ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)
ConditionAcute Hepatic Porphyria
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age12+
SponsorAlnylam Pharmaceuticals
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Documented diagnosis of AHP, per physician's determination
Disqualifiers
Currently enrolled in a clinical trial for any investigational agent
Trial population
Patients with all types of AHP, including patients with hepatic and/or renal impairment, adolescents, elderly patient, and pregnant or lactating women.
Trial design
Design model
Cohort
Time perspective
Prospective
Treatments tested in this trial
Not listed
Trial groups
150 Participants
are grouped into 1 trial groupGroup A: Patients with AHP
Trial outcomes
Primary outcomes
1
Incidence of Selected Events of Interest in AHP Patients
Selected events of interest are defined as hepatic events (including hepatic neoplasia), renal events, thromboembolic events, and pancreatitis.
Time frame
Up to 6 yearsSecondary outcomes
1
Annualized Rate of Porphyria Attacks
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.
Time frame
Up to 6 years2
12-Item Short Form Health Survey Version 2 (SF-12 V2) (Standard Version)
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.
Time frame
Up to 6 yearsSponsors and contacts
Click on the lead sponsor to view all of their trials.
Source ClinicalTrials.gov