CTRI/2017/08/009359进行中（未招募）3 期
A Phase 3, Open-Label, Randomized, Multicenter, 12 Months,Efficacy And Safety Study Of Weekly Mod-4023 Compared ToDaily Genotropin® Therapy In Pre-Pubertal Children WithGrowth Hormone Deficiency And A 12-Month Open-LabelExtension To Assess The Efficacy And Safety Of Mod-4023.
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 220
- 试验地点
- 14
- 主要终点
- Annual HV in cm/year after 12 months of treatment.
研究概览
简要总结
The study will consist of a 12 month, open-label, randomized, active controlled, parallel group study comparing the efficacy and safety of weekly MOD-4023 to daily growth hormone, Genotropin. After a screening period lasting up to 8 weeks, patients meeting the eligibility criteria, will be randomized 1:1 ratio to MOD-4023 (0.66 mg/kg/week) or growth hormone Genotropin (0.034 mg/kg/daily) for 7 injections over a week) for 12 months.
The total duration of patient participation in the study will be up to 15 months (12 months of treatment and up to 8 (+4) weeks of screening). The study will be conducted at approximately 150-180 sites in 30-40 countries worldwide.
研究设计
- 研究类型
- Interventional
- 分配方式
- Computer generated randomization
- 盲法
- Open Label
入排标准
- 年龄范围
- 3.00 Year(s) 至 12.00 Year(s)（—）
- 性别
- All
入选标准
- •Pre-pubertal children aged equal or elder than 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.
- •Confirmed diagnosis of GHD by two different GH provocation tests defined as a peak plasma GH level of equal or lesser then 10 ng/mL, determined by local or central laboratory using a validated assay.
- •Global study MM may accept prior local laboratory results, subject to pre-approval and if the tests were conducted as recommended in the protocol Appendix B.
- •BA is not older than CA and should be lesser than 10 for girls and lesser than 11 for boys.
- •Without prior exposure to any recombinant hGH (r-hGH) therapy (naive patients).
- •Impaired Ht velocity defined as: a.
- •Annualized HV below the 25 percentile for CA (HV lesser than -0.7 SDS) and gender according to the OPKO HV (Tanner, Prader and Hermanussen) calculator, provided.
- •The interval between 2 Ht measurements should be at least 6 months, but should not exceed, 18 months prior to inclusion.
- •Baseline IGF-1 level of at least 1 standard deviation (SD) below the mean IGF-1 level standardized for age and sex (IGF-1 SDS equal or lesser than lesser than -1) according to the central laboratory reference values.
- •A single re-test will be allowed (subject to discussion with MM) if all other criteria are met.
- •Normal calculated glomerular filtration rate (GFR) based on updated bedside Schwartz formula for pediatric patients (calculation is recommended below).
- •Creatine Clearance Rate (CrCL) (mL per min per 1.73 m 2) equals to 0.413 by Ht per serum creatine (Scr) Ht- in cm Scr- in mg per dL.
- •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.
- •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).
- •Inclusion into the OLE:
- •Completion of the main study (12 months of treatment) with adequate compliance.
- •Agreement to refrain from sexual activity during the OLE i.e. observe complete sexual abstinence as the only acceptable contraceptive measure during the OLE (for pubertal and post-pubertal patients).
排除标准
- •Children with prior history of leukemia, lymphoma, sarcoma or any other forms of cancer.
- •History of radiation therapy or chemotherapy.
- •Malnourished children defined as BMI lesser than -2 SDS for age and sex.
- •Children with psychosocial dwarfism.
- •Children born small for gestational age (SGA – birth weight and/or birth length lesser than -2 SDS for gestational age).
- •Any clinically significant (CS) 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.
- •Types 1 and 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.(Criteria for controlled diabetes are defined in Appendix F).
- •Chromosomal abnormalities including Turner syndrome, Laron syndrome, Noonan syndrome, Prader-Willi syndrome, Russell-Silver syndrome, SHOX mutations/deletions and skeletal dysplasias.
- •Concomitant administration of other treatments that may have an effect on growth such as anabolic steroids, or sex steroids, with the exception of Attention-Deficit-Hyperactivity Disorder (ADHD) drugs or hormone replacement therapies (thyroxin, hydrocortisone, desmopressin [DDAVP]).
- •Children requiring glucocorticoid therapy (e.g. for asthma) that are taking chronically a dose greater than 400 μg/day of inhaled budesonide or equivalent as provided in Appendix J.
- •Major medical conditions and or presence of contraindication to r-hGH treatment.
- •More than 1 closed epiphyses.
- •Known or suspected Human Immunodeficiency Virus (HIV)-positive patient, or patient with advanced diseases such as Acquired Immunodeficiency Syndrome (AIDS) or tuberculosis.
- •Drug, substance, or alcohol abuse.
- •Known hypersensitivity to the components of study medication.
- •Other causes of short stature such as celiac disease, uncontrolled primary hypothyroidism and rickets.
- •The patient and or the parent or legal guardian are likely to be non-compliant in respect to study conduct.
- •Participation in any other study of an investigational agent within 30 days prior to ICF signature (including administration of investigational agent).
- •Study enrollment requirements have been met or the study has been closed by the Sponsor prior to the completion of screening process.
- •Exclusion during the OLE:
- •Concomitant administration of other treatments that may have an effect on growth such as anabolic steroids, or sex steroids (other than for hormonal replacement), with the exception of ADHD drugs or hormone replacement therapies (thyroxin, hydrocortisone, testosterone, estrogen and progesterone, desmopressin [DDAVP])
- •Change in medical condition during the treatment period (such as, but not limited to, development of a serious inter-current critical illness, a severe adverse drug reaction, etc.)
- •Positive pregnancy test.
- •Unresolved drug related (MOD-4023 or Genotropin) SAE from the treatment period as per MM judgement.
结局指标
主要结局
Annual HV in cm/year after 12 months of treatment.
时间窗: After 12 months of treatment
次要结局
- Secondary efficacy endpoints (Auxology/Clinical):(1. Annualized HV after 6 months of treatment.)
- Secondary endpoints (Biochemical)(1. Absolute IGF-1 and IGF-1 SDS levels on Day 4 (-1) after MOD-4023 dosing)
- OLE Endpoints:(Biochemical Endpoints)
研究者
研究点 (14)
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