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

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.

Recruiting

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Key information

Conditions

Acute Hepatic Porphyria Congenital, Hereditary, and Neonatal Diseases and Abnormalities Coproporphyria, Hereditary Digestive System Diseases Genetic Diseases, Inborn Liver Diseases Metabolic Diseases Nutritional and Metabolic Diseases Porphyrias Porphyrias, Hepatic Skin Diseases Skin Diseases, Genetic Skin and Connective Tissue Diseases

Age range

12 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Clinical Trial Site, Brussels, Belgium

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

Documented diagnosis of AHP, per physician's determination

Exclusion criteria

Currently enrolled in a clinical trial for any investigational agent

Treatment and study plan

Primary outcomes

Incidence of Selected Events of Interest in AHP Patients

Time frame: Up to 6 years

Selected events of interest are defined as hepatic events (including hepatic neoplasia), renal events, thromboembolic events, and pancreatitis.

Secondary outcomes

Annualized Rate of Porphyria Attacks

Time frame: 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)

Time frame: 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.

Study contacts

Contact information is provided by the study sponsor or research team.

Alnylam Clinical Trial Information Line

CONTACT

[email protected]

1-877-ALNYLAM

Alnylam Clinical Trial Information Line

CONTACT

[email protected]

1-877-256-9526

Sponsors and collaborators

Lead sponsor

Alnylam Pharmaceuticals

Industry

Registry information

Official study title

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

Acronym: ELEVATE

Important dates

Study start: 2021
Primary completion: 2027
Study completion: 2027
First posted: May 12, 2021
Registry last updated: Apr 27, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed