EUCTR2011-004553-60-HU进行中（未招募）1 期
Safety and dose finding study of different MOD-4023 dose levelscompared to daily r-hGH therapy in pre-pubertal growth hormonedeficient children
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 56
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •1.Pre-pubertal child aged = 3 yrs old and not above 10 years for girls or 11 years for boys with either isolated GHD, or GH insufficiency as part of multiple pituitary hormone deficiency.
- •2. Confirmed diagnosis of GHD by two different GH provocation tests defined as a peak plasma GH level of =10 ng/ml, determined by central laboratory using a validated assay. If the patient has already been tested locally and reserve samples that were taken at appropriate time-points are available, these will be reanalyzed by the central laboratory. Historical tests missing the -30 minutes time point will be accepted. If no reserve samples are kept (only for tests performed prior to site initiation), then the details of the locally performed tests will be reviewed by the Coordinating Investigator: if the results cannot be
- •accepted, the patient will undergo both stimulation tests during the screening period and the samples will be analyzed by the central laboratory. At least one of the two stimulation tests (and preferably both) will be analyzed by the central laboratory. If the patient requires sex hormone priming (due to the age), and both stimulation tests must be performed during the Screening (no historical samples kept, or test was without priming), it is recommended to perform stimulation tests in consecutive setting in one day, or in two consecutive days, to avoid priming the patient twice. Local historical tests without sex-steroid priming will not be accepted for patients that require sex steroid priming according to the protocol.
- •3.Bone age (BA) is not older than chronological age and should be no greater than 9 years for girls and 10 years for boys.
- •4.Without prior exposure to any r-hGH therapy.
- •5.Impaired height and height velocity defined as:
- •a.Height (HT) of at least 2.0 standard deviations (SD) below the mean height for chronological age (CA) and gender according to the standards from Prader et. al, 1989 , (HT SDS = -2.0).
- •b.Annualized height velocity (HV) below the 25th percentile for CA (HV <-0.7 SDS) and gender according to the standards of Prader et al (1989). The interval between two height measurements should be at least 6 months, but should not exceed 18 months prior to inclusion.
- •6.BMI must be within ±2 SD of mean BMI for the chronological age and sex according to the 2000 CDC standards.
- •7.Baseline IGF-I level of at least 1 SD below the mean IGF-I level standardized for age and sex (IGF-I SDS = -1.0) according to the central laboratory reference values;
- •8.Children with normal fundoscopy (ophthalmoscopy) at screening (without signs/symptoms of intracranial hypertension as assessed by fundoscopy);
- •9.Children with multiple hormonal deficiencies must be on stable replacement therapies for other hypothalamo-pituitary-organ axes for at least 3 months and 6 months for thyroid replacement therapy prior to the first study drug administration;
- •10.Normal 46 XX karyotype for girls
- •11. Written informed consent of the parent or legal guardian of the patient and assent of the patient for the Main study and for the OLE.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 56
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1.Children with past or present intracranial tumor growth as confirmed by an MRI scan (with contrast).
- •2.History of radiation therapy or chemotherapy.
- •3.Malnourished children defined as:
- •a.Serum albumin below the lower limit of normal (LLN) according to the reference ranges of central laboratory;
- •b.Serum iron below the lower limit of normal (LLN) according to the reference ranges of central laboratory;
- •c.BMI < -2 SD for age and sex;
- •4.Children with psychosocial dwarfism.
- •5.Children born small for gestational age (SGA – birth weight and/or birth length < -2 SD for gestational age).
- •6.Children with idiopathic short stature.
- •7.Presence of anti-hGH antibodies at screening.
- •8.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.
- •9.Patients with diabetes mellitus.
- •10.Patients with impaired fasting sugar (based on WHO; fasting blood sugar >110 mg/dl or 6.1 mmol/l) after repeated blood analysis.
- •11.Chromosomal abnormalities and medical syndromes” (Turner’s syndrome, Laron syndrome, Noonan syndrome, Prader-Willi Syndrome, Russell-Silver Syndrome, SHOX mutations/deletions and skeletal dysplasias), with the exception of septo-optic dysplasia.
- •12.Closed epiphyses.
- •13.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))
- •14.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.
- •15.Major medical conditions and/or presence of contraindication to r-hGH treatment.
- •16.Known or suspected HIV-positive patient, or patient with advanced diseases such as AIDS or tuberculosis.
- •17.Drug, substance, or alcohol abuse.
- •18.Known hypersensitivity to the components of study medication.
- •19.Other causes of short stature such as coeliac disease, hypothyroidism and rickets.
- •20.The patient and/or the parent/legal guardian are likely to be non-compliant in respect to study conduct.
- •21.Participation in any other trial of an investigational agent within 30 days prior to Screening.
研究者
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