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Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria

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ClinicalTrials.gov Identifier: NCT04056481
Expanded Access Status : Available
First Posted : August 14, 2019
Last Update Posted : October 11, 2019
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals

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

Condition or disease Intervention/treatment
Acute Hepatic Porphyria Drug: Givosiran

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 Design
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Study Type : Expanded Access
Expanded Access Type : Treatment IND/Protocol
Official Title: Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria (AHP)

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Porphyria

Interventions
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Intervention Details:
  • Drug: Givosiran
    givosiran (ALN-AS1) administered as a subcutaneous (SC) injection
    Other Name: ALN-AS1
Eligibility Criteria
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Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Criteria

Inclusion Criteria:

  • Diagnosed with Acute Hepatic Porphyria (Acute Intermittent Porphyria, Hereditary Coproporhyria, Variegate Porphyria, ALA dehydratase deficient porphyria)
  • Experienced a porphyria attack and be considered by their treating physician to be at risk for having an attack in the future.
  • 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) <30 mL/min/1.73 m2
  • On an active liver transplantation waiting list
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04056481


Contacts
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Contact: Alnylam EAP Hotline 1-877-256-9526 eap@alnylam.com

Locations
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United States, Florida
Nicklaus Children's Hospital
Miami, Florida, United States, 33155
France
Program Site
Colombes, France
Sponsors and Collaborators
Alnylam Pharmaceuticals
Investigators
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Study Director: Mary-Jean Fanelli Alnylam Pharmaceuticals
More Information
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Additional Information:
Information on the Early Access Program for Givosiran  This link exits the ClinicalTrials.gov site

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Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT04056481     History of Changes
Other Study ID Numbers: ALN-AS1-005
First Posted: August 14, 2019    Key Record Dates
Last Update Posted: October 11, 2019
Last Verified: October 2019
Keywords provided by Alnylam Pharmaceuticals:
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
Additional relevant MeSH terms:
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Porphyrias, Hepatic
Coproporphyria, Hereditary
Porphyria, Erythropoietic
Porphyrias
Metabolic Diseases
 Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases
Liver Diseases
Digestive System Diseases