Humatrope Treatment to Final Height in Turner's Syndrome
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 154
- 试验地点
- 1
- 主要终点
- Height Standard Deviation Score (SDS) (National Center for Health Statistics [NCHS]), Change From Baseline to Last Measurement, As Randomized Population
研究概览
简要总结
A randomized, controlled trial in girls with Turner syndrome at least 7 years old and younger than 13 at study entry, to determine the efficacy and safety of Humatrope (somatropin) treatment in promoting linear growth to final height.
详细描述
A randomized, controlled trial of Humatrope (somatropin) treatment in girls with Turner syndrome at least 7 years old and younger than 13 at study entry.
Core study objectives are to determine the efficacy of Humatrope in promoting linear growth to final height in girls with Turner syndrome, and to assess the safety of this treatment. Core study completion criteria (protocol final height) are that the patient has both a height velocity < 2 cm per year and a bone age of 14 years or greater.
Addendum 1 provides the option of Humatrope treatment to patients who were randomized to the Control arm of the Core study and who discontinued from the study on or after December 19, 1997.
Addendum 2 objectives are: 1) to collect true final height data; 2) to evaluate hearing, tympanic membrane function and other specific areas of interest with respect to the safety of growth hormone therapy in Turner syndrome; 3) to evaluate pancreatic beta cell function (glucose metabolism) in patients previously enrolled in the Core study.
Addendum 3 objective is to determine the parental origin of the retained X chromosome of an appropriate subset of patients currently or previously enrolled in the Core study, and to determine whether this parental origin holds any predictive value for spontaneous growth or for response to growth hormone therapy.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 7 Years 至 13 Years（Child）
- 性别
- Female
- 接受健康志愿者
- 否
入选标准
- •girl with Turner syndrome
- •prepubertal, Tanner stage I breast
- •height velocity less than 6 cm/year and height less than or equal to the tenth percentile for sex and age in general population
- •at least 6 months (preferably 12 months) of accurate height measurements available for calculation of pre-study height velocity
- •if thyroxine deficient, to have received replacement therapy, and for six months prior to enrollment have had normal thyroid function tests
排除标准
- •prior treatment with growth hormone
- •presence of a Y component in karyotype with gonads in situ
- •diabetes mellitus
研究组 & 干预措施
Control
Control arm; untreated with Humatrope. Ethinyl estradiol (escalating doses to 20 mcg daily) after age 13, and medroxyprogesterone acetate (10 mg tablets ten days monthly) after age 15. Subject continues until Core study completion criteria are met (protocol final height).
干预措施: Ethinyl estradiol (Drug)
Control
Control arm; untreated with Humatrope. Ethinyl estradiol (escalating doses to 20 mcg daily) after age 13, and medroxyprogesterone acetate (10 mg tablets ten days monthly) after age 15. Subject continues until Core study completion criteria are met (protocol final height).
干预措施: Medroxyprogesterone acetate (Drug)
Humatrope
Humatrope (0.05 mg/kg/dose) by subcutaneous injection 6 times per week. Ethinyl estradiol (escalating doses to 20 mcg daily) after age 13, and medroxyprogesterone acetate (10 mg tablets ten days monthly) after age 15. Subject continues until Core study completion criteria are met (protocol final height).
干预措施: Somatropin (Drug)
Humatrope
Humatrope (0.05 mg/kg/dose) by subcutaneous injection 6 times per week. Ethinyl estradiol (escalating doses to 20 mcg daily) after age 13, and medroxyprogesterone acetate (10 mg tablets ten days monthly) after age 15. Subject continues until Core study completion criteria are met (protocol final height).
干预措施: Ethinyl estradiol (Drug)
Humatrope
Humatrope (0.05 mg/kg/dose) by subcutaneous injection 6 times per week. Ethinyl estradiol (escalating doses to 20 mcg daily) after age 13, and medroxyprogesterone acetate (10 mg tablets ten days monthly) after age 15. Subject continues until Core study completion criteria are met (protocol final height).
干预措施: Medroxyprogesterone acetate (Drug)
结局指标
主要结局
Height Standard Deviation Score (SDS) (National Center for Health Statistics [NCHS]), Change From Baseline to Last Measurement, As Randomized Population
时间窗: Baseline, and end of 4-year addendum
Value analyzed is change from baseline to the most mature height measurement available. The terms Standard Deviation Score (SDS) and National Center for Health Statistics (NCHS) were defined in baseline characteristics. Greater height SDS values indicate greater height; positive values of change from baseline indicate increased height.
Height Standard Deviation Score (SDS) (National Center for Health Statistics [NCHS]), Last Measurement After Attainment of Final Height
时间窗: at completion of core study, or at end of 4-year addendum
SDS report the number of standard deviations from the mean for age and sex for an individual measurement (normal range: -2 to +2 SDS). Height SDS \[NCHS\] uses the NCHS US general female population reference height values for age (Kuczmarski RJ et al. 2000) as the population mean and standard deviation. Calculation of Height SDS is provided in Height SDS \[Lyon\] description (Baseline). Since data reported by Kuczmarski RJ et al provides US general female population standards, values of Height SDS \[NCHS\] for untreated patients with Turner syndrome tend to be below zero e.g, -2.0 to -4.0 SDS.
次要结局
- Height Standard Deviation Score (SDS) (National Center for Health Statistics [NCHS]), Change From Baseline, As-Treated Population(every 3 months during core study, and at start and end of 4-year addendum)
- Height (Centimeters [cm])(every 3 months during core study, and at start and end of 4-year addendum)
- Number of Participants With an Abnormal Pure Tone Audiometry, Audiologist Assessment(at completion of core study or beginning of addendum)
- Number of Participants With Abnormal Speech Audiometry, Audiologist Assessment(at completion of core study or beginning of addendum)
- Number of Participants With Abnormal Impedance Tympanometry, Audiologist Assessment(at completion of core study or beginning of addendum)
- Number of Participants With Hearing Loss, Audiologist Assessment(at completion of core study or beginning of addendum)
- Fasting Glucose, Change From Baseline(At core study baseline, and at end of 4-year addendum)
- Maximum Fasting Glucose Value(At start and through end of 4-year addendum (up to an additional 2 years))
- Number of Participants With Any Abnormal Fasting Glucose Value(At start and through end of 4-year addendum)
- Maximum Fasting Insulin Values(At start and through end of 4-year addendum (up to an additional 2 years))
- Number of Participants With Any Abnormal Fasting Insulin Value(At start and through end of 4-year addendum)
- Minimum Fasting Glucose/Insulin Ratio Values(At start and through end of 4-year addendum (up to an additional 2 years))
- Number of Participants With Any Abnormal Fasting Glucose/Insulin Ratio Value(At start and through end of 4-year addendum)
- Glycosylated Hemoglobin, Change From Baseline(At core study baseline, and at end of 4-year addendum)
- Maximum Glycosylated Hemoglobin(At start and through end of 4-year addendum (up to an additional 2 years))
- Number of Participants With Any Abnormal Glycosylated Hemoglobin (HbA1c) Value(At start and through end of 4-year addendum)