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Trial record 1 of 1 for:    NCT04883905
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ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP) (ELEVATE)

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ClinicalTrials.gov Identifier: NCT04883905
Recruitment Status : Recruiting
First Posted : May 12, 2021
Last Update Posted : November 8, 2022
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals

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.

Condition or disease
Acute Hepatic Porphyria

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Study Type : Observational
Estimated Enrollment : 150 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: ELEVATE, a Global Observational Longitudinal Prospective Registry of Patients With Acute Hepatic Porphyria (AHP)
Actual Study Start Date : April 26, 2021
Estimated Primary Completion Date : April 2027
Estimated Study Completion Date : April 2027


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.



Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.

  2. 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.



Information from the National Library of Medicine

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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
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.
Criteria

Inclusion Criteria:

  • Documented diagnosis of AHP, per physician's determination

Exclusion Criteria:

  • Currently enrolled in a clinical trial for any investigational agent

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04883905


Contacts
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Contact: Alnylam Clinical Trial Information Line 1-877-ALNYLAM clinicaltrials@alnylam.com
Contact: Alnylam Clinical Trial Information Line 1-877-256-9526 clinicaltrials@alnylam.com

Locations
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United States, Alabama
Clinical Trial Site Recruiting
Birmingham, Alabama, United States, 35233
United States, Arizona
Clinical Trial Site Recruiting
Gilbert, Arizona, United States, 85234
United States, California
Clinical Trial Site Recruiting
San Francisco, California, United States, 90074
United States, Massachusetts
Clinical Trial Site Recruiting
Boston, Massachusetts, United States, 02114
United States, Minnesota
Clinical Trial Site Recruiting
Minneapolis, Minnesota, United States, 55455
United States, New York
Clinical Trial Site Recruiting
New York, New York, United States, 10029
United States, North Carolina
Clinical Trial Site Recruiting
Winston-Salem, North Carolina, United States, 27157
United States, Oklahoma
Clinical Trial Site Recruiting
Oklahoma City, Oklahoma, United States, 73120
United States, Pennsylvania
Clinical Trial Site Recruiting
Philadelphia, Pennsylvania, United States, 19107
United States, Texas
Clinical Trial Site Recruiting
Galveston, Texas, United States, 77555
United States, Utah
Clinical Trial Site Recruiting
Salt Lake City, Utah, United States, 84112
United States, Washington
Clinical Trial Site Recruiting
Seattle, Washington, United States, 98195
France
Clinical Trial Site Recruiting
Bordeaux, France
Clinical Trial Site Recruiting
Paris, France
Clinical Trial Site Recruiting
Toulouse, France
Germany
Clinical Trial Site Recruiting
Chemnitz, Germany
Clinical Trial Site Recruiting
Münster, Germany
Clinical Trial Site Recruiting
Würzburg, Germany
Italy
Clinical Trial Site Recruiting
Milan, Italy
Clinical Trial Site Recruiting
Modena, Italy
United Kingdom
Clinical Trial Site Recruiting
Cardiff, United Kingdom
Clinical Trial Site Recruiting
London, United Kingdom
Sponsors and Collaborators
Alnylam Pharmaceuticals
Investigators
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Study Director: Medical Director Alnylam Pharmaceuticals
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Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT04883905    
Other Study ID Numbers: ALN-AS1-006
First Posted: May 12, 2021    Key Record Dates
Last Update Posted: November 8, 2022
Last Verified: November 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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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
Keywords provided by Alnylam Pharmaceuticals:
Givosiran
AHP
Acute hepatic porphyria
Additional relevant MeSH terms:
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Porphyrias, Hepatic
Coproporphyria, Hereditary
Porphyria, Erythropoietic
Porphyrias
Metabolic Diseases
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases
Liver Diseases
Digestive System Diseases