Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria

NCT ID: NCT04056481

Last Updated: 2024-05-20

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

APPROVED_FOR_MARKETING

Study Classification

EXPANDED_ACCESS

Brief Summary

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The purpose of this study is to provide expanded access of givosiran to patients with Acute Hepatic Porphyria (AHP).

Detailed Description

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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.

Conditions

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Acute Hepatic Porphyria

Interventions

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Givosiran

givosiran (ALN-AS1) administered as a subcutaneous (SC) injection

Intervention Type DRUG

Other Intervention Names

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ALN-AS1

Eligibility Criteria

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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
Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Alnylam Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Medical Director

Role: STUDY_DIRECTOR

Alnylam Pharmaceuticals

Other Identifiers

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ALN-AS1-005

Identifier Type: -

Identifier Source: org_study_id

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