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The purpose of this study is to provide expanded access of givosiran to patients with Acute Hepatic Porphyria (AHP).
Choosing to participate in an expanded access program is an important personal decision. Talk with your doctor and family members or friends about deciding to join a research study. To learn more about this study, please have your doctor contact the study research staff using the Contacts provided. For general information, see the link provided in More Information.
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Givosiran | Drug | givosiran (ALN-AS1) administered as a subcutaneous (SC) injection |
|
Inclusion Criteria:
Exclusion Criteria:
Previously or currently participating in a givosiran clinical trial
Has any of the following laboratory parameter assessments at Screening:
On an active liver transplantation waiting list
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Alnylam Pharmaceuticals | Study Director |
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| ID | Term |
|---|---|
| D046349 | Coproporphyria, Hereditary |
| D017118 | Porphyria, Acute Intermittent |
| D011164 | Porphyrias |
| D046350 | Porphyria, Variegate |
| ID | Term |
|---|---|
| D017094 | Porphyrias, Hepatic |
| D008107 | Liver Diseases |
| D004066 | Digestive System Diseases |
| D012873 | Skin Diseases, Genetic |
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| ID | Term |
|---|---|
| C000630124 | givosiran |
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| 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 |