ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)

ELEVATE, a Global Observational Longitudinal Prospective Registry of Patients With Acute Hepatic Porphyria (AHP)

Status

Recruiting

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT04883905

Acronym

ELEVATE

Enrollment

150

Registered

2021-05-12

Start date

2021-04-26

Completion date

2030-12-31

Last updated

2026-09-08

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Acute Hepatic Porphyria

Keywords

Givosiran, AHP, Acute hepatic porphyria

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.

Interventions

None listed

Study design

Observational model

COHORT

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

12 Years to No maximum

Healthy volunteers

Inclusion criteria

* Documented diagnosis of AHP, per physician's determination

Exclusion criteria

* Currently enrolled in a clinical trial for any investigational agent

Design outcomes

Primary

Measure	Time frame	Description
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

Measure	Time frame	Description
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.

Countries

Belgium, France, Germany, Italy, Spain, Sweden, Switzerland, Taiwan, United Kingdom, United States

Contacts

CONTACTAlnylam Clinical Trial Information Line

clinicaltrials@alnylam.com1-877-ALNYLAM

STUDY_DIRECTORMedical Director

Alnylam Pharmaceuticals

Outcome results