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Rare disease

Von Willebrand's disease

  • 6 clinical trials
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6 Clinical trials

Phase 2 Study of ALN-6400 Safety and Tolerability in Female Patients with Von Willebrand Disease and Heavy Menstrual Bleeding

InvestigationalNo placebo
Countries:GermanyGermany
  • Participants:18–64 years
  • Substances:RNA, (UM-SP-(2′-DEOXY-2′-FLUORO)A-SP-GM-AM-AM-(2′-DEOXY-2′-FLUORO)A-CM-UM-CM-AM-UM-AM-GM-(2′-DEOXY-2′-FLUORO)C-GM-(2′-DEOXY-2′-FLUORO)A-UM-UM-GM-CM-AM-SP-CM-SP-AM), COMPLEX WITH RNA (UM-SP-GM-SP-CM-AM-AM-UM-(2′-DEOXY-2′-FLUORO)C-GM-(2′-DEOXY-2′-FLUORO)C-(2′-DEOXY-2′-FLUORO)U-(2′-DEOXY-2′-FLUORO)A-UM-GM-AM-GM-UM-UM-UM-CM-UM-AM) 3′-[[(2S,4R)-1-[29-[[2-(ACETYLAMINO)-2-DEOXY-Β-D-GALACTOPYRANOSYL]OXY]-14,14-BIS[[3-[[3-[[5-[[2-(ACETYLAMINO)-2-DEOXY-Β-D-GALACTOPYRANOSYL]OXY]-1-OXOPENTYL]AMINO]PROPYL]AMINO]-3-OXOPROPOXY]METHYL]-1,12,19,25-TETRAOXO-16-OXA-13,20,24-TRIAZANONACOS-1-YL]-4-HYDROXY-2-PYRROLIDINYL]METHYL HYDROGEN PHOSPHATE] (1:1)
  • Sponsor:Alnylam Pharmaceuticals Inc.

A Study of VGA039 to Prevent Bleeding in Teenagers and Adults with von Willebrand Disease Who Have Not Used Regular Preventive Treatment Before

InvestigationalNo placebo
Countries:AustriaAustria
  • Participants:18–64 years · 65+ years
  • Substances:VGA039
  • Sponsor:Vega Therapeutics Inc.

Study on the Safety and Effectiveness of Emicizumab for Patients with TYPE 3 Von Willebrand Disease

Registered drugNo placebo
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Efmoroctocog Alfa
  • Sponsor:F. Hoffmann-La Roche AG

Study on Preventing Bleeding in Children with Severe von Willebrand Disease Using Vonicog Alfa and Octocog Alfa

Registered drugNo placebo
Countries:FranceFrance
  • Participants:0–17 years
  • Substances:Octocog Alfa
  • Sponsor:Takeda Development Center Americas Inc.

Study on the Effects of BT200 (Rondaptivon Pegol) for Patients with Type 2B von Willebrand Disease

Investigational
Countries:AustriaAustria
  • Participants:18–64 years · 65+ years
  • Substances:Rondaptivon Pegol
  • Sponsor:Medical University Of Vienna

Study on the Effectiveness and Safety of Vonicog Alfa with or without Octocog Alfa for Children with Severe von Willebrand Disease Experiencing Bleeding or Undergoing Surgery

Registered drugNo placebo
Countries:AustriaAustria
  • Participants:0–17 years
  • Substances:Octocog Alfa
  • Sponsor:Baxalta Innovations GmbH
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