Genetics
Protocol:110-504
Condition or Disease: Mucopolysaccharidosis Type IVA
Study Type: Observational
Status: Open for enrollment
Study Title: Morquio A Registry Study (MARS)
Principal I investigator: Angela Berg, DNP, APRN, CPN
Genetics
Protocol:110-504
Condition or Disease: Mucopolysaccharidosis Type IVA
Study Type: Observational
Status: Open for enrollment
Study Title: Morquio A Registry Study (MARS)
Principal I investigator: Angela Berg, DNP, APRN, CPN
Bleeding and Clotting Disorders
Protocol:CSL627_3001
Condition or Disease: Hemophilia A
Study Type: Interventional (Clinical Trial)
Status: Open for enrollment
Study Title: A Phase III Open Label, Multicenter, Extension Study to Assess the Safety and Efficacy of Recombinant Coagulation Factor VIII (rVIII SingleChain, CSL627) in Subjects with Severe Hemophilia A
Principal I investigator: Joseph Lasky III, MD
Protocol:PRO15070638
Condition or Disease: Von Willebrand disease (vWD)
Study Type: Interventional (Clinical Trial)
Status: Opening Late 2018
Study Title: The VWD Minimize Menorrhagia (VWDMin) Trial
Principal I investigator: Joseph Lasky III, MD
Bleeding and Clotting Disorders
Protocol:ALN-AT3SC-003
Condition or Disease: Hemophilia A or B
Study Type: Interventional (Clinical Trial)
Status: Open for enrollment
Study Title: ATLAS-INH: A Phase 3 Study to Evaluate the Efficacy and Safety of Fitusiran in Patients with Hemophilia A or B, with Inhibitory Antibodies to Factor VIII or IX
Principal I investigator: Joseph Lasky III, MD
More Info: //clinicaltrials.gov/ct2/show/NCT03417102
Bleeding and Clotting Disorders
Protocol:ALN-AT3SC-004
Condition or Disease: Hemophilia A or B
Study Type: Interventional (Clinical Trial)
Status: Open for enrollment
Study Title: ATLAS-A/B: A Phase 3 Study to Evaluate the Efficacy and Safety of Fitusiran in Patients With Hemophilia A or B, Without Inhibitory Antibodies to Factor VIII or IX
Principal I investigator: Joseph Lasky III, MD
More Info: //clinicaltrials.gov/ct2/show/NCT03417245
Bleeding and Clotting Disorders
Protocol:GENA-25
Condition or Disease: Hemophilia A
Study Type: Observational
Status: Open for enrollment
Study Title: PRactical utilization of Octapharma FVIII Concentrates in Previously Untreated and Minimally Treated Haemophilia A Patients Entering Routine Clinical Treatment (with Nuwiq, Octanate or Wilate) ‰ÛÒ A Real- World Efficacy and Safety Observational Study
Principal I investigator: Joseph Lasky III, MD
More Info: //clinicaltrials.gov/ct2/show/NCT03695978
Bleeding and Clotting Disorders
Protocol:1160.106
Condition or Disease: Venous thrombosis
Study Type: Interventional (Clinical Trial)
Status: Open for enrollment
Study Title: Open-label, randomized, parallel-group, active-controlled, multi-centre, non-inferiority study of dabigatran etexilate versus standard of care for venous thromboembolism treatment in children from birth to less than 18 years of age: The DIVERSITY study
Principal I investigator: Alan Ikeda, MD
More Info: //clinicaltrials.gov/ct2/show/NCT01895777
Bleeding and Clotting Disorders
Protocol:1160.108
Condition or Disease: Venous Thromboembolism, Secondary Prevention
Study Type: Interventional (Clinical Trial)
Status: Open for enrollment
Study Title: Open label, single arm safety prospective cohort study of dabigatran etexilate for secondary prevention of venous thromboembolism in children from 0 to less than 18 years
Principal I investigator: Alan Ikeda, MD
More Info: //clinicaltrials.gov/ct2/show/NCT02197416
Bleeding and Clotting Disorders
Protocol:1321.7
Condition or Disease: Hemorrhage
Study Type: Interventional (Clinical Trial)
Status: Open for enrollment
Study Title: Single dose, open label, uncontrolled, safety trial of intravenous administration of idarucizumab to paediatric patients enrolled from ongoing phase IIb/III clinical trials with dabigatran etexilate for the treatment and secondary prevention of venous thromboembolism.
Principal I investigator: Alan Ikeda, MD
More Info: //clinicaltrials.gov/ct2/show/NCT02815670
Genetics
Protocol:DIREGC07009
Condition or Disease: Gaucher
Study Type:
Status: Open for enrollment
Study Title: Gaucher Registry
Principal I investigator: Alan Ikeda, MD
More Info: //clinicaltrials.gov/ct2/show/NCT00358943