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. - None
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
入选标准
- •1. 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.
- •2. 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.
- •3. BA is not older than CA and should be lesser than 10 for girls and lesser than 11 for boys.
- •4. Without prior exposure to any recombinant hGH (r-hGH) therapy (naive patients).
- •5. 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.
- •b. The interval between 2 Ht measurements should be at least 6 months, but should not exceed, 18 months prior to inclusion.
- •6. 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.
- •7. 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)
- •Scr- in mg per dL.
- •8. 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.
- •9. Normal 46XX karyotype for girls.
- •10. 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:
- •11. Completion of the main study (12 months of treatment) with adequate
- •compliance.
- •12. 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).
- •13. 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
- •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 lesser than -2 SDS for age and sex.
- •4. Children with psychosocial dwarfism.
- •5. Children born small for gestational age (SGA ââ?¬â?? birth weight and/or birth
- •length lesser than -2 SDS for gestational age).
- •6. Presence of anti-hGH Ab at screening.
- •7. 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.
- •8. 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).
- •9. Chromosomal abnormalities including Turner 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
- •Attention-Deficit-Hyperactivity Disorder (ADHD) drugs or hormone
- •replacement therapies (thyroxin, hydrocortisone, desmopressin
- •11. Children requiring glucocorticoid therapy (e.g. for asthma) that are taking
- •chronically a dose greater than 400 Ã?¼g/day of inhaled budesonide or
- •equivalent as provided in Appendix J.
- •12. Major medical conditions and or presence of contraindication to r-hGH
- •13. More than 1 closed epiphyses.
- •14. Known or suspected Human Immunodeficiency Virus (HIV)-positive
- •patient, or patient with advanced diseases such as Acquired
- •Immunodeficiency Syndrome (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 or legal guardian are likely to be non-compliant
- •in respect to study conduct.
- •19. Participation in any other study of an investigational agent within 30 days
- •prior to ICF signature (including administration of investigational agent).
- •20. 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:
- •21. 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])
- •22. 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.)
- •23. Positive pregnancy test.
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