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

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Observational

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.

Detailed Summary:
Sponsor: Alnylam Pharmaceuticals

Current Primary Outcome:

  • Medical history of AHP patients [ Time Frame: Baseline to 6-Month Follow-Up Visit ]
  • Medication usage of AHP patients [ Time Frame: Baseline to 6-Month Follow-Up Visit ]


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Plasma biomarkers [ Time Frame: 0, 6 months and During Attacks ]
  • Porphyria signs and symptoms [ Time Frame: 0, 2, 4, 6 months ]
  • Quality of Life [ Time Frame: 0, 6 months ]
  • Urine biomarkers [ Time Frame: 0, 6 months and During Attacks ]
  • Healthcare Utilization [ Time Frame: 0, 6 months ]


Original Secondary Outcome: Same as current

Information By: Alnylam Pharmaceuticals

Dates:
Date Received: September 11, 2014
Date Started: August 2014
Date Completion: November 2016
Last Updated: December 11, 2015
Last Verified: December 2015