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

Completed

• Conditions: Acute Hepatic Porphyria

• Registration Number: NCT02240784
• Lead Sponsor: Alnylam Pharmaceuticals

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2014-08
• Study First Submitted: 2014-09-11
• Study First Submitted QC: 2014-09-12
• Study First Posted: 2014-09-16
• Primary Completion: 2021-04-26
• Study Completion: 2021-04-26
• Status Verified: 2021-05
• Last Update Submitted: 2021-05-14
• Last Update Posted: 2021-05-17

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 136

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 & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Medical history of AHP patients (Part A only)	Baseline to 6-Month Follow-Up Visit
Medication usage of AHP patients (Part A only)	Baseline to 6-Month Follow-Up Visit

Secondary Outcome Measures

Name	Time	Method
Plasma biomarkers (Part A only)	0, 6 months and During Attacks
Change in disease activity as measured by Quality of Life (Part B only)	0, 6, 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)	0, 6, 12, 18, 24, 30, 36 months
Quality of Life (Part A only)	0, 6 months
Change in disease activity as measured by Brief Pain Inventory form (Part B only)	0, 3, 6, 9, 12, 18, 24, 30, 36 months
Urine biomarkers (Part A only)	0, 6 months and During Attacks
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
Porphyria signs and symptoms (Part A only)	0, 2, 4, 6 months
Healthcare Utilization (Part A only)	0, 6 months

Trial Locations

Locations (1)

Clinical Trial Site; 🇬🇧 London, United Kingdom