CTRI/2024/08/072953招募中3 期
A Multicenter, Open-Label, Single-Arm, Three-Cohort, Phase 3, Clinical Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Eliglustat Sublingual Film in Patients with Gaucher Disease Type 1 and Type 3.
Amneal Pharmaceuticals Pvt Ltd10 个研究点 分布在 1 个国家目标入组 30 人开始时间: 2024年9月2日最近更新:
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- 入组人数
- 30
- 试验地点
- 10
- 主要终点
- Percentage change in spleen volume (in MN)
研究概览
简要总结
This study is a phase 3, non-randomized, multicenter, open-label, single-arm, two-cohort study to evaluate the efficacy, safety, and pharmacokinetics (PK) of eliglustat in patients with Gaucher disease Type 1 and Type 3. The primary outcome measure will be the percentage change in spleen volume (in MN) at Week 39 vs. Baseline. The secondary outcomes will include the percentage change in liver volume (in MN) at Weeks 13, 26, and 39 vs. Baseline; absolute change in hemoglobin level at Weeks 13, 26, and 39 vs. Baseline; percentage change in platelet count at Weeks 13, 26, and 39 vs. Baseline; percentage change in spleen volume (in MN) at Weeks 13 and 26 vs. Baseline; comparison of Gaucher disease assessments at Weeks 13, 26, and 39 vs. Baseline; change in Gaucher disease severity score (GD-DS3) at Week 39 vs. Baseline; biomarker assessment (chitotriosidase) at Weeks 4, 13, 26, and 39 vs. Baseline; type, incidence, severity, seriousness, and relatedness of adverse events (AEs); assessment of Cmax, Tmax, AUC0-tau, Vd, CL, and t1/2 on Day 1 for eliglustat; assessment of AUC0-tau, Cmax,ss, Tmax,ss, Vss, CL, and t1/2 at steady state on Day 28 (Week 4), Week 13, and Week 39 for eliglustat; and Cmin,ss (Ctrough,ss) prior to dose on Day 14 (Week 2), Day 28 (Week 4), Week 13, and Week 39 for eliglustat. Percentage change in GL-1 (Glucosylceramide) & Lyso GL-1 (Glucosylsphingosine.) at week 4, 13,26 and 39 vs baseline.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 盲法
- None
入排标准
- 年龄范围
- 6.00 Year(s) 至 64.00 Year(s)（—）
- 性别
- All
入选标准
- •The patient (and/or their parent/legal guardian) is willing and able to provide signed informed consent (assent for 12 to 17 years age group) (written assent for 12-17 years age group and oral assent for 6-11 years age group) prior to any study-related procedures to be performed.
- •Male and female patients aged greater than and equal to 18years (cohort I) or 12 to 17 (cohort II) or more than 6 years (Cohort III) completed years at the time of screening.
- •The patient has a diagnosis of Gaucher disease type 1 and Type 3 confirmed by a documented deficiency of acid beta-glucosidase activity by enzyme assay or Glucocerebrosidase (GBA) genotype.
- •The patient has the following symptoms of Gaucher disease during the Screening period: i.
- •At least one of the following laboratory abnormalities: Hemoglobin level of 8.0 to 11.0 g/dL if female or 8.0 to 12.0 g/dL if male Platelet count of 50,000 to 130,000/mm3 ii.
- •Splenomegaly (spleen volume of more than 3 MN).
- •If hepatomegaly is present, the liver volume must be less than 3MN.
- •Consents to provide a blood sample for genotyping for GD, chitotriosidase, and CYP2D6 (to categorize the patients predicted rate of metabolism), unless the patients genotypes for GD, chitotriosidase, and CYP2D6 are already available.
- •Female patients of childbearing potential must have a documented negative pregnancy test prior to enrollment.
- •In addition, all female patients of childbearing potential must use a medically accepted form of contraception throughout the study (either a barrier method or hormonal contraceptive with ethinyl estradiol and norethindrone or similar active components).
排除标准
- •The patient has had a partial or total splenectomy.
- •The patient has received substrate reduction therapies for Gaucher disease within 3 months prior to enrollment.
- •The patient has any evidence of moderate to severe uncontrolled neurologic (e.g., peripheral neuropathy, tremor, seizures, Parkinsonism, or cognitive impairment) or pulmonary involvement (e.g., pulmonary hypertension, interstitial lung disease) as related to Gaucher disease.
- •The patient has current symptomatic bone disease such as bone pain attributable to osteonecrosis and/or pathologic fracture, or has had a bone crisis in the 12 months prior to enrollment.
- •The patient is transfusion-dependent.
- •The patient has the following laboratory abnormalities during the Screening period: Hemoglobin level less than 8 g/dL and Platelet count of less than 50,000/mm3
- •The patient has documented anemia due to causes other than Gaucher disease that requires treatment not yet initiated or not yet stable under treatment for at least 3 months (e.g., iron, vitamin B12, and/or folate deficiency) prior to enrollment.
- •The patient has documented thalassemia minor or sickle cell trait with a platelet count of less than 50,000 or greater than 130,000/mm
- •The patient has ever had any radiation treatment in the abdominal region.
- •The patient has documented prior esophageal varices or liver infarction or current liver enzymes (alanine aminotransferase [ALT]/ aspartate aminotransferase [AST]) or total bilirubin greater than 2 times the upper limit of normal (ULN), unless the patient has a diagnosis of Gilbert Syndrome.
- •The patient has any clinically significant disease, other than Gaucher disease, including cardiovascular, renal, hepatic, gastrointestinal, pulmonary, neurologic, endocrine, metabolic (e.g. hypokalemia, hypomagnesemia), or psychiatric disease, other medical conditions, or serious intercurrent illnesses that may preclude participation in the study.
- •The patient is known to have any of the following: Clinically significant coronary artery disease including history of myocardial infarction [MI] or ongoing signs or symptoms consistent with cardiac ischemia or heart failure; or clinically significant arrhythmias or conduction defect such as 2nd or 3rd degree atrioventricular (AV) block, complete bundle branch block, prolonged QTc interval, or sustained ventricular tachycardia (VT).
- •The patient has tested positive for the human immunodeficiency virus (HIV) antibody, Hepatitis C antibody, or Hepatitis B surface antigen.
- •The patient has received an investigational product within 30 days prior to enrollment.
- •The patient is scheduled for in-patient hospitalization, including elective surgery, during the study.
- •The patient has a history of cancer within 5 years of enrollment, with the exception of basal cell carcinoma.
- •The patient is pregnant or lactating.
- •The patient has received any medication that may cause QTc interval prolongation within 30 days prior to enrollment.
- •The patient is chronic tobacco chewer, smoker, alcoholic or using drugs of abuse.
- •The patient has received (acute or chronic) treatment with a cytochrome P450 3A4 (CYP3A4) inducer within 30 days prior to enrollment.
- •The patient is not a CYP2D6 poor metabolizer or is an indeterminate metabolizer with one allele identified as active, and has received any medication that is a strong inhibitor of CYP3A4 or CYP2D6 within 30 days prior to enrollment, except where a patient has been receiving chronic treatment with either a strong inhibitor of CYP3A4 or a strong inhibitor of CYP2D6 (but not both medications) for at least 30 days prior to enrollment.
- •The patient is a CYP2D6 poor metabolizer or an indeterminate metabolizer with neither allele known to be active and has received (acute or chronic) treatment with a strong inhibitor of CYP3A4 within 30 days prior to enrollment.
结局指标
主要结局
Percentage change in spleen volume (in MN)
时间窗: Week 39 vs Baseline (day 0)
次要结局
- Percentage change in liver volume (in MN)(Week 13, 26 and 39 vs Baseline (day 0))
- Absolute change in Hemoglobin level(Week 13, 26 and 39 vs Baseline (day 0))
- Percentage change in platelet count(Week 13, 26 and 39 vs Baseline (day 0))
- Percentage change in spleen volume (in MN)(Week 13 and Week 26 vs Baseline (day 0))
- Comparison of Gaucher disease assessments(Week 13, Week 26 and Week 39 vs Baseline (day 0))
- Change in Gaucher disease severity score (GD-DS3)(Week 39 and vs Baseline (day 0))
- Biomarkers assessment (chitotriosidase)(Week 4, 13, 26 and 39 vs Baseline (day 0))
- Type, incidence, severity, seriousness, and relatedness of AEs(From initiation (day 0) to closure of study (week 39))
- Assessment of Cmax, Tmax, AUC0-tau, Vd, CL & t1/2(day 1 for (cohort 1)eliglustat)
- Assessment of AUC0-tau, Cmax,ss, Tmax,ss, Vss, CL and t1/2(steady state at day 28 (week 4), Week 13 and Week 39 for eliglustat)
- Cmin,ss (Ctrough,ss) prior to dose(day 14 (Week 2), day 28 (week 4), Week 13 and 39 for eliglustat)
- Cmin,ss (Ctrough,ss) prior to dose & Cpost 1.5h(Day 14 (Week 2), day 28 (week 4), Week 13, Week 26 & 39 for eliglustat for Cohort 3 patients)
- Percentage change in GL-1((glucosylceramide) & lyso-GL-1)
研究者
Dr Prayag Shah
Amneal Pharmaceuticals Pvt. Ltd.
研究点 (10)
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