NCT03519646已完成不适用
Evaluation of the Safety in the Combination Usage of Cerdelga and Cerezyme in Type III Gaucher Disease Patients and the Efficacy on Soft Tissue Diseases.
适应症
干预措施
相关药物
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 4
- 试验地点
- 1
- 主要终点
- Adverse Events
研究概览
简要总结
Evaluation of the safety in the combination usage of Cerdelga and Cerezyme in type III Gaucher disease patients and the efficacy on soft tissue diseases.
详细描述
- This is a 3-year study and the enrollment time of this study is 24 months.
- The participants have to receive the investigational agent:Cerdelga.(Cerdelga have 21 mg、42 mg and 84 mg capsule.)
- The participants have to go back to the hospital and receive the investigational agent and take the Gaucher related biomarkers test before receiving Cerdelga, and 2 weeks, 1, 3, 6, 12, 18 and 24 months after receiving Cerdelga.
- The participants have to inform if any adverse events happened.
- The investigators will follow up by phone if adverse events happened in the participants after one months start the treatment.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 6 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Gaucher disease patients diagnosed by low B-glucocerebrosidase deficiency and GBA mutation .
- •The participant is at least 6 years old at time of enrollment.
- •Under stable Cerezyme dosage for at least for 3 months.
- •Presence of lymphadenopathy.
- •Patient (and/or their parent/legal guardian) is willing to participate and able to provide signed informed consent.
排除标准
- •The participant is CYP2D6 ultra-rapid metabolizer.
- •The participant had received substrate reduction therapy for Gaucher disease within 3 months of enrollment.
- •The participant had any clinically significant disease other than GD, including cardiovascular (especially arrhythmia), renal, liver, pulmonary, endocrinopathy, hypokalemia, or hypomagnesemia that may confound the study result.
- •The participant is pregnant or lactating.
- •The participant is known to be allergy to Cerdelga.
- •The participant use drugs that will strongly inhibit CYP2D6 or CYP3A activity .
研究组 & 干预措施
Experimental Case_Eiglustat
Experimental
Besides regular ERT, patients also need to take Eiglustat for 24 months.
干预措施: Eliglustat (Drug)
结局指标
主要结局
Adverse Events
时间窗: From date of enrollment with information consent form until 24 months or date of death from any cause, whichever came first.
Number of adverse events in patients.
次要结局
- Assessment of Gaucher related biomarkers test :Lyso GL1(30% decrease)(Baseline,1,3,6,12,18 and 24 months after receiving Cerdelga.)
- Assessment of Gaucher related biomarkers test :CCL18 (30% decrease)(Baseline,1,3,6,12,18 and 24 months after receiving Cerdelga.)
- Assessment of Gaucher related biomarkers test:Chitotriosidase(30% decrease)(Baseline,1,3,6,12,18 and 24 months after receiving Cerdelga.)
- Change in lymphadenopathy manifestations.(From date of enrollment with information consent form until 24 months or date of death from any cause, whichever came first.)
- Pharmacokinetics(Plasma concentration-time data will be obtained pre-dose (within 30 minutes prior to dosing) and at 1, 2, 6, 12, 26, 36 hours after 1st dosing, and 1,3,6,12,18 and 24 months thereafter.)
研究者
研究点 (1)
Loading locations...
相似试验
进行中（未招募）
2 期
Tucatinib+Trastuzumab+Eribulin in HER2+ MBCPretreated Unresectable Locally Advanced or Metastatic HER2+ Breast CancerBreast CancerNCT05458674Criterium, Inc.17
尚未招募
2 期
Neoadjuvant/Adjuvant Sintilimab, Nab-paclitaxel, and Gemcitabine for Resectable/Borderline Resectable Pancreatic CancerPancreatic Cancer, Stage IBPancreatic Cancer, Stage IIAPancreatic Cancer, Stage IIBPancreatic Cancer Stage IIINCT05562297Shanghai Zhongshan Hospital50
已完成
4 期
Study to Evaluate the Safety of Co-administration of GSK Biologicals' Poliorix and Infanrix in Healthy Children in RussiaPoliomyelitisNCT01094171GlaxoSmithKline400
已完成
1 期
Study to Evaluate the Safety, Pharmacokinetics, and Activity of GDC-1971 in Combination With Either Osimertinib in Participants With Unresectable, Locally Advanced, or Metastatic Non-Small Cell Lung Cancer, or With Cetuximab in Participants With Metastatic Colorectal CancerColorectal CancerNon-Small Cell Lung CancerNCT05954871Genentech, Inc.10
Unknown
2 期
New Combination Regimen of L-asparaginase, Dexamethasone, Ifosfamide, Cisplatin and Etoposide in NK/T-Cell LymphomaLymphomaNCT00933673Fudan University76