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Clinical trial Recruiting at 28 sites Observational Ages 12 and up

Registry for patients with Acute Hepatic Porphyria

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

Run by Alnylam Pharmaceuticals · NCT04883905 · Data updated 2026-11-12

This study is creating a registry to track the experiences and treatment results of people with Acute Hepatic Porphyria to see how well givosiran and other approved therapies work in real life.

Quick facts

Study typeObservational
Ages12 Years and up
SexAll
Enrollment150 (estimated)
SponsorAlnylam Pharmaceuticals
Industry-sponsored
Locations30 sites (Birmingham, Alabama and 29 other locations)
StatusRecruiting
StartedApril 26, 2021
Main results expectedDecember 31, 2030
Study endsDecember 31, 2030
Registry record updatedOctober 6, 2026
Trial IDNCT04883905 on ClinicalTrials.gov

Is this trial a fit for you?

Likely a good fit

Ideal candidates for this registry are individuals with a documented diagnosis of Acute Hepatic Porphyria.

Probably not a fit

Patients currently enrolled in a clinical trial for any investigational agent may not benefit from this registry.

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This global patient registry aims to characterize the natural history and real-world clinical management of patients diagnosed with Acute Hepatic Porphyria (AHP). It will also evaluate the real-world safety and effectiveness of givosiran and other approved therapies for AHP. By collecting data from various clinical sites, the registry seeks to provide insights into patient experiences and treatment outcomes over time.

Why it matters

Potential benefit: If successful, this registry could enhance understanding of AHP and improve treatment strategies for patients.

How similar studies have gone: Other studies have shown success in utilizing patient registries to gather valuable data on rare diseases, making this approach promising.

Who can join: the full rules

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* Documented diagnosis of AHP, per physician's determination

Exclusion Criteria:

* Currently enrolled in a clinical trial for any investigational agent

Where it's running

  • Clinical Trial Site
    Birmingham, Alabama, United States     Recruiting
  • Clinical Trial Site
    Gilbert, Arizona, United States     Recruiting
  • Clinical Trial Site
    San Francisco, California, United States     Recruiting
  • Clinical Trial Site
    Baltimore, Maryland, United States     Recruiting
  • Clinical Trial Site
    Boston, Massachusetts, United States     Recruiting
  • Clinical Trial Site
    Minneapolis, Minnesota, United States     Recruiting
Show 24 more locations
  • Clinical Trial Site
    New York, New York, United States     Recruiting
  • Clinical Trial Site
    Oklahoma City, Oklahoma, United States     Recruiting
  • Clinical Trial Site
    Galveston, Texas, United States     Recruiting
  • Clinical Trial Site
    Salt Lake City, Utah, United States     Recruiting
  • Clinical Trial Site
    Seattle, Washington, United States     Recruiting
  • Clinical Trial Site
    Brussels, Belgium     Recruiting
  • Clinical Trial Site
    Leuven, Belgium     Recruiting
  • Clinical Trial Site
    Bordeaux, France     Recruiting
  • Clinical Trial Site
    Paris, France     Recruiting
  • Clinical Trial Site
    Toulouse, France     Recruiting
  • Clinical Trial Site
    Berlin, Germany     Recruiting
  • Clinical Trial Site
    Chemnitz, Germany     Recruiting
  • Clinical Trial Site
    Würzburg, Germany     Recruiting
  • Clinical Trial Site
    Milan, Italy     Recruiting
  • Clinical Trial Site
    Modena, Italy     Recruiting
  • Clinical Trial Site
    Madrid, Spain     Recruiting
  • Clinical Trial Site
    Stockholm, Sweden     Recruiting
  • Clinical Trial Site
    Zurich, Switzerland     Recruiting
  • Clinical Trial Site
    Taichung, Taiwan     Recruiting
  • Clinical Trial Site
    Taipei, Taiwan     Recruiting
  • Clinical Trial Site
    Cardiff, United Kingdom     Recruiting
  • Clinical Trial Site
    London, United Kingdom     Recruiting
  • Clinical Trial Site
    Winston-Salem, North Carolina, United States     Completed
  • Clinical Trial Site
    Philadelphia, Pennsylvania, United States     Completed

Study contacts

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

  • Using a carbohydrate-rich diet to treat acute intermittent porphyria

    This study is testing whether a high-carbohydrate diet can help people with acute intermittent porphyria feel better by looking at changes in their symptoms and health markers.

    Recruiting at 3 sites Nordlandssykehuset HF · Bodø, Nordland and 2 other locations · NCT06273644

  • Identifying genes that affect Acute Intermittent Porphyria

    This study is trying to find out which genes might make people more likely to have attacks of Acute Intermittent Porphyria or help protect them from it, and it's for anyone aged 12 and older who has symptoms or is related to someone with the condition.

    Recruiting Icahn School of Medicine at Mount Sinai · New York, New York · NCT05502133

How to take part

  1. Review the eligibility criteria above with your treating physician.
  2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
  3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.
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Last reviewed 2026-11-14 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.