Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria
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ClinicalTrials.gov Identifier: NCT04056481 |
Expanded Access Status :
No longer available
First Posted : August 14, 2019
Last Update Posted : July 18, 2022
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Condition or disease | Intervention/treatment |
---|---|
Acute Hepatic Porphyria | Drug: Givosiran |
Study Type : | Expanded Access |
Expanded Access Type : | Treatment IND/Protocol |
See clinical trials of the intervention/treatment in this expanded access record. | |
Official Title: | Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria (AHP) |

- Drug: Givosiran
givosiran (ALN-AS1) administered as a subcutaneous (SC) injectionOther Name: ALN-AS1

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Ages Eligible for Study: | 12 Years and older (Child, Adult, Older Adult) |
Sexes Eligible for Study: | All |
Inclusion Criteria:
- Diagnosed with Acute Hepatic Porphyria (Acute Intermittent Porphyria, Hereditary Coproporhyria, Variegate Porphyria, ALA dehydratase deficient porphyria)
- Have adequate venous access for program sample collections as judged by the Investigator for study sample collections
Exclusion Criteria:
- Previously or currently participating in a givosiran clinical trial
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Has any of the following laboratory parameter assessments at Screening:
- Alanine aminotransferase (ALT) >2×ULN
- Total bilirubin >1.5×ULN. Patients with elevated total bilirubin that is secondary to documented Gilbert's syndrome are eligible if the total bilirubin is <2×ULN
- Estimated Glomerular Filtration Rate (eGFR) <15 mL/min/1.73 m2
- On an active liver transplantation waiting list

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04056481
Study Director: | Medical Director | Alnylam Pharmaceuticals |
Responsible Party: | Alnylam Pharmaceuticals |
ClinicalTrials.gov Identifier: | NCT04056481 |
Other Study ID Numbers: |
ALN-AS1-005 |
First Posted: | August 14, 2019 Key Record Dates |
Last Update Posted: | July 18, 2022 |
Last Verified: | July 2022 |
Acute Hepatic Porphyria (AHP) Acute Intermittent Porphyria (AIP) Porphyria Acute Intermittent Acute Porphyria Hereditary Coproporphyria (HCP) |
Variegate Porphyria (VP) ALA Dehydratase Deficient Porphyria (ADP) Givosiran Expanded Access EAP |
Porphyrias, Hepatic Coproporphyria, Hereditary Porphyria, Erythropoietic Porphyrias Metabolic Diseases |
Skin Diseases, Genetic Genetic Diseases, Inborn Skin Diseases Liver Diseases Digestive System Diseases |