Skip to main content
OpenTrials
Completed

NCT Number: NCT02949830

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

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).

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Acute Intermittent Porphyria Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Genetic Diseases, Inborn Liver Diseases Metabolic Diseases Nutritional and Metabolic Diseases Porphyria, Acute Intermittent Porphyrias Porphyrias, Hepatic Skin Diseases Skin Diseases, Genetic Skin and Connective Tissue Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Clinical Trial Site, Stockholm, Sweden

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Givosiran

Drug

Givosiran by subcutaneous (SC) injection.

Other names: ALN-AS1, Givlaari

Primary outcomes

  1. Percentage of Participants With Adverse Events (AEs)

    Time frame: Through Month 49

    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.

Secondary outcomes

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

    Time frame: Baseline; Month 48

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

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

    Time frame: Baseline; Month 48

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

  3. Annualized Rate of Composite Porphyria Attacks

    Time frame: Through Month 48

    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.

  4. Annualized Rate of Hemin Administration

    Time frame: Through Month 49

    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.

Sponsors and collaborators

Lead sponsor

Alnylam Pharmaceuticals

Industry

Registry information

Official study title

A Multicenter, Open-label Extension Study to Evaluate the Long-term Safety and Clinical Activity of Subcutaneously Administered ALN-AS1 in Patients With Acute Intermittent Porphyria Who Have Completed a Previous Clinical Study With ALN-AS1

Important dates

Study start
2016
Primary completion
2021
Study completion
2021
First posted
Oct 31, 2016
Registry last updated
Mar 12, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.