Phase I and Phase II Clinical Trials: Hematology, Oncology, and Stem Cell Transplantation

Contacts

General Inquiries Christina Baggott, RN, PhD [email protected]

AML, ALL Catherine Aftandilian, MD, MS [email protected] Norman Lacayo, MD [email protected] Gary Dahl, MD [email protected]

Bone and Soft Tissue Sarcomas, Sheri Spunt, MD, MBA [email protected] Renal Tumors

HLH Michael Jeng, MD [email protected]

Hodgkin’s Lymphoma Michael Link, MD [email protected]

Immunotherapy Crystal Mackall, MD [email protected] Kara Davis, DO [email protected]

Liver Tumors, Germ Cell Tumors Arun Rangaswami, MD [email protected]

Neuroblastoma Robbie Majzner, MD [email protected]

Neuro-Oncology Sonia Partap, MD [email protected] Paul Fisher, MD [email protected] Cynthia Campen, MD [email protected] , MD [email protected]

Non-Hodgkin’s Lymphoma Sandra Luna-Fineman, MD [email protected]

Stem Cell Transplantation Maria Grazia Roncarolo, MD [email protected] Rajni Agarwal-Hashmi, MD [email protected]

Oncology: /Lymphoma

NCT #/Phase Title Sponsor Investigator Contact

Phase I TINI: Total Therapy for Infants with Acute Lymphoblastic St. Jude Norman Christina (NCT02553460) Leukemia (ALL) Lacayo Baggott, PhD, baggott@ stanford.edu

Phase II HLHR13: Testing the Substitution of Brentuximab Vedotin for St. Jude Michael Christina (NCT01920932) Vincristine in the Usual Chemotherapy and Radiation in High-Risk Link Baggott, PhD, Pediatric Hodgkin Lymphoma baggott@ stanford.edu

725 Welch Rd, Palo Alto, CA 94304 | (650) 497-8953 | basscenter.stanfordchildrens.org 029584 | 05/2017 Phase I and Phase II Clinical Trials: Hematology, Oncology, and Stem Cell Transplantation 2

NCT #/Phase Title Sponsor Investigator Contact

Phase II AAML1522: A Phase II, Multicenter, Single-arm, Open-label Study to COG Sheri Spunt Christina (NCT02538965) Evaluate the Activity, Safety and Pharmacokinetics of Lenalidomine Baggott, PhD, in Pediatric Subjects from 1 to 18 Years of Age with Relapse/ baggott@ Refractory Acute Myeloid Leukemia stanford.edu

Phase I/II SELHEM: A Phase I/II Study of the Selective Inhibitor of Nuclear St. Jude Norman Christina (NCT02212561) Export Selinexor (KPT-330) in Combination with Fludarabine Lacayo Baggott, PhD, and Cytarabine in Pediatric Patients with Refractory or Relapsed baggott@ Leukemia or Myelodysplastic Syndrome stanford.edu

Phase I PANAML: A Phase I and Dose Expansion Cohort Study Of St. Jude Norman Christina (NCT02676323) Panobinostat in Combination with Fludarabine and Cytarabine Lacayo Baggott, PhD, In Pediatric Patients with Refractory Or Relapse Acute Myeloid baggott@ Leukemia or Myelodysplastic Syndrome stanford.edu

Phase I/II LYM3003: A Randomized, Open-label, Safety and Efficacy Study Janssen Michael Christina (NCT02703272) of Ibrutinib in Pediatric and Young Adult Patients With Relapsed or Link Baggott, PhD, Refractory Mature B-cell non-Hodgkin Lymphoma baggott@ stanford.edu

Phase II ANHL12P1: A Randomized Phase II Trial of Brentuximab Vedotin or COG Sheri Spunt Christina (NCT01979536) Crizotinib in Combination with Chemotherapy for Newly Diagnosed Baggott, PhD, Patients with Anaplastic Large Cell Lymphoma (ALCL) baggott@ stanford.edu

Phase I POE-14: Phase I Study of Carfilzomib in combination with POETIC Norman Christina (NCT02512926) Cyclophosphamide and Etoposide for Children with Relapsed or Lacayo Baggott, PhD, Refractory Solid Tumors and baggott@ stanford.edu

Phase II CCTL019B2205J: A Phase II, single arm, multicenter trial to Novartis Kara Davis Christina (NCT02228096) determine the efficacy and safety of CTL019 in pediatric patients Baggott, PhD, with relapsed and refractory B-cell acute lymphoblastic leukemia baggott@ stanford.edu

Part of 2205J CCTL019B2206: A multicenter study of apheresis collection of Novartis Kara Davis Christina peripheral blood mononuclear cells (PBMC) in patients with CD19 Baggott, PhD, expressing malignancies who could be eligible for a CTL019 clinical baggott@ research trial stanford.edu

Phase II (planned AHOD1721: Risk-based, response-adapted, Phase II open-label COG/BMS Sheri Spunt Christina to open 6/17) trial of nivolumab + brentuximab vedotin (N+ Bv) for children, Baggott, PhD, adolescents,and young adults with relapsed/refractory (R/R) baggott@ CD30 + classic Hodgkin lymphoma (cHL)after failure of first- stanford.edu linetherapy,followed by brentuximab + bendamustine (Bv + B) for participantswith a suboptimal response. CheckMate 744: CHECKpoint pathway and nivolumab clinical Trial Evaluation

725 Welch Rd, Palo Alto, CA 94304 | (650) 497-8953 | basscenter.stanfordchildrens.org 029584 | 05/2017 Phase I and Phase II Clinical Trials: Hematology, Oncology, and Stem Cell Transplantation 3

Oncology:

NCT #/Phase Title Sponsor Investigator Contact

Phase I Macrogenics: A Phase 1, Open-label, Dose Escalation Study of Macro- Crystal Christina (NCT02475213) MGA271 in Pediatric Patients with B7-H3-Expressing Relapsed or Genics Mackall Baggott, PhD, Refractory Solid Tumors baggott@ stanford.edu

Phase II Novartis 2205J: A Phase II, single arm, multicenter trial to determine Novartis Kara Davis Christina (NCT02228096) the efficacy and safety of CTL019 in pediatric patients with relapsed Baggott, PhD, and refractory B-cell acute lymphoblastic leukemia baggott@ stanford.edu

Oncology: Neuro-Oncology

NCT #/Phase Title Sponsor Investigator Contact

Phase II/III SJATRT: Phase 2 Study Of Alisertib as a Single Agent in Recurrent St. Jude Sonia Christina (NCT02114229) or Progressive Central Nervous System (CNS) Aypicial Teratoid Partap Baggott, PhD, Rhabdoid Tumors (AT/RTs) and Extra-CNS Malignant Rhabdoid baggott@ Tumors (MRTs) and in Combination Therapy in Newly Diagnosed stanford.edu AT/RT

Phase II/III SJMB12: A Clinical and Molecular Risk-Directed Therapy for Newly St. Jude Sonia Christina (NCT01878617) Diagnosed Medulloblastoma Partap Baggott, PhD, baggott@ stanford.edu

Phase I/II PBTC-029B: A Phase I and Phase II and Re-Treatment Study of PBTC Paul Fisher Christina (NCT01089101) AZD6244 for Recurrent or Refractory Pediatric Low Grade Glioma Baggott, PhD, baggott@ stanford.edu

Phase I/II PBTC-042: Phase I study of CDK 4-6 inhibitor PD-0332991 in PBTC Paul Fisher Christina (NCT02255461) children with recurrent, progressive or refractory central nervous Baggott, PhD, system tumors baggott@ stanford.edu

Phase I/II PBTC-045: A Safety and Preliminary Efficacy trial of MK-3475 PBTC Paul Fisher Christina (NCT02359565) (pembrolizumab; anti-PD-1) in Children with recurrent, progressive Baggott, PhD, or refractory high-grade gliomas (HGG), DIPGs and hypermutated baggott@ brain tumors stanford.edu

Phase I/II PBTC-047: Phase 1 Trial of Panobinostat in Children with Diffuse PBTC Paul Fisher Christina (NCT02717455) Intrinsic Pontine Glioma Baggott, PhD, baggott@ stanford.edu

Phase II INTELLANCE 2: ABT-414 alone or ABT-414 plus temozolomide versus AbbVie Sonia Christina (NCT02343406) lomustine or temozolomide for recurrent glioblastoma: a randomized Partap Baggott, PhD, phase II study of the EORTC Brain Tumor Group baggott@ stanford.edu

725 Welch Rd, Palo Alto, CA 94304 | (650) 497-8953 | basscenter.stanfordchildrens.org 029584 | 05/2017 Phase I and Phase II Clinical Trials: Hematology, Oncology, and Stem Cell Transplantation 4

Oncology: Solid Tumors

NCT #/Phase Title Sponsor Investigator Contact

Phase II NANT 2011-01: A Randomized Phase II Study of 131-1-MIBG vs 131-1- NANT Sheri Spunt Christina (NCT02035137) MIBG with Vincristine and Irinotecan vs 131-1-MIBG with Vorinostat Baggott, PhD, for Resistant/Relapsed Neuroblastoma baggott@ stanford.edu

Phase I NANT2011-04: A Phase I Study of Lenalidomide and Anti-GD2 Mab NANT Sheri Spunt Christina (NCT01711554) CH14.18 +/- Isotretinoin in Patients with Refractory/Recurrent Baggott, PhD, Neuroblastoma baggott@ stanford.edu

Phase I NANT2012-01: A Phase I Study of Difluoromethylornithine (DFMO) NANT Sheri Spunt Christina (NCT02030964) and Celecoxib Cyclophosphamide/Topotecan for Patients with Baggott, PhD, Relapsed or Refractory Neuroblastoma baggott@ stanford.edu

Phase I NANT 2013-01: Immunotherapy of Relapsed Neuroblastoma with NANT Sheri Spunt Christina (NCT02573896) expanded NK cells, Lenalidomide and ch14.18 Baggott, PhD, baggott@ stanford.edu

Phase I NANT 2013-02: A Phase I Study of Sorafenib and NANT Sheri Spunt Christina (NCT02298348) Cyclophosphamide/Topotecan in Patients with Relapsed and Baggott, PhD, Refractory Neuroblastoma baggott@ stanford.edu

Phase I NANT 2014-01: Phase I Study of SF1126 for Patients with Relapsed NANT Sheri Spunt Christina (NCT02337309) or Refactory Neuroblastoma Baggott, PhD, baggott@ stanford.edu

Phase I/II NANT 2015-02: Phase 1/2 Study of PF-06463922, an Oral Small NANT Sheri Spunt Christina Molecule Inhibitor of ALK/ROS1, for patients with Alk-Driven Baggott, PhD, Relapsed or Refractory Neurblastoma baggott@ stanford.edu

Phase I/II LOXO: A Phase 1/2 Study of the Oral TRK Inhibitor LOXO 101 in Loxo Sheri Spunt Christina (NCT02637687) Pediatric Patients with Advanced Solid or Primary Central Nervous Baggott, PhD, System Tumors baggott@ stanford.edu

Phase II ADVL1322: A Phase II Study of Pazopanib (GW786034, NSC# COG Sheri Spunt Christina (NCT02452554) 737754) in Children, Adolescents and Young Adults with Refractory Baggott, PhD, Solid Tumors baggott@ stanford.edu

Phase I/II ADVL1412: A Phase 1/2 Study of Nivolumab (IND# 124729) COG Sheri Spunt, Christina (NCT02304458) in Children, Adolescents, and Young Adults with Recurrent or Crystal Baggott, PhD, Refractory Solid Tumors as a Single Agent and in Combination Mackall, baggott@ with Ipilimumab Kara Davis stanford.edu

725 Welch Rd, Palo Alto, CA 94304 | (650) 497-8953 | basscenter.stanfordchildrens.org 029584 | 05/2017 Phase I and Phase II Clinical Trials: Hematology, Oncology, and Stem Cell Transplantation 5

NCT #/Phase Title Sponsor Investigator Contact

Phase II ADVL1522: A Phase 2 Study of IMGN901 (Lorvotuzumab Mertansine; COG Sheri Spunt Christina (NCT02452554) IND# 126953, NSC# 783609) in Children with Relapsed or Baggott, PhD, Refractory Wilms Tumor, Rhabdomyosarcoma, Neuroblastoma, baggott@ Pleuropulmonary Blastoma, Malignant Peripheral Nerve Sheath stanford.edu Tumor (MPNST) or Synovial Sarcoma

Phase I A Phase 1, Open-Label, Dose Escalation Study of MGA271 in Pediatric Macro- Crystal Christina (NCT02475213) Patients with B7-H3-Expressing Relapsed or Refractory Solid Tumors Genics Mackall Baggott, PhD, baggott@ stanford.edu

Phase II AEWS1221: Randomized Phase II Trial Evaluating the Addition of the COG Sheri Spunt Christina (NCT02306161) IGF-1R Monoclonal Antibody Ganitumab (AMG 479, NSC# 750008, Baggott, PhD, IND# 120449) to Multiagent Chemotherapy for Patients with Newly baggott@ Diagnosed Metastatic Ewing Sarcoma stanford.edu

Phase II AOST1321: Phase II Study of Denosumab (IND# 127430, NSC# COG Sheri Spunt Christina (NCT02470091) 744010), a RANK Ligand Antibody, for Recurrent or Refractory Baggott, PhD, Osteosarcoma baggott@ stanford.edu

Phase II AOST1421: A Phase II Study of Human-Mouse Chimeric Anti- COG Sheri Spunt Christina (NCT02484443) Disialoganglioside Monoclonal Antibody ch14.18 (Dinutuximab, Baggott, PhD, NSC#764038, IND# 4308) in Combination with GM-CSF in Patients baggott@ with Recurrent Osteosarcoma stanford.edu

Phase II AOST1521: A Phase 2 Study of GPNMB-targeted Antibody-Drug COG Sheri Spunt Christina (NCT02487979) Conjugate, CDX-011 (Glembatumumab Vedotin, CR011-vcMMAE; Baggott, PhD, IND# 128248, NSC# 763737), in Recurrent or Refractory baggott@ Osteosarcoma stanford.edu

Phase II/III ARST1321: Pazopanib Neoadjuvunt Trial on Non-Rhabdomyosarcoma COG Sheri Spunt Christina (NCT02180867) Soft Tissue Sarcomas (PAZNTIS): A Phase II/III Randomized Trial Baggott, PhD, of Preoperative Chemoradiation or Preoperative Radiation Plus or baggott@ Minus Pazopanib (NSC# 737754, IND# 118613) stanford.edu

Phase II ANBL1221: A Phase II Randomized Trial of Irinotecan/Temozolomide COG Sheri Spunt Christina (NCT01767194) with Temsirolimus (NSC# 683864, IND# 61010) or Chimeric 14.18 Baggott, PhD, Antibody (ch14.18) (NSC# 623408, IND# 4308) in Children with baggott@ Refractory, Relapsed or Progressive Neuroblastoma stanford.edu

Phase I POE-014: Phase I Study of Carfilzomib in combination with POETIC Norman Christina (NCT02512926) Cyclophosphamide and Etoposide for Children with Relapsed or Lacayo Baggott, PhD, Refractory Solid Tumors and Leukemias baggott@ stanford.edu

Phase I/II POETIC (GO29665): A Phase I/II, Multicenter, Open-Label, Dose- POETIC Norman Christina (NCT02639546) Escalation Study of the Safety and Pharmacokinetics of Cobimetinib Lacayo Baggott, PhD, (IND# 124,530) in Pediatric and Young Adult Patients with Previously baggott@ Treated Solid Tumors stanford.edu

Phase I POETIC (GO29664): An Early-Phase, Multicenter, Open-label POETIC Norman Christina (NCT02541604) Study of the Safety and Pharmacokinetics of Atezolizumab Lacayo Baggott, PhD, (MPDL3280A) In Pediatric and Young Adult Patients with baggott@ Previously Treated Solid Tumors. stanford.edu

725 Welch Rd, Palo Alto, CA 94304 | (650) 497-8953 | basscenter.stanfordchildrens.org 029584 | 05/2017 Phase I and Phase II Clinical Trials: Hematology, Oncology, and Stem Cell Transplantation 6

Stem Cell Transplantation

NCT #/Phase Title Sponsor Investigator Contact

Phase I A Study to Evaluate the Safety and Tolerability of Tandemly Purified Stanford Rajni Prianka (NCT02963064) Allogeneic CD34+ CD90+ Hematopoietic Stem Cells (HSC) Agarwal- Kumar, RN, Administered Following Conditioning with AMG 191 to Achieve Hashmi prianka. Engraftment and Immune Reconstitution in Patients with Severe kumar@ Combined Immunodeficiency (SCID) stanford.edu

Phase I/II Caspacide T Cells from an HLA Partially Matched Related Bellicum Rajni Prianka (NCT02065869) Donor After Negative Selection of TCR Aß+T and B Cells in Agarwal- Kumar, RN, Pediatric Patients Hashmi prianka. kumar@ stanford.edu

Phase IB An Open-Label, Randomized, Adaptive, Two-Arm, Multicenter Trial Roche Ami Shah Kirstin (NCT01715909) to Evaluate Pharmacokinetics and Pharmacodynamics of Two Doses Dougall, of Oseltamivir Phosphate (Tamiflu) in the Treatment of Influenza in kirstin. Immunocompromised Children Younger Than 13 Years of Age with dougall@ Confirmed Influenza Infection stanford.edu

Expanded Access An Intermediate-Size, Expanded Access Protocol to Provide Chimerix, Rajni Peter Chase, (NCT02596997) Brincidofovir for the Treatment of Serious Adenovirus Infection Inc. Agarwal- peter.chase@ or Disease Hashmi stanford.edu

Phase I/II Pilot Safety and Feasibility Trial of Sirolimus and Mycophenolate Stanford Rajni Leigh Shinn, (NCT02728700) for Prevention of GVHD in Mismatched Unrelated and Related Agarwal- RN, Donor Hematopoietic Stem Cell Transplantation for Hematologic Hashmi leigh.shinn@ Malignancies stanford.edu

Phase I/II Use of T-allo10 cell infusions combined with mismatched related Stanford Rajni Leigh Shinn, (Opening 5/17) or unrelated donor hematopoietic stem cell transplantation (HSCT) Agarwal- RN, for hematological malignancies Hashmi leigh.shinn@ stanford.edu

725 Welch Rd, Palo Alto, CA 94304 | (650) 497-8953 | basscenter.stanfordchildrens.org 029584 | 05/2017 Phase I and Phase II Clinical Trials: Hematology, Oncology, and Stem Cell Transplantation 7

Hematology

NCT #/Phase Title Sponsor Investigator Contact

Phase I A Phase I, Open-Label, Single Dose, Non-Randomized Study to Daiichi Michael Elaine (NCT02303431) Evaluate Pharmakokinetics and Pharmacodynamics of Edoxaban Sankyo Jeng Macasiray, RN, in Pediatric Patients emacasiray@ stanford.edu

Phase II AG348-C-003: A Phase 2, Open Label Randomized, Dose Ranging, Agios Bert Glader Elaine (NCT02476916) Safety, Efficacy, Pharmacokinetic and Pharmacodynamic Study of Macasiray, RN, AG-348 in Adult Patients with Pyruvate Kinase Deficiency emacasiray@ stanford.edu

Phase II SIR-DA-1202: A Randomized Phase 2 Study of Vincristine Pfizer and Michael Elaine (NCT02110069) versus Sirolimus to treat High Risk Kaposiform Boston Jeng Macasiray, RN, Hemangioendothelioma (KHE) Children's emacasiray@ Hospital stanford.edu

Phase II A Phase II, Open-Label, Non-Controlled, Intra-Patient Dose- Novartis Bert Glader Elaine (NCT030256698) Escalation Study to Characterize the Pharmacokinetics after and Boston Macasiray, RN, Oral Administration of Eltrombopag in Pediatric Patients With Children's emacasiray@ Refractory, Relapsed, or Treatment Naaive Severe Aplastic Anemia or Hospital stanford.edu Recurrent Aplastic Anemia

Phase II/III A Phase II/III Open-label, Single Arm Multicenter Study to Assess NovImmune Michael Elaine (NCT02069899) Safety, Tolerability, Pharmacokinetics and Efficacy of Intravenous Jeng Macasiray, RN, Multiple Administrations of NI-0501, an Anti-interferon Gamma emacasiray@ (Anti-IFN) Monoclonal Antibody in Pediatric Patients with Primary stanford.edu Hemophagocytic Lymphohistiocytosis(HLH)

725 Welch Rd, Palo Alto, CA 94304 | (650) 497-8953 | basscenter.stanfordchildrens.org 029584 | 05/2017