Acute Hepatic Porphyria
Conditions
Keywords
Porphyria, AIP, VP, HCP, Variegate, Coproporphyria
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.
Interventions
None listed
Sponsors
Alnylam Pharmaceuticals
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
12 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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
| Measure | Time frame |
|---|---|
| Quality of Life (Part A only) | 0, 6 months |
| Urine biomarkers (Part A only) | 0, 6 months and During Attacks |
| Healthcare Utilization (Part A only) | 0, 6 months |
| Plasma 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 |
| Change in disease activity as measured by Brief Pain Inventory form (Part B only) | 0, 3, 6, 9, 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 |
| Change in disease activity as measured by Quality of Life (Part B only) | 0, 6, 12, 18, 24, 30, 36 months |
| Porphyria signs and symptoms (Part A only) | 0, 2, 4, 6 months |
Countries
Australia, Belgium, Bulgaria, Czechia, Finland, France, Germany, Israel, Italy, Mexico, Netherlands, Norway, Poland, South Africa, South Korea, Spain, Switzerland, Taiwan, United Kingdom, United States
Outcome results
None listed