EUCTR2014-000350-11-DE进行中（未招募）不适用
An open label, randomized study to investigate the safety of weekly pentosan polysulfate injections in adult patients with Mucopolysaccharidosis Type I receiving enzyme replacement therapy.
Multiplex Pharma Holdings LLC0 个研究点目标入组 6 人开始时间: 2014年3月10日最近更新:
适应症
相关药物
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 6
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
- •1. Male or female.
- •2. Age =18 years.
- •3.Diagnosed as having Mucopolysaccharidosis Type I (alpha-L-Iduronidase deficiency) and have been treated with enzyme replacement therapy (ERT) for a minimum of 24 months.
- •4. Joint pain and/or reduced joint range of motion at time of enrolment according to the investigator.
- •5. Written informed consent upon enrolment.
- •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 6
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1. History of hypersensitivity to pentosan polysulfate or any of the excipients.
- •2. Known allergic reaction to heparin or pentosan polysulfate e.g. heparin induced thrombocytopenia.
- •3. Clinically relevant concomitant severe condition (with the exception of signs and symptoms relating to MPSI) e.g. gastrointestinal (including gastrointestinal ulcerations, polyps, or diverticula), renal, hepatic (ALT and/or AST >10 times upper limit of reference range), pancreatic, pulmonary, cardiovascular (including endocarditis lenta), aneurysm, cerebral hemorrhage, hematological (including thrombocytopenia, hemophilia, hemorrhagic diathesis), endocrinological, suspected tumors with risk of bleeding, neurological, or psychiatric conditions in the opinion of the investigator.
- •4. Planned invasive procedure other than blood sampling.
- •5. History of or planned bone marrow transplant.
- •6. Clinically relevant abnormal laboratory test results including liver enzymes, thrombocytopenia, prolonged clotting time.
- •7. Intake of anti-inflammatory agents e.g. corticosteroids, non-steroidal anti-inflammatory drugs (NSAIDs) other than ibuprofen or paracetamol within 10 half lives (t½) of the drug prior to first dosing and throughout the study.
- •8. Intake of anti-thrombotic agents e.g. heparin, tissue plasminogen activator (t-PA), streptokinase, aspirin within 10 half lives (t½) of the drug prior to first dosing and throughout the study. Coumarin anticoagulants are permitted.
- •9. Known history or presence of alcohol or substance abuse.
- •10. Known hepatitis B or C or human immunodeficiency virus (HIV) or syphilis seropositivity. Note: testing for hepatitis and HIV will be performed as part of the screening procedures.
- •11. For females: pregnancy or lactation.
- •12. For females of childbearing potential: not willing and able to use a contraceptive method, which the investigator considers reliable. Reliable methods for women are hormonal contraceptives, surgical intervention (e.g. tubal ligation), intrauterine device (IUD) and sexual abstinence. Women must use a reliable method from 6 weeks before the first administration of study medication until 3 weeks after the last administration of the study medication.
- •13. Participation in another clinical study involving a new chemical entity within 3 months and for studies involving biologicals 6 months prior to enrolment in this study.
- •14. The subject has received vaccination(s) within 1 month prior to enrollment, or is unwilling to postpone vaccinations for the duration of the study.
- •15. Subjects who are unable to comply with the requirements of the study or who in the opinion of the investigator should not participate in the study.
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