EUCTR2016-001145-11-IT进行中（未招募）1 期
A multicenter, Phase 3, randomized, open-label, active-controlled, parallel-group trial investigating the safety, tolerability, and efficacy of TransCon hGH administered once a week versus standard daily hGH replacement therapy over 52 weeks in prepubertal children with growth hormone deficiency (GHD)
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 162
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) Prepubertal children with GHD (either isolated or as part of a multiple pituitary hormone deficiency) in Tanner stage 1 aged:
- •o Boys: 3-12 years, inclusive
- •o Girls: 3-11 years, inclusive
- •2) Impaired height (HT) defined as at least 2.0 standard deviations (SD) below the mean height for chronological age and sex (HT SDS =-2.0) according to the 2000 CDC Growth Charts for the United States Methods and Development
- •3) BMI within ±2.0 SD of the mean BMI for chronological age and sex according to 2000 CDC standards
- •4) Diagnosis of GHD confirmed by 2 different GH stimulation tests, defined as a peak GH level of =10 ng/mL, determined with a validated assay. One or 2 well documented historical tests (with properly recorded sampling times and results as well as documented euthyroid status of the subject) performed within 6 months prior to Screening can be accepted to replace 1 or both GH stimulation tests. The highest GH level determines eligibility.
- •5) Bone age (BA) at least 6 months less than chronological age (X-ray may have been taken within 6 months prior to Screening, the X-ray or digital image should be sent to the central reader).
- •6) Baseline IGF-1 level of at least 1.0 SD below the mean IGF-1 level standardized for age and sex (IGF-1 SDS =-1.0) according to the central laboratory reference values.
- •7) Normal fundoscopy at Screening (without signs/symptoms of intracranial hypertension).
- •8) Children with multiple hormonal deficiencies must be on stable replacement therapy (stable dose and normal blood hormone levels) for other hypothalamo-pituitary axes for at least 3 months. Thyroid replacement therapy for thyroid hormone deficiency must be instituted at least 6 months (and be stable for at least 3 months) prior to Screening. Temporary adjustment of glucocorticoid replacement therapy, as appropriate, is acceptable.
- •9) Normal 46 XX karyotype for girls (results prior to Screening may be accepted).
- •10) Written, signed informed consent of the parent(s) or legal guardian(s) of the subject and written assent of the subject (if the subject is able to read, understand, and sign).
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 150
- •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 a body weight below 12 kg
- •2) Prior exposure to recombinant hGH or IGF-1 therapy
- •3) Children with past or present intracranial tumor growth as confirmed by a sellar MRI scan (with contrast dye recommended) at Screening (MRI results from up to 6 months prior to Screening may be accepted)
- •4) Children born small for gestational age (SGA) (ie, birth weight and/or birth length) =-2.0 SD for gestational age
- •5) Malnutrition, defined as:
- •o Serum albumin level below the lower limit of normal (LLN) according to the reference ranges of the central laboratory, and
- •o Serum iron below the lower limit of normal (LLN) according to the reference ranges of the central laboratory, and
- •o BMI =-2.0 SD for age and sex
- •6) Children with psychosocial dwarfism
- •7) Children with idiopathic short stature
- •8) Other causes of short stature such as coeliac disease (confirmed by anti-transglutaminase antibodies test), hypothyroidism, or rickets
- •9) History or presence of malignant disease; any evidence of present tumor growth
- •10) Any clinically significant abnormality likely to affect growth or the ability to evaluate growth (eg, chronic diseases like renal insufficiency, spinal cord irradiation)
- •11) Subjects with poorly controlled diabetes mellitus (HbA1c =8.0%) or diabetic complications
- •12) Known chromosomal abnormalities and other named medical syndromes (eg, Turner syndrome, Laron syndrome, Noonan syndrome, Prader-Willi syndrome,Russell-Silver syndrome, SHOX mutations/deletions and skeletal dysplasias) with the exception of septo-optic dysplasia
- •13) Closed epiphyses
- •14) Tanner stage >1 (scant pubic hair alone does not exclude the subject)
- •15) Concomitant administration of other treatments that may have an effect on growth such as anabolic steroids with the exception of hormone replacement therapies (thyroxine, hydrocortisone, desmopressin)
- •16) Children requiring glucocorticoid therapy (eg, asthma) who are taking a dose of greater than 400 µg/d of inhaled budesonide or equivalents for longer than 1 month during a calendar year
- •(Note: Approximately equivalent doses: fluticasone: 264 µg/d; beclomethasone: 504 µg/d; flunisolide 1,000 µg/d; triamcinolone: 1,000 µg/d; mometasone: 211 µg/d; ciclesonide 264 µg/d)
- •17) Major medical conditions and/or presence of contraindication to hGH treatment
- •18) Known or suspected HIV-positive subject
- •19) Known hypersensitivity to the components of the study drug
- •20) The subject and/or the parent/legal guardian are likely to be non-compliant with respect to trial conduct
- •21) Participation in any other trial of an investigational agent within 3 months prior to Screening
- •22) Any other reason that in the opinion of the investigator would prevent the subject from completing participation or following the trial schedule
研究者
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