EXPLORE: A Natural History Study of Acute Hepatic Porphyria (AHP)

Acute Hepatic Porphyria Clinical Trial

Official title:

A Natural History Study of Acute Hepatic Porphyria (AHP) Patients With Recurrent Attacks

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02240784
• Other study ID #: ALN-AS1-NT-001
• Status: Completed
• Start date: August 2014
• Est. completion date: April 26, 2021

Study information

• Verified date: May 2021
• Source: Alnylam Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

The purpose of this study is to characterize the natural history and clinical management of Acute Hepatic Porphyria (AHP) patients with recurring attacks.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 136
• Est. completion date: April 26, 2021
• Est. primary completion date: April 26, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years and older

Eligibility

Inclusion Criteria:

• Males and females aged >/= 12 years

• Diagnosis of AHP [acute intermittent porphyria (AIP), variegate porphyria (VP), hereditary coproporphyria (HCP) and aminolevulinic acid dehydratase (ALAD) deficient porphyria (ADP)]

• Porphyria attacks in the past 12 months or receiving treatment to prevent attacks

• Willing to provide written informed consent, medical records, and to comply with study requirements

Exclusion Criteria:

• Current participation in a clinical trial of an investigational product

Related Conditions & MeSH terms

• Acute Hepatic Porphyria
• Porphyria, Erythropoietic
• Porphyrias

Locations

Country	Name	City	State
Australia	Clinical Trial Site	Camperdown	New South Wales
Belgium	Clinical Trial Site	Leuven
Bulgaria	Clinical Trial Site	Sofia
Czechia	Clinical Trial Site	Prague
Finland	Clinical Trial Site	Helsinki
France	Clinical Trial Site	Paris
Germany	Clinical Trial Site	Chemnitz
Israel	Clinical Trial Site	Petach Tikva
Italy	Clinical Trial Site	Milano
Italy	Clinical Trial Site	Modena
Korea, Republic of	Clinical Trial Site	Seoul
Mexico	Clinical Trial Site	Mexico City
Netherlands	Clinical Trial Site	Rotterdam
Norway	Clinical Trial Site	Bergen
Poland	Clinical Trial Site	Warsaw
South Africa	Clinical Trial Site	Cape Town
Spain	Clinical Trial Site	Barcelona
Spain	Clinical Trial Site	Murcia
Switzerland	Clinical Trial Site	Zurich
Taiwan	Clinical Trial Site	Taipei City
United Kingdom	Clinical Trial Site	Cardiff	Wales
United Kingdom	Clinical Trial Site	London
United States	Clinical Trial Site	Ann Arbor	Michigan
United States	Clinical Trial Site	Galveston	Texas
United States	Clinical Trial Site	Miami	Florida
United States	Clinical Trial Site	New York	New York
United States	Clinical Trial Site	Philadelphia	Pennsylvania
United States	Clinical Trial Site	Salt Lake City	Utah
United States	Clinical Trial Site	San Francisco	California
United States	Clinical Trial Site	Winston-Salem	North Carolina

Sponsors (1)

Lead Sponsor	Collaborator
Alnylam Pharmaceuticals

Countries where clinical trial is conducted

United States,  Australia,  Belgium,  Bulgaria,  Czechia,  Finland,  France,  Germany,  Israel,  Italy,  Korea, Republic of,  Mexico,  Netherlands,  Norway,  Poland,  South Africa,  Spain,  Switzerland,  Taiwan,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Medical history of AHP patients (Part A only)		Baseline to 6-Month Follow-Up Visit
Primary	Medication usage of AHP patients (Part A only)		Baseline to 6-Month Follow-Up Visit
Secondary	Plasma biomarkers (Part A only)		0, 6 months and During Attacks
Secondary	Porphyria signs and symptoms (Part A only)		0, 2, 4, 6 months
Secondary	Quality of Life (Part A only)		0, 6 months
Secondary	Urine biomarkers (Part A only)		0, 6 months and During Attacks
Secondary	Healthcare Utilization (Part A only)		0, 6 months
Secondary	Change in disease activity as measured by Quality of Life (Part B only)		0, 6, 12, 18, 24, 30, 36 months
Secondary	Change in disease activity as measured by Porphyria Screening/Follow Up Questionnaire (Part B only)		0, 3, 6, 9, 12, 18, 24, 30, 36 months
Secondary	Change in disease activity as measured by Brief Pain Inventory form (Part B only)		0, 3, 6, 9, 12, 18, 24, 30, 36 months
Secondary	Change in disease activity as measured by European Organization for Research and Treatment of Cancer (EORTC) Questionnaire (Part B only)		0, 6, 12, 18, 24, 30, 36 months