Acute Hepatic PorphyriaELEVATENCT04883905
ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)
An observational study of Acute Hepatic Porphyria, run by Alnylam Pharmaceuticals at 30 sites in 10 countries.
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. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Acute Hepatic Porphyria
- How many people
- 150 participants
- Where
- 30 sitesin 10 countries
- Study dates
- 26 Apr 2021to 31 Dec 2030
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients with all types of AHP, including patients with hepatic and/or renal impairment, adolescents, elderly patient, and pregnant or lactating women.
Who may be able to join
- Documented diagnosis of AHP, per physician's determination
Who cannot join
- Currently enrolled in a clinical trial for any investigational agent
Trial locations and contacts
30 sites in 10 countries. Contact the study team or a site near you to ask about taking part.
30 sites in 10 countries
United States13 sites, 11 recruiting
- Recruiting
Clinical Trial Site
Baltimore, Maryland, 21205
- Recruiting
Clinical Trial Site
Birmingham, Alabama, 35233
- Recruiting
Clinical Trial Site
Boston, Massachusetts, 02114
- Recruiting
Clinical Trial Site
Galveston, Texas, 77555
- Recruiting
Clinical Trial Site
Gilbert, Arizona, 85234
- Recruiting
Clinical Trial Site
Minneapolis, Minnesota, 55455
- Recruiting
Clinical Trial Site
New York, New York, 10029
- Recruiting
Clinical Trial Site
Oklahoma City, Oklahoma, 73120
- Completed
Clinical Trial Site
Philadelphia, Pennsylvania, 19107
- Recruiting
Clinical Trial Site
Salt Lake City, Utah, 84112
- Recruiting
Clinical Trial Site
San Francisco, California, 90074
- Recruiting
Clinical Trial Site
Seattle, Washington, 98195
- Completed
Clinical Trial Site
Winston-Salem, North Carolina, 27157
France3 sites, 3 recruiting
- Recruiting
Clinical Trial Site
Bordeaux
- Recruiting
Clinical Trial Site
Paris
- Recruiting
Clinical Trial Site
Toulouse
Germany3 sites, 3 recruiting
- Recruiting
Clinical Trial Site
Berlin
- Recruiting
Clinical Trial Site
Chemnitz
- Recruiting
Clinical Trial Site
Würzburg
Belgium2 sites, 2 recruiting
- Recruiting
Clinical Trial Site
Brussels
- Recruiting
Clinical Trial Site
Leuven
Italy2 sites, 2 recruiting
- Recruiting
Clinical Trial Site
Milan
- Recruiting
Clinical Trial Site
Modena
Taiwan2 sites, 2 recruiting
- Recruiting
Clinical Trial Site
Taichung
- Recruiting
Clinical Trial Site
Taipei
United Kingdom2 sites, 2 recruiting
- Recruiting
Clinical Trial Site
Cardiff
- Recruiting
Clinical Trial Site
London
About this study
Official title: 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.
- Conditions
- Acute Hepatic Porphyria
- Keywords
- Givosiran, AHP, Acute hepatic porphyria
- Medical subject headings
- Coproporphyria, Hereditary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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 being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Selected Events of Interest in AHP Patients
Selected events of interest are defined as hepatic events (including hepatic neoplasia), renal events, thromboembolic events, and pancreatitis.
Measured: Up to 6 years
Secondary outcomes (2)
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.
Measured: Up to 6 years
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.
Measured: Up to 6 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 May 2021
- Study start26 Apr 2021
- Primary completion31 Dec 2030 (estimated)
- Study completion31 Dec 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Hepatic PorphyriaStudies of the same condition, open and under way.
- More trials run by Alnylam PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 150 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Alnylam Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Alnylam Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04883905
- Sponsor's protocol ID
- ALN-AS1-006
- Other IDs
- EUPAS43201
- Acronym
- ELEVATE
- Status last verified
- Oct 2026
- First submitted
- 7 May 2021
- Last updated
- 6 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- ELEVATE, a Global Observational Longitudinal Prospective Registry of Patients With Acute Hepatic Porphyria (AHP)
- ClinicalTrials.gov ID
- NCT04883905
- Acronym / sponsor ID
- ELEVATE / ALN-AS1-006
- Conditions
- Acute Hepatic Porphyria
- Keywords
- Givosiran, AHP, Acute hepatic porphyria
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 150 participants (estimated)
- Lead sponsor
- Alnylam PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 26 Apr 2021 (actual)
- Primary completion
- 31 Dec 2030 (estimated)
- Study completion
- 31 Dec 2030 (estimated)
- First posted
- 12 May 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 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. |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with all types of AHP, including patients with hepatic and/or renal impairment, adolescents, elderly patient, and pregnant or lactating women.
Key inclusion criteria (1 listed)
- Documented diagnosis of AHP, per physician's determination
Key exclusion criteria (1 listed)
- Currently enrolled in a clinical trial for any investigational agent
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Incidence of Selected Events of Interest in AHP Patients
Time frame: Up to 6 years
Selected events of interest are defined as hepatic events (including hepatic neoplasia), renal events, thromboembolic events, and pancreatitis.
2 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Oct 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Medical Director study director
Alnylam Pharmaceuticals
30 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Acute Hepatic Porphyria, run by Alnylam Pharmaceuticals at 30 sites in 10 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 6 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 30 sites in United States, France, Germany, Belgium, Italy and 5 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Alnylam Clinical Trial Information Line (1-877-ALNYLAM, clinicaltrials@alnylam.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Selected Events of Interest in AHP Patients (time frame: Up to 6 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Oct 2026.