EUCTR2020-003120-17-DE进行中（未招募）1 期
A 3-part study to evaluate the efficacy and safety of venglustat in combination with Cerezyme in adult and pediatric patients with Gaucher disease Type 3 (GD3) with open-label long-term treatment - LEAP2IT
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 12
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
- •- GD3 adult participant =18 years of age.
- •- GD3 pediatric participant is =12 years of age <18 years and =30 kg of weight. For male patients 12 to <18 years of age: There is a potential risk for venglustat to impact male fertility particularly when used in patients who have not reached a certain stage of sexual maturation. If a patient has not reached Tanner Stage 3 by the screening visit, the Investigator should assess and discuss with the patient the potential benefits and risks when considering eligibility for study participation. In this benefit/risk assessment-discussion, the parent(s)/legal guardian(s), the patient (if considered able by local regulation), and the Sponsor’s medical representative (if considered appropriate) shall be consulted.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 10
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 24
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •- Substrate reduction therapy or chaperone therapy for GD within 6 months prior to enrollment.
- •- Participant has had a partial or total splenectomy within 3 years prior to randomization.
- •- Participant is blood transfusion-dependent.
- •- Prior esophageal varices or liver infarction or current liver enzymes (alanine aminotransferase [ALT]/ aspartate aminotransferase [AST]) or total bilirubin >2 times the upper limit of normal, unless
- •the patient has a diagnosis of Gilbert Syndrome.
- •- Participant has any clinically significant disease, other than GD, including cardiovascular (congenital cardiac defect, coronary artery disease, valve disease or left sided heart failure; clinically significant arrhythmias or conduction defect), hepatic, gastrointestinal, pulmonary, neurologic, endocrine, metabolic (eg, hypokalemia, hypomagnesemia) or psychiatric disease, other medical conditions, or serious intercurrent illnesses that may preclude participation.
- •- Participant has renal insufficiency, as defined by an estimated glomerular filtration rate <30 mL/min/1.73m2 at the screening visit.
- •- Participant has received an investigational product within 30 days prior to enrollment.
- •- Participant has a history of cancer, with the exception of basal cell carcinoma.
- •- Participant has myoclonic seizures.
- •- Participant is pregnant or lactating.
- •- Participant has, according to World Health Organization (WHO) Grading, a cortical cataract >onequarter of the lens circumference (Grade cortical cataract-2) or a posterior subcapsular cataract >2
- •mm (Grade posterior subcapsular cataract-2). Patients with nuclear cataracts will not be excluded.
- •- Participant requires use of invasive ventilatory support.
- •- Participant requires use of noninvasive ventilator support while awake for longer than 12 hours daily.
- •- Participant is unable to receive treatment with Cerezyme due to a known hypersensitivity or is unwilling to receive Cerezyme treatment to ensure maintenance of Gaucher treatment goals.
- •- Participant is currently receiving potentially cataractogenic medications (corticosteroids, psoralens used in dermatology with ultraviolet light therapy [PUVA], typical antipsychotics, and glaucoma
- •medications) or any medication that may worsen the vision of a patient with cataract (eg, alphaadrenergic glaucoma medications).
- •- Participant has received strong or moderate inducers or inhibitors of CYP3A within 15 days or 5 half-lives from screening, whichever is longer, prior to enrolment in Part 2. This also includes the
- •consumption of grapefruit, grapefruit juice, or grapefruit containing products within 72 hours of starting venglustat administration in Parts 2 and 3.
- •- Participant is scheduled for in-patient hospitalization including elective surgery, during the study.
- •- Participant has had a major organ transplant (e.g., bone marrow or liver).
- •- Participant, in the opinion of the investigator, is unable to adhere to the requirements of the study or unable to undergo study assessments (e.g., contraindications for magnetic resonance imaging).
研究者
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