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

Sponsor

Alnylam Pharmaceuticals (Industry)

Overall Status

Recruiting

CT.gov ID

NCT04883905

Collaborator

(none)

150

Enrollment

Locations

71.2

Anticipated Duration (Months)

6.8

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

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	Intervention/Treatment	Phase
Acute Hepatic Porphyria

Study Design

Study Type:

Observational

Anticipated 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 :

Apr 26, 2021

Anticipated Primary Completion Date :

Apr 1, 2027

Anticipated Study Completion Date :

Apr 1, 2027

Arms and Interventions

Arm	Intervention/Treatment
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.

Outcome Measures

Primary Outcome Measures

Incidence of Selected Events of Interest in AHP Patients [Up to 6 years]
Selected events of interest are defined as hepatic events, renal events, and pancreatitis.

Secondary Outcome Measures

Annualized Rate of Porphyria Attacks [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.
12-Item Short Form Health Survey Version 2 (SF-12 V2) (Standard Version) [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.

Eligibility Criteria

Criteria

Ages Eligible for Study:

12 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Documented diagnosis of AHP, per physician's determination

Exclusion Criteria:

Currently enrolled in a clinical trial for any investigational agent

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Clinical Trial Site	Birmingham	Alabama	United States	35233
2	Clinical Trial Site	Gilbert	Arizona	United States	85234
3	Clinical Trial Site	San Francisco	California	United States	90074
4	Clinical Trial Site	Boston	Massachusetts	United States	02114
5	Clinical Trial Site	Minneapolis	Minnesota	United States	55455
6	Clinical Trial Site	New York	New York	United States	10029
7	Clinical Trial Site	Winston-Salem	North Carolina	United States	27157
8	Clinical Trial Site	Oklahoma City	Oklahoma	United States	73120
9	Clinical Trial Site	Philadelphia	Pennsylvania	United States	19107
10	Clinical Trial Site	Galveston	Texas	United States	77555
11	Clinical Trial Site	Salt Lake City	Utah	United States	84112
12	Clinical Trial Site	Seattle	Washington	United States	98195
13	Clinical Trial Site	Bordeaux		France
14	Clinical Trial Site	Paris		France
15	Clinical Trial Site	Toulouse		France
16	Clinical Trial Site	Chemnitz		Germany
17	Clinical Trial Site	Münster		Germany
18	Clinical Trial Site	Würzburg		Germany
19	Clinical Trial Site	Milan		Italy
20	Clinical Trial Site	Modena		Italy
21	Clinical Trial Site	Cardiff		United Kingdom
22	Clinical Trial Site	London		United Kingdom