Studies
| Study First Submitted Date | 2019-08-12 |
| Study First Posted Date | 2019-08-14 |
| Last Update Posted Date | 2022-07-18 |
| Verification Month Year | July 2022 |
| Verification Date | 2022-07-31 |
| Last Update Posted Date | 2022-07-18 |
Detailed Descriptions
| Sequence: | 20772765 |
| Description | 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. |
Conditions
| Sequence: | 52301943 |
| Name | Acute Hepatic Porphyria |
| Downcase Name | acute hepatic porphyria |
Id Information
| Sequence: | 40252815 |
| Id Source | org_study_id |
| Id Value | ALN-AS1-005 |
Countries
| Sequence: | 42671618 | Sequence: | 42671619 | Sequence: | 42671620 | Sequence: | 42671621 | Sequence: | 42671622 | Sequence: | 42671623 | Sequence: | 42671624 | Sequence: | 42671625 | Sequence: | 42671626 | Sequence: | 42671627 |
| Name | Australia | Name | Belgium | Name | Canada | Name | France | Name | Germany | Name | Luxembourg | Name | Netherlands | Name | Spain | Name | Sweden | Name | United States |
| Removed | True | Removed | True | Removed | True | Removed | True | Removed | True | Removed | True | Removed | True | Removed | True | Removed | True | Removed | True |
Interventions
| Sequence: | 52613988 |
| Intervention Type | Drug |
| Name | Givosiran |
| Description | givosiran (ALN-AS1) administered as a subcutaneous (SC) injection |
Keywords
| Sequence: | 80052013 | Sequence: | 80052003 | Sequence: | 80052004 | Sequence: | 80052005 | Sequence: | 80052006 | Sequence: | 80052007 | Sequence: | 80052008 | Sequence: | 80052009 | Sequence: | 80052010 | Sequence: | 80052011 | Sequence: | 80052012 |
| Name | EAP | Name | Acute Hepatic Porphyria (AHP) | Name | Acute Intermittent Porphyria (AIP) | Name | Porphyria | Name | Acute Intermittent | Name | Acute Porphyria | Name | Hereditary Coproporphyria (HCP) | Name | Variegate Porphyria (VP) | Name | ALA Dehydratase Deficient Porphyria (ADP) | Name | Givosiran | Name | Expanded Access |
| Downcase Name | eap | Downcase Name | acute hepatic porphyria (ahp) | Downcase Name | acute intermittent porphyria (aip) | Downcase Name | porphyria | Downcase Name | acute intermittent | Downcase Name | acute porphyria | Downcase Name | hereditary coproporphyria (hcp) | Downcase Name | variegate porphyria (vp) | Downcase Name | ala dehydratase deficient porphyria (adp) | Downcase Name | givosiran | Downcase Name | expanded access |
Browse Conditions
| Sequence: | 193987240 | Sequence: | 193987241 | Sequence: | 193987242 | Sequence: | 193987243 | Sequence: | 193987244 | Sequence: | 193987245 | Sequence: | 193987246 | Sequence: | 193987247 | Sequence: | 193987248 | Sequence: | 193987249 |
| Mesh Term | Porphyrias, Hepatic | Mesh Term | Coproporphyria, Hereditary | Mesh Term | Porphyria, Erythropoietic | Mesh Term | Porphyrias | Mesh Term | Metabolic Diseases | Mesh Term | Skin Diseases, Genetic | Mesh Term | Genetic Diseases, Inborn | Mesh Term | Skin Diseases | Mesh Term | Liver Diseases | Mesh Term | Digestive System Diseases |
| Downcase Mesh Term | porphyrias, hepatic | Downcase Mesh Term | coproporphyria, hereditary | Downcase Mesh Term | porphyria, erythropoietic | Downcase Mesh Term | porphyrias | Downcase Mesh Term | metabolic diseases | Downcase Mesh Term | skin diseases, genetic | Downcase Mesh Term | genetic diseases, inborn | Downcase Mesh Term | skin diseases | Downcase Mesh Term | liver diseases | Downcase Mesh Term | digestive system diseases |
| Mesh Type | mesh-list | Mesh Type | mesh-list | Mesh Type | mesh-list | Mesh Type | mesh-list | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor |
Sponsors
| Sequence: | 48442134 |
| Agency Class | INDUSTRY |
| Lead Or Collaborator | lead |
| Name | Alnylam Pharmaceuticals |
Overall Officials
| Sequence: | 29355853 |
| Role | Study Director |
| Name | Medical Director |
| Affiliation | Alnylam Pharmaceuticals |
Eligibilities
| Sequence: | 30841524 |
| Gender | All |
| Minimum Age | 12 Years |
| Maximum Age | N/A |
| Criteria | Inclusion Criteria:
Diagnosed with Acute Hepatic Porphyria (Acute Intermittent Porphyria, Hereditary Coproporhyria, Variegate Porphyria, ALA dehydratase deficient porphyria) Exclusion Criteria: Previously or currently participating in a givosiran clinical trial Has any of the following laboratory parameter assessments at Screening: Alanine aminotransferase (ALT) >2×ULN |
| Adult | True |
| Child | True |
| Older Adult | True |
Calculated Values
| Sequence: | 254178611 |
| Registered In Calendar Year | 2019 |
| Were Results Reported | False |
| Has Single Facility | False |
| Minimum Age Num | 12 |
| Minimum Age Unit | Years |
Intervention Other Names
| Sequence: | 26732096 |
| Intervention Id | 52613988 |
| Name | ALN-AS1 |
Responsible Parties
| Sequence: | 28953836 |
| Responsible Party Type | Sponsor |