The purpose of this study is to characterize the natural history and clinical management of Acute Hepatic Porphyria (AHP) patients with recurring attacks.
Study Type
OBSERVATIONAL
Enrollment
136
Clinical Trial Site
San Francisco, California, United States
Medical history of AHP patients (Part A only)
Time frame: Baseline to 6-Month Follow-Up Visit
Medication usage of AHP patients (Part A only)
Time frame: Baseline to 6-Month Follow-Up Visit
Plasma biomarkers (Part A only)
Time frame: 0, 6 months and During Attacks
Porphyria signs and symptoms (Part A only)
Time frame: 0, 2, 4, 6 months
Quality of Life (Part A only)
Time frame: 0, 6 months
Urine biomarkers (Part A only)
Time frame: 0, 6 months and During Attacks
Healthcare Utilization (Part A only)
Time frame: 0, 6 months
Change in disease activity as measured by Quality of Life (Part B only)
Time frame: 0, 6, 12, 18, 24, 30, 36 months
Change in disease activity as measured by Porphyria Screening/Follow Up Questionnaire (Part B only)
Time frame: 0, 3, 6, 9, 12, 18, 24, 30, 36 months
Change in disease activity as measured by Brief Pain Inventory form (Part B only)
Time frame: 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)
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Clinical Trial Site
Miami, Florida, United States
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Ann Arbor, Michigan, United States
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New York, New York, United States
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Winston-Salem, North Carolina, United States
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Philadelphia, Pennsylvania, United States
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Galveston, Texas, United States
Clinical Trial Site
Salt Lake City, Utah, United States
Clinical Trial Site
Camperdown, New South Wales, Australia
Clinical Trial Site
Leuven, Belgium
...and 20 more locations
Time frame: 0, 6, 12, 18, 24, 30, 36 months