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

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

Group / Cohort

A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
Acute Hepatic Porphyria

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
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

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
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)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
August 1, 2014

Primary Completion (ACTUAL)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
April 26, 2021

Study Completion (ACTUAL)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
April 26, 2021

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
September 11, 2014

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
September 12, 2014

First Posted (ESTIMATE)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
September 16, 2014

Study Record Updates

Last Update Posted (ACTUAL)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
May 17, 2021

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 14, 2021

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
May 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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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