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Recruiting NCT04883905

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

Observational Acute Hepatic Porphyria

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
This is an observational study: the protocol does not assign a study treatment.
Who it may be relevant to
Registry conditions: Acute Hepatic Porphyria. Basic parameters: from 12 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States, Belgium, France, Germany, Italy +5
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Official title

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

Overview

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.

Primary outcome measures

  • Incidence of Selected Events of Interest in AHP Patients [Time frame: Up to 6 years]
Secondary outcome measures (2)
  • Annualized Rate of Porphyria Attacks [Time frame: Up to 6 years]
  • 12-Item Short Form Health Survey Version 2 (SF-12 V2) (Standard Version) [Time frame: Up to 6 years]

Eligibility criteria

Inclusion criteria

  • Documented diagnosis of AHP, per physician's determination

Exclusion criteria

  • Currently enrolled in a clinical trial for any investigational agent

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model
Cohort

Study locations

United States · 13 centers
  • Clinical Trial Site — Birmingham
  • Clinical Trial Site — Gilbert
  • Clinical Trial Site — San Francisco
  • Clinical Trial Site — Baltimore
  • Clinical Trial Site — Boston
  • Clinical Trial Site — Minneapolis
  • Clinical Trial Site — New York
  • Clinical Trial Site — Winston-Salem
  • … and 5 more centers
France · 3 centers
  • Clinical Trial Site — Bordeaux
  • Clinical Trial Site — Paris
  • Clinical Trial Site — Toulouse
Germany · 3 centers
  • Clinical Trial Site — Berlin
  • Clinical Trial Site — Chemnitz
  • Clinical Trial Site — Würzburg
Belgium · 2 centers
  • Clinical Trial Site — Brussels
  • Clinical Trial Site — Leuven
Italy · 2 centers
  • Clinical Trial Site — Milan
  • Clinical Trial Site — Modena
Taiwan · 2 centers
  • Clinical Trial Site — Taichung
  • Clinical Trial Site — Taipei
United Kingdom · 2 centers
  • Clinical Trial Site — Cardiff
  • Clinical Trial Site — London
Spain · 1 center
  • Clinical Trial Site — Madrid
Sweden · 1 center
  • Clinical Trial Site — Stockholm
Switzerland · 1 center
  • Clinical Trial Site — Zurich

Identifiers

NCT: NCT04883905 · ALN-AS1-006 · EUPAS43201

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗