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The purpose of this study is to characterize the natural history and clinical management of Acute Hepatic Porphyria (AHP) patients with recurring attacks.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Acute Hepatic Porphyria |
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| Measure | Description | 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 |
| Measure | Description | Time Frame |
|---|---|---|
| Plasma biomarkers (Part A only) | 0, 6 months and During Attacks | |
| Porphyria signs and symptoms (Part A only) | 0, 2, 4, 6 months | |
| Quality of Life (Part A only) |
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Inclusion Criteria:
Exclusion Criteria:
• Current participation in a clinical trial of an investigational product
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Acute Hepatic Porphyria
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Alnylam Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Clinical Trial Site | San Francisco | California | 94143 | United States | ||
| Clinical Trial Site |
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Urine and plasma samples for known proteins associated with porphyria attacks.
| 0, 6 months |
| Urine biomarkers (Part A only) | 0, 6 months and During Attacks |
| Healthcare Utilization (Part A only) | 0, 6 months |
| Change in disease activity as measured by Quality of Life (Part B only) | 0, 6, 12, 18, 24, 30, 36 months |
| 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 |
| Miami |
| Florida |
| 33136 |
| United States |
| Clinical Trial Site | Ann Arbor | Michigan | 48109 | United States |
| Clinical Trial Site | New York | New York | 10029 | United States |
| Clinical Trial Site | Winston-Salem | North Carolina | 27157 | United States |
| Clinical Trial Site | Philadelphia | Pennsylvania | 19107 | United States |
| Clinical Trial Site | Galveston | Texas | 77555 | United States |
| Clinical Trial Site | Salt Lake City | Utah | 84112 | United States |
| Clinical Trial Site | Camperdown | New South Wales | Australia |
| Clinical Trial Site | Leuven | Belgium |
| Clinical Trial Site | Sofia | Bulgaria |
| Clinical Trial Site | Prague | Czechia |
| Clinical Trial Site | Helsinki | Finland |
| Clinical Trial Site | Paris | France |
| Clinical Trial Site | Chemnitz | Germany |
| Clinical Trial Site | Petah Tikva | Israel |
| Clinical Trial Site | Milan | Italy |
| Clinical Trial Site | Modena | Italy |
| Clinical Trial Site | Mexico City | Mexico |
| Clinical Trial Site | Rotterdam | Netherlands |
| Clinical Trial Site | Bergen | Norway |
| Clinical Trial Site | Warsaw | Poland |
| Clinical Trial Site | Cape Town | South Africa |
| Clinical Trial Site | Seoul | South Korea |
| Clinical Trial Site | Barcelona | Spain |
| Clinical Trial Site | Murcia | Spain |
| Clinical Trial Site | Zurich | Switzerland |
| Clinical Trial Site | Taipei | Taiwan |
| Clinical Trial Site | Cardiff | Wales | United Kingdom |
| Clinical Trial Site | London | United Kingdom |
| ID | Term |
|---|---|
| D046349 | Coproporphyria, Hereditary |
| D011164 | Porphyrias |
| C535469 | Coproporphyria |
| ID | Term |
|---|---|
| D017094 | Porphyrias, Hepatic |
| D008107 | Liver Diseases |
| D004066 | Digestive System Diseases |
| D012873 | Skin Diseases, Genetic |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
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