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

May 14, 2021 updated by: Alnylam Pharmaceuticals

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

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

Study Overview

Status

Completed

Conditions

Study Type

Observational

Enrollment (Actual)

136

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
    • New South Wales
      • Camperdown, New South Wales, Australia
        • Clinical Trial Site
  • Belgium
      • Leuven, Belgium
        • Clinical Trial Site
  • Bulgaria
      • Sofia, Bulgaria
        • Clinical Trial Site
  • Czechia
      • Prague, Czechia
        • Clinical Trial Site
  • Finland
      • Helsinki, Finland
        • Clinical Trial Site
  • France
      • Paris, France
        • Clinical Trial Site
  • Germany
      • Chemnitz, Germany
        • Clinical Trial Site
  • Israel
      • Petach Tikva, Israel
        • Clinical Trial Site
  • Italy
      • Milano, Italy
        • Clinical Trial Site
      • Modena, Italy
        • Clinical Trial Site
  • Korea, Republic of
      • Seoul, Korea, Republic of
        • Clinical Trial Site
  • Mexico
      • Mexico City, Mexico
        • Clinical Trial Site
  • Netherlands
      • Rotterdam, Netherlands
        • Clinical Trial Site
  • Norway
      • Bergen, Norway
        • Clinical Trial Site
  • Poland
      • Warsaw, Poland
        • Clinical Trial Site
  • South Africa
      • Cape Town, South Africa
        • Clinical Trial Site
  • Spain
      • Barcelona, Spain
        • Clinical Trial Site
      • Murcia, Spain
        • Clinical Trial Site
  • Switzerland
      • Zurich, Switzerland
        • Clinical Trial Site
  • Taiwan
      • Taipei City, Taiwan
        • Clinical Trial Site
  • United Kingdom
      • London, United Kingdom
        • Clinical Trial Site
    • Wales
      • Cardiff, Wales, United Kingdom
        • Clinical Trial Site
  • United States
    • California
      • San Francisco, California, United States, 94143
        • Clinical Trial Site
    • Florida
      • Miami, Florida, United States, 33136
        • Clinical Trial Site
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • Clinical Trial Site
    • New York
      • New York, New York, United States, 10029
        • Clinical Trial Site
    • North Carolina
      • Winston-Salem, North Carolina, United States, 27157
        • Clinical Trial Site
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19107
        • Clinical Trial Site
    • Texas
      • Galveston, Texas, United States, 77555
        • Clinical Trial Site
    • Utah
      • Salt Lake City, Utah, United States, 84112
        • Clinical Trial Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

12 years and older (ADULT, OLDER_ADULT, CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Acute Hepatic Porphyria

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Acute Hepatic Porphyria

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Medical history of AHP patients (Part A only)
Time Frame: Baseline to 6-Month Follow-Up Visit
Baseline to 6-Month Follow-Up Visit
Medication usage of AHP patients (Part A only)
Time Frame: Baseline to 6-Month Follow-Up Visit
Baseline to 6-Month Follow-Up Visit

Secondary Outcome Measures

Outcome Measure
Time Frame
Plasma biomarkers (Part A only)
Time Frame: 0, 6 months and During Attacks
0, 6 months and During Attacks
Porphyria signs and symptoms (Part A only)
Time Frame: 0, 2, 4, 6 months
0, 2, 4, 6 months
Quality of Life (Part A only)
Time Frame: 0, 6 months
0, 6 months
Urine biomarkers (Part A only)
Time Frame: 0, 6 months and During Attacks
0, 6 months and During Attacks
Healthcare Utilization (Part A only)
Time Frame: 0, 6 months
0, 6 months
Change in disease activity as measured by Quality of Life (Part B only)
Time Frame: 0, 6, 12, 18, 24, 30, 36 months
0, 6, 12, 18, 24, 30, 36 months
Change in disease activity as measured by Porphyria Screening/Follow Up Questionnaire (Part B only)
Time Frame: 0, 3, 6, 9, 12, 18, 24, 30, 36 months
0, 3, 6, 9, 12, 18, 24, 30, 36 months
Change in disease activity as measured by Brief Pain Inventory form (Part B only)
Time Frame: 0, 3, 6, 9, 12, 18, 24, 30, 36 months
0, 3, 6, 9, 12, 18, 24, 30, 36 months
Change in disease activity as measured by European Organization for Research and Treatment of Cancer (EORTC) Questionnaire (Part B only)
Time Frame: 0, 6, 12, 18, 24, 30, 36 months
0, 6, 12, 18, 24, 30, 36 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Alnylam Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (ACTUAL)

August 1, 2014

Primary Completion (ACTUAL)

April 26, 2021

Study Completion (ACTUAL)

April 26, 2021

Study Registration Dates

First Submitted

September 11, 2014

First Submitted That Met QC Criteria

September 12, 2014

First Posted (ESTIMATE)

September 16, 2014

Study Record Updates

Last Update Posted (ACTUAL)

May 17, 2021

Last Update Submitted That Met QC Criteria

May 14, 2021

Last Verified

May 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ALN-AS1-NT-001

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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