An Open-Label Multiple-Dose Study of RZ358 in Patients with Congenital Hyperinsulinism

Rezolute, Inc.0 个研究点目标入组 32 人开始时间: 2019年10月7日最近更新: 2022年5月9日

适应症

试验速览

阶段: 1 期
状态: 进行中（未招募）
发起方: Rezolute, Inc.
入组人数: 32

研究概览入排标准研究者记录与标识符相似试验

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. Provide written informed consent and, as applicable, assent, before any study specific procedures are performed
•2. At Screening, aged =2 years and =45 years
•3. An established clinical diagnosis, with or without a genetic diagnosis, of congenital hyperinsulinism
•4. Patient is currently receiving SOC medications (i.e corticosteroids, diazoxide, lanreotide, sirolimus, and octreotide) and/or nutritional supplementation for CHI, with at least 2 months of stable treatment; OR is not on treatment, at the time of Screening.
•5. Glucose values <70 mg/dL (3.9 mmol/L) for =4% of the overall monitored CGM time with at least 3 severe hypoglycemia events by CGM threshold of <50mg/dL (2.8mmol/L) during the last 7 days of a 10-day Screening CGM evaluation period; A discrete severe hypoglycemia event by CGM is defined by having at least 3 consecutive (15 monitored minutes) hypoglycemia readings <70 mg/dL (3.9 mmol/L) with at least one of those readings <50 mg/dL (2.8 mmol/L) and with at least 60 monitored minutes of glucose values = 70 mg/dL (3.9 mmol/L) in between events.
•Experiencing = 3 hypoglycemia events (<70 mg/dL) per week by SMBG and/or according to the Investigator's evaluation.
•6. Hepatic ultrasound at Screening without clinically significant findings, including clinically significant gallstones as judged by the Investigator (e.g. large size, obstructive, biliary colic, or LFT abnormalities exceeding protocol thresholds) or evidence of peliosis hepatitis
•7. For female patients of childbearing potential, a negative serum or urine pregnancy test within 7 days before dosing Females of childbearing potential are defined as fertile following menarche and until becoming postmenopausal or permanently sterile. Note: Postmenopausal is defined as absence of vaginal bleeding or spotting for at least 1 year. Permanently sterile is defined as having had a hysterectomy, bilateral salpingectomy, or bilateral oophorectomy.
•8. For female patients of childbearing potential, a willingness to use highly effective* contraceptive measures adequate to prevent a new pregnancy for the duration of the study, including for at least 105 days after receiving the last dose of study drug. For women with reproductive potential who use a hormonal method of contraception, concurrent use of a second (barrier) method is recommended. (*Highly effective methods of birth control are defined as those that result in a low failure rate [ie, less than 1% per year] when used consistently and correctly [eg, implants, injectables, oral contraceptives, some intrauterine devices, bilateral tubal occlusion, true sexual abstinence in line with the preferred and usual lifestyle of the patient, or vasectomized partner.])
•9. For sexually active males, a willingness to use contraceptive measures, e.g. a condom, for the duration of the study, including for at least 105 days after receiving the last dose of study drug. In addition, males must agree not to donate sperm over the same study period.
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 30
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 2
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

排除标准

•1. Any out-of-range laboratory value at Screening that has not been reviewed, approved, and documented as not clinically significant by the Investigator, with the exception of liver function tests for total bilirubin, ALT, AST, and ALP, which must be within 1.5X the upper limit of normal (ULN) for the reference range
•2. Body mass index = 35 kg/m2 for patients aged 18 years and above, or BMI = 99% (percentile) per CDC growth charts for patients >12 and <18 years of age.
•3. History of malignancy within 3 years before Screening other than carcinoma in situ of the cervix or adequately treated non-metastatic squamous or basal cell carcinoma of the skin
•4. History of seropositivity for HIV antibody, hepatitis B, or hepatitis C antibody
•5. Major general surgery within 3 months before Screening or anticipated during the study period
•6. Use of systemic corticosteroids within 30 days before Screening
•7. Known allergy or sensitivity to RZ358 or any component of the study drug
•8. Treatment with an investigational drug or device within 30 days or 5 half-lives of the investigational drug before Day 1 of Week 1, whichever is longer. Participation in registries and purely diagnostic studies is allowed.
•9. Female patients who are pregnant, planning to become pregnant during the course of the study, have recently delivered (within 3 months before Screening), or are breastfeeding
•10. Male patients who are planning a pregnancy with a female partner during the course of the study or within 105 days after administration of study drug
•11. Any organ condition, concomitant disease (eg, psychiatric illness, severe alcoholism, or drug abuse, cardiac, hepatic, or kidney disease), or other abnormality that itself, or the treatment of which, could interfere with the conduct of the study (eg, may affect absorption, distribution, metabolism, or elimination of the study drug) or that, in the opinion of the Investigator and/or Sponsor’s medical monitor, would pose an unacceptable risk to the patient in the study.

研究者

发起方

Rezolute, Inc.

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