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

Contacts

Stefania Chirita [email protected] General Inquiries: Oncology Liza Reichert [email protected]

General Inquiries: Hematology Liza Reichert [email protected]

General Inquiries: SCTIntakeCoordinators@ Alex Palau Charles, RN Stem Cell Transplantation stanfordchildrens.org

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

Bone and Soft Tissue Sarcomas, Allison Pribnow, MD [email protected] Renal Tumors, Liver Tumors, Sheri Spunt, MD, MBA [email protected] Germ Cell Tumors

HLH Michael Jeng, MD [email protected]

Michael Link, MD [email protected] Hodgkin’s and Non-Hodgkin’s Lymphoma Lianna Marks, MD [email protected]

Kara Davis, DO [email protected] Crystal Mackall, MD [email protected] Liora Schultz, MD [email protected]

Neuroblastoma Robbie Majzner, MD [email protected]

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

Rajni Agarwal-Hashmi, MD [email protected] Alice Bertaina, MD [email protected] Stem Cell Transplantation Agnieszka Czechowicz, MD, PhD [email protected] Maria Grazia Roncarolo, MD [email protected] Ami Shah, MD [email protected]

Oncology: /Lymphoma

NCT #/Phase Title Sponsor Investigator Contact

Phase II/III TOT17: Total Therapy XVII for Newly Diagnosed Patients St. Jude Norman Regina Dagher, NP, (NCT03117751) With Acute Lymphoblastic Leukemia and Lymphoma Lacayo [email protected]

Phase II AML16: Phase II Trial of Epigenetic Priming in Patients St. Jude Norman Stefania Chirita, (NCT03164057) with Newly Diagnosed Acute Myeloid Leukemia Lacayo [email protected]

Phase I TINI: Total Therapy for Infants with Acute Lymphoblastic St. Jude Norman Stefania Chirita, (NCT02553460) Leukemia (ALL) Lacayo [email protected]

Phase II Pediatric Classical Hodgkin Lymphoma Consortium St. Jude Michael Stefania Chirita, (NCT03755804) Study: cHOD17 Link [email protected]

Phase I POE 14-01: Study of Carfilzomib in combination with POETIC Norman Sarah Cardone, RN, (NCT02512926) Cyclophosphamide and Etoposide for Children with Lacayo sarahcardone@ Relapsed or Refractory Solid Tumors and stanford.edu

Phase I/II POE 16-01: A Phase I/II study of Neratinib in Pediatric POETIC Norman Sarah Cardone, RN, (NCT02932280) Patients with Relapse/Refractory Solid Tumors or Lacayo sarahcardone@ Hematologic Malignancies stanford.edu

Phase II A Phase 2 Study of the JAK1/JAK2 Inhibitor Ruxolitinib Incyte/ Jay Shamanth Kuramkote, (NCT02723994) With Chemotherapy in Children With De Novo High-Risk COG Balagtas skuramkote@ CRLF2-Rearranged and/or JAK Pathway–Mutant Acute stanford.edu Lymphoblastic Leukemia

Phase II A Phase 2 Study of the MEK inhibitor Trametinib (IND COG Jay Shamanth Kuramkote, (NCT03190915) #119346, NSC# 763093) in Children with Relapsed or Balagtas skuramkote@ Refractory Juvenile Myelomonocytic Leukemia stanford.edu

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NCT #/Phase Title Sponsor Investigator Contact

Phase II ANHL12P1: A Randomized Phase II Trial of Brentuximab COG Jay Nancy Sweeters, RN, (NCT01979536) Vedotin or Crizotinib in Combination with Chemotherapy Balagtas nancy.sweeters@ for Newly Diagnosed Patients with Anaplastic Large Cell stanford.edu Lymphoma (ALCL)

Phase I A Phase 1 Study of Palbociclib (IND#141416), a CDK 4/6 COG Jay Stefania Chirita, (NCT03792256) Inhibitor, in Combination with Chemotherapy in Children Balagtas [email protected] with Relapsed Acute Lymphoblastic Leukemia (ALL) or Lymphoblastic Lymphoma (LL)

Phase I/II IDASA: A Phase I/II, Multicenter, Open- Genentech Norman Sarah Cardone, RN, Label, Multi-Arm Study Evaluating the Safety, Lacayo sarahcardone@ Tolerability, Pharmacokinetics, and Preliminary stanford.edu Activity of Idasanutlin In Combination with Either Chemotherapy or Venetoclax in the Treatment of Pediatric and Young Adult Patients with Relapsed/ Refractory Acute Leukemias or Solid Tumors

Oncology: Immunotherapy

NCT #/Phase Title Sponsor Investigator Contact

Phase I Phase I Clinical Trial of Autologous GD2 Chimeric Stanford Crystal Christina Baggott, PhD, (NCT04196413) Antigen Receptor (CAR) T cells (GD2CART) for Mackall [email protected] Diffuse Intrinsic Pontine Gliomas (DIPG) and and Spinal Diffuse Midline Glioma (DMG) Michelle Monje

Phase IB Phase IB Clinical Trial of Autologous CD22 Chimeric Stanford Liora Christina Baggott, PhD, (NCT03448393) Antigen Receptor (CAR) T Cells in Children and Young Schultz [email protected] Adults With Recurrent or Refractory B Cell Malignancies (GENE TRANSFER)

Phase I Phase 1 Dose Escalation Study of CD19/CD22 Chimeric Stanford Crystal Christina Baggott, PhD, (NCT03241940) Antigen Receptor (CAR) T Cells in Children and Young Mackall [email protected] Adults with Recurrent or Refractory B Cell Malignancies

Phase II AALL1721/CASSIOPEIA: A phase II trial of Novartis/ Kara Davis Christina Baggott, PhD, (NCT03876769) tisagenlecleucel in first-line high-risk (HR) pediatric and COG [email protected] young adult patients with B-cell acute lymphoblastic leukemia (B-ALL) who are minimal residual disease (MRD) positive at the end of consolidation (EOC) therapy

Phase I CCT5027: A Phase 1 Study Evaluating the Safety, Janssen Lori Christina Baggott, PhD, (NCT03904069, Tolerability, and Efficacy of FLT3 Chimeric Antigen Muffly, [email protected] coming soon) Receptor T-cell (CAR-T) AMG 553 in Subjects With Liora Relapsed/Refractory Acute Myeloid Leukemia Schultz

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NCT #/Phase Title Sponsor Investigator Contact

Phase II An Open-label, Multicenter, Phase 2 Study Evaluating Janssen Liora Christina Baggott, PhD, (NCT03384654) the Efficacy and Safety of Daratumumab in Pediatric Schultz [email protected] and Young Adult Subjects ≥1 and ≤30 Years of Age With Relapsed/Refractory Precursor B-cell or T-cell Acute Lymphoblastic Leukemia or Lymphoblastic Lymphoma

Oncology: Neuro-Oncology

NCT #/Phase Title Sponsor Investigator Contact

Phase II/III SJATRT: Phase 2 Study Of Alisertib as a Single Agent St. Jude Sonia Stefania Chirita, (NCT02114229) in Recurrent or Progressive Central Nervous System Partap [email protected] (CNS) Aypicial Teratoid Rhabdoid Tumors (AT/RTs) and Extra-CNS Malignant Rhabdoid Tumors (MRTs) and in Combination Therapy in Newly Diagnosed AT/RT

Phase II/III SJMB12: A Clinical and Molecular Risk-Directed Therapy St. Jude Sonia Stefania Chirita, (NCT01878617) for Newly Diagnosed Medulloblastoma Partap [email protected]

Phase II A Phase 2 Study of Reduced Therapy for Newly Diagnosed COG Jay Tiffany Tang, (NCT02724579) Average-Risk WNT-Driven Medulloblastoma (ACNS1422) Balagtas tiffanytang@ stanford.edu

Phase II ADVL1622: Phase 2 Trial of XL184 (Cabozantinib) an Oral COG Jay Erica Velasco, (NCT02867592) Small-Molecule Inhibitor of Multiple Kinases, in Children Balagtas [email protected] and Young Adults with Refractory Sarcomas, Wilms Tumor and Other Rare Tumors

Phase II ACNS1721: A Phase 2 Study of Veliparib (ABT-888, IND # COG Jay Stefania Chirita, (NCT03581292) 139199) and Local Irradiation, Followed by Maintenance Balagtas [email protected] Veliparib and Temozolomide, in Patients with Newly Diagnosed High-Grade Glioma (HGG) without H3 K27M or BRAFV600E Mutations COG

Phase I/II Phase I/II trial of Dabrafenib, Trametinib, and PBTC Sonia Tiffany Tang, (NCT04201457) Hydroxychloroquine (HCQ) for BRAF V600E-mutant or Partap tiffanytang@ Trametinib and HCQ for BRAF fusion/duplication positive stanford.edu or NF1-associated recurrent or progressive gliomas in children and young adults

Phase I PBTC-56: A phase I study of the ADAM-10 inhibitor PBTC Michelle Erica Velasco, (NCT04295759) INCB7839 in children with recurrent/progressive high- Monje [email protected] grade gliomas to target microenvironmental neuroligin-3

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NCT #/Phase Title Sponsor Investigator Contact

Phase I PBTC-051: Phase I Study to Evaluate the Safety and PBTC Michelle Stefania Chirita, (NCT03389802) Tolerability of the CD40 Agonistic Monoclonal Antibody Monje [email protected] APX005M in Pediatric Subjects With Recurrent/ Refractory Brain Tumors and Newly Diagnosed Brain Stem Glioma

Phase I PBTC-49: A Phase I study of Savolitinib in Recurrent, PBTC Michelle Erica Velasco, (NCT03598244) Progressive or Refractory Medulloblastoma, High-Grade Monje [email protected] Glioma, or Diffuse Intrinsic Pontine Glioma

Feasibility Study PBTC-48: Feasibility trial of Optune for children with PBTC Michelle Stefania Chirita, (NCT03033992) recurrent or progressive supratentorial high-grade glioma Monje [email protected] and ependymoma

Phase I/II PBTC-047: Phase 1 Trial of Panobinostat in Children with PBTC Michelle Erica Velasco, (NCT02717455) Diffuse Intrinsic Pontine Glioma Monje [email protected]

Phase I/II PBTC-045: A Safety and Preliminary Efficacy trial of PBTC Michelle Stefania Chirita, (NCT02359565) MK-3475 (pembrolizumab; anti-PD-1) in Children with Monje [email protected] recurrent, progressive or refractory high-grade gliomas (HGG), DIPGs and hypermutated brain tumors

Phase IIb A Phase 2b Trial of the MEK 1/2 Inhibitor (MEKi) Spring- Cynthia Erica Velasco, (NCT03962543) PD-0325901 in Adult and Pediatric Patients with Works Campen [email protected] Neurofibromatosis Type 1 (NF1)-Associated Inoperable Plexiform Neurofibromas (PNs) that are Progressing or Causing Significant Morbidity

Oncology: Solid Tumors

NCT #/Phase Title Sponsor Investigator Contact

Phase I/II POE 16-01: A Phase I/II study of Neratinib in Pediatric POETIC Norman Sarah Cardone, RN, (NCT02932280) Patients with Relapse/Refractory Solid Tumors or Lacayo sarahcardone@ Hematologic Malignancies stanford.edu

Phase I POE 14-01: Phase I Study of Carfilzomib in combination POETIC Norman Sarah Cardone, RN, (NCT02512926) with Cyclophosphamide and Etoposide for Children with Lacayo sarahcardone@ Relapsed or Refractory Solid Tumors and Leukemias stanford.edu

Phase II A Phase 2 Randomized Study of Irinotecan/ COG Jay Richard Fu, (NCT037943490) Temozolomide/Dinutuximab with or without Eflornithine Balagtas [email protected] (DFMO) (IND# 141913) in Children with Relapsed, Refractory or Progressive Neuroblastoma (ANBL1821)

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NCT #/Phase Title Sponsor Investigator Contact

Phase II AREN1921: A Study of Combination Chemotherapy COG Jay Richard Fu, (NCT04322318) for Patients With Newly Diagnosed DAWT and Balagtas [email protected] Relapsed FHWT

Phase I/II IDASA: A Phase I/II, Multicenter, Open-Label, Genentech Norman Sarah Cardone, RN, (NCT04029688) Multi-Arm Study Evaluating the Safety, Lacayo sarahcardone@ Tolerability, Pharmacokinetics, and Preliminary stanford.edu Activity of Idasanutlin In Combination with Either Chemotherapy or Venetoclax in the Treatment of Pediatric and Young Adult Patients with Relapsed/ Refractory Acute Leukemias or Solid Tumors

Phase I Cellectar: A Phase 1, Open-Label, Dose Escalation Study Cellectar Norman Richard Fu, (NCT03478462) of CLR 131 in Children and Adolescents with Select Solid Lacayo [email protected] Tumors, Lymphoma, and Malignant Brain Tumors

Phase I/II A Phase 1/2 Study of the Oral RET Inhibitor LOXO-292 Loxo Sheri Richard Fu, (NCT03899792) in Pediatric Patients with Advanced RET-Altered Solid or Spunt [email protected] Primary Central Nervous System Tumors

Phase II ADVL1921: Phase 1 study to evaluate the safety COG/ Jay Richard Fu, (NCT03709680) and pharmacokinetics of palbociclib (IBRANCE®) Pfizer Balagtas [email protected] in combination with irinotecan and temozolomide in pediatric patients with recurrent or refractory solid tumors

Stem Cell Transplantation

NCT #/Phase Title Sponsor Investigator Contact

Phase II Allogeneic hematopoietic stem cell transplantation from Stanford Alice Nivedita Kunte, (NCT04249830) an HLA-partially matched related or unrelated donor Bertaina [email protected] after TCR αβ+T cells/CD19+ B cell depletion in children and young adults affected by malignant or non-malignant hematological disorders.

Expanded Access An Expanded Access Study Using the CliniMACS System Stanford Rajni Nivedita Kunte, (NCT02162511) to Offer Therapeutic Manipulated Grafts that are CD34 Agarwal- [email protected] Cell Enriched and T Cell Depleted for Allogeneic Stem Hashmi Cell Recipients (BMT 271)

Phase I A Study to Evaluate the Safety and Tolerability Jasper Rajni Elisabeth Merkel, RN, (NCT02963064) of Tandemly Purified Allogeneic CD34+ CD90+ Thera- Agarwal- [email protected] Hematopoietic Stem Cells (HSC) Administered Following peutics Hashmi Conditioning with AMG 191 to Achieve Engraftment and Immune Reconstitution in Patients with Severe Combined Immunodeficiency (SCID)

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NCT #/Phase Title Sponsor Investigator Contact

Phase I/II Use of T-allo10 cell infusions combined with mismatched Stanford Rajni Nivedita Kunte, (NCT03198234) related or unrelated donor hematopoietic stem cell Agarwal- [email protected] transplantation (HSCT) for hematological malignancies Hashmi

Pilot Study Unrelated Donor Transplant Versus Immune Therapy Pediatric Alice Alicia Harnett, (NCT02845596) in Pediatric Severe Aplastic Anemia (TransIT) Blood and Bertaina [email protected] Marrow Transplant Consortium

Phase 2 A randomized trial of low versus moderate Pediatric Alice Alicia Harnett, (NCT03619551) exposure busulfan for infants with severe combined Blood and Bertaina [email protected] immunodeficiency (SCID) receiving TCRab+/CD19+ Marrow depleted transplantation: A Phase II Study (CSIDE) Transplant Consortium

Phase II KIR Favorable Mismatched Haplo Transplant and KIR Pediatric Alice Nivedita Kunte, (NCT02646839) Polymorphism in ALL/AML/MDS Allo-HCT Children Blood and Bertaina [email protected] Marrow Transplant Consortium

Phase I A Study of T-allo10 Infusion After HLA-Partially Stanford Alice Nivedita Kunte, (NCT04640987) Matched Related or Unrelated TCR αβ+ T-cell/ CD19+ Bertaina [email protected] B-cell Depleted Allogeneic Hematopoietic Stem Cell Transplantation (αβ Depleted-HSCT) in Children and Young Adults Affected by Hematologic Malignancies

Phase I Gene Therapy for Pyruvate Kinase Deficiency (PKD): A Rocket Ami Shah Elisabeth Merkel, RN, (NCT04105166) Phase I Clinical Trial to Evaluate the Safety of the Infusion Pharma- [email protected] of Autologous CD34 Cells Transduced with a Lentiviral ceuticals Vector Carrying the Codon Optimized Red Cell Pyruvate Kinase (coRPK) Gene in Adult and Pediatric Subjects with PKD. (GENE TRANSFER)

Phase III A Phase 3 Study of Lenti­D Drug Product After bluebird Ami Shah Elisabeth Merkel, RN, (NCT01896102) Myeloablative Conditioning Using Busulfan and bio [email protected] Fludarabine in Subjects 17 Years of Age and Under With Cerebral Adrenoleukodystrophy (CALD) (GENE TRANSFER)

Phase I/II A Phase 1/2 Study to Evaluate the Safety and Efficacy Vertex Ami Shah Elisabeth Merkel, RN, (NCT03745287) of a Single Dose of Autologous CRISPRCas9­ Modified [email protected] CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Severe Sickle Cell Disease (GENE TRANSFER)

Phase I/II A Phase 1/2 Study of the Safety and Efficacy of a Single Vertex Ami Shah Elisabeth Merkel, RN, (NCT03655678) Dose of Autologous CRISPRCas9­ Modified CD34+ [email protected] Human Hematopoietic Stem and Progenitor Cells (hHSPCs) in subjects with Transfusion-Dependent­ Beta ­ Thalassemia (GENE TRANSFER)

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Hematology

NCT #/Phase Title Sponsor Investigator Contact

Phase 3 Gene Therapy: An Open-Label, Non-Investigational Pfizer May Chien Liza Reichert, (NCT03587116 Product, Multi-Center, Lead-In Study to Evaluate at [email protected] —coming soon) Least 6 Months of Prospective Efficacy and Selected Safety Data of Current Factor IX (FIX) or Factor VIII (FVIII) Prophylaxis Replacement Therapy in the Usual Care Setting of Moderately Severe to Severe Adult Hemophilia B subjects (FIX:C≤2%) Who Are Negative for Neutralizing Antibodies to Adeno-Associated Virus Vector-Spark100 (Benegene-1) and Moderately Severe to Severe Hemophilia A Adult Subjects (FVIII:C≤1%) Who Are Negative for Neutralizing Antibodies to Adeno- Associated Virus Vector SB-525 capsid (AAV6), Prior to the Respective Therapeutic Phase 3 Gene Therapy Studies

Phase 3 Gene Therapy: Open-label, Single-Arm Study to Pfizer Caroline Liza Reichert, (NCT04370054 Evaluate the Efficacy and Safety of PF 07055480 Berube [email protected] —coming soon) (Recombinant AAV2/6 Human Factor VIII Gene Therapy) in Adult Male Participants with Moderately Severe to Severe Hemophilia A (FVIII:C≤1%)

725 Welch Rd, Palo Alto, CA 94304 | (650) 497-8953 | basscenter.stanfordchildrens.org 041672| 07/21