Recombinant Human Growth Hormone (rhGH) and Recombinant Human Insulin-like Growth Factor-1 rhIGF-1) Combination Therapy in Children With Short Stature Associated With IGF-1 Deficiency: A Six-year, Randomized, Multi-center, Open-label, Parallel-group, Active Treatment Controlled, Dose Selection Trial
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 发起方
- Ipsen
- 入组人数
- 106
- 试验地点
- 1
- 主要终点
- Height Velocity
研究概览
简要总结
IGF-1 (insulin-like growth factor-1) is a hormone that is normally produced in the body in response to another hormone called growth hormone. Growth Hormone is produced by a small gland at the base of the brain (the pituitary). Together IGF-1 and GH are large contributors to growth during infancy, childhood, and adolescence.
Children with IGF Deficiency are short and have an imbalance in the levels of growth hormone and IGF-1 that their body produces. Their growth hormone levels are normal or even high, but IGF-1 levels do not increase normally in response to growth hormone. As a result, they have a type of growth hormone insensitivity and an inability to grow normally.
This study is a test to see whether daily dosing with a combination of rhIGF-1 and rhGH will help children with IGFD grow taller more quickly than children treated with rhGH alone. The study medications, rhIGF-1 and rhGH, are approved by the US Food and Drug Administration (FDA) for use in some growth disorders in children, but the combination of rhIGF-1 and rhGH in children with IGF-1 deficiency (IGFD) is investigational.
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
- 5 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Parents or legally authorized representatives must give signed informed consent before any trial-related activities
- •IGF-1 SDS of ≤ -1 for age and gender
- •Short stature, as defined by a height SDS of ≤ -2 for age and gender
- •Chronological age ≥ 5 years
- •Bone age ≤ 11 years in boys and ≤ 9 years in girls
- •GH sufficiency, defined as a maximal stimulated GH response of greater than or equal to 10 ng/mL at Visit 2 (note: upon approval of the Medical Monitor, the result of a prior GH stimulation test may satisfy this requirement).
- •Prepubertal status
- •Adequate nutrition as evidenced by a body mass index (BMI) greater than or equal to the 5th percentile for age and gender
排除标准
- •Severe Primary IGFD (defined as height and IGF-1 SDS ≤ 3, and stimulated GH response greater than or equal to 10 ng/mL)
- •Prior or current use of medications with the potential to alter growth patterns including GH, IGF-1, IGFBP-3, gonadotrophin agonists (e.g., Lupron), aromatase inhibitors, androgens and estrogens
- •Known or suspected allergy to rhGH, rhIGF-1 or a constituent of their formulations
- •Current use of medications for attention deficit disorder
- •A chronic health condition that requires anti-inflammatory steroids or daily medication unless approved by the Medical Monitor
研究组 & 干预措施
1. rhGH Alone
干预措施: NutropinAq® (Somatropin [rDNA origin]) (Drug)
2. Combination Dose
干预措施: Increlex® (Mecasermin [rDNA origin] injection) + NutropinAq® (Somatropin [rDNA origin]) (Drug)
3. Combination Dose
干预措施: Increlex® (Mecasermin [rDNA origin] injection) + NutropinAq® (Somatropin [rDNA origin]) (Drug)
4. Combination Dose
干预措施: Increlex® (Mecasermin [rDNA origin] injection) + NutropinAq® (Somatropin [rDNA origin]) (Drug)
结局指标
主要结局
Height Velocity
时间窗: First year of treatment
次要结局
- Height Velocity(Second, third and fourth year)
- Cumulative Change in Height Standard Deviation Score (SDS)(First, second, third and fourth year)
- Predicted Adult Height (PAH)(At baseline (Day 1), year 1,2,3 and 4)
- Changes From Baseline (Day 1) in Serum Concentrations of Insulin-Like Growth Factor Binding Protein-3 (IGFPB-3)(At Baseline (Day 1), Year 1,2,3 and 4)
- Total Change From Baseline (Day 1) in BMI SDS(At year 1,2,3,4 and end of study (visit 23) versus baseline (day 1))
- Skeletal Maturation(At baseline(day 1), year 1,2,3 and 4)
- Changes From Baseline (Day 1) in Serum Concentrations of Growth Hormone (GH)(At Baseline (Day 1), Year 1,2,3 and 4)
- Changes From Baseline (Day 1) in Serum Concentrations of Growth Hormone Binding Protein (GHBP)(At Baseline (Day 1), Year 1,2,3 and 4)
- Changes From Baseline (Day 1) in Serum Concentrations of Insulin-Like Growth Factor Binding Protein-1 (IGFBP-1)(At Baseline (Day 1), Year 1,2,3 and 4)
- Changes From Baseline (Day 1) in Serum Concentrations of Insulin-Like Growth Factor-1 (IGF-1)(At Baseline (Day 1), Year 1,2,3 and 4)
- Changes From Baseline (Day 1) in Serum Concentrations of Acid-Labile Subunit (ALS)(At Baseline (Day 1), Year 1,2,3 and 4)
- Summary of Adverse Events With Number of Occurrences(Approximately up to 4 years.)