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Hematology/Oncology Department
Research Studies Open for Enrollment
Date: 01/19/2022
Title ID/Protocol NCT/Phase Sponsor P.I. Contact
THALASSEMIA
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, MD
STEM CELL TRANSP 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, MD
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, MD
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 1/2 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 1/2
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
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
Thalassemia
Evaluating Efficacy and Safety of Gene Therapy in Transfusion-dependent Beta-Thal Beta0/Beta0 Genotype by Autologous Transplantation of CD34+ Stem Cells Transduced Ex Vivo with Lentiviral BA-T87Q Globin Vector in Subjects < 50 YearsHgb-212 NCT02906202
Phase 3
Bluebird BioAshutosh Lal, MD
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 thalassemiaST-400-01 NCT03432364
Phase 1/2
Sangamo Therapeutics, Inc.Mark Walters, MD
SICKLE CELL DISEASE
A randomized, placebo-controlled, double blind, single ascending and multiple ascending dose study to assess the safety, pharmacokinetics and pharmacodynamics of FT-4202 in healthy volunteers and sickle cell disease patientsFORMA N/A
Phase 1
FORMAWard Hagar, MD
Anti-Sickling AgentsAnti-Sickling N/A
UCSF BCHOAnne 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 BCHOLynne Neumayr, MD
Engaging Parents of Children with Sickle Cell Disease and their Providers in Shared-Decision Making for Hydroxyurea (ENGAGE HU)Engage-Hydroxyurea NCT03442114
N/A
Patient-Centered Outcomes Research InstituteLynne Neumayr, 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
N/A
National Heart, Lung and Blood InstituteMarsha Treadwell, PhD
Sickle Cell
A Phase IIb Randomized, Double-Blind, Placebo-Controlled Multi-Center Study to Assess the Safety, Tolerability, and Efficacy of Riociguat in Patients with Sickle Cell DiseasesSTERIO-SCD N/A
Phase 2b
University of Pittsburgh Investigator InitiatedWard Hagar, MD
ONCOLOGY: EARLY PHASE
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, MD
Oncology
A Study Using Molecular Guided Therapy with Induction Chemotherapy followed by a Randomized Controlled Trial of standard Immunotherapy with or w/o DFMO followed by DFMO maintenance for Newly Diagnosed High-Risk NeuroblastomaNMTRC012 NCT02559778
Phase 2
Beat Childhood Cancer (BCC)Anu Agrawal, MD
Oncology, Neuro-Onc, Oncology: Early Phase, Lymphoma, Solid Tumors
NCI-COG PEDIATRIC MATCH (MOLECULAR ANALYSIS FOR THERAPY CHOICE) - PHASE 2 SUBPROTOCOL OF AG-120 (IVOSIDENIB) IN PATIENTS WITH TUMORS HARBORING IDH1 MUTATIONSAPEC1621K NCT04195555
Phase 2
COGCarla Golden, MD
Oncology, Oncology: Early Phase
Open-Label, Multicenter, Phase 2 Study Evaluating Efficacy and Safety of Daratumumab in Ped and Young Adults >=1 and =<30 Years with Relapsed/Refractory Precursor B-cell or T-cell ALLDaratumumab APEC1621B NCT03210714
Phase 2
COGCarla Golden, MD
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
COGCarla Golden, MD
NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) - Phase 2 subprotocol of LY3023414 in Solid TumorsAPEC1621D NCT03155620
Phase 2
COGCarla Golden, MD
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 NCT03526250
Phase 2
COGCarla Golden, MD
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
COGCarla Golden, MD
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
COGCarla Golden, MD
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
COGCarla Golden, MD
Oncology, Solid Tumors
Neuroblastoma Maintenance Therapy Trial Using DifluoromethylornithineNMTRC-014 NCT02679144
Phase 2
Beat Childhood Cancer (BCC)Anu Agrawal, MD
Solid Tumors
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, MD
ONCOLOGY
Key Adverse Events after Childhood CancerALTE03N1 NCT00082745
N/A
COGCarla Golden, MD
Neuropsychological, Social, Emotional and Behavioral Outcomes in Children with CancerALTE07C1 NCT00772200
N/A
COGCarla Golden, MD
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 Progenitor Cell TransplantationACCL1633 NCT03057054
Phase 3
COGCarla Golden, MD
Use of a Clinical Trial Screening Tool to Address Cancer Health Disparities in the NCI Community Oncology Research ProgramDCP-001 N/A
N/A
NCORPCarla Golden, MD
Leukemia
An Open-Label, Multicenter Study of RC-P in Patients with Acute Lymphoblastic Leukemia (ALL)/Lymphoblastic Lymphoma (LBL) Following Hypersensitivity to E. coli-derived AsparaginasesAALL1931 NCT04145531
Phase 2/3
COG/GJazz PharmaceuticalsCarla Golden, MD
International Phase 3 Trial in Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia Ph+ ALL Testing Imatinib in Combination with Two Different Cytotoxic Chemotherapy BackbonesAALL1631 NCT03007147
Phase 3
COGCarla Golden, MD
Phase 3 Trail of IND# 117467 (Blinatumomab) in Combination with Chemotherapy in Patients with Newly Diagnosed Stanrd Risk/Down Syndrome B-ALL and Localized B-LlyAALL1731 NCT03914625
Phase 3
COGCarla Golden, MD
Risk-stratified Therapy for Acute Myeloid Leukemia in Down SyndromeAAML1531 NCT02521493
Phase 3
COGCarla Golden, MD
Leukemia, Lymphoma, Solid Tumors
A Randomized Web-based Physical Activity Intervention among Children and Adolescents with CancerALTE1631 NCT03223753
Phase 3
COGCarla Golden, MD
Neuro-Onc, Leukemia, Lymphoma, Solid Tumors
Project:EveryChild A Registry, Eligibility Screening, Biology and Outcome StudyAPEC14B1 NCT02402244
N/A
COGCarla Golden, MD
Oncology, Leukemia
A phase 3 randomized trial for patients with de novo AML comparing standard therapy including gemtuzumab ozogamicin GO to CPX-351 with GO, and the addition of the FLT3 inhibitor gilteritinib for patients with FLT3 mutationsAAML1831 NCT04293562
Phase 3
COGCarla Golden, MD
Oncology, Neuro-Onc, Leukemia, Lymphoma, Solid Tumors
Establishing Continuous Cell Lines and xenografts from Pediatric Cancers for Biological and Pre-Clinical Therapeutic StudiesABTR04B1 NCT00898755
COGCarla Golden, MD
Oncology, Oncology: Early Phase, Solid Tumors
A Safety, Pharmacokinetic and Efficacy Study of a ?-Secretase Inhibitor, Nirogacestat (PF 03084014; IND# 146375), in Children and Adolescents with Progressive, Surgically Unresectable Desmoid TumorsARST1921 NCT04195399
Phase 2
COGCarla Golden, MD
Oncology, Solid Tumors
Utilizing Response- and Biology-Based Risk Factors to Guide Therapy in Patients with Non-High-Risk NeuroblastomaANBL1232 NCT02176967
Phase 3
COGCarla Golden, MD
Oncology: Early Phase, Lymphoma, Solid Tumors
NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) Screening ProtocolAPEC1621SC NCT03155620
Phase 2
COGCarla Golden, MD
Oncology: Early Phase, Solid Tumors
Master NCI COG Pediatric MATCH (Molecular Analysis for Therapy Choice)APEC1621 N/A
Phase 2
COGCarla Golden, MD
Solid Tumors
Effects of Modern Chemotherapy Regimens on Spermatogenesis and Steroidogenesis in Adolescent and Young Adult Osteosarcoma SurvivorsALTE16C1 NCT03206450
N/A
COGCarla Golden, MD
Neuroblastoma Classification Biology StudiesANBL00B1 NCT00904241
COGCarla Golden, MD
Pediatric Hepatic Malignancy International Therapeutic Trial (PHITT)AHEP1531 NCT03533582
Phase 3
COGCarla Golden, MD
Phase 3 Study of Active Surveillance for Low Risk and a Randomized Trial of Carboplatin vs. Cisplatin for Standard Risk Pediatric and Adult Patients with Germ Cell TumorsAGCT1531 NCT03067181
Phase 3
COGCarla Golden, MD
Randomized Phase 3 Study of Vincristine, Dactinomycin, Cyclophosphamide (VAC) Alt. w. Vincristine/Irinotecan (VI) Vs VAC/VI Plus Temsirolimus in Patients with Intermediate Risk RhabdomyosarcomaARST1431 NCT02567435
Phase 3
COGCarla Golden, MD
Renal Tumors Classification, Biology, and Banking StudyAREN03B2 NCT00945009
Phase 3
COGCarla Golden, MD
NEURO-ONCOLOGY
Genomic Analysis in Pediatric MalignanciesGenomics N/A
N/ATabitha Cooney, 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, MD
Oncology, Neuro-Onc
Phase 2 Study of Reduced Therapy for Newly Diagnosed Average-Risk WNT-Driven Medulloblastoma PatientsACNS1422 NCT02724579
Phase 2
COGCarla Golden, MD
Oncology, Neuro-Onc, Solid Tumors
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'sCaroline Hastings, MD

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