• Click on a protocol’s title to view its description in ClinicalTrials.gov.
  • Click on (pdf) to view the protocol (WUSTL key password required).
Antiphospholipid Syndrome (APS)
PI / ContactProtocol (Sponsor)Status
PI: Amir Mahmoud / Kristen Sanfilippo
Contact: Kim Kramer
kramerka@wustl.edu
557-747-2942
Efgartigimod in Antiphospholipid Antibody Syndrome Patients with Elevated IgG Antibodies: A pilot feasibility trial (Washington University in St. Louis) Not yet open to enrollment
Atypical HUS
PI / ContactProtocol (Sponsor)Status
PI: Elaine Majerus
Contact: Sabrina Barber
s.barber@wustl.edu
314-273-1440
Atypical Hemolytic-Uremic Syndrome (aHUS) Registry (Alexion)Open to enrollment
(pdf – password protected)
Bleeding and clotting disorders
PI / ContactProtocol (Sponsor)Status
PI: Elaine Majerus
Contact: Bill Berger
bergerwb@wustl.edu
314-362-9996
CDC Community Counts: Surveillance Project for Patients with Bleeding and Blood Clotting Disorders (CDC Division of Blood Disorders)Open
Essential Thrombocythemia
PI / ContactProtocol (Sponsor)Status
PI: Stephen Oh
Contact: Nicole Gaudin
nrgaudin@wustl.edu
314-747-7960
A Phase 3 Study of P1101 vs Anagrelide in Essential Thrombocythemia Patients who Failed Hydroxyurea (P1101ET) (PharmaEssentia Corp)Open to enrollment
(pdf – password protected)
PI: Stephen Oh
Contact: Nicole Gaudin
nrgaudin@wustl.edu
314-747-7960
A Phase 2b Open Label Study of Ropeginterferon (P1101) in Essential Thrombocythemia Patients (PharmaEssentia)Pending Open
(pdf – password protected)
Hemophilia A
PI / ContactProtocol (Sponsor)Status
PI: Elaine Majerus
Contact: Rachel Cody
rcody@wustl.edu
314-362-7306
A Phase 3 Study of Valoctocogene Roxaparvovec in Hemophilia A Patients (BioMarin Pharmaceutical)Open
PI: Elaine Majerus
Contact: Sabrina Barber
s.barber@wustl.edu
314-273-1440
Emicizumab In Patients With Acquired Hemophilia A: Multicenter, Single-Arm, Open-Label Clinical Trial (Genetech)Enrollment pending
(pdf – password protected)
PI: Elaine Majerus
Contact: Sabrina Barber
s.barber@wustl.edu
314-273-1440
Efficacy and Safety of Concizumab prophylaxis in patients with haemophilia A or B with inhibitors (Novo Nordisk Pharmaceuticals, Inc.)Open to enrollment
(pdf – password protected)
Hemophilia A, Hemophilia B, and Factor VII Deficiency
PI / ContactProtocol (Sponsor)Status
PI: Elaine Majerus
Contact: Kim Kramer
kramerka@wustl.edu
557-747-2942
A Phase 1 Single and Multiple Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics,
Pharmacodynamics, and Efficacy of SR604 in Participants with Hemophilia A or Hemophilia B or Factor VII deficiency (Part B only)
(Equilibra Bioscience LLC)
Open to enrollment
(pdf – password protected)
Hemophilia/Bleeding
PI / ContactProtocol (Sponsor)Status
PI: Elaine Majerus
Contact: Bill Berger
bergerwb@wustl.edu
314-362-9996
American Thrombosis and Hemostasis Network Data Collection (CDC Division of Blood Disorders)Open
ITP
PI / ContactProtocol (Sponsor)Status
PI: Morey Blinder
Contact: Kim Kramer
kramerka@wustl.edu
557-747-2942
A study to assess the tolerability of ianalumab (VAY736) with investigator’s choice thrombopoietin receptor agonist (IC TPO-RA) in patients with primary immune thrombocytopenia (ITP) (Novartis Pharmaceuticals)Not yet open to enrollment
(pdf – password protected)
Low Grade MDS and CCUS
PI / ContactProtocol (Sponsor)Status
PI: Amber Afzal
Contact: Kim Kramer
kramerka@wustl.edu
557-747-2942
Pilot Study of Statins in Patients with Clonal Cytopenia of Undetermined Significance (CCUS) and Myelodysplastic Syndromes (MDS) (Washington University in St. Louis)Closed to enrollment
Myelofibrosis
PI / ContactProtocol (Sponsor)Status
PI: Dr. Amy Zhou
Contact: Nicole Gaudin 
nrgaudin@wustl.edu 
314-747-7960
RSK1 Targeting in Myelofibrosis (WashU Medicine)Enrolling
(pdf – password protected)
PI: Stephen Oh
Contact: Karyn Gordon
kdgordon@wustl.edu
314-362-0156
A Phase 3 Study of Pelabresib (CPI-0610) plus Ruxolitinib vs Placebo plus Ruxolitinib in Myelofibrosis Patients (Constellation Pharmaceuticals)Ongoing; closed to enrollment
(pdf – password protected)
PI: Amy Zhou
Contact: Nicole Gaudin
nrgaudin@wustl.edu
314-747-7960
A Phase 1b/2a Study of DISC-0974 in Myelofibrosis Patients with Anemia (DISC-0974-102) (DISC Medicine)Pending open
(pdf – password protected)
PNH
PI / ContactProtocol (Sponsor)Status
PI: Elaine Majerus
Contact: Sabrina Barber
s.barber@wustl.edu
314-273-1440
Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry (Alexion)Open to enrollment
PI: Elaine Majerus
Contact: Sabrina Barber
s.barber@wustl.edu
314-273-1440
A multicenter, single arm, open-label trial to evaluate efficacy and safety of oral, twice daily iptacopan in adult PNH patients who have Hb≥10 g/dL in response to anti-C5 antibody and switch to iptacopan (Novartis)Pending enrollment
(pdf – password protected)
Polycythemia Vera
PI / ContactProtocol (Sponsor)Status
PI: Stephen Oh
Contact: Karyn Gordon
kdgordon@wustl.edu
314-362-0156
A Phase 2a Study of ISIS 702843 in Phlebotomy Dependent Polycythemia Vera Patients (ISIS 702843-CS4) (Ionis Pharmaceuticals)Open to enrollment
(pdf – password protected)
PI: Stephen Oh
Contact: Karyn Gordon
kdgordon@wustl.edu
314-362-0156
A Phase 3b Study of Ropeginterferon (P1101) in Polycythemia Vera Patients (PharmaEssentia)Pending Open
(pdf – password protected)
Sickle Cell Disease
PI / ContactProtocol (Sponsor)Status
PI: Allison King
Contact: Lisa Garrett
garrett_l@wustl.edu
314-362-0051
Sickle Cell Disease Patient Registry (SCDIC-II) (NHLBI)Enrolling
(pdf – password protected)
PI: Allison King
Contact: Lisa Garrett
garrett_l@wustl.edu
314-362-0051
The Epidemiology of Silent and Overt Strokes in Adults with Sickle Cell Disease (Global Blood Therapeutics)Enrolling
(pdf – password protected)
PI: Allison King
Contact: Lisa Garrett
garrett_l@wustl.edu
314-362-0051
Sickle Cell Disease and CardiovAscular Risk-Red cell Exchange Trial (SCD-CARRE) (NHLBI)Enrolling
(pdf – password protected)
PI: Sana Saif Ur Rehman
Contact: Isuru Gamlath
isuru.gamlath@wustl.edu
314-362-9998
Patient Characteristics and Treatment Patterns from Early Crizanlizumab Use in Real-world Setting (National Alliance of Sickle Cell Centers)(pdf – password protected)
PI: Sana Saif Ur Rehman
Contact: Isuru Gamlath
isuru.gamlath@wustl.edu
314-362-9998
Globin research Network for DATA and Discovery (GRNDaD) (Global Blood Therapeutics)(pdf – password protected)
PI: Sana Saif Ur Rehman
Contact: Isuru Gamlath
isuru.gamlath@wustl.edu
314-362-9998
Globin research Network for DATA and Discovery (GRNDaD) substudy – HUGS (Global Blood Therapeutics)(pdf – password protected)
Systemic Mastocytosis
PI / ContactProtocol (Sponsor)Status
PI: Stephen Oh
Contact: Nicole Gaudin
nrgaudin@wustl.edu
314-747-7960
A Phase 2 Study of CGT9486 in Patients with Nonadvanced Systemic Mastocytosis (Summit) (Cogent Biosciences)Open to enrollment
(pdf – password protected)
PI: Stephen Oh
Contact: Nicole Gaudin
nrgaudin@wustl.edu
314-747-7960
A Phase 2 Study of Avapritinib (BLU-285) in Nonadvanced Systemic Mastocytosis Patients with Inadequately Controlled Symptoms (BLU-285-2203) (Blueprint Medicines)Ongoing; closed to enrollment
(pdf – password protected)
Von Willebrand Disease
PI / ContactProtocol (Sponsor)Status
PI: Elaine Majerus
Contact: Sabrina Barber
s.barber@wustl.edu
314-273-1440
A Natural History Cohort Study of the Safety, Effectiveness, and Practice of Treatment for People with Severe Von Willebrand Disease (VWD) (American Thrombosis and Hemostasis Network (ATHN))Open to enrollment
(pdf – password protected)