EUCTR2005-001651-37-HU进行中（未招募）不适用
Open-label, non-comparative, multi-center study to evaluate the long-term efficacy, safety and tolerability of oral miglustat as a maintenance therapy after a switch from enzyme replacement therapy in adult patients with stable type 1 Gaucher disease.
适应症
相关药物
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 50
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
- •Eligible patients must meet all of the following inclusion criteria:
- •1.Male or female patients aged 18 years or older.
- •2.Type 1 Gaucher disease, diagnosed by glucocerebrosidase assay or molecular analysis of the glucocerebrosidase gene.
- •3.Treatment with ERT for at least 3 years, with a stable dose regimen for at least the last 6 months.
- •4.Clinically and biologically stable disease for at least the previous 2 years, with at least two time point assessments (including Baseline as one potential time point), defined as:
- •Stable organomegaly, assessed by magnetic resonance imaging (MRI) or computed tomography (CT):
- •-Liver volume within 10% of the mean.
- •-Spleen volume within 10% of the mean.
- •Free of documented symptomatic bone disease.
- •Mean hemoglobin level > 11g/dl.
- •Mean platelet count > 100x10^9 /l.
- •Chitotriosidase activity within 20% of the mean.
- •If chitotriosidase is not available (in the case of chitotriosidase deficiency, or if it was not determined), other relevant biomarkers (e.g., ACE, TRAP and ferritin) could be considered.
- •5.Written informed consent.
- •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
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range
排除标准
- •Eligible patients must meet none of the following exclusion criteria:
- •1.History or evidence of oculomotor gaze palsy, ataxia or other clinical manifestations typically associated with neuronopathic type 3 Gaucher disease.
- •2.Not ambulant patients, or with documented symptomatic bone disease.
- •3.Splenectomy before 18 years of age for splenomegaly and/or thrombocytopenia.
- •4.Peripheral polyneuropathy (not mononeuropathy) documented both by compatible clinical signs and/or symptoms, and electrodiagnosis (EDX).
- •5.Patients (males and females) who do not agree to use reliable contraception throughout the study and for 3 months after study drug discontinuation.
- •6.Female patients who are pregnant or breast feeding, or without a pregnancy test prior to Day 1.
- •7.History of significant lactose intolerance.
- •8.Clinically significant diarrhea (>3 liquid stools per day for >7 days) without definable cause within 6 months prior to Day 1, or a history of clinically relevant gastrointestinal disorders.
- •9.History of cataracts, or known increased risk of cataract formation.
- •10.Severe renal impairment with a creatinine clearance <30 ml/min/1.73mP2P
- •11.Concomitant active medical condition such as HIV or hepatitis B/C that would render patients unsuitable for study.
- •12.Previous treatment with miglustat.
- •13.Known hypersensitivity to miglustat or any excipients.
- •14.Patients with current alcohol or drug abuse or dependence.
- •15.Current treatment with another investigational drug, or within 3 months prior to Day 1.
研究者
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Open-label, non-comparative, multi-center study to evaluate the long-term efficacy, safety and tolerability of oral miglustat as a maintenance therapy after a switch from enzyme replacement therapy in adult patients with stable type 1 Gaucher disease. - MAINTENANCEGaucher disease is the most common of the glycosphingolipid storage diseases and has autosomal recessive inheritance. For a more precise description, please refer to page 16 of the protocol:1BACKGROUND AND RATIONALE1.1DiseaseMedDRA version: 8.1Level: LLTClassification code 10018048Term: Gaucher's diseaseEUCTR2005-001651-37-CZActelion Pharmaceuticals Ltd50
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