EUCTR2016-003874-42-ES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
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 assaya. Global Medical Monitor may accept
- •prior local laboratory results; subject to pre-approval and if the tests were
- •conducted as recommended in the protocol Appendix B.
- •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
- •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)a 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.
- •11. 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.(Criteria for
- •controlled diabetes are defined in Appendix F).
- •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
- •as provided in Appendix J.
- •12. Major medical conditions and/or presence of contraindication to r-hGH
- •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.
- •19. 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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