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

Acute Hepatic Porphyria Clinical Trial

— ELEVATE  

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04883905
• Other study ID #: ALN-AS1-006
• Status: Recruiting
• Start date: April 26, 2021
• Est. completion date: April 2027

Study information

• Verified date: May 2024
• Source: Alnylam Pharmaceuticals
• Contact: Alnylam Clinical Trial Information Line
• Phone: 1-877-ALNYLAM
• Email: clinicaltrials[@]alnylam.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 150
• Est. completion date: April 2027
• Est. primary completion date: April 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years and older

Eligibility

Inclusion Criteria:

• Documented diagnosis of AHP, per physician's determination

Exclusion Criteria:

• Currently enrolled in a clinical trial for any investigational agent

Related Conditions & MeSH terms

• Acute Hepatic Porphyria
• Porphyria, Erythropoietic
• Porphyrias

Locations

Country	Name	City	State
Belgium	Clinical Trial Site	Leuven
France	Clinical Trial Site	Bordeaux
France	Clinical Trial Site	Paris
France	Clinical Trial Site	Toulouse
Germany	Clinical Trial Site	Chemnitz
Germany	Clinical Trial Site	Münster
Germany	Clinical Trial Site	Würzburg
Italy	Clinical Trial Site	Milan
Italy	Clinical Trial Site	Modena
Sweden	Clinical Trial Site	Stockholm
Switzerland	Clinical Trial Site	Zürich
United Kingdom	Clinical Trial Site	Cardiff
United Kingdom	Clinical Trial Site	London
United States	Clinical Trial Site	Birmingham	Alabama
United States	Clinical Trial Site	Boston	Massachusetts
United States	Clinical Trial Site	Galveston	Texas
United States	Clinical Trial Site	Gilbert	Arizona
United States	Clinical Trial Site	Minneapolis	Minnesota
United States	Clinical Trial Site	New York	New York
United States	Clinical Trial Site	Oklahoma City	Oklahoma
United States	Clinical Trial Site	Philadelphia	Pennsylvania
United States	Clinical Trial Site	Salt Lake City	Utah
United States	Clinical Trial Site	San Francisco	California
United States	Clinical Trial Site	Seattle	Washington
United States	Clinical Trial Site	Winston-Salem	North Carolina

Sponsors (1)

Lead Sponsor	Collaborator
Alnylam Pharmaceuticals

Countries where clinical trial is conducted

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

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of Selected Events of Interest in AHP Patients	Selected events of interest are defined as hepatic events, renal events, and pancreatitis.	Up to 6 years
Secondary	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.	Up to 6 years
Secondary	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.	Up to 6 years