NCT04883905

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.

Trial Health

88
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
150

participants targeted

Target at P50-P75 for all trials

Timeline
11mo left

Started Apr 2021

Longer than P75 for all trials

Geographic Reach
10 countries

30 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress85%
Apr 2021Apr 2027

Study Start

First participant enrolled

April 26, 2021

Completed
11 days until next milestone

First Submitted

Initial submission to the registry

May 7, 2021

Completed
5 days until next milestone

First Posted

Study publicly available on registry

May 12, 2021

Completed
5.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 1, 2027

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

April 1, 2027

Last Updated

April 27, 2026

Status Verified

April 1, 2026

Enrollment Period

5.9 years

First QC Date

May 7, 2021

Last Update Submit

April 23, 2026

Conditions

Acute Hepatic Porphyria

Keywords

GivosiranAHPAcute hepatic porphyria

Outcome Measures

Primary Outcomes (1)

  • 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.

    Up to 6 years

Secondary Outcomes (2)

  • Annualized Rate of Porphyria Attacks

    Up to 6 years

  • 12-Item Short Form Health Survey Version 2 (SF-12 V2) (Standard Version)

    Up to 6 years

Study Arms (1)

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.

Eligibility Criteria

Age12 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-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.

You may qualify if:

  • Documented diagnosis of AHP, per physician's determination

You may not qualify if:

  • Currently enrolled in a clinical trial for any investigational agent

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (30)

Clinical Trial Site

Birmingham, Alabama, 35233, United States

RECRUITING

Clinical Trial Site

Gilbert, Arizona, 85234, United States

RECRUITING

Clinical Trial Site

San Francisco, California, 90074, United States

RECRUITING

Clinical Trial Site

Baltimore, Maryland, 21205, United States

RECRUITING

Clinical Trial Site

Boston, Massachusetts, 02114, United States

RECRUITING

Clinical Trial Site

Minneapolis, Minnesota, 55455, United States

RECRUITING

Clinical Trial Site

New York, New York, 10029, United States

RECRUITING

Clinical Trial Site

Winston-Salem, North Carolina, 27157, United States

COMPLETED

Clinical Trial Site

Oklahoma City, Oklahoma, 73120, United States

RECRUITING

Clinical Trial Site

Philadelphia, Pennsylvania, 19107, United States

COMPLETED

Clinical Trial Site

Galveston, Texas, 77555, United States

RECRUITING

Clinical Trial Site

Salt Lake City, Utah, 84112, United States

RECRUITING

Clinical Trial Site

Seattle, Washington, 98195, 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

MeSH Terms

Conditions

Coproporphyria, Hereditary

Condition Hierarchy (Ancestors)

Porphyrias, HepaticLiver DiseasesDigestive System DiseasesSkin Diseases, GeneticGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesSkin DiseasesSkin and Connective Tissue DiseasesPorphyriasMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

  • Medical Director

    Alnylam Pharmaceuticals

    STUDY DIRECTOR

Central Study Contacts

Alnylam Clinical Trial Information Line

CONTACT

1-877-ALNYLAMclinicaltrials@alnylam.com

Alnylam Clinical Trial Information Line

CONTACT

1-877-256-9526clinicaltrials@alnylam.com

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 7, 2021

First Posted

May 12, 2021

Study Start

April 26, 2021

Primary Completion (Estimated)

April 1, 2027

Study Completion (Estimated)

April 1, 2027

Last Updated

April 27, 2026

Record last verified: 2026-04

Data Sharing

IPD Sharing
Will not share

Locations

DE(3)ES(1)IT(2)CH(1)FR(3)GB(2)SE(1)BE(2)US(13)TW(2)