Expanded Access Protocol of Givosiran for Patients with Acute Hepatic Porphyria

Recruitment & Eligibility

Status: Complete

Sex: All

Target Recruitment: 10

Inclusion Criteria

1. Male or female, >=12 years of age.
2. Documented diagnosis of AIP, HCP, VP, or ADP.
3. Has adequate venous access as judged by the Investigator for study sample collections.
4. Patient is able to understand and is willing and able to comply with the study requirements and to provide written informed consent. In patients under the age of legal consent, the legal guardian(s) must provide informed consent and the patient should provide assent per local requirements.
5. Be willing to comply with the contraceptive requirements during the study period.

Exclusion Criteria

1. Has any of the following laboratory parameter assessments at screening
a. ALT >2xULN
b. Total bilirubin (TBL) >1.5xULN. Patients with elevated TBL that is secondary to documented Gilbert's syndrome are eligible if the TBL is <2xULN
c. eGFR <15 mL/min/1.73 m2 using the Modification of Diet in Renal Disease (MDRD) formula or the Schwartz Bedside Formula Appendix 1.(Levey 2009)
2. Currently enrolled in another investigational device or drug study, or less than 30 days or 5 half-lives, whichever is longer, since ending another investigational device or drug study(s), or receiving other investigational agent(s).
3. On an active liver transplantation waiting list.
4. Active infection with hepatitis B or hepatitis C previously identified by serology.
5. Known human immunodeficiency virus (HIV) infection.
6. History of multiple drug allergies or history of allergic reaction to an oligonucleotide or to GalNAc.
7. History of intolerance to SC injection(s).
8.Any condition (eg, medical concern or alcohol or substance abuse) which, in the opinion of the Investigator, would make the patient unsuitable for dosing or which could interfere with the study compliance or the patient's safety. This includes significant active and poorly controlled (unstable) cardiovascular, neurologic, gastrointestinal, endocrine, renal, or psychiatric disorders unrelated to porphyria identified by key laboratory abnormalities or medical history.
9. Has a major surgery planned during the first 6 months of the study.
10. History of serious infection within one month prior to Screening.
11. Had a malignancy within 2 years prior to screening, except for basal or squamous cell carcinoma of the skin, cervical in-situ carcinoma, or breast ductal carcinoma, that has been successfully treated.
12. Female patient is pregnant or breastfeeding.

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
- Urinary aminolevulinic acid (ALA) levels<br>- Urinary porphobilinogen (PBG) levels<br>- Rates of porphyria attacks and hemin administration<br>- Patient-reported outcomes, assessed by the Givosiran Patient Experience Questionnaire (GPEQ) at end of study<br>- Frequency of adverse events

Secondary Outcome Measures

Name	Time	Method

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