ELEVATE AHP Registry
Research type
Research Study
Full title
ELEVATE, a global observational longitudinal prospective registry of patients with acute hepatic porphyria (AHP)
IRAS ID
295247
Contact name
Charles Rees
Contact email
Sponsor organisation
Alnylam Pharmaceuticals Inc.
Clinicaltrials.gov Identifier
N/A, N/A
Duration of Study in the UK
5 years, 3 months, 15 days
Research summary
The purpose of this prospective observational study is to have a better understanding of the natural history and clinical management of AHP (acute hepatic porphyria) as part of routine care. This protocol will not recommend the use of any specific treatments, visits, or procedures. No medication is provided as part of registry participation.
Patients are expected to contribute data for the duration of the registry or until study discontinuation (eg, due to death, withdrawal of consent, loss to follow-up, enrollment in a clinical trial with an investigative treatment during the study). Registry data will be collected at the time of a routine clinical encounter or by referencing the medical record and entered in to the electronic data capture (EDC) at least once every 12 months using electronic case report forms (eCRFs).Porphyria attacks will be collected for the period of 12 months prior to study enrollment and during follow-up including the following: number of porphyria attacks; duration and treatment for each attack (if available); location of care (eg, hospital, urgent healthcare, home).
Approximately 150 people with AHP will be included in this study at several different study centres internationally. There will be about 25 study centres participating worldwide.REC name
East of England - Cambridge East Research Ethics Committee
REC reference
21/EE/0137
Date of REC Opinion
2 Jul 2021
REC opinion
Further Information Favourable Opinion