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

Alnylam Pharmaceuticals

Status: Completed

Conditions: Acute Hepatic Porphyria

Study type: Observational

Funder types: Industry

Identifiers

NCT02240784

ALN-AS1-NT-001

Details and patient eligibility

About

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

Enrollment: 136 patients

Sex: All

Ages: 12+ years old

Volunteers: No Healthy Volunteers

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

Trial design

136 participants in 1 patient group

Acute Hepatic Porphyria