EUCTR2015-001578-17-DE进行中（未招募）1 期
An Open-label, Multicenter, Single-arm, Phase 4 Study of the Effect ofTreatment with Velaglucerase alfa on Bone-related Pathology inTreatment-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
- 40
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 only dry blood spot test is insufficient. 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 or BMD T-score of < -1 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 =18 and =70 years of age.
- •5. Female patients of childbearing potential must agree to use a medically acceptable method of contraception at all times during the study. (See Section 6.8.3 for acceptable methods of contraception).
- •6. The patient, or patient’s legally authorized representative(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? no
- •Number of subjects for this age range:
- •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 10
排除标准
- •Patients who meet any of the following criteria will be excluded from the
- •1. Neurological symptoms indicating that the patient may have 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.
- •8. Severe vitamin D deficiency to the level that would be expected to
- •result in osteomalacia (vitamin D < 10 ng/mL [25 nmol/L]). If there is
- •mild vitamin D insufficiency at screening (vitamin D > 10 and < 30
- •ng/mL) treat with 4000 IU vitamin D per day for 1 month and rescreen.
- •9. The patient has previously interrupted ERT for safety reasons.
- •10. The patient has had hypersensitivity to the active substance or to
- •any of the excipients.
研究者
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