Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria

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Variegate porphyria
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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)

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.

Study Type

Expanded Access

Condition

Acute Hepatic Porphyria

Intervention

Givosiran

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status

Drug

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:

               1. Alanine aminotransferase (ALT) >2×ULN

               2. 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

               3. Estimated Glomerular Filtration Rate (eGFR) <15 mL/min/1.73 m2

          -  On an active liver transplantation waiting list

Gender

All

Ages

12 Years - N/A

Contacts

Medical Director, ,

Location Countries

Australia

Location Countries

Australia

Administrative Informations

NCT ID

NCT04056481

Organization ID

ALN-AS1-005

Responsible Party

Sponsor

Study Sponsor

Alnylam Pharmaceuticals

Study Sponsor

Medical Director, Study Director, Alnylam Pharmaceuticals

Verification Date

September 2021