EUCTR2012-002787-27-FR进行中（未招募）1 期
A multicenter, Phase 2, randomized, open label, active-controlled, parallel-group study investigating the safety, tolerability, and efficacy of different dose levels of ACP-001 administered once weekly versus standard daily rhGH replacement therapy in pre-pubertal children with Growth Hormone Deficiency (GHD)
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 52
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 with GHD (either isolated or as part of a multiple pituitary hormone deficiency) in Tanner stage I aged:
- •a. Boys: 3 years = boy’s age = 12 years
- •b. Girls: 3 years = girl’s age = 11 years;
- •2. Diagnosis of GHD confirmed by two different GH provocation tests, defined as a peak GH level of = 10 ng/mL, determined by a central laboratory using a validated assay. One well documented historical test (with properly recorded sampling times and results as well as euthyroid status of the patient) performed within 3 months prior to Screening can be accepted to replace one GH stimulation test analyzed by the central laboratory;
- •3. Bone age (BA) not greater than the chronological age;
- •4. Impaired height and height velocity defined as:
- •a. Height (HT) of at least 2.0 standard deviations (SD) below the mean height for chronological age and sex (HT SDS = - 2.0) according to the CDC standards of 2000
- •b. Height velocity (HV) of at least 1 SD below the mean HV for chronological age and sex (HV SDS = - 1.0) according to the standards of Prader et al. of 1989, whereas the time between 2 height measurements is not less than 6 months;
- •5. BMI within ±2 SD of the mean BMI for chronological age and sex according to the 2000 CDC standards;
- •6. Baseline IGF-I level of at least 1 SD below the mean IGF-I level standardized for age and sex (IGF-I 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;
- •10. Written informed consent of the parent or legal guardian of the patient and written assent of the patient (if the patient is able to read, understand, and sign).
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 52
- •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 11 kg;
- •2. Children with hematocrit at Screening below 34%;
- •3. Prior exposure to rhGH or IGF-I therapy;
- •4. Children with past or present intracranial tumor growth as confirmed
- •by a sellar MRI scan (with contrast) at Screening (if no MRI was
- •performed within the last 6 months prior to Screening);
- •5. Children born small for gestational age (SGA), i.e. birth weight and/or
- •birth length < -2 SD for gestational age;
- •6. Malnutrition, defined as:
- •a. Serum albumin level below the lower limit of normal (LLN) according
- •to the reference ranges of the central laboratory, and
- •b. Serum iron below the lower limit of normal (LLN) according to the
- •reference ranges of the central laboratory, and
- •c. BMI < -2 SD for age and sex;
- •7. Children with psychosocial dwarfism;
- •8. Children with idiopathic short stature;
- •9. Other causes of short stature such as coeliac disease (confirmed by
- •anti-transglutaminase antibodies test), hypothyroidism, or rickets;
- •10. Presence of anti-hGH binding antibodies at Screening;
- •11. History or presence of malignant disease; any evidence of present
- •tumor growth;
- •12. Any clinically significant abnormality likely to affect growth or the
- •ability to evaluate growth, e.g. chronic diseases like renal insufficiency,
- •spinal cord irradiation, etc.;
- •13. Patients with diabetes mellitus or impaired fasting sugar (based on
- •WHO standards);
- •14. Chromosomal abnormalities and medical syndromes (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;
- •15. Closed epiphyses;
- •16. Concomitant administration of other treatments that may have an
- •effect on growth such as anabolic steroids or methylphenidate for
- •attention deficit hyperactivity disorder (ADHD) with the exception of
- •hormone replacement therapies (thyroxine, hydrocortisone,
- •desmopressin);
- •17. Children requiring glucocorticoid therapy (e.g. 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;
- •18. Major medical conditions and/or presence of contraindication to rhGH treatment;
- •19. Known or suspected HIV-positive patient;
- •20. Known hypersensitivity to the components of the study medication;
- •21. The patient and/or the parent/legal guardian are likely to be
- •noncompliant in respect to study conduct;
- •22. Participation in any other trial of an investigational agent within 30
- •days prior to Screening.
研究者
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