EUCTR2016-003874-42-DE进行中（未招募）1 期
A phase 3, open-label, randomized, multicenter, 12 months, efficacy and safety study of weekly mod-4023 compared to daily genotropin® therapy in pre-pubertal children with growth hormone deficiency - N/A
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
- 220
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 children aged =3 years , and not yet 11 years for girls (10 years and 364 days) or not yet 12 years (11 years and 364 days) for boys, (on the date of ICF signature), 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 local or central laboratory using a validated assay . Global Medical Monitor may accept prior local laboratory results; subject to pre-approval and if the tests were conducted as recommended in the protocol
- •3. Bone age (BA) is not older than chronological age and should be less than 10 for girls and less than 11 for boys.
- •4. Without prior exposure to any rhGH therapy (naïve patients).
- •5. Impaired height velocity defined as:
- •Annualized height velocity (HV) below the 25th percentile for CA (HV < -0.7 SDS) and gender according to Prader HV standard tables, Tanner HV curves, or local primary care provider standard.
- •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 SDS of mean BMI for the chronological age and sex.
- •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) according to the central laboratory reference values. A single re-test will be allowed (subject to discussion with medical monitor) if all other criteria are met.
- •8. Normal calculated GFR based on updated bedside” Schwartz formula for pediatric patients (calculation is recommended below):
- •CrCL (mL/min/1.73 m2) =0.413 * Ht / Scr
- •Ht: height in cm;
- •Scr: serum creatinine in mg/dL;
- •9. Children with multiple hormonal deficiencies must be on stable replacement therapies (no change in dose) for other hypothalamo-pituitary organ axes for at least 3 months prior to ICF signing
- •10. Normal 46XX karyotype for girls.
- •Willing and able to provide written informed consent of the parent or legal guardian of the patient and written assent from pediatric patients (where applicable based on age and country regulation).
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 220
- •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 prior history of leukemia, lymphoma, sarcoma or any other forms of cancer.
- •2. History of radiation therapy or chemotherapy
- •3. Malnourished children defined as BMI < -2 SDS for age and sex
- •4. Children with psychosocial dwarfism
- •5. Children born small for gestational age (SGA – birth weight and/or birth length <-2 SDS for gestational age)
- •6. Presence of anti-hGH antibodies at screening
- •7. 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.
- •8. Type 1 and type 2 diabetic patients who, in the opinion of the investigator, are not receiving standard of care treatment, or are non-compliant with their prescribed treatment or who are in poor metabolic control
- •9. Chromosomal abnormalities including Turner’s syndrome, Laron syndrome, Noonan syndrome, Prader-Willi syndrome, Russell-Silver syndrome, SHOX mutations/deletions and skeletal dysplasias.
- •10. Concomitant administration of other treatments that may have an effect on growth such as anabolic steroids, or sex steroids, with the exception of ADHD drugs or hormone replacement therapies (thyroxin, hydrocortisone, desmopressin [DDAVP])
- •11. Children requiring glucocorticoid therapy (e.g. for asthma) that are taking chronically a dose greater than 400 µg/d of inhaled budesonide or equivalent
- •12. Major medical conditions and/or presence of contraindication to r-hGH treatment.
- •13. Closed epiphyses
- •14. Known or suspected HIV-positive patient, or patient with advanced diseases such as AIDS or tuberculosis.
- •15. Drug, substance, or alcohol abuse.
- •16. Known hypersensitivity to the components of study medication.
- •17. Other causes of short stature such as celiac disease, uncontrolled primary hypothyroidism and rickets.
- •18. 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 ICF signature (including administration of investigational agent).
研究者
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