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PB2314 : OLIPUDASE ALFA FOR ADULTS WITH ACID SPHINGOMYELINASE DEFICIENCY: IMPROVEMENTS IN CROSSOVER PLACEBO PATIENTS AND FURTHER IMPROVEMENTS IN ORIGINAL OLIPUDASE ALFA PATIENTS AFTER 2 YEARS IN ASCEND TRIAL

Researchers

Presenter

  • Jesus Villarrubia

Principal Investigators

  • Antonio Barbato

  • Renata C Gallagher

  • Roberto Giugliani

  • Norberto B Guelbert

  • Julia B Hennermann

  • Carla Hollak

  • Takayuki Ikezoe

  • Robin Lachmann

  • Olivier Lidove

  • Paulina Mabe

  • Eugen Mengel

  • Maurizio Scarpa

  • Ebubekir Senates

  • Michel Tchan

  • Beth L. Thurberg

  • Abhimanyu Yarramaneni

  • Andreea M. Rawlings

  • Yong Kim

  • Monica Kumar

Medical Centers

  • Sanofi, Cambridge, United States

  • Clinical Medicine and Surgery,Federico II University Hospital,Naples,Italia

  • Regional Coordinator Centre for Rare Diseases,University Hospital of Udine,Udine,Italie

  • INAGEMP,Porto Alegre,Brésil

  • Pediatric and Adolescent Medicine,Villa Metabolica, University Medical Center,Mainz,Germany

  • Pediatric and Adolescent Medicine,Villa Metabolica, University Medical Center,Mainz,Germany

  • Croix Saint-Simon Hospital, Paris, France

  • Reumatologia, Hospital Universitairo Ramon y Cajal, Madrid, Spain

  • Sanofi,Paris,Frankrijk

  • Childrens Hospital at Montefiore, Albert Einstein College of Medicine, Bronx, NY

  • Childrens Hospital at Montefiore, Albert Einstein College of Medicine, Bronx, NY

  • Pediatrics, University of California, San Francisco, CA, USA

  • Regional Coordinator Centre for Rare Diseases,University Hospital of Udine,Udine,Italie

  • Gastroenterology, Fukushima Medical University, Fukushima, Japan

  • Pediatric and Adolescent Medicine,Villa Metabolica, University Medical Center,Mainz,Germany

  • Charles Dent Metabolic Unit,National Hospital for Neurology and Neurosurgery,London,Соединённое Королевство

  • La Croix St Simon Hospital,Paris,Frankrike

  • Childrens Hospital at Montefiore, Albert Einstein College of Medicine, Bronx, NY

  • Pediatrics, University of California, San Francisco, CA, USA

  • Clinical Science for LSD,SpinCS,Hochheim,Tyskland

  • Clinical Medicine and Surgery,Federico II University Hospital,Naples,Italia

  • Sanofi,Paris,França

  • Pediatrics,University of California,San Francisco, CA,Vereinigte Staaten

  • Med Genet Serv &amp

  • Hematology Department,Fukushima Medical University,Fukushima,Japan

  • Endocrinology and Metabolism,Amsterdam UMC, University of Amsterdam,Amsterdam,Nederländerna

  • Sanofi,Paris,Frankrijk

  • Charles Dent Metabolic Unit,National Hospital for Neurology and Neurosurgery,London,Royaume-uni

  • Clinical Science for LSD,SpinCS,Hochheim,Германия

  • Clinical Medicine and Surgery,Federico II University Hospital,Naples,Italia

  • Hematology,Hospital Universitario La Paz,Madrid,España

  • Clinical Science for LSD,SpinCS,Hochheim,Tyskland

  • La Croix St Simon Hospital,Paris,Frankrike

  • Charles Dent Metabolic Unit,National Hospital for Neurology and Neurosurgery,London,Royaume-uni

  • Sanofi Bridgewater NJ United States

  • Reumatologia, Hospital Universitairo Ramon y Cajal, Madrid, Spain

  • Clinical Medicine and Surgery,Federico II University Hospital,Naples,Italia

  • Clinical Science for LSD,SpinCS,Hochheim,Tyskland

  • Regional Coordinator Centre for Rare Diseases,University Hospital of Udine,Udine,Italie

  • Pediatrics, University of California, San Francisco, CA, USA

  • Istanbul Medeniyet University Hospital, Istanbul, Turkey

  • La Croix St Simon Hospital,Paris,Frankrike

  • Clinica Santa Maria,Santiago,Чили

  • Hematology,Hospital Universitario La Paz,Madrid,España

  • Sanofi,Cambridge, MA,Estados Unidos

  • Regional Coordinator Centre for Rare Diseases,University Hospital of Udine,Udine,Italie

  • Endocrinology and Metabolism,Amsterdam UMC, University of Amsterdam,Amsterdam,Nederländerna

  • Pediatric and Adolescent Medicine,Villa Metabolica, University Medical Center,Mainz,Germany

  • Endocrinology and Metabolism,Amsterdam UMC, University of Amsterdam,Amsterdam,Nederländerna

  • INAGEMP,Porto Alegre,Brésil

  • Endocrinology and Metabolism,Amsterdam UMC, University of Amsterdam,Amsterdam,Nederländerna

  • Childrens Hospital at Montefiore, Albert Einstein College of Medicine, Bronx, NY

  • Sanofi,Bridgewater,États-unis

  • Sanofi,Bridgewater, NJ,Vereinigte Staaten

  • Hematology Department,Fukushima Medical University,Fukushima,Japan

  • Reina Fabiola University Clinic,Córdoba,Argentine

  • Clinical Medicine and Surgery,Federico II University Hospital,Naples,Italia

  • Reina Fabiola University Clinic,Córdoba,Аргентина

  • Sanofi,Cambridge, MA,Vereinigte Staaten

  • Pediatric and Adolescent Medicine,Villa Metabolica, University Medical Center,Mainz,Germany

  • Sanofi,Cambridge, MA,Vereinigte Staaten

  • Reina Fabiola University Clinic,Córdoba,Argentine

  • INAGEMP,Porto Alegre,Бразилия

  • Charles Dent Metabolic Unit,National Hospital for Neurology and Neurosurgery,London,Royaume-uni

  • Genetic Medicine,Westmead Hospital,Sydney,Australië

  • INAGEMP,Porto Alegre,Brésil

  • Reumatologia, Hospital Universitairo Ramon y Cajal, Madrid, Spain

  • Pediatrics, University of California, San Francisco, CA, USA

  • University of California, San Francisco, United States

  • Regional Coordinator Centre for Rare Diseases,University Hospital of Udine,Udine,Italie

  • Hematology,Fukushima Medical University,Fukushima,Giappone

  • Clinical Medicine and Surgery,Federico II University Hospital,Naples,Italia

  • Sanofi, Bridgewater, NJ

  • Hematology Department,Fukushima Medical University,Fukushima,Japan

  • Genetic Medicine,Westmead Hospital,Sydney,Australië

  • Hematology,Fukushima Medical University,Fukushima,Giappone

  • Clinical Medicine and Surgery, Federico II University of Naples, Napoli, Italy

  • Endocrinology and Metabolism,Amsterdam UMC, University of Amsterdam,Amsterdam,Nederländerna

  • Sanofi,Paris,Frankrijk

  • INAGEMP,Porto Alegre,Brésil

  • La Croix St Simon Hospital,Paris,Франция

  • Charles Dent Metabolic Unit,National Hospital for Neurology and Neurosurgery,London,Royaume-uni

  • Charles Dent Metabolic Unit, National Hospital for Neurology & Neurosurgery London United Kingdom

  • Genetic Medicine,Westmead Hospital,Sydney,Австралия

  • Clinica Santa Maria, Santiago, Chile

  • Sanofi,Bridgewater, NJ,Stati Uniti

  • INAGEMP,Porto Alegre,Brésil

  • Childrens Hospital at Montefiore, Albert Einstein College of Medicine, Bronx, NY

  • INAGEMP,Porto Alegre,巴西

  • Pediatric and Adolescent Medicine,Villa Metabolica, University Medical Center,Mainz,Germany

  • Endocrinology and Metabolism,Amsterdam UMC, University of Amsterdam,Amsterdam,Nederländerna

  • Endocrinology and Metabolism,Amsterdam UMC, University of Amsterdam,Amsterdam,Nederländerna

  • Clinical Science for LSD,SpinCS,Hochheim,Tyskland

  • Clinical Science for LSD,SpinCS,Hochheim,Tyskland

  • Genetic Medicine,Westmead Hospital,Sydney,Australië

  • Charles Dent Metabolic Unit, National Hospital for Neurology & Neurosurgery London United Kingdom

  • Clinica Santa Maria, Santiago, Chile

  • Clinical Science for LSD,SpinCS,Hochheim,Tyskland

  • Reina Fabiola University Clinic,Córdoba,Argentine

  • Hematology,Hospital Universitario La Paz,Madrid,España

  • Sanofi, Cambridge, MA

  • Endocrinology and Metabolism,Amsterdam UMC, University of Amsterdam,Amsterdam,Nederländerna

  • Sanofi,Paris,Франция

  • La Croix St Simon Hospital,Paris,Frankrike

  • Clinical Science for LSD,SpinCS,Hochheim,Tyskland

  • Pediatric and Adolescent Medicine,Villa Metabolica, University Medical Center,Mainz,Germany

  • Clinical Science for LSD,SpinCS,Hochheim,Tyskland

  • Reumatologia, Hospital Universitairo Ramon y Cajal, Madrid, Spain

  • Pediatric and Adolescent Medicine,Villa Metabolica, University Medical Center,Mainz,Germany

  • La Croix St Simon Hospital,Paris,Frankrike

  • Sanofi,Cambridge, MA,États-unis

  • Childrens Hospital at Montefiore, Albert Einstein College of Medicine, Bronx, NY

  • Sanofi,Bridgewater, NJ,Estados Unidos

  • Pediatrics,University of California,San Francisco, CA,Vereinigte Staaten

  • Clinica Santa Maria, Santiago, Chile

  • Genetic Medicine,Westmead Hospital,Sydney,Australië

  • Reina Fabiola University Clinic,Córdoba,Argentine

  • Charles Dent Metabolic Unit, National Hospital for Neurology & Neurosurgery London United Kingdom

  • Hospital Universitario Ramón y Cajal,Madrid,Испания

  • Sanofi,Cambridge, MA,Stati Uniti

  • Genetic Medicine,Westmead Hospital,Sydney,Australië

  • Regional Coordinator Centre for Rare Diseases,University Hospital of Udine,Udine,Italie

  • Regional Coordinator Centre for Rare Diseases,University Hospital of Udine,Udine,Italie

  • Endocrinology and Metabolism,Amsterdam UMC, University of Amsterdam,Amsterdam,Nederländerna

  • Sanofi, Paris, France

  • DR BRASIL Research Grp, HCPA, Dept Genetics,UFRGS &amp

  • Childrens Hospital at Montefiore, Albert Einstein College of Medicine, Bronx, NY

  • Sanofi, Paris, France

  • Sanofi,Bridgewater, NJ,Vereinigte Staaten

  • Pediatric and Adolescent Medicine,Villa Metabolica, University Medical Center,Mainz,Germany

Locations

  • Spain

  • United States

  • Italy

  • Brazil

  • Mexico

  • Argentina

  • Germany

  • Japan

  • United Kingdom

  • France

  • Philippines

  • Chile

  • Turkey

  • Canada

  • Australia

Companies

  • N/A

Study Components

Therapeutic Area

  • Genetic Disorder

  • Endocrine/metabolic Diseases (ME)

Disease

  • Niemann-Pick disease type A

Biomarkers

  • N/A

Drug/Treatment

  • N/A

Outcome

  • N/A

Study Design

  • Placebo Control

  • Cross Over

  • Dose Escalation

Phase

  • II/III

Study Id's

  • NCT02004691

Sponsors

  • N/A

Result

  • N/A

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