JPRN-jRCT2031200340进行中（未招募）3 期
A multicenter, Phase 3, randomized, open-label, active-controlled, parallel-group trial investigating the efficacy, safety, and tolerability of lonapegsomatropin (TransCon hGH) administered once a week versus standard daily hGH replacement therapy over 52 weeks in Japanese prepubertal hGH-treatment naive children with growth hormone deficiency(GHD) - riGHt
适应症
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 50
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
- 年龄范围
- >= 3age old 至 <= 16age old（—）
- 性别
- All
入选标准
- •Main period
- •1) Prepubertal Japanese children with GHD (either isolated or as part of a multiple pituitary hormone deficiency) in Tanner stage 1 aged (at randomization):
- •Boys: 3 - 12 years, inclusive
- •Girls: 3 - 11 years, inclusive
- •2) Impaired HT:
- •A. Defined as at least 2.0 standard deviation score (SDS) below the mean HT for chronological age and sex (HT SDS <= -2.0) according to the Japanese year 2000 national growth survey reference data
- •B. For subjects with HT SDS > -2.0, who have benign, organic, intracranial lesions (such as Rathke's cleft cyst) causing deficiency of at least one pituitary hormone in addition to GH, if
- •a. the growth velocity is <= 1.5 SD below the age-appropriate mean in the normal population over at least 1 year (or 6 months with medical monitor confirmation) prior to screening
- •b. and with appropriate documentation of growth velocity as confirmed by the medical monitor
- •3) Body mass index (BMI) within +/- 2.0 SD of the mean BMI for chronological age and sex according to the Japanese year 2000 national growth survey, or BMI within +/- 2.0 SD of the mean BMI for bone age and sex
- •4) Diagnosis of GHD confirmed by GH stimulation tests:
- •A. For all subjects except those listed in section B below:
- •Confirmed by at least two different GH stimulation tests with a peak GH level of <= 6.0 ng/mL (for tests using insulin, arginine, clonidine, glucagon, or L-Dopa as the stimuli), or <= 16 ng/mL [when growth hormone-releasing peptide 2 (GHRP-2) is the stimulus], determined with a validated assay.
- •Well-documented historical tests (with properly recorded sampling times and results as well as documented euthyroid status of the subject) performed within approximately 6 months prior to Screening can be accepted to replace any or all of the GH stimulation tests.
- •B. For subjects with organic brain disease, deficiency of a pituitary hormone other than GH or a documented history of symptomatic hypoglycemia:
- •Confirmation as in A above however, if deemed appropriate by the Medical Monitor, with one GH stimulation test rather than two
- •5) Bone age at least 6 months less than chronological age (X ray may have been taken within approximately 6 months prior to Screening may be accepted, 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 approximately 3 months. Thyroid replacement therapy for thyroid hormone deficiency must be instituted approximately 6 months (and be stable for approximately 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 parents or legally acceptable representatives of the subject and written assent of the subject (if the subject is able to read, understand, and sign)
- •1) Children who have completed main period
- •2) Children who have not permanently discontinued investigational product in the main period
- •3) Written, signed informed consent of
排除标准
- •Main period
- •1) Children with a weight below 5.5 kg
- •2) Prior exposure to recombinant hGH or IGF-1 therapy
- •3) Children with a history of benign intracranial tumors unless there is documentation(within 6 Month(M) prior to Screening)to confirm no growth within the past 2 years. Children with non-malignant meningiomas are not eligible
- •4) Children born small for gestational age(i.e., birth weight <= -2.0 SD for gestational age, with or without a birth length < -2.0 SD for gestational age)
- •5) Malnutrition, defined as:
- •- Serum albumin level below the lower limit of normal(LLN)according to the reference ranges of the central laboratory, and
- •- Serum iron below the LLN according to the reference ranges of the central laboratory, and
- •- 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; children with GHD and clinically cured tumors may be eligible after consultation with the Medical Monitor(MM)
- •10) Any clinically significant abnormality likely to affect growth or the ability to evaluate growth with standing height measurement(e.g., chronic diseases like renal insufficiency, spinal cord irradiation or significant spinal abnormalities including but not limited to spina bifida variants or significant kyphosis or scoliosis)
- •11) Subjects with poorly controlled diabetes mellitus(DM) (HbA1c >= 8.0%)or diabetic complications
- •12) Known chromosomal abnormalities and other named medical syndromes known to impact growth(e.g., 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 and including methylphenidate and other stimulant medications used for ADHD. With the exception of hormone replacement therapies(e.g., levothyroxine or hydrocortisone when used for hormone replacement). Medication for ADHD will be allowed for subjects to start after enrollment. Glucocorticoids not given for hormone replacement therapy are discussed separately below.
- •16) Children requiring systemic glucocorticoids for reasons other than as hormone replacement therapy, inhaled, or mid- to high-potency topical glucocorticoids as follows:
- •A. Children requiring inhaled glucocorticoid therapy(e.g., asthma)who used a dose of greater than 400 microgram/d of inhaled budesonide or equivalent for more than 28 days cumulatively over the course of the 12M prior to screening(or who will likely require treatment with more than 400 microgram/d of inhaled budesonide or equivalent for more than 28 days cumulatively in a calendar year during the trial). Chronic use of lower doses of inhaled glucocorticoids is not a reason for exclusion.
- •(Note: Approximately equivalent doses to budesonide DPI 400 microgram/d: beclomethasone[HFA; extra fine particles], 240 microgram/d; beclomethasone[HFA, standard particles], 400 microgram/day; ciclesonide[HFA], 240 microgram/d; flunisolide[HFA], 240 microgram/d; fluticasone propionate[HFA], 220 microgram/d; fluticasone propionate
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