NCT04552691Approved For Marketing不适用
Expanded Access Treatment With Open-Label Pegunigalsidase Alfa for Fabry Patients
适应症
相关药物
试验速览
- 阶段
- 不适用
- 状态
- Approved For Marketing
- 试验地点
- 11
研究概览
简要总结
The objective of this treatment protocol is to provide guidance to Treating Physicians who seek access to pegunigalsidase alfa for Fabry patients whose clinical condition, in the opinion of the Treating Physician, requires treatment with enzyme replacement therapy (ERT) with pegunigalsidase alfa and a) cannot be adequately treated with currently approved FDA products and/or b) are not able or willing to participate in any of the on-going clinical trials in the United States.
研究设计
- 研究类型
- Expanded Access
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •In the opinion of the Treating Physician, the patient cannot be adequately treated with any FDA approved drugs for Fabry and is not able to enroll in any current clinical trial for Fabry disease.
- •Patient (or legal guardian) is able to sign an informed consent prior to treatment.
- •A documented diagnosis of Fabry disease.
- •Preferably two, but at minimum 1, historical serum creatinine evaluations in the last 2 years with the latest value within the last 6 months.
- •Female patients and male patients whose co-partners are of child-bearing potential agree to use a medically acceptable method of contraception, not including the rhythm method. Acceptable methods of contraception include hormonal products, intrauterine device, or male or female condoms. Contraception should be used for 90 days after treatment discontinuation.
排除标准
- •Patients enrolled and currently treated in Study PB-102-F20, and patients enrolled and currently treated in Extension Study PB-102-F60
- •Patients who currently are on treatment under any other ongoing clinical trials of PRX-102
- •History of Type 1 (anaphylaxis or anaphylactoid like) life-threatening hypersensitivity during previous exposure to other ERTs which could not be handled with medication
- •Women who are breastfeeding may not participate unless they agree to stop breastfeeding.
- •Women who are currently pregnant.
研究者
研究点 (11)
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