Evidence-based Assessment of Medication Sensitivity in Acute Hepatic Porphyria
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 60
- 试验地点
- 1
- 主要终点
- Use of Medications of Interest Form
研究概览
简要总结
This is an observational study collecting patient/caregiver reports on suspected medication/drug-induced acute porphyria attacks, as well as safe use of drugs previously labeled "unsafe" or with unknown risk. Participants will be recruited through the RDCRN Contact Registry for the Porphyrias Consortium. The study will be advertised on the Consortium website and through the American Porphyria Foundation's social media network.
详细描述
Contact Registrants will be sent an email invitation describing the study. A unique link will be generated for each registrant and included in the email invitation. After 2 weeks, a second email invitation will be sent to the registrants who have not yet participated. After 1 month, a third email invitation will be sent to the registrants who have not yet participated. We may send additional emails periodically (e.g. every 6-12 months) to subjects enrolled to remind them to complete new surveys if they have taken any other medications of interest or have experienced any new reactions/attacks.
If the registrant wants to participate in the study, he/she will follow the survey link in the email invitation, which directs him/her to an IRB-approved online consent form. If the participant consents to participate, he/she will be directed to online forms to report general information (ex. age, sex, test results, family history), suspected medication-related acute attacks, and "uneventful use of possibly risky medication". If a patient has multiple medication-related acute attacks, the patient/caregiver will be able to submit multiple report forms that will be linked. In addition to the report forms, patients will be asked to review a list of "Medications of Interest" and report current or past use.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Other
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Able to read and write in English
- •Enrolled in the RDCRN PC Contact Registry
- •Self-reported patient or caregiver of patient with acute porphyria
- •There are no restrictions on age, sex or ethnicity. However, the following criteria will be applied to those reports that are considered evaluable (analyzed and scored):
- •Medication name must be specified
- •The data elements required in the scoring system must be complete
排除标准
- •Unable to provide informed consent and complete forms
结局指标
主要结局
Use of Medications of Interest Form
时间窗: 5 years from study start
Designed to assess medications used by category and effects of each.
Acute Attack Form
时间窗: 5 years from study start
Designed to capture PBG levels, characteristics of the acute attack, timing of medication administration, and pharmacological information on the medication.
Uneventful Use of Possible Risky Medication Form
时间窗: 5 years from study start
Designed to capture details of medication in question, ALA/PGB results during use of medication in questions, concomitant medication use, diet history, and alcohol use.
次要结局
未报告次要终点