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222 : Eighteen-Month Interim Analysis of Efficacy and Safety of Givosiran, an RNAi Therapeutic for Acute Hepatic Porphyria, in the Envision Open Label Extension

Researchers

Presenter

  • David J. Kuter

Principal Investigators

  • Sioban Keel

  • Charles J. Parker

  • David C. Rees

  • Ulrich Stlzel

  • Paolo Ventura

  • Manisha Balwani

  • Laurent Gouya

  • Amy Simon

  • Shangbin Liu

  • John J Ko

  • Sean Rhyee

  • Samuel M. Silver

Medical Centers

  • Nephrology Division, Massachusetts General Hospital, Boston, MA

  • Dept of Medicine, Univ of Washington, Seattle, WA

  • University of Utah School of Medicine, Salt Lake City, UT

  • Department of Oncology, King's College Hospital, London, United Kingdom

  • Klinikum Chemnitz, Chemnitz, Germany

  • Università degli Studi di Modena e Reggio Emilia, Emilia-Romagna, Italy

  • Icahn School of Medicine at Mount Sinai, New York, NY

  • Centre Francais des Porphyries, Paris, France

  • Merrimack Pharmaceuticals, Cambridge, MA

  • University of Michigan, Ann Arbor, MI

Locations

  • United States

  • United Kingdom

  • Germany

  • Italy

  • France

Companies

  • N/A

Study Components

Therapeutic Area

  • Genetic Disorder

Disease

  • Acute hepatic porphyria

Biomarkers

  • N/A

Drug/Treatment

  • ALN-AS1

  • hemin

Outcome

  • N/A

Study Design

  • Randomized

  • Placebo Control

  • Open Label

  • Controlled

Phase

  • III

Study Id's

  • NCT03338816

Sponsors

  • N/A

Result

  • N/A

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