A Study to Evaluate Long-term Safety and Clinical Activity of Givosiran (ALN-AS1) in Patient With Acute Intermittent Porphyria (AIP)

Alnylam Pharmaceuticals16 enrolled

Overview

The purpose of this study is to determine the long-term safety, tolerability and pharmacokinetics of givosiran (ALN-AS1) in AIP patients who completed study ALN-AS1-001 (NCT02452372).

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Acute Intermittent Porphyria

Interventions

GivosiranDRUG

Givosiran by subcutaneous (SC) injection.

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Completed participation in Part C of study ALN-AS1-001 (NCT02452372) * Not on a scheduled regimen of hemin * Women of child bearing potential must have a negative serum pregnancy test, not be nursing, and use acceptable contraception * Willing and able to comply with the study requirements and to provide written informed consent Exclusion Criteria: * Clinically significant abnormal laboratory results * Received an investigational agent (other than ALN-AS1) within 90 days before the first dose of study drug or are in follow-up of another clinical study * History of multiple drug allergies or intolerance to subcutaneous injection

Locations (5)

Clinical Trial Site

San Francisco, California, United States

Clinical Trial Site

New York, New York, United States

Clinical Trial Site

Galveston, Texas, United States

Clinical Trial Site

Stockholm, Sweden

Outcomes

Primary Outcomes

Percentage of Participants With Adverse Events (AEs)

An AE is any untoward medical occurrence in a participant or clinical investigational patient administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment.

Time frame: Through Month 49

Secondary Outcomes

The Pharmacodynamic (PD) Effect of Givosiran on Urine Levels of Delta-aminolevulinic Acid (ALA) as Measured by Percent Decrease From Baseline

The PD effect of givosiran was evaluated by spot urine ALA levels normalized to spot urine creatinine levels.

Time frame: Baseline; Month 48

The Pharmacodynamic (PD) Effect of Givosiran on Urine Levels of Porphobilinogen (PBG) as Measured by Percent Decrease From Baseline

The PD effect of givosiran was evaluated by spot urine PBG levels normalized to spot urine creatinine levels.

Time frame: Baseline; Month 48

Annualized Rate of Composite Porphyria Attacks

Porphyria attacks were defined as meeting all of the following criteria: an acute episode of neurovisceral pain in the abdomen, back, chest, extremities and/or limbs, no other medically determined cause, and required treatment with intravenous (IV) dextrose or hemin, carbohydrates, or analgesics, or other medications such as antiemetics at a dose or frequency beyond the participant's usual daily porphyria management. Composite porphyria attacks included porphyria attacks that required hospitalization, urgent healthcare visit, or intravenous (IV) hemin administration at home. The annualized attack rate (AAR) was calculated as the number of composite porphyria attacks/total person-years.

Time frame: Through Month 48

Annualized Rate of Hemin Administration

The annualized rate of hemin administration was evaluated by annualized days of hemin use, which is calculated as the number of doses of hemin administered/total person-years.

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.