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Hematology/Oncology Department
Research Studies Open for Enrollment 		Date: 12/12/2018

Oncology:

Title ID/Protocol NCT/Phase Sponsor P.I. Contact
SICKLE CELL DISEASE
A Phase 2a, Randomised, Double-Blind, Placebo-Controlled Study of IMR-687 in Adult Patients with Sickle Cell Anaemia (Homozygous HbSS or Sickle-Beta-0 Thalassemia)Imara NCT03401112
Phase 2a		ImaraCarolyn Hoppe, MD .
Alloimmunization in Sickle Cell DiseaseAlloimmunization N/A
UnknownCarolyn Hoppe, MD .
Anti-Sickling AgentsAnti-Sickling N/A
UCSF Benioff Children's Hospital OaklandAnne Marsh, MD .
Application of the HemeChip Point of Care Device for Real-time Monitoring of Hemoglobin S Levels in Chronically Transfused Patients with Sickle Cell DiseaseHemeChip N/A
Doris Duke Charitable FoundationCarolyn Hoppe, MD .
Association between Low Bone Mineral Density (BMD) and Avascular Necrosis (AVN) Chart Review StudySCD-BMD-AVN N/A
UCSF Benioff Children's Hospital OaklandLynne Neumayr, MD .
Cross-UC Campus Data Harmonization for Pediatric OutcomeHarmony N/A
Health Resources and Services Administration (HRSA)Lynne Neumayr, MD .
Cutaneous Pain Fiber Stimulation by Laser in Patients with Sickle Cell DiseaseLaser N/A
UnknownWard Hagar, MD .
Dissemination and Implementation of Stroke Prevention Looking at the Care Environment (DISPLACE)DISPLACE N/A
UCSF Benioff Children's Hospital OaklandCarolyn Hoppe, MD .
Efficacy and Safety of Rivipansel in the Treatment of Vaso-Occlusive Crisis in Hospitalized Subjects With Sickle Cell DiseaseGMI-1070 NCT02187003
Phase 3		PfizerLynne Neumayr, MD .
Engaging Parents of Children with Sickle Cell Disease and their Providers in Shared-Decision Making for Hydroxyurea (ENGAGE HU)Engage-Hydroxyurea NCT03442114
Patient-Centered Outcomes Research InstituteLynne Neumayr, MD .
Hydroxyurea for the Prevention of Brain Injury in Sickle Cell Disease (HU Prevent)HU Prevent N/A
National Heart, Lung & Blood Institute (NHLBI)Lynne Neumayr, MD .
IMPLEmenting MEasures NeTwork for Child Health (IMPLEMENT for Child Health) AIM 2IMPLEMENT N/A
UnknownAnne Marsh, MD .
Natural history of overt or silent strokes in adults with sickle cell anemia treated with blood transfusion or hydroxyurea therapyStroke-HU N/A
BCHOCarolyn Hoppe, MD .
Open-label, Single and Multiple Dose Study to Evaluate the Pharmacokinetics, Safety, Tolerability and Treatment Effect of Voxelotor (GBT440) in Pediatric Participants with Sickle Cell DiseaseGBT440-Phase2a NCT02850406
Phase 2a		Global Blood TherapeuticsCarolyn Hoppe, MD .
Phase 1/2 Study of Hematopoietic Stem Cell Therapy for Young Adults with Severe Sickle Cell DiseaseHSCTSCD N/A
Phase 1/2		National Heart, Lung & Blood Institute (NHLBI)Mark Walters, MD .
Prospective Study to Evaluate Disease Status in Patients with Sickle Cell Disease: Global Regional Network for Database and Discovery (GRNDaD)GRNDaD N/A
Doris Duke Charitable FoundationCarolyn Hoppe, MD .
Randomized, Double-Blind, Placebo-Controlled Multi-Center Study to Assess the Safety, Tolerability, and Efficacy of Riocigut in Patients with Sickle Cell DiseasesSTERIO NCT02633397
Phase 2b		University of PittsburghWard Hagar, MD .
Sickle Cell Disease Implementation Consortium Registry: Collecting and Tracking Health Information for Patients with Sickle Cell DiseaseSCCCI N/A
National Heart, Lung and Blood InstituteMarsha Treadwell, PhD .
Sickle Cell Disease Longitudinal Data CollectionSCDLong N/A
UnknownLynne Neumayr, MD .
Specimen Banking for Future Sickle Cell Disease ResearchSpecimen N/A
UCSF Benioff Children's Hospital OaklandCarolyn Hoppe, MD .
Thalassemia
Iron and Cardiac Function in Sickle Cell Disease and ThalassemiaIronCardiac N/A
UnknownWard Hagar, MD .
STEM CELL TRANSPLANTATION AND CELLULAR THERAPY
Sickle Cell
Bone Marrow Transplantation Compared to Standard Care in Adolescents and Young Adults with Severe Sickle Cell DiseaseBMT-CTN-1503-STRIDE2 NCT02766465
Phase 2		BMT-CTN, Medical College of WisconsinMark Walters, MD .
Phase 1 Study Evaluating Gene Therapy by Transplantation of Autologous CD34 Stem Cells Transduced Ex Vivo with the LentiGlobin BB305 Lentiviral Vector in Subjects with Severe Sickle Cell DiseaseHGB-206 02140554
Phase 1		bluebird bio, Inc.Mark Walters, MD .
Reduced Intensity Conditioning for Haploidentical Bone Marrow Transplantation in Patients with Symptomatic Sickle Cell DiseaseBMT-CTN-1507 NCT03263559
Phase 2		BMT-CTN, Medical College of WisconsinMark Walters, MD .
Sickle Cell, Thalassemia
Allogeneic Stem Cell Transplantation of CordIn (Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells) in Patients with HemoglobinopathiesCordIn NCT02504619
Phase 1/2		Gamida Cell LtdMark Walters, MD .
Long Term Follow Up for Patients who have received Allogeneic Stem Cell Transplantation of NiCord/CordIn, Umbilical Cord Blood-derived Ex Vivo Expanded Stem and Progenitor CellsNiCord N/A
Gamida Cell LtdMark Walters, MD .
Longterm Follow-up of Subjects with Hemoglobinopathies Treated With Ex Vivo Gene Therapy Using Autologous Hematopoietic Stem Cells Transduced With a Lentiviral VectorLentiviral N/A
bluebird bio, Inc.Mark Walters, MD .
Sickle Cell, Thalassemia, Hematology
Sample Collection of Bone Marrow, Blood, Peripheral Blood Stem Cells or Umbilical Cord Blood from Patients with Genetic Diseases Affecting the Blood CellsBMT-repository N/A
California Institute for Regenerative MedicineMark Walters, MD .

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