JPRN-jRCT2080223906已完成3 期
A PHASE 3, OPEN-LABEL, RANDOMIZED, MULTICENTER, 12-MONTH, EFFICACY AND SAFETY STUDY OF WEEKLY MOD-4023 COMPARED TO DAILY GENOTROPIN THERAPY IN JAPANESE PRE-PUBERTAL CHILDREN WITH GROWTH HORMONE DEFICIENCY
OPKO Health Inc. (ICCC: EPS Corporation)0 个研究点目标入组 44 人开始时间: 2018年5月17日最近更新:
适应症
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 44
研究概览
简要总结
All 44 patients assigned to receive the study drug were included in the safety analysis set and the FAS, which was the primary efficacy analysis set. For efficacy, the primary endpoint of annual growth rate (cm/year) at 12 months of treatment was 9.65+/-0.29 in the MOD-4023 group and 7.8+/-0.29 in the Genotropin group, and point estimates of differences between the groups exceeded the pre-specified threshold of -1.8 cm/year, indicating a similar improvement trend in the MOD-4023 and Genotropin groups.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized Intervention Model: Parallel Assignment Masking: Single (Outcomes Assessor) Primary Purpose: Other
入排标准
- 年龄范围
- >= 3age old 至 <= 11age old（—）
- 性别
- All
入选标准
- •1.Pre-pubertal child aged >= 3 years old, and not yet 10 years for girls (9 years and 364days) or not yet 11 years for boys (10 years and 364 days),on the date of ICF
- •signature, with either isolated GHD, or GH insufficiency as part of multiple ituitary
- •hormone deficiency.
- •2.Confirmed diagnosis of GHD by 2 different types of GH provocation tests (standardized on growth foundation data):defined as a peak serum GH level of <= 6.0
- •ng/mL or <= 16 ng/mL when conducting GHRP-2 provocation test.
- •Prior local laboratory results will be accepted subject to pre-approval by the study
- •medical monitor and if the tests were conducted according to 1 of the protocols in
- •3.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 r-hGH therapy.
- •5.Height SD score <= -2.0 at screening.
- •6.Impaired height velocity defined as:Annualized HV below the 25th percentile for CA (HV < -0.7 SDS) and gender according to the local primary care provider standard. The interval between 2 height measurements should be at least 6 months, but should not exceed 18 months prior to inclusion.
- •7.BMI must be within +- 2SDS of mean BMI for the chronological age and sex.
- •8.Baseline IGF-1 level of at least 1 SD below the mean IGF-1 level standardized for age and sex (IGF-1 SDS <= -1) according to the central laboratory reference values. A
- •single re-test will be allowed (subject to approval from the study medical monitor) if all other criteria are met.
- •9.Normal creatinine levels according to common practice reference ranges per age.
- •10.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 ICF signing.
- •11.Clinical presentation of normal 46XX karyotype for girls.
- •12.Willing and able to provide written informed consent of the parent or legal guardian of the patient and written assent from pediatric patients (when applicable based on age and Japan regulation).
排除标准
- •1.Children with prior history of leukemia, lymphoma, sarcoma or any other 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 by the discretion of the investigator.
- •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.Children with diabetes mellitus.
- •9.Known or suspected chromosomal abnormalities including Turner`s syndrome, Laron
- •syndrome, Noonan syndrome, Prader-Willi syndrome, Russell-Silver syndrome, SHOX mutations/deletions and skeletal dysplasia`s, with the exception of septo-optic
- •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 Micro g/d of inhaled budesonide or equivalent as described in Appendix.
- •12. Major medical conditions and/or presence of contraindication to r-hGH treatment.
- •13. Known or suspected HIV-positive patient, or patient with advanced diseases such as AIDS or tuberculosis.
- •14. Drug, substance, or alcohol abuse.
- •15. Known hypersensitivity to the components of study medication.
- •16. Other causes of short stature such as celiac disease, uncontrolled primary
- •hypothyroidism and rickets.
- •17. The patient and/or the parent/legal guardian are likely to be non-compliant in respect to study conduct.
- •18 Participation in any other trial of an investigational agent within 30 days prior to screening and throughout the entire study period (including administration of investigational agent).
研究者
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