Clinical trial NCT04883905: ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)
Official title: ELEVATE, a Global Observational Longitudinal Prospective Registry of Patients With Acute Hepatic Porphyria (AHP)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Acute Hepatic Porphyria
Keywords: Givosiran
AHP
Acute hepatic porphyria
Lead sponsor: Alnylam Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2021-05-12
Enrollment: 150
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with all types of AHP, including patients with hepatic and/or renal impairment, adolescents, elderly patient, and pregnant or lactating women.

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.


Eligibility criteria: Inclusion Criteria:

* Documented diagnosis of AHP, per physician's determination

Exclusion Criteria:

* Currently enrolled in a clinical trial for any investigational agent

Primary outcomes:
measure: Incidence of Selected Events of Interest in AHP Patients
timeFrame: Up to 6 years
Secondary outcomes:
measure: Annualized Rate of Porphyria Attacks
timeFrame: Up to 6 years
measure: 12-Item Short Form Health Survey Version 2 (SF-12 V2) (Standard Version)
timeFrame: Up to 6 years
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: Clinical Trial Site
state: Alabama
city: Gilbert
country: United States
facility: Clinical Trial Site
state: Arizona
city: San Francisco
country: United States
facility: Clinical Trial Site
state: California
city: Baltimore
country: United States
facility: Clinical Trial Site
state: Maryland
city: Boston
country: United States
facility: Clinical Trial Site
state: Massachusetts
city: Minneapolis
country: United States
facility: Clinical Trial Site
state: Minnesota
city: New York
country: United States
facility: Clinical Trial Site
state: New York
city: Winston-Salem
country: United States
facility: Clinical Trial Site
state: North Carolina
city: Oklahoma City
country: United States
facility: Clinical Trial Site
state: Oklahoma
city: Philadelphia
country: United States
facility: Clinical Trial Site
state: Pennsylvania
city: Galveston
country: United States
facility: Clinical Trial Site
state: Texas
city: Salt Lake City
country: United States
facility: Clinical Trial Site
state: Utah
city: Seattle
country: United States
facility: Clinical Trial Site
state: Washington
city: Leuven
country: Belgium
facility: Clinical Trial Site
city: Bordeaux
country: France
facility: Clinical Trial Site
city: Paris
country: France
facility: Clinical Trial Site
city: Toulouse
country: France
facility: Clinical Trial Site
city: Chemnitz
country: Germany
facility: Clinical Trial Site
city: Münster
country: Germany
facility: Clinical Trial Site
city: Würzburg
country: Germany
facility: Clinical Trial Site
city: Milan
country: Italy
facility: Clinical Trial Site
city: Modena
country: Italy
facility: Clinical Trial Site
city: Stockholm
country: Sweden
facility: Clinical Trial Site
city: Zürich
country: Switzerland
facility: Clinical Trial Site
city: Taichung
country: Taiwan
facility: Clinical Trial Site
city: Taipei
country: Taiwan
facility: Clinical Trial Site
city: Cardiff
country: United Kingdom
facility: Clinical Trial Site
city: London
country: United Kingdom
facility: Clinical Trial Site
Study officials:
affiliation: Alnylam Pharmaceuticals
name: Medical Director
role: STUDY_DIRECTOR
References: