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Hematology/Oncology Department
Research Studies Open for Enrollment
Date: 10/22/2018
Title ID/Protocol NCT/Phase Sponsor P.I. Contact
THALASSEMIA
Evaluate the Efficacy and Safety of RBCs Derived from Mirasol-treated Whole Blood Compared with Conventional RBCs in Patients Requiring Chronic Transfusion SupportPRAISE NCT03329404
TerumoAshutosh Lal, MDKacie Smith (510-428-3885 x2752)
Multi-center, randomized, open-label, parallel-group study with LPC-401 for the treatment of myocardial iron overload in adult patients with transfusion-dependent beta-thalassemiaHEPCIDIN NCT03381833
Phase 2
La Jolla Pharmaceutical CompanyAshutosh Lal, MDJennifer Ferguson, RN, MSN, FNP (510-428-3885 x5324)
Single-Center, Non-Randomized Study of the Safety and Efficacy of In Utero Hematopoietic Stem Cell Transplantation for the Treatment of Fetuses with Alpha Thalassemia MajorIUHCT NCT02986698
Phase 1
UC San FranciscoElliott Vichinsky, MDElliott Vichinsky, MD (510-428-3885 x3651)
STEM CELL TRANSPLANTATION AND CELLULAR THERAPY
Oncology
Role of KIR-favorably Mismatched Haploidentical Transplantation and KIR-polymorphisms in Determining Outcomes of Children with ALL/AML/MDS Undergoing Allogeneic Hematopoietic Cell TransplantationONC1401 NCT02646839
Phase 1/2
Pediatric Blood and Marrow Transplatn ConsortiumNahal Rose Lalefar, MDMarci Moriarty, RN (510-428-3385 x5396)
Oncology: Early Phase, Leukemia
Pediatric and Young Adult Leukemia Adoptive Therapy. A Phase 1/2 Feasibility and Safety Study of CD19-CAR T Cell Immunotherapy for CD19+ LeukemiaPLAT-02 NCT02028455
Phase 1/2
Seattle Children's HospitalAnu Agrawal, MDMarci Moriarty, RN (510-428-3385 x5396)
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, MDMarci Moriarty, RN (510-428-3385 x5396)
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, MDMarci Moriarty, RN (510-428-3385 x5396)
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, MDMarci Moriarty, RN (510-428-3385 x5396)
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, MDMarci Moriarty, RN (510-428-3385 x5396)
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, MDJulie Decaris (510-428-3385 x2739)
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, MDMarci Moriarty, RN (510-428-3385 x5396)
Thalassemia
Evaluating Efficacy and Safety of Gene Therapy in Transfusion-dependent Beta-Thalassemia Beta0/Beta0 Genotype by Autologous Transplantation of CD34+ Stem Cells Transduced Ex Vivo with Lentiviral BA-T87Q Globin VectorHgb-212 NCT02906202
Phase 3
Bluebird BioAshutosh Lal, MDMarci Moriarty, RN (510-428-3385 x5396)
Open-label, Single-arm Study to Assess Safety, Tolerability, and Efficacy of ST-400 Autologous Hematopoietic Stem Cell Transplant for Treatment of Transfusion-dependent Beta thalassemia (TDT)ST-400-01 NCT03432364
Phase 1/2
Sangamo Therapeutics, Inc.Mark Walters, MDMarci Moriarty, RN (510-428-3385 x5396)
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, MDSmith, Kacie
Anti-Sickling AgentsAnti-Sickling N/A
UCSF Benioff Children's Hospital OaklandAnne Marsh, MDKacie Smith (510-428-3885 x2752)
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, MDCarolyn Hoppe, MD (510-428-3885 x3193)
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, MDMariam Hameed (510-428-3885 x4151)
Dissemination and Implementation of Stroke Prevention Looking at the Care Environment (DISPLACE)DISPLACE N/A
UCSF Benioff Children's Hospital OaklandCarolyn Hoppe, MDMariam Hameed (510-428-3885 x4151)
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, MDKacie Smith (510-428-3885 x2752)
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, MDMariam Hameed (510-428-3885 x4151)
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, MDKacie Smith (510-428-3885 x2752)
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, MDMariam Hameed (510-428-3885 x4151)
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, MDMariam Hameed (510-428-3885 x4151)
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, PhDTeleah Slater (510-428-3691)
ONCOLOGY: EARLY PHASE
Leukemia
Groupwide Pilot Study to Test the Tolerability and Biologic Activity of the Addition of Azacitidine to Chemotherapy in Infants with Acute Lymphoblastic Leukemia (ALL) and KMT2A (MLL) Gene RearrangementAALL15P1 NCT02828358
Phase 2
Children's Oncology GroupCarla Golden, MDChristine Ho (510-428-3885 x 2609)
Leukemia, Lymphoma
Pilot Study of Mitoxantrone in combination with Clofarabine in Children, Adolescents and Young Adults with Refractory/Relapsed Acute Leukemia or High Grade Non-Hodgkin LymphomaMITCL NCT01842672
Phase 1/2a
New York Medical CollegeJennifer Michlitsch, MDAnu Agrawal, MD (510-428-3885 x3539)
Lymphoma
Randomized Phase 2 Trial of Brentuximab Vedotin (SGN35), or Crizotinib (commercially labeled) in Combination with Chemotherapy for Newly Diagnosed Patients with Anaplastic Large Cell LymphomaANHL12P1 NCT01979536
Phase 2
Children's Oncology GroupCarla Golden, MDChristine Ho (510-428-3885 x 2609)
Risk adapted, response based trial of nivolumab and brentuximab vedotin followed by brentuximab vedotin and bendamustine for pediatric and young adults with relapsed or refractory CD30 positive classic Hodgkin lymphomaAHOD1721 NCT02927769
Phase 2
Children's Oncology GroupCarla Golden, MDChristine Ho (510-428-3885 x 2609)
Lymphoma, Solid Tumors
NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) - Phase 2 subprotocol of JNJ-42756493 (Erdafitinib) in patients with tumors harboring FGFR1/2/3/4 alterationsAPEC1621B NCT03210714
Phase 2
Children's Oncology GroupCarla Golden, MDChristine Ho (510-428-3885 x 2609)
NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) - Phase 2 subprotocol of LOXO-101 (larotrectinib) in patients with tumors harboring actionable NTRK fusionsAPEC1621A NCT03213704
Phase 2
Children's Oncology GroupCarla Golden, MDChristine Ho (510-428-3885 x 2609)
NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) - Phase 2 subprotocol of LY3023414 in Solid TumorsAPEC1621D NCT03155620
Phase 2
Children's Oncology GroupCarla Golden, MDChristine Ho (510-428-3885 x 2609)
NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) - Phase 2 subprotocol of olaparib in patients with tumors harboring defects in DNA damage repair genesAPEC1621H NCT03233204
Phase 2
Children's Oncology GroupCarla Golden, MDChristine Ho (510-428-3885 x 2609)
NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) - Phase 2 subprotocol of tazemetostat for patients with tumors harboring alterations in EZH2 or members of the SWI/SNF complexAPEC1621C NCT03213665
Phase 2
Children's Oncology GroupCarla Golden, MDChristine Ho (510-428-3885 x 2609)
NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice)- Phase 2 subprotocol of Ensartinib in patients with tumors harboring ALK or ROS1 genomic alterationsAPEC1621F NCT03213652
Phase 2
Children's Oncology GroupCarla Golden, MDChristine Ho (510-428-3885 x 2609)
NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice)- Phase 2 subprotocol of Vemurafenib in patients with tumors harboring actionable BRAF V600 mutationsAPEC1621G NCT03220035
Phase 2
Children's Oncology GroupCarla Golden, MDChristine Ho (510-428-3885 x 2609)
NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice): Phase 2 subprotocol of Selumetinib in patients with Tumors harboring activating MAPK pathway mutationsAPEC1621E NCT03213691
Phase 2
Children's Oncology GroupCarla Golden, MDChristine Ho (510-428-3885 x 2609)
Solid Tumors
NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) - Phase 2 subprotocol of Palbociclib in Patients with Tumors Harboring Activating Alterations in Cell Cycle GenesAPEC1621I N/A
Phase 2
Children's Oncology GroupCarla Golden, MDChristine Ho (510-428-3885 x 2609)
Neuroblastoma Maintenance Therapy Trial Using DifluoromethylornithineNMTRC-014-DFMO NCT02679144
Phase 2
Beat Childhood Cancer (BCC)Anu Agrawal, MDAnu Agrawal, MD (510-428-3885 x3539)
Pazopanib in Combination with Irinotecan and Temozolomide for Children and Young Adults with Relapsed or Refractory SarcomaPAZIT NCT03139331
Phase 1
University of California, San FranciscoJennifer Michlitsch, MDAnu Agrawal, MD (510-428-3885 x3539)
Phase 2 Trial of XL184 (Cabozantinib) an Oral Small-Molecule Inhibitor of Multiple Kinases, in Children and Young Adults with Refractory Sarcomas, Wilms Tumor, and Other Rare TumorsADVL1622 NCT02867592
Phase 2
Children's Oncology GroupCarla Golden, MDChristine Ho (510-428-3885 x 2609)
Randomized Phase 3 Trial Evaluating the Addition of the IGF-1R Monoclonal Antibody Ganitumab (AMG 479) to Multiagent Chemotherapy for Patients with Newly Diagnosed Metastatic Ewing SarcomaAEWS1221 NCT02306161
Phase 3
Children's Oncology GroupCarla Golden, MDChristine Ho (510-428-3885 x 2609)
Two-Part Study of TB-403 in Pediatric Subjects with Relapsed or Refractory Medulloblastoma, Neuroblastoma, Ewing Sarcoma or Alveolar RhabdomyosarcomaTB-403 NCT02748135
Phase 1/2a
Beat Childhood Cancer (BCC) and Oncurious NVJennifer Michlitsch, MDJennifer Michlitsch, MD (510-428-3885 x3264)
ONCOLOGY
Pediatric Tissue Bank at the Children's Hospital & Research Center OaklandONC Tissue Bank N/A
Jon Rowland, MD
The Effectiveness of Lactobacillus plantarum (LBP, IND# 17339) in Preventing Acute Graft-versus-Host Disease (GvHD) in Children undergoing Alternative Hematopoietic Progenitor Cell Transplantation (HCT)ACCL1633 N/A
Phase 3
Children's Oncology Group (COG)Carla Golden, MDChristine Ho (510-428-3885 x 2609)
Solid Tumors
Pediatric Hepatic Malignancy International Therapeutic Trial (PHITT)AHEP1531 N/A
Phase 3
Children's Oncology Group (COG)Carla Golden, MDChristine Ho (510-428-3885 x 2609)
NEURO-ONCOLOGY
Clinical/Molecular Risk-Tailored Intensive, Compressed Induction Chemotherapy Followed by Consolidation Randomization To Single-Cycle or 3 Tandem Cycles of Marrow-Ablative Chemo with Hematopoietic Progenitor Cell RescueHead-Start-4 NCT02875314
Phase 4
Next Consortium, Nationwide Children'sJoseph Torkildson, MDJoseph Torkildson, MD
Genomic Analysis in Pediatric MalignanciesGenomics N/A
N/ATabitha Cooney, MDTabitha Cooney, MD
H3.3.K27M specific peptide vaccine combined with poly-ILCL for the treatment of newly diagnosed HLA-A2+ H3.3K27M positive diffuse intrinsic pontine glioma and other newly diagnosed HLA-A2+ H3.3K27M positive gliomasPNOC007 NCT02960230
Phase 1
Pacific Pediatric Neuro-Oncology Consortium (PNOC)Tabitha Cooney, MDTabitha Cooney, MD (510-428-3885 x3398)
Phase 2 Study of everolimus for recurrent or progression low-grade gliomas in childrenPNOC001 NCT01734512
Phase 2
Pacific Pediatric Neuro-Oncology Consortium (PNOC)Joseph Torkildson, MDJoseph Torkildson, MD
Prospective multi-institutional study to assess the risk of radiation induced vasculopathy and stroke as well as stroke recurrence in children with cancer who received radiation therapy to the neck and/or brainRadART-Pro N/A
LaRoche FamilyJoseph Torkildson, MDJoseph Torkildson, MD
GENERAL HEMATOLOGY
Randomized, open-label, active controlled, safety and extrapolated efficacy study in pediatric subjects requiring anticoagulation for treatment of a venous thromboembolic eventApixaban NCT02464969
Phase 4
PfizerTiti Singer, MDSheila Contapay-Tabilin, RN (510-428-3885 x4754)

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