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

Brief Title

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

Official Title

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

Brief Summary

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



Study Type

Observational


Primary Outcome

Medical history of AHP patients (Part A only)

Secondary Outcome

 Plasma biomarkers (Part A only)

Condition

Acute Hepatic Porphyria


Study Arms / Comparison Groups

 Acute Hepatic Porphyria
Description:  

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information



Estimated Enrollment

136

Start Date

August 2014

Completion Date

April 26, 2021

Primary Completion Date

April 26, 2021

Eligibility Criteria

        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
      

Gender

All

Ages

12 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Medical Director, , 

Location Countries

Australia

Location Countries

Australia

Administrative Informations


NCT ID

NCT02240784

Organization ID

ALN-AS1-NT-001


Responsible Party

Sponsor

Study Sponsor

Alnylam Pharmaceuticals


Study Sponsor

Medical Director, Study Director, Alnylam Pharmaceuticals


Verification Date

May 2021