Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria
May 16, 2024 updated by: Alnylam Pharmaceuticals
Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria (AHP)
The purpose of this study is to provide expanded access of givosiran to patients with Acute Hepatic Porphyria (AHP).
Study Overview
Status
Approved for marketing
Conditions
Intervention / Treatment
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.
Study Type
Expanded Access
Expanded Access Type
- Treatment IND/Protocol
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
8 years and older (Child, Adult, Older Adult)
Accepts Healthy Volunteers
N/A
Description
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
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Study Director: Medical Director, Alnylam Pharmaceuticals
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Helpful Links
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Registration Dates
First Submitted
August 12, 2019
First Submitted That Met QC Criteria
August 12, 2019
First Posted (Actual)
August 14, 2019
Study Record Updates
Last Update Posted (Actual)
May 20, 2024
Last Update Submitted That Met QC Criteria
May 16, 2024
Last Verified
May 1, 2024
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- ALN-AS1-005
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.