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

April 17, 2024 updated by: Alnylam Pharmaceuticals

ELEVATE, a Global Observational Longitudinal Prospective 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.

Study Overview

Status

Recruiting

Conditions

Study Type

Observational

Enrollment (Estimated)

150

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • Belgium
      • Leuven, Belgium
        • Recruiting
        • Clinical Trial Site
  • France
      • Bordeaux, France
        • Recruiting
        • Clinical Trial Site
      • Paris, France
        • Recruiting
        • Clinical Trial Site
      • Toulouse, France
        • Recruiting
        • Clinical Trial Site
  • Germany
      • Chemnitz, Germany
        • Recruiting
        • Clinical Trial Site
      • Münster, Germany
        • Recruiting
        • Clinical Trial Site
      • Würzburg, Germany
        • Recruiting
        • Clinical Trial Site
  • Italy
      • Milan, Italy
        • Recruiting
        • Clinical Trial Site
      • Modena, Italy
        • Recruiting
        • Clinical Trial Site
  • Sweden
      • Stockholm, Sweden
        • Recruiting
        • Clinical Trial Site
  • United Kingdom
      • Cardiff, United Kingdom
        • Recruiting
        • Clinical Trial Site
      • London, United Kingdom
        • Recruiting
        • Clinical Trial Site
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35233
        • Recruiting
        • Clinical Trial Site
    • Arizona
      • Gilbert, Arizona, United States, 85234
        • Recruiting
        • Clinical Trial Site
    • California
      • San Francisco, California, United States, 90074
        • Recruiting
        • Clinical Trial Site
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Recruiting
        • Clinical Trial Site
    • Minnesota
      • Minneapolis, Minnesota, United States, 55455
        • Recruiting
        • Clinical Trial Site
    • New York
      • New York, New York, United States, 10029
        • Recruiting
        • Clinical Trial Site
    • North Carolina
      • Winston-Salem, North Carolina, United States, 27157
        • Recruiting
        • Clinical Trial Site
    • Oklahoma
      • Oklahoma City, Oklahoma, United States, 73120
        • Recruiting
        • Clinical Trial Site
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19107
        • Recruiting
        • Clinical Trial Site
    • Texas
      • Galveston, Texas, United States, 77555
        • Recruiting
        • Clinical Trial Site
    • Utah
      • Salt Lake City, Utah, United States, 84112
        • Recruiting
        • Clinical Trial Site
    • Washington
      • Seattle, Washington, United States, 98195
        • Recruiting
        • Clinical Trial Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

12 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

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.

Description

Inclusion Criteria:

  • Documented diagnosis of AHP, per physician's determination

Exclusion Criteria:

  • Currently enrolled in a clinical trial for any investigational agent

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
Patients with AHP
Patients with a diagnosis of AHP will be eligible for the study and will be managed and treated per routine clinical practice.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Incidence of Selected Events of Interest in AHP Patients
Time Frame: Up to 6 years
Selected events of interest are defined as hepatic events, renal events, and pancreatitis.
Up to 6 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
Up to 6 years
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.
Up to 6 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Alnylam Pharmaceuticals

Investigators

  • Study Director: Medical Director, Alnylam Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

April 26, 2021

Primary Completion (Estimated)

April 1, 2027

Study Completion (Estimated)

April 1, 2027

Study Registration Dates

First Submitted

May 7, 2021

First Submitted That Met QC Criteria

May 7, 2021

First Posted (Actual)

May 12, 2021

Study Record Updates

Last Update Posted (Actual)

April 18, 2024

Last Update Submitted That Met QC Criteria

April 17, 2024

Last Verified

April 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ALN-AS1-006

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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