Overview

This trial is active, not recruiting.

Condition acute hepatic porphyria
Sponsor Alnylam Pharmaceuticals
Start date August 2014
End date November 2015
Trial size 112 participants
Trial identifier NCT02240784, ALN-AS1-NT-001

Summary

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

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Observational model cohort
Time perspective prospective
Arm

Primary Outcomes

Measure
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

Secondary Outcomes

Measure
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

Eligibility Criteria

Male or female participants at least 18 years old.

Inclusion Criteria: - Males and females aged > 18 years - Diagnosis of AHP [acute intermittent porphyria (AIP), variegate porphyria (VP), and hereditary coproporphyria (HCP)] - 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

Additional Information

Official title A Natural History Study of Acute Hepatic Porphyria (AHP) Patients With Recurrent Attacks
Trial information was received from ClinicalTrials.gov and was last updated in December 2015.
Information provided to ClinicalTrials.gov by Alnylam Pharmaceuticals.