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| ID | Type | Description | Link |
|---|---|---|---|
| EUPAS43201 | Registry Identifier | EU PAS REGISTER |
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This global patient registry is being conducted to characterize the natural history and real-world clinical management of patients with AHP, and to further characterize the real-world safety and effectiveness of givosiran and other approved AHP therapies.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Patients with AHP | Patients with a diagnosis of AHP will be eligible for the study and will be managed and treated per routine clinical practice. |
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| Measure | Description | Time Frame |
|---|---|---|
| Incidence of Selected Events of Interest in AHP Patients | Selected events of interest are defined as hepatic events (including hepatic neoplasia), renal events, thromboembolic events, and pancreatitis. | Up to 6 years |
| Measure | Description | Time Frame |
|---|---|---|
| Annualized Rate of Porphyria Attacks | Porphyria attacks are defined as meeting all of the following criteria: an acute episode of neurovisceral pain in the abdomen, back, chest, extremities and/or limbs, no other medically determined cause, and requires treatment with intravenous (IV) dextrose or hemin, carbohydrates, or analgesics, or other medications such as antiemetics at a dose or frequency beyond the participant's usual daily porphyria management. |
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Inclusion Criteria:
Exclusion Criteria:
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Patients with all types of AHP, including patients with hepatic and/or renal impairment, adolescents, elderly patient, and pregnant or lactating women.
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Alnylam Clinical Trial Information Line | Contact | 1-877-ALNYLAM | clinicaltrials@alnylam.com | |
| Alnylam Clinical Trial Information Line | Contact | 1-877-256-9526 | clinicaltrials@alnylam.com |
| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Alnylam Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Clinical Trial Site | Recruiting | Birmingham | Alabama | 35233 | United States | |
| Clinical Trial Site |
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| Up to 6 years |
| 12-Item Short Form Health Survey Version 2 (SF-12 V2) (Standard Version) | S-12 V2 is a 12-question measure capturing global quality of life and overall health status and evaluates the following 8 domains: physical functioning, role physical, bodily pain, general health, vitality, social functioning, role emotional, and mental health. | Up to 6 years |
| Recruiting |
| Gilbert |
| Arizona |
| 85234 |
| United States |
| Clinical Trial Site | Recruiting | San Francisco | California | 90074 | United States |
| Clinical Trial Site | Recruiting | Baltimore | Maryland | 21205 | United States |
| Clinical Trial Site | Recruiting | Boston | Massachusetts | 02114 | United States |
| Clinical Trial Site | Recruiting | Minneapolis | Minnesota | 55455 | United States |
| Clinical Trial Site | Recruiting | New York | New York | 10029 | United States |
| Clinical Trial Site | Completed | Winston-Salem | North Carolina | 27157 | United States |
| Clinical Trial Site | Recruiting | Oklahoma City | Oklahoma | 73120 | United States |
| Clinical Trial Site | Completed | Philadelphia | Pennsylvania | 19107 | United States |
| Clinical Trial Site | Recruiting | Galveston | Texas | 77555 | United States |
| Clinical Trial Site | Recruiting | Salt Lake City | Utah | 84112 | United States |
| Clinical Trial Site | Recruiting | Seattle | Washington | 98195 | United States |
| Clinical Trial Site | Recruiting | Brussels | Belgium |
| Clinical Trial Site | Recruiting | Leuven | Belgium |
| Clinical Trial Site | Recruiting | Bordeaux | France |
| Clinical Trial Site | Recruiting | Paris | France |
| Clinical Trial Site | Recruiting | Toulouse | France |
| Clinical Trial Site | Recruiting | Berlin | Germany |
| Clinical Trial Site | Recruiting | Chemnitz | Germany |
| Clinical Trial Site | Recruiting | Würzburg | Germany |
| Clinical Trial Site | Recruiting | Milan | Italy |
| Clinical Trial Site | Recruiting | Modena | Italy |
| Clinical Trial Site | Recruiting | Madrid | Spain |
| Clinical Trial Site | Recruiting | Stockholm | Sweden |
| Clinical Trial Site | Recruiting | Zurich | Switzerland |
| Clinical Trial Site | Recruiting | Taichung | Taiwan |
| Clinical Trial Site | Recruiting | Taipei | Taiwan |
| Clinical Trial Site | Recruiting | Cardiff | United Kingdom |
| Clinical Trial Site | Recruiting | London | United Kingdom |
| ID | Term |
|---|---|
| D046349 | Coproporphyria, Hereditary |
| 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 |
| D011164 | Porphyrias |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
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