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SUN-325 : SAFETY AND EFFICACY OF LUMASIRAN, AN INVESTIGATIONAL RNA INTERFERENCE (RNAi THERAPEUTIC, IN ADULT AND PEDIATRIC PATIENTS WITH PRIMARY HYPEROXALURIA TYPE 1

Researchers

Presenter

  • W. van't Hoff

Principal Investigators

  • P. Cochat

  • J. Groothoff

  • J. Harambat

  • Y. Frishberg

  • S. Hulton

  • D. Magen

  • B. Hoppe

  • J. Lieske

  • D.S. Milliner

  • G. Deschenes

Medical Centers

  • Great Ormond Street Hospital, Paediatric Gastroenterology, London, United Kingdom

  • Shaare Zedek Medical Center, Pediatric Nephrology, Jerusalem, Israel

  • Hospital Robert Debre/Pediatric Nephrology, Paris, France

  • Amsterdam UMC, Nephrology, Amsterdam, Netherlands

  • University Hospital Bonn, Pediatric Nephrology, Bonn, Germany

  • Université Claude Bernard, Centre de Référence des Maladies Rares Néphrogones, Lyon, France

  • Nottingham Children's hospital, Paediatric Gastroenterology, Nottingham, United Kingdom

  • Rambam Health Care Campus, Pediatric Nephrology, Haifa, Israel

  • Mayo Clinic, Nephrology and Hypertension, Rochester, USA

  • Bordeaux University Hospital, Hematology, Bordeaux, France

Locations

  • United Kingdom

  • Israel

  • France

  • Netherlands

  • Germany

  • United States

Companies

  • N/A

Study Components

Therapeutic Area

  • Genetic Disorder

  • Nephrology

Disease

  • Primary hyperoxaluria type 1

Biomarkers

  • N/A

Drug/Treatment

  • ALN-GO1

Outcome

  • N/A

Study Design

  • Randomized

  • Placebo Control

  • Double Blind/Blinded

  • Multicenteric

  • Controlled

Phase

  • II

Study Id's

  • N/A

Sponsors

  • N/A

Result

  • N/A

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