Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria

July 14, 2022 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

No longer available

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

10 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

N/A

Genders Eligible for Study

All

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:

    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

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

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)

July 18, 2022

Last Update Submitted That Met QC Criteria

July 14, 2022

Last Verified

July 1, 2022

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.

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