Safety and Dose Finding Study of Different MOD-4023 Dose Levels Compared to Daily R-human Growth Hormone (hGH) Therapy in Pre-pubertal Growth Hormone Deficient Children
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 48
- 试验地点
- 13
- 主要终点
- Annualized Height Velocity
研究概览
简要总结
Protocol CP-4-004-OLE (Open Label Extension) is designed as a long-term, open-label extension using single patient use, multi-dose, disposable pre-filled pen.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 3 Years 至 11 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients who completed the first year of treatment in the main study are allowed to enter the (Long Term) Open Label Extension (OLE) study.
排除标准
- •Children with past or present intracranial tumor growth as confirmed by an MRI scan (with contrast).
- •History of radiation therapy or chemotherapy.
- •Malnourished children defined as:
- •Serum albumin below the lower limit of normal (LLN) according to the reference ranges of central laboratory;
- •Serum iron below the lower limit of normal (LLN) according to the reference ranges of central laboratory;
- •BMI < -2 Standard Deviation for age and sex;
- •Children with psychosocial dwarfism.
- •Children born small for gestational age (SGA - birth weight and/or birth length < -2 SD for gestational age).
- •Presence of anti-hGH antibodies at screening.
- •Any clinically significant abnormality likely to affect growth or the ability to evaluate growth, such as, but not limited to, chronic diseases like renal insufficiency, spinal cord irradiation, etc.
- •Patients with diabetes mellitus.
- •Patients with impaired fasting sugar (based on WHO; fasting blood sugar >110 mg/dl or 6.1 mmol/l) after repeated blood analysis.
- •Chromosomal abnormalities and medical "syndromes" (Turner's syndrome, Laron syndrome, Noonan syndrome, Prader-Willi Syndrome, Russell-Silver Syndrome, short stature homeobox-containing gene (SHOX) mutations/deletions and skeletal dysplasias), with the exception of septo-optic dysplasia.
- •Closed epiphyses.
- •Concomitant administration of other treatments that may have an effect on growth such as anabolic steroids and methylphenidate for attention deficit hyperactivity disorder (ADHD), with the exception of hormone replacement therapies (thyroxine, hydrocortisone, desmopressin (DDAVP))
- •Children requiring glucocorticoid therapy (e.g. asthma) who are taking a dose of greater than 400 µg/d of inhaled budesonide or equivalents for longer than 1 month during a calendar year.
- •Major medical conditions and/or presence of contraindication to r-hGH treatment.
- •Known or suspected HIV-positive patient, or patient with advanced diseases such as AIDS or tuberculosis.
- •Drug, substance, or alcohol abuse.
- •Known hypersensitivity to the components of study medication.
- •Other causes of short stature such as coeliac disease, hypothyroidism and rickets.
- •The patient and/or the parent/legal guardian are likely to be non-compliant in respect to study conduct.
- •Participation in any other trial of an investigational agent within 30 days prior to Screening.
研究组 & 干预措施
MOD-4023
Once weekly injection of long acting r-hGH (MOD-4023) provided as a solution for injection containing 20 or 50 mg/mL MOD-4023 in a single patient use, multi-dose, disposable pre-filled pen (PEN).
干预措施: MOD-4023 (Drug)
结局指标
主要结局
Annualized Height Velocity
时间窗: 8 years
A summary of the annualized HV at the end of each year for Periods III/IV (OLE Years 1 - 4) and V (PEN Years 1 - 5).
Delta Height SDS Every 12 Months
时间窗: 8 years
A summary of annual change in height SDS at the end of each year for Periods III/IV (OLE Years 1 - 4) and V (PEN Years 1 - 5). Height SDS (Standard Deviation Score) reflects how an individual's height compares to the average for their age and sex, calculated using Growth Analyzer and based on Switzerland 1989 (Prader) reference. A z-score of 0 indicates a height equal to the population mean, while positive scores indicate above-average heights and negative scores reflect below-average heights.
次要结局
- Summary of IGF-1 SDS(8 years)