Registry for Acute Hepatic Porphyria
(ELEVATE Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to gather real-world information about acute hepatic porphyria (AHP), a rare liver condition, from patients. Researchers seek to understand how AHP affects individuals over time and how treatments, such as givosiran (an RNA interference therapy) and other approved therapies, perform in everyday life. The trial seeks participants diagnosed with AHP by their doctor who are not part of another trial. This information will help improve care for those living with AHP. As a Phase 4 trial, this research focuses on understanding how the already FDA-approved treatment benefits more patients in real-world settings, offering valuable insights for those considering participation.
Why are researchers excited about this trial?
Researchers are excited about the ELEVATE registry for patients with Acute Hepatic Porphyria (AHP) because it aims to gather comprehensive data that could lead to better understanding and management of this rare condition. Unlike standard treatments that often focus on managing symptoms, this registry could provide insights into patient experiences, long-term outcomes, and the effectiveness of various treatments over time. This could pave the way for developing more targeted therapies, improving patient care, and potentially introducing innovative treatments that go beyond traditional symptom control.
Who Is on the Research Team?
Medical Director
Principal Investigator
Alnylam Pharmaceuticals
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Registry Observation
Participants are observed to characterize the natural history and real-world clinical management of AHP
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Find a Clinic Near You
Who Is Running the Clinical Trial?
Alnylam Pharmaceuticals
Lead Sponsor
Dr. Yvonne Greenstreet
Alnylam Pharmaceuticals
Chief Executive Officer since 2021
MD from the University of Leeds, MBA from INSEAD
Dr. Pushkal Garg
Alnylam Pharmaceuticals
Chief Medical Officer since 2016
MD from Columbia University