is detectable on red blood cells. The normal range for HbA1c is 4 percent (%) to 5.9%. Number of participants, who had abnormal HbA1c levels any time during the assessment, were reported. | Posted | Number | participants | Baseline up to Year 2 |
|
|
|
| Secondary | Difference Between Bone Age (BA) and Chronological Age (CA) (BA-CA) | BA was determined using left wrist and hand X-ray. CA was determined using the date of birth. Difference of BA and CA (BA-CA) was reported. | All treated participants from r-hGH arm. This outcome measure was only planned in the r-hGH arm. Overall number of participants analyzed = participants with available data for this outcome; number analyzed = participants with available data for this outcome at specified timepoint. | Posted | Mean | Standard Deviation | months | Baseline, Year 1, Year 2 |
|
|
|
| Secondary | Number of Participants With Anti r-hGH Antibodies | Data for this outcome was not collected from any participant. | Posted | Baseline up to Year 2 |
|
|
| Secondary | Number of Participants With Abnormal Insulin-Like Growth Factor 1 (IGF1) Levels | The normal range for IGF1 levels is 45 to 117 nanogram per milliliter (ng/mL) for girls aged less than (<) 3 years and 80 to 236 ng/mL for girls aged 3 to 6 years. Values outside the normal range were considered abnormal. Number of participants, who had abnormal IGF1 levels any time during the assessment, were reported. | All treated participants from r-hGH arm. This outcome measure was only planned in the r-hGH arm. | Posted | Number | participants | Baseline up to Year 2 |
|
|
|
| Secondary | Number of Participants Who Reached Normal Height at Year 4 | Participants with normal height were those who attained a height which was within +/- 2 height SDS of reference population standard. 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. | All treated participants from r-hGH arm. This outcome measure was only planned in the r-hGH arm. | Posted | Number | participants | Year 4 |
|
|
|
| 8 |
| 61 |
| 47 |
| 61 |
| Trauma leading to scalp surgery | Surgical and medical procedures | No Coding Applied | Non-systematic Assessment |
|
| Measles | Skin and subcutaneous tissue disorders | No Coding Applied | Non-systematic Assessment |
|
| Serious otitis | Nervous system disorders | No Coding Applied | Non-systematic Assessment |
|
| Immunoglobulin G4 deficiency | Immune system disorders | No Coding Applied | Non-systematic Assessment |
|
| Arterial hypertension | Respiratory, thoracic and mediastinal disorders | No Coding Applied | Non-systematic Assessment |
|
| Chronic persistent cytolysis | Congenital, familial and genetic disorders | No Coding Applied | Non-systematic Assessment |
|
| Surgery to remove large mass (meningocele/benign cyst) | Surgical and medical procedures | No Coding Applied | Non-systematic Assessment |
|
| Transtympanic drainage tube placement | Surgical and medical procedures | No Coding Applied | Non-systematic Assessment |
|
| Tonsillectomy/adenoidectomy | Surgical and medical procedures | No Coding Applied | Non-systematic Assessment |
|
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| 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 |
| D006728 |
| Hormones |
| D006730 | Hormones, Hormone Substitutes, and Hormone Antagonists |
| D010455 | Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |
|
| Year 2 |
|
|