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Completed

NCT Number: NCT02240784

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

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

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Key information

Conditions

Acute Hepatic Porphyria Congenital, Hereditary, and Neonatal Diseases and Abnormalities Coproporphyria Coproporphyria, Hereditary Digestive System Diseases Genetic Diseases, Inborn Liver Diseases Metabolic Diseases Nutritional and Metabolic Diseases Porphyrias Porphyrias, Hepatic Skin Diseases Skin Diseases, Genetic Skin and Connective Tissue Diseases

Age range

12 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Clinical Trial Site, Camperdown, New South Wales, Australia

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Primary outcomes

  1. Medical history of AHP patients (Part A only)

    Time frame: Baseline to 6-Month Follow-Up Visit

  2. Medication usage of AHP patients (Part A only)

    Time frame: Baseline to 6-Month Follow-Up Visit

Secondary outcomes

  1. Plasma biomarkers (Part A only)

    Time frame: 0, 6 months and During Attacks

  2. Porphyria signs and symptoms (Part A only)

    Time frame: 0, 2, 4, 6 months

  3. Quality of Life (Part A only)

    Time frame: 0, 6 months

  4. Urine biomarkers (Part A only)

    Time frame: 0, 6 months and During Attacks

  5. Healthcare Utilization (Part A only)

    Time frame: 0, 6 months

  6. Change in disease activity as measured by Quality of Life (Part B only)

    Time frame: 0, 6, 12, 18, 24, 30, 36 months

  7. 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

  8. 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

  9. 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

Sponsors and collaborators

Lead sponsor

Alnylam Pharmaceuticals

Industry

Registry information

Official study title

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

Important dates

Study start
2014
Primary completion
2021
Study completion
2021
First posted
Sep 16, 2014
Registry last updated
May 17, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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