Sickle Cell
Protocol:D5136C00009
Condition or Disease: Sickle Cell Disease
Study Type: Interventional (Clinical Trial)
Status: Open for enrollment
Study Title: A randomised, double-blind, parallel-group, multicenter, phase III study to evaluate the effect of ticagrelor bid versus placebo in reducing the number of VOCs in paediatric patients with sickle cell disease.
Principal I investigator: Nik Farahana Nik Abdul Rashid, MD
More Info: //clinicaltrials.gov/ct2/show/NCT03615924
Genetics
Protocol:VTS-270-303
Condition or Disease: Niemann-Pick Disease, Type C
Study Type: Interventional (Clinical Trial)
Status: Opening Mid 2019
Study Title: An Open-label, Multicenter Safety and Tolerability Study of VTS-270 (2-hydroxypropyl-ë_-cyclodextrin) in Pediatric Subjects Aged < 4 Years With Neurologic Manifestations of Niemann-Pick Type C (NPC) Disease
Principal I investigator: Alan Ikeda, MD
More Info: //clinicaltrials.gov/ct2/show/NCT03687476
Genetics
Protocol:VTS-270-001
Condition or Disease: Niemann-Pick Disease, Type C
Study Type: Expanded Access
Status: Open for enrollment
Study Title: An Open-label Expanded Access Treatment Protocol for VTS-270 (2-hydroxypropyl-ë_-cyclodextrin) in Patients With Neurologic Manifestations of Niemann-Pick Type C Disease (NPC)
Principal I investigator: Alan Ikeda, MD
More Info: //clinicaltrials.gov/ct2/show/NCT03643562
Genetics
Protocol:VTS-270 IND
Condition or Disease: Niemann-Pick Disease, Type C
Study Type: Expanded Access
Status: Open for enrollment
Study Title: Intrathecal 2-Hydroxypropyl-ë_-cyclodextrin for Neurological Decline in Patients with Niemann-Pick Disease Type C1
Principal I investigator: Alan Ikeda, MD
Sickle Cell
Protocol:GBT440-034
Condition or Disease: Sickle Cell Disease
Study Type: Interventional (Clinical Trial)
Status: Open for enrollment
Study Title: An Open Label Extension Study GBT440 administered Orally to Patients with Sickle Cell Disease Who Have Participate in GBR440 Clinical Trials.
Principal I investigator: Alan Ikeda, MD
More Info: //clinicaltrials.gov/ct2/show/NCT03573882
Sickle Cell
Protocol:GBT440-031
Condition or Disease: Sickle Cell Disease
Study Type: Interventional (Clinical Trial)
Status: Enrollment Paused
Study Title: A Double-blind, Randomized, Placebo-controlled, Multicenter Study of GBT440 Administered Orally to Subjects with Sickle Cell Disease
Principal I investigator: Alan Ikeda, MD
More Info: //clinicaltrials.gov/ct2/show/NCT03036813
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
