A Phase IV, Open Label, Multicenter, Case-controlled Study of Growth in Patients Using the Nutropin AQ® Nuspin®
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 入组人数
- 18
- 主要终点
- Annualized Height Velocity in Response to rhGH Treatment Using the NuSpin Device After Two Years of Treatment
研究概览
简要总结
This is a Phase IV, open label, case-controlled, observational study to evaluate the age-adjusted cumulative height velocity in pre-pubertal isolated growth hormone deficient (IGHD) and idiopathic short stature (ISS) participants receiving daily doses of somatropin (recombinant human growth hormone [rhGH]; Nutropin) using NuSpin device compared to historical controls from the national cooperative growth study (NCGS).
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 2 Years 至 14 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Bone age less than or equal to (</=) 8 years (females) or </= 10 years (males) as determined by x-ray of the left hand and wrist obtained within six months before enrollment
- •Prepubertal males and females by physical exam
- •Naive to rhGH therapy
- •Diagnosis of IGHD or ISS by standard pharmacologic testing and no other discernable etiology for short stature
- •Height standard deviation score (Ht SDS) </= -1.5 (</= 5th percentile) for IGHD participants; Ht SDS </= -2.25 (</= 1.2 percentile) for ISS participants
排除标准
- •Short stature etiologies other than IGHD or ISS
- •Participants receiving chronic corticosteroid therapy (greater than [>] 3 months) for other medical conditions
- •Participants with active malignancy or any other condition that the investigator believes would pose a significant hazard to the participant if rhGH were initiated
- •Females with turner syndrome
- •Any previous rhGH treatment
- •Participation in another simultaneous medical investigation or trial
- •Pediatric participants with closed epiphyses
- •Participants prescribed rhGH doses outside the variance of NCGS control participant dosing
研究组 & 干预措施
Cohort 1: IGHD participants
Isolated growth hormone deficient (IGHD) participants who initiated somatropin (Deoxyribonucleic acid [DNA] origin) (recombinant human growth hormone [rhGH]) using the NuSpin device, will be observed for at least 2 years. The choice of initiation of NuSpin treatment and the treatment regimen will be as per treating physician's discretion, the study protocol does not enforce or specify any treatment regimen.
干预措施: NuSpin (Device)
Cohort 1: IGHD participants
Isolated growth hormone deficient (IGHD) participants who initiated somatropin (Deoxyribonucleic acid [DNA] origin) (recombinant human growth hormone [rhGH]) using the NuSpin device, will be observed for at least 2 years. The choice of initiation of NuSpin treatment and the treatment regimen will be as per treating physician's discretion, the study protocol does not enforce or specify any treatment regimen.
干预措施: Somatropin (Drug)
Cohort 2: ISS participants
Idiopathic short stature (ISS) participants who initiated somatropin (DNA origin) (rhGH) using the NuSpin device, will be observed for at least 2 years. The choice of initiation of NuSpin treatment and the treatment regimen will be as per treating physician's discretion, the study protocol does not enforce or specify any treatment regimen.
干预措施: NuSpin (Device)
Cohort 2: ISS participants
Idiopathic short stature (ISS) participants who initiated somatropin (DNA origin) (rhGH) using the NuSpin device, will be observed for at least 2 years. The choice of initiation of NuSpin treatment and the treatment regimen will be as per treating physician's discretion, the study protocol does not enforce or specify any treatment regimen.
干预措施: Somatropin (Drug)
结局指标
主要结局
Annualized Height Velocity in Response to rhGH Treatment Using the NuSpin Device After Two Years of Treatment
时间窗: Baseline up to Month 24 (Year 2)
The annualized height velocity (cm per year \[cm/yr\]) over 2 years was calculated as: \[(height in cm at t24 minus (-) height in cm at t0) divided by (date at t24 - date at t0)\] multiplied by 365.25, where, t0 is the baseline measurement visit and t24 is the 2-year measurement visit.
次要结局
- Annualized Height Velocity in Response to rhGH Treatment Using the NuSpin Device for First Year of Treatment(Baseline up to Month 12 (Year 1))
- Annualized Height Velocity in Response to rhGH Treatment Using the NuSpin Device During the Second Year of Therapy(Month 12 to Month 24 (Year 1 to Year 2))