Back to resultsExpanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria
Primary Purpose
Acute Hepatic Porphyria
Status
No longer available
Phase
Locations
Study Type
Expanded Access
Intervention
Givosiran
Sponsored by
About this trial
This is an expanded access trial for Acute Hepatic Porphyria focused on measuring 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
Eligibility 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
Sites / Locations
Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT04056481
Brief Title
Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria
Official Title
Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria (AHP)
Study Type
Expanded Access
2. Study Status
Record Verification Date
July 2022
Overall Recruitment Status
No longer available
Study Start Date
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Primary Completion Date
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Study Completion Date
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3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Alnylam Pharmaceuticals
4. Oversight
5. Study Description
Brief Summary
The purpose of this study is to provide expanded access of givosiran to patients with Acute Hepatic Porphyria (AHP).
Detailed 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Acute Hepatic Porphyria
Keywords
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
7. Study Design
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
Givosiran
Other Intervention Name(s)
ALN-AS1
Intervention Description
givosiran (ALN-AS1) administered as a subcutaneous (SC) injection
10. Eligibility
Sex
All
Minimum Age & Unit of Time
12 Years
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Director
Organizational Affiliation
Alnylam Pharmaceuticals
Official's Role
Study Director
12. IPD Sharing Statement
Learn more about this trial
Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria
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