Symptoms and General Pathology

All Conditions

Neoplasms

Blood and Lymph Conditions

Rare Diseases

Syndrome

Preleukemia

Myelodysplastic Syndromes

Bone Marrow Diseases

Hematologic Diseases

Precancerous Conditions

All Drugs and Chemicals

Immunomodulating Agents

Rafael Bejar, MD

Tiffany Tanaka, MD

Chao Family Comprehensive Cancer Center

Jessica Limson

Deepa Jeyakumar, M.D.

Colin McCarthy

Kimberly Aguilar

Rafael Bejar, M.D., Ph.D.

Tiffany Tanaka, M.D.

The purpose of this study is to collect information and bone marrow, blood, saliva, cheek cells and skin to be used in the laboratory to assist the sponsor in identifying a new way of treating MDS.

Goals of the study:

The purpose of this study is to collect the blood and marrow samples, and non-involved fibroblasts, that are required to identify the unique, personalized array of mutation-driven neoantigens that are expressed by the subject's MDS cells and to assess the feasibility of immunizing and expanding one or more of the patient's T cells ex vivo for investigation of their use as adoptive cellular immunotherapy.

We intend to obtain cells from bone marrow, peripheral blood, epithelial tissue, and saliva from patients who are likely to be candidates for MDS treatment in the near future.

Collection of Samples From Patients With MDS

Collection of Bone Marrow, Peripheral Blood (PB), Epithelial Tissue, and Saliva Samples From Patients With Myelodysplastic Syndromes (MDS) to Identify MDS-Specific Antigens (MSA) for Use in Cellular Immunotherapy.

To sequence the exome and transcriptome obtained from MDS hematopoietic cells and the exome from non-hematopoietic cells (e.g. fibroblasts).

To select variants by comparing MDS versus non-MDS cell exome sequences. MDS-specific variant sequences are defined as those that differ between the two and are not common polymorphisms. We will also compare myeloid and lymphoid hematopoietic cells and assess the number of myeloid-specific vs myeloid and lymphoid MDS-related variants

To select putative mutation-driven neoantigen-related peptides, which represent the sequences obtained from Aim 2, according to their ability to bind to the patient's MHC using PersImmune's licensed and proprietary algorithms.

To test the neoantigen peptides for their in vitro immunogenicity for autologous T lymphocytes.

To test the potency and specificity of neoantigen peptide-stimulated T cells for the patient's MDS cells that express the defined neoantigens.

To create a database summarizing the data obtained.

University of California, San Diego

PersImmune, Inc

Antineoplastic Agents

Azacitidine

Antimetabolites

Enzyme Inhibitors

1. Lemzoparlimab is administered by intravenous (IV) infusion once a week at a dose of 30 mg/kg in 28-day treatment cycles
2. Azacitidine (AZA): AZA is administered by subcutaneous injection once a day at a dose of 75 mg/m2 on D1 to D7 of each treatment cycle in 28-day treatment cycles.

Lemzoparlimab+Azacitidine (AZA)

AZA is administered by subcutaneous injection once a day at a dose of 75 mg/m2 on D1 to D7 of each treatment cycle in 28-day treatment cycles.

Azacitidine (AZA)

Shuang Li

Yanli Yang, Doctor

Ruixiang Xia, Doctor

Baolin Tang, Doctor

Jian Li, Doctor

Tong Wu, Doctor

Hongmei Jing, Doctor

Shuangnian Xu, Doctor

Xi Zhang, Doctor

Li Wang, Doctor

Quanyi Lu, Doctor

Danian Nie, Doctor

Yanjie He, Doctor

Qifa Liu, Doctor

Jianyu Weng, Doctor

Xin Du, Doctor

Ling Qin, Doctor

Hu Zhou, Doctor

Zhongxing Jiang, Doctor

Zunming Zhu, Doctor

Tiejun Gong, Doctor

Fankai Meng, Doctor

zhichao Chen, Doctor

Zhichao Chen, Doctor

zengmei Sheng, Doctor

Sujun Gao, Doctor

Feng Zhu, Doctor

Fei Li, Doctor

Xiaojing Yan, Doctor

Yi Wang, Doctor

Aili He, Doctor

Ming Hou, Doctor

Wei Wang, Doctor

Zhirong Zhang, Doctor

Qingliang Teng, Doctor

Liangming Ma, Doctor

Sujiang Zhang, Doctor

Xiuqin Wang, Doctor

Hua Zhong, Doctor

Chunkang Chang, Doctor

Yu Wu, Doctor

Xuelei Ma, Doctor

Jin Wei, Doctor

Rong Fu, Doctor

Zhijian Xiao, Doctor

Hongyan Tong, Doctor

Songfu Jiang, Doctor

This phase3 study is a randomized, controlled, open-label, multicenter study to evaluate the efficacy and safety of Lemzoparlimab for injection in combination with AZA versus AZA monotherapy as first-line therapy in treatment-naïve subjects with intermediate- and high-risk MDS.

A Phase 3 Clinical Study to Evaluate Lemzoparlimab for Injection in Combination With Azacitidine (AZA) Versus AZA Monotherapy in Treatment-naïve Patients With Higher-risk Myelodysplastic Syndrome (MDS)

A Randomized, Controlled, Open-label, Multicenter Phase 3 Clinical Study to Evaluate Lemzoparlimab for Injection in Combination With Azacitidine (AZA) Versus AZA Monotherapy in Treatment-naïve Patients With Higher-risk Myelodysplastic Syndrome (MDS)

TJ Biopharma Co., Ltd.

Leukemia

Leukemia, Myeloid

Leukemia, Myeloid, Acute

Neoplasms by Histologic Type

Myeloid Leukemia

Acute Myeloid Leukemia

Acute Non Lymphoblastic Leukemia

Acute Graft Versus Host Disease

This is an open-label Phase 1/2a clinical study. The study will be conducted in two parts: Part I: Phase 1 dose escalation, TJ011133 is tentatively scheduled to be administered once weekly in 28-day treatment cycles;Part II: Phase 2a study TJ011133 will be administered at a dose of 30 mg/kg once weekly, and AZA will be administered at a dose of 75 mg/m2 by subcutaneous injection for 7 consecutive days from D1 to D7 in 28-day treatment cycles.

TJ011133

This study is a phase I/II study of TJ011133 as Monotherapy and in Combination with Azacitidine (AZA) in Patients with Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS). This study include Phase I and Phase IIa study. Phase I study ClinicalTrials.gov ID is NCT04202003 and this is for phase IIa study. Phase IIa study is designed to preliminarily assess the efficacy and safety of TJ011133 in combination with AZA as first-line treatment in patients with newly diagnosed AML who are intolerant to standard induction chemotherapy or patients with treatment naive, intermediate and high-risk MDS.

A Study to Evaluate the Safety and Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of TJ011133 as Monotherapy and in Combination With Azacitidine (AZA) in Patients With Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS)

A Multicenter, Open-label, Phase 1/2a Study to Evaluate the Safety and Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of TJ011133 as Monotherapy and in Combination With Azacitidine (AZA) in Patients With Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS)

To evaluate the safety and tolerance of TJ011133 monotherapy in patients with r/r AML or MDS

To explore the maximum tolerable dose (MTD) of TJ011133 monotherapy for patients with r/r AML or MDS, the recommended phase II dose (RP2D) and the optimal dosage regimen.

Preliminary efficacy endpoints (only for Phase 2a): for response assessment in AML/MDS patients, the complete response rate (CR rate) will be evaluated according to the ELN2017/IWG 2006 criteria

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Collection of Samples From Patients With MDS	NCT03072498	Myelodysplastic...		18 Years -	PersImmune, Inc
A Phase 3 Clinical Study to Evaluate Lemzoparlimab for Injection in Combination With Azacitidine (AZA) Versus AZA Monotherapy in Treatment-naïve Patients With Higher-risk Myelodysplastic Syndrome (MDS)	NCT05709093	Myelodysplastic...	Lemzoparlimab+A... Azacitidine (AZ...	18 Years -	TJ Biopharma Co., Ltd.
A Study to Evaluate the Safety and Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of TJ011133 as Monotherapy and in Combination With Azacitidine (AZA) in Patients With Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS)	NCT04202003	Acute Myeloid L... Myelodysplastic...	TJ011133	18 Years - 70 Years	TJ Biopharma Co., Ltd.
A Study to Evaluate the Safety and Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of TJ011133 as Monotherapy and in Combination With Azacitidine (AZA) in Patients With Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS)	NCT04202003	Acute Myeloid L... Myelodysplastic...	TJ011133	18 Years - 70 Years	TJ Biopharma Co., Ltd.

Spots Global Cancer Trial Database for myelodysplastic syndromes(mds)