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800 ⋆ : Phase 1, Randomized, Placebo Controlled Study of Givosiran, an Investigational RNA Interference (RNAi) Therapeutic, in Patients with Acute Intermittent Porphyria: Interim study results

Researchers

Presenter

  • Eliane Sardh

Principal Investigators

  • Manisha Balwani

  • Penny Stein

  • David Rees

  • Joseph R. Bloomer

  • D Montgomery Bissell

  • Robert J. Desnick

  • Charles Parker

  • John Phillips

  • Herbert L. Bonkovsky

  • Nabil Al-Tawil

  • Craig penz

  • Amy Chan

  • Chang-Hoek Soh

  • William Querbes

  • Amy Simon

  • Stephanie Rock

  • Karl E. Anderson

Medical Centers

  • Rheumatology Unit, Karolinska University Hospital, Karolinska Institute, Stockholm, Sweden

  • Porphyria Centre Sweden, Stockholm, Sweden

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

  • Kings College London, London, United Kingdom

  • University of Alabama, Birmingham, AL

  • University of California, San Francisco, CA

  • University of Utah, Salt Lake City, UT

  • Wake Forest University, Winston-Salem, NC

  • Clinical Development, Alnylam, Cambridge, MA

  • The University of Texas Medical Branch, Galveston, TX

  • Alnylam, Cambridge, MA

Locations

  • Sweden

  • United States

  • United Kingdom

Companies

  • N/A

Study Components

Therapeutic Area

  • Genetic Disorder

Disease

  • Acute intermittent porphyria

Biomarkers

  • N/A

Drug/Treatment

  • ALN-AS1

Outcome

  • N/A

Study Design

  • Randomized

  • Placebo Control

  • Pharmacokinetics

  • Pharmacodynamics

  • Single Ascending Dose

  • Multiple Ascending Dose

  • Controlled

Phase

  • I

Study Id's

  • NCT02452372

Sponsors

  • N/A

Result

  • N/A

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