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Expanded 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
Alnylam Pharmaceuticals
About
Eligibility
Locations
Outcomes
Full info

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

12 Years - undefined (Child, Adult, Older Adult)All Sexes

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

Sites / Locations

    Outcomes

    Primary Outcome Measures

    Secondary Outcome Measures

    Full Information

    First Posted
    August 12, 2019
    Last Updated
    July 14, 2022
    Sponsor
    Alnylam Pharmaceuticals
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    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
    undefined (undefined)
    Primary Completion Date
    undefined (undefined)
    Study Completion Date
    undefined (undefined)

    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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