ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)
- Conditions
- Acute Hepatic Porphyria
- Registration Number
- NCT04883905
- Lead Sponsor
- Alnylam Pharmaceuticals
- Brief Summary
This global patient registry is being conducted to characterize the natural history and real-world clinical management of patients with AHP, and to further characterize the real-world safety and effectiveness of givosiran and other approved AHP therapies.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 150
- Documented diagnosis of AHP, per physician's determination
- Currently enrolled in a clinical trial for any investigational agent
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Incidence of Selected Events of Interest in AHP Patients Up to 6 years Selected events of interest are defined as hepatic events (including hepatic neoplasia), renal events, thromboembolic events, and pancreatitis.
- Secondary Outcome Measures
Name Time Method Annualized Rate of Porphyria Attacks Up to 6 years Porphyria attacks are defined as meeting all of the following criteria: an acute episode of neurovisceral pain in the abdomen, back, chest, extremities and/or limbs, no other medically determined cause, and requires treatment with intravenous (IV) dextrose or hemin, carbohydrates, or analgesics, or other medications such as antiemetics at a dose or frequency beyond the participant's usual daily porphyria management.
12-Item Short Form Health Survey Version 2 (SF-12 V2) (Standard Version) Up to 6 years S-12 V2 is a 12-question measure capturing global quality of life and overall health status and evaluates the following 8 domains: physical functioning, role physical, bodily pain, general health, vitality, social functioning, role emotional, and mental health.
Trial Locations
- Locations (1)
Clinical Trial Site
🇬🇧London, United Kingdom