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Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria

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)
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

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

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