IL-12 Genetically Engineered Myeloid Cells in Participants With Relapsed, Refractory Solid Tumors
Phase I Trial of IL-12 Genetically Engineered Myeloid Cells in Participants With Relapsed, Refractory Solid Tumors
Background:
Myeloid cells are a type of immune cell found in most tumors. Interleukin 12 (IL-12) is a protein that helps the immune system kill tumor cells. Researchers want to know if myeloid cells that have been genetically engineered to produce IL-12 (IL-12 GEMys) can activate the immune system to attack cancer cells in solid tumors.
Objective:
To test IL-12 GEMys in people with cancer.
Eligibility
People aged 18 years and older with cancer that returned or failed to respond to treatment.
Design:
Participants will be screened. They will have a physical exam with blood tests. They will have tests of their heart and lung function. They will have imaging scans of their tumors. A sample of tumor tissue may be taken.
Participants will have daily injections for few days to prepare them to undergo leukapheresis: Blood will be taken from the body through a needle inserted into a vein. The blood will pass through a machine that separates out stem cells. The remaining blood will be returned to the body through a different needle. The collected stem cells will be modified in a lab to create IL-12 GEMys.
Participants will check in to the hospital. They will receive drugs for 5 days to prepare their body for the treatment. Then they will have their own IL-12 GEMys infused through a needle inserted into a vein. They will stay in the hospital until they are well enough to go home. This may be 7 to 14 days or longer.
Some participants may receive a second treatment with IL-12 GEMys within 2 years after the first.
Participants will have follow-up visits for about 5 years. These will include imaging scans and blood tests.
...
Visão geral do estudo
Status
Status
Condições
Condições
Intervenção / Tratamento
Intervenção / Tratamento
Descrição detalhada
Background:
- Myeloid cells are found in virtually all tumors, and they can play diverse roles in the tumor microenvironment including phagocytosing dying tumor cells, presenting antigen, suppressing anti-tumor immunity, and limiting natural killer (NK) and dendritic cell (DC) differentiation and function.
- Hematopoietic stem and progenitor cells (HSPCs) and myeloid cells can be genetically modified to express cytokines, such as the anti-tumor cytokine interleukin (IL)-12, which can be delivered locally by recruited myeloid cells in tumor and metastatic tissues.
- IL-12 is an important immunostimulatory cytokine. It can promote multiple effectors in adaptive immunity including enhancing major histocompatibility complex (MHC) class I expression, activation of NK cells, and DC maturation, and is essential for the T-helper 1 (Th1) response in T cells.
- A human lentiviral vector encoding single chain IL-12 gene and a truncated human epidermal growth factor receptor (EGFRt) have been developed. This lentivirus encodes a single chain bioactive IL-12 obtained by fusing the p35 and p40 subunits with a flexible (Gly4Ser)3 linker and can be used to transduce apheresed CD34+ HSPCs.
- Genetically engineered myeloid (GEMy) cells that secrete IL-12 (IL-12 GEMys) show efficacy in metastatic murine models including sarcomas, pancreatic carcinoma, and breast carcinoma. When IL-12 GEMys are combined with cyclophosphamide and fludarabine conditioning, long-term cure of large primary and metastatic solid tumors in mice occurs. These results suggest that IL-12 GEMys may have anti-tumor metastatic activity in humans.
Objectives:
- Part A (escalation): To determine the recommended phase 2 dose (RP2D) of IL-12 GEMys in participants with relapsed or refractory solid tumors following a cyclophosphamide and fludarabine conditioning regimen or without conditioning regimen.
- Part B (expansion): To assess whether IL-12 or IFN(gamma) levels, or both, increase in tumors post-treatment at the RP2D compared to levels seen in pre-treatment tumors.
Eligibility:
- Participants >= 18 years with relapsed or refractory solid tumor malignancies except primary tumors of the central nervous system (CNS) for whom standard measures do not exist or are no longer effective and have measurable or evaluable disease.
- There are no limits on prior therapy.
- Participants must be willing to undergo mandatory pre- and post-treatment tumor biopsies (Part B only).
Design:
- This is a Phase I, dose escalation/de-escalation trial design.
- Participants will undergo leukapheresis to obtain CD34+ cells that will then be genetically modified to express an IL-12 lentiviral vector with EGFRt. Differentiation to myeloid cells will occur after transduction.
- Depending on Dose Level, participants may or may not receive a lymphocyte-depleting therapy consisting of cyclophosphamide and fludarabine with the intent of enhancing the activity of the infused IL-12 GEMys.
- There will be an expansion cohort at the RP2D.
- On Day 0, participants will receive an infusion of IL-12 GEMys.
- Participants will be evaluated sequentially for toxicity, antitumor effects, and other biological correlatives.
- If an adequate number of additional cells are available for an additional dose, the participant may be offered the option of retreatment.
Tipo de estudo
Tipo de estudo
Inscrição (Estimado)
Inscrição
Estágio
Estágio
- Fase 1
Contactos e Locais
Contato de estudo
Contato de estudo
- Nome: Rosandra N Kaplan, M.D.
- Número de telefone: (240) 760-6198
- E-mail: kaplanrn@mail.nih.gov
Estude backup de contato
- Nome: NCI POB Solid Tumor Referral Team
- Número de telefone: (240) 858-7013
- E-mail: ncipobstreferrals@mail.nih.gov
Locais de estudo
-
-
Maryland
-
Bethesda, Maryland, Estados Unidos, 20892
- National Institutes of Health Clinical Center
-
Contato:
- NCI POB Solid Tumor Referral Team
- Número de telefone: 240-858-7013
- E-mail: ncipobstreferrals@mail.nih.gov
-
-
Critérios de participação
Critérios de elegibilidade
Critérios de elegibilidade
Idades elegíveis para estudo
- Adulto
- Adulto mais velho
Aceita Voluntários Saudáveis
Descrição
- INCLUSION CRITERIA:
- Relapsed or refractory solid tumor malignancies for whom standard measures do not exist or are no longer effective. Must have histologic confirmation of original diagnosis or relapse.
- Participants must have evaluable (measurable or not measurable) disease.
Part B only: Participants must:
- be willing to undergo mandatory pre- and post-treatment tumor biopsies. Tumor tissue should either be taken from non-target lesions or from target lesions where sampling can be done without impacting lesion measurement
and
--have disease amenable to biopsy to allow to perform pre- and post-tumor biopsies.
- Participants with treated brain metastases are eligible if follow-up brain imaging after central nervous system (CNS)-directed therapy and close monitoring for over 3 months shows no active progression.
- At least one prior cancer treatment when upfront standard therapy exists. Note: There is no limit to the number or type of prior treatment regimens.
- The indicated time must have elapsed since any systemic anti-cancer therapy prior to leukapheresis.
- Age >= 18 years.
- Eastern Cooperative Oncology Group (ECOG) performance status <= 2.
Note: Participants who are unable to walk because of paralysis, but who are able to maintain supine position independently in a wheelchair, will be considered ambulatory for the purpose of performance status.
Participants must have adequate organ and marrow function as defined below:
- Peripheral absolute neutrophil count (ANC) >= 1000/mm^3
- Platelet count >= 100,000/mm^3
- Hemoglobin (Hgb) >= 8 g/dL (transfusion independent)
- Prothrombin time (PT) <= 1.5 X institutional upper limit of normal (ULN) (Part B only, participants undergoing biopsy)
- Creatinine clearance >= 60 mL/minute/1.73m^2 (calculated by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula)
- Aspartate Aminotransferase (AST) <= 3 X institutional ULN
- Alanine Aminotransferase (ALT) <= 3 X institutional ULN
- Total bilirubin <= 1.5 institutional ULN. Note: In the case of Gilbert's syndrome total bilirubin <= 3 X ULN
- Serum albumin >= 2.7 g/dL
- Ejection fraction of >= 45% and no evidence of hemodynamically significant pericardial effusion as determined by an echocardiogram (ECHO).
- QTc interval < 480 msec
- Oxygen saturation >92% on room air at rest
- Participants with a clinical history of prolonged smoking (>= 10 pack-years), lung disease, or current or recent history of respiratory symptoms must have a forced expiratory volume in the first second (FEV1) > 50%.
- Participants seropositive for human immunodeficiency virus (HIV) must have an undetectable HIV viral load.
- Participants seropositive for Hepatitis C virus (HCV) must have an undetectable HCV viral load
- Participants positive for Hepatitis B surface antigen (HbsAg) must have an undetectable Hepatitis B virus (HBV) viral load.
- Women of childbearing potential (WOCBP) must agree to use highly effective contraception (hormonal, intrauterine device [IUD], abstinence, surgical sterilization) at the study entry and up to 12 months after the last dose of combined chemotherapy.
Note: WOCBP is defined as any woman who has experienced menarche and who has not undergone successful surgical sterilization or who is not postmenopausal.
Men able to father a child must agree to use a highly effective method of contraception (surgical sterilization, abstinence or man may request that partner uses the highly effective form of contraception to fulfill this requirement) at the study entry and up to 7 months after the last dose of study drugs. Men able to father a child must not freeze or donate sperm within the same period.
- Nursing participants must be willing to discontinue nursing from study treatment initiation through 12 months after the last dose of the study drug(s).
- Ability and willingness of participant to enroll on protocol 15-C-0028, Follow-Up Evaluation for Gene-Therapy Related Delayed Adverse Events after Participation in Pediatric Oncology Branch Clinical Trials after 5 years.
- Ability of the participant to understand and the willingness to sign a written informed consent document.
EXCLUSION CRITERIA:
- Participants with history of primary CNS tumors or leptomeningeal disease.
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to cyclophosphamide, fludarabine, IL-12, or other agents used in the study.
- Concurrent untreated opportunistic infections as evidenced by history, blood test or imaging at screening.
- Active systemic infections requiring anti-infective treatment.
- Any form of primary immunodeficiency (such as Severe Combined Immunodeficiency Disease) or secondary/acquired immunodeficiency requiring steroids or other non-steroid immunosuppressive agents.
- History of clonal hematopoiesis of indeterminate potential (CHIP) or myelodysplasia/myelodysplastic syndrome (MDS) or monoclonal gammopathy of undetermined significance (MGUS).
- Participants with symptomatic pleural effusions requiring intervention or with recent history (within 3 months) of pleural effusions that required intervention.
- Participants with ischemic symptoms (may include chest pain/pressure, shortness of breath, nausea, vomiting, sweating, and/or pain in the neck, shoulder, jaw or arm) confirmed by stress test OR a history of coronary revascularization (unless the participant has a normal cardiac stress test after revascularization and within 12 months prior to leukapheresis).
- Any form of diagnosed autoimmune disease requiring immune suppression as well as participants with active autoimmune skin diseases such as psoriasis or any history of active systemic autoimmune disease (e.g., Crohn's, rheumatoid arthritis, systemic lupus) requiring systemic immunosuppression/systemic disease-modifying agents within the last 2 years. Exceptions will be allowed for vitiligo and hypothyroidism that has been stable on thyroid replacement medications for > 6 weeks prior to leukapheresis.
- Any participant who developed autoimmunity (>= grade 3 per CTCAE v. 6.0) with checkpoint inhibitor use. Note: Exceptions will be allowed for vitiligo and hypothyroidism that has been stable on thyroid replacement medications for > 6 weeks prior to leukapheresis.
- Participants who have a major surgical procedure, other than for diagnosis, within 4 weeks prior to leukapheresis or anticipated to need a major surgical procedure during the study.
- History of prior solid organ transplantation.
- Participants that require urgent therapy due to tumor mass effects or spinal cord compression within 2 weeks prior to leukapheresis.
- Any investigational therapy within 2 weeks prior to leukapheresis.
- Pregnancy confirmed with Beta-human chorionic gonadotropin (Beta-HCG) serum or urine pregnancy test in WOCBP at screening. Note: In case of a suspected false-positive serum or urine test result, additional evaluation must be done to rule out pregnancy.
- Uncontrolled intercurrent illness or medical condition(s) evaluated by medical history, physical exam, or situations that would unacceptably increase risk for the participant or impair the ability to evaluate the endpoints of the study.
Plano de estudo
Como o estudo é projetado?
Detalhes do projeto
- Finalidade Principal: Tratamento
- Alocação: Não randomizado
- Modelo Intervencional: Atribuição sequencial
- Mascaramento: Nenhum (rótulo aberto)
Número de braços
Armas e Intervenções
Grupo de Participantes / BraçoGrupo de Participantes / Braço |
Intervenção / TratamentoIntervenção / Tratamento |
|---|---|
|
Experimental: Arm 1
Escalating/de-escalating doses of IL-12 GEMys with or without conditioning (cyclophosphamide and fludarabine)
|
Cell therapy generated from autologous CD34+ cells.
Administered on Day 0 as an IV infusion not to exceed 20ml/kg or 40ml/kg depending on DMSO levels.
Lymphodepletive chemotherapy administered as 30 mg/kg IV infusion over 1 hour daily on days -6 and -5.
Lymphodeleptive chemotherapy administered as 25 mg/m^2 IV infusion over 30 minutes on days -6 through -2.
Administered as IV infusion at 500 mg/m^2, if needed.
|
|
Experimental: Arm 2
RP2D of IL-12 GEMys with or without conditioning (cyclophosphamide and fludarabine)
|
Cell therapy generated from autologous CD34+ cells.
Administered on Day 0 as an IV infusion not to exceed 20ml/kg or 40ml/kg depending on DMSO levels.
Lymphodepletive chemotherapy administered as 30 mg/kg IV infusion over 1 hour daily on days -6 and -5.
Lymphodeleptive chemotherapy administered as 25 mg/m^2 IV infusion over 30 minutes on days -6 through -2.
Administered as IV infusion at 500 mg/m^2, if needed.
|
O que o estudo está medindo?
Medidas de resultados primários
Medidas de resultados primários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
|
Part A (Escalation): Determine the recommended phase 2 dose (RP2D) of IL-12
Prazo: 0-28 Days
|
Maximum dosage of GEMys IL-12 with which no more than 1 participant experience dose limiting toxicity as assessed by grade of adverse event
|
0-28 Days
|
|
Part B (Expansion): Assess whether IL-12 or IFNy levels, or both increase in tumors post treatment at the RP2D
Prazo: 1 week
|
Increased IL-12 and/or IFNy levels and IL-12 production as measured by ELISA, using a paired t-test or Wilcoxon signed rank test
|
1 week
|
Medidas de resultados secundários
Medidas de resultados secundários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
|
Determine the feasibility of manufacturing IL-12 GEMys that express a truncated epidermal growth factor receptor (EGFRt) meeting release criteria
Prazo: Time of cell infusion
|
Number of participants at each dose level for whom the target number of cells at that dose level can be manufactured and assessed prior to infusion
|
Time of cell infusion
|
|
Determine the safety of IL-12 GEMys
Prazo: 0-12 months
|
Safety data will be analyzed per standard methods and interpreted descriptively for each dose level.
Ongoing analysis of toxicity using CTCAE and RCL collected from the blood
|
0-12 months
|
|
Assess the antitumor activity of IL-12 GEMys
Prazo: 0-5 years
|
Overall response rate (ORR) (PR + CR) per RECIST 1.1
|
0-5 years
|
|
Assess the re-treatment utility of IL-12 GEMys
Prazo: 0-5 years
|
Assessment of clinical response per RECIST
|
0-5 years
|
Colaboradores e Investigadores
Patrocinador
Patrocinador
Investigadores
Investigadores
- Investigador principal: Rosandra N Kaplan, M.D., National Cancer Institute (NCI)
Publicações e links úteis
Links úteis
Datas de registro do estudo
Datas Principais do Estudo
Início do estudo (Estimado)
Início do estudo
Conclusão Primária (Estimado)
Conclusão Primária
Conclusão do estudo (Estimado)
Conclusão do estudo
Datas de inscrição no estudo
Enviado pela primeira vez
Enviado pela primeira vez
Enviado pela primeira vez que atendeu aos critérios de CQ
Enviado pela primeira vez que atendeu aos critérios de CQ
Primeira postagem (Real)
Primeira postagem
Atualizações de registro de estudo
Última Atualização Postada (Real)
Última Atualização Postada
Última atualização enviada que atendeu aos critérios de controle de qualidade
Última atualização enviada que atendeu aos critérios de controle de qualidade
Última verificação
Última verificação
Mais Informações
Termos relacionados a este estudo
Palavras-chave
Termos MeSH relevantes adicionais
- Aminoácidos, peptídeos e proteínas
- Proteínas
- Produtos químicos orgânicos
- Hidrocarbonetos
- Anticorpos, monoclonais, humanizados
- Anticorpos, monoclonais
- Anticorpos
- Imunoglobulinas
- Imunoproteínas
- Proteínas sanguíneas
- Globulinas de soro
- Globulins
- Mustarazas de fosforamida
- Compostos de mostarda de nitrogênio
- Compostos de mostarda
- Hidrocarbonetos, halogenados
- Fosforamidas
- Compostos organofosforos
- Cetuximabe
- Ciclofosfamida
- fludarabina
Outros números de identificação do estudo
Outros números de identificação do estudo
- 10002091
- 002091-C
Plano para dados de participantes individuais (IPD)
Planeja compartilhar dados de participantes individuais (IPD)?
Descrição do plano IPD
Prazo de Compartilhamento de IPD
Critérios de acesso de compartilhamento IPD
Tipo de informação de suporte de compartilhamento de IPD
- PROTOCOLO DE ESTUDO
- SEIVA
- CIF
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