EUCTR2015-001578-17-ES进行中（未招募）1 期
An Open-label, Multicenter, Single-arm, Phase 4 Study of the Effect of Treatment with Velaglucerase alfa on Bone-related Pathology in Treatment-naïve Patients with Type 1 Gaucher Disease
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 21
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
- •Each patient must meet the following criteria to be enrolled in this study.
- •1. The patient has a documented diagnosis of type 1 Gaucher disease, as documented by
- •deficient GCB activity in leukocytes (whole blood only) or cultured skin fibroblasts.
- •Diagnosis by dry blood spot test is not sufficient. Diagnosis may be based on results obtained
- •prior to screening if documented in the patient?s medical history.
- •2. Patients must have a LS BMD Z-score <-1 as measured by DXA during the screening phase.
- •3. Patient is treatment-naïve, ie, has not received ERT or SRT in the 12 months prior to
- •enrollment.
- •4. The patient is ?16 and ?65 years of age. For patients ?16 and <18 years of age a Tanner
- •stage of ?4 must be established.
- •5. Female patients of child bearing potential must agree to use a medically acceptable method
- •of contraception at all times during the study.
- •6. The patient, and the patient?s parent(s) or legal guardian(s), if applicable, understands the
- •nature, scope, and possible consequences of the study and has provided written informed
- •consent that has been approved by the Institutional Review Board/Independent Ethics
- •Committee (IRB/IEC).
- •7. The patient must be sufficiently cooperative to participate in this clinical study as judged by
- •the investigator.
- •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 30
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •Patients who meet any of the following criteria will be excluded from the study.
- •1. Neurological symptoms indicating that the patient has type 3 Gaucher disease.
- •2. A significant comorbidity, which, as determined by the investigator, might affect study data
- •or confound the study results (eg, malignancies, primary biliary cirrhosis, autoimmune liver
- •disease, etc).
- •3. Any osteoporosis-specific treatment (eg, bisphosphonates) or treatment with erythropoietin
- •(or erythropoietin-like substances) during the past year.
- •4. Structural, joint-associated bone damage of such extent and severity that the investigator
- •deems it could impact participation in the study and assessment of relevant study endpoints
- •(eg, pain).
- •5. The patient is pregnant or lactating.
- •6. The patient has had a splenectomy.
- •7. The patient is enrolled in another clinical study that involves clinical investigations or use of
- •any investigational product (drug or device) within 30 days prior to study enrollment or at
- •any time during the study.
研究者
相似试验
进行中（未招募）
1 期
An Open-Label, Phase IV Study of Velaglucerase alfa on Bone Related Pathology in Adult, Treatment-Naïve Patients with Type 1 Gaucher DiseaseGaucher DiseaseMedDRA version: 20.0Level: PTClassification code 10075697Term: Gaucher's disease type ISystem Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2015-001578-17-ITSHIRE HUMAN GENETIC THERAPIES, INC40
进行中（未招募）
1 期
An Open-Label, Phase IV Study of Velaglucerase alfa on Bone Related Pathology in Adult, Treatment-Naïve Patients with Type 1 Gaucher DiseaseMedDRA version: 20.0Level: PTClassification code 10075697Term: Gaucher's disease type ISystem Organ Class: 10010331 - Congenital, familial and genetic disordersGaucher DiseaseEUCTR2015-001578-17-DEShire Human Genetic Therapies, Inc.40
进行中（未招募）
1 期
An Open-Label, Phase IV Study of Velaglucerase alfa on Bone Related Pathology in Adult, Treatment-Naïve Patients with Type 1 Gaucher DiseaseMedDRA version: 20.0Level: PTClassification code 10075697Term: Gaucher's disease type ISystem Organ Class: 10010331 - Congenital, familial and genetic disordersGaucher DiseaseEUCTR2015-001578-17-GBShire Human Genetic Therapies, Inc.19
进行中（未招募）
1 期
A study on the immune response and safety of a vaccine against Neisseria meningitidis serogroup B infection in healthy infants from 2 months of ageHealthy volunteers (active immunization of infants against invasive meningococcal disease (IMD) due to N. meningitidis serogroup B infection).EUCTR2023-000764-58-Outside-EU/EEAGlaxoSmithKline Biologicals SA
招募中
4 期
A Study of Romosozumab (EVENITY®) in Postmenopausal Women in India with Osteoporosis at a High Risk of FractureCTRI/2023/03/050576Amgen Technology Pvt. Ltd.