Neoplasms

All Conditions

Adenocarcinoma

Carcinoma

Neoplasms, Glandular and Epithelial

Neoplasms by Histologic Type

Antineoplastic Agents

All Drugs and Chemicals

Pembrolizumab

Antineoplastic Agents, Immunological

Immune Checkpoint Inhibitors

BXCL701 0.3 mg, orally, twice a day on days 1-14 every 21 days

BXCL701

Pembrolizumab 200 mg intravenous (IV) on day 1 every 21 days.

Princess Jones

Benjamin Weinberg, MD

Single-arm, open label study to determine the 18 week progression-free survival rate of the combination of BXCL701 and pembrolizumab in patients with pancreatic ductal adenocarcinoma in the second-line metastatic setting.

BXCL701 and Pembrolizumab in Patients With Metastatic Pancreatic Ductal Adenocarcinoma

Phase II Trial of BXCL701 and Pembrolizumab in Patients With Metastatic Pancreatic Ductal Adenocarcinoma

BioXcel Therapeutics Inc

Merck Sharp & Dohme LLC

Georgetown University

Symptoms and General Pathology

Blood and Lymph Conditions

Rare Diseases

Syndrome

Leukemia

Leukemia, Myeloid

Leukemia, Myeloid, Acute

Preleukemia

Myelodysplastic Syndromes

Anemia, Refractory, with Excess of Blasts

Anemia

Anemia, Refractory

Hematologic Diseases

Bone Marrow Diseases

Precancerous Conditions

Myeloid Leukemia

Acute Myeloid Leukemia

Acute Non Lymphoblastic Leukemia

Acute Graft Versus Host Disease

Myelodysplastic Syndrome With Excess Blasts

Dose escalation will occur using a 3+3 dose escalation approach, evaluating 4 different dose levels of BXCL701.

During each 28 day study cycle participants will take BXCL701 2x daily for up to 12 cycles.

Eric S Winer, MD

Eric S Winter, MD

Eric s Winer, MD

Eric S. Winer, MD

The goal of this research study is to find the safest and most effective dose of the study drug, BXCL701, for the treatment of Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS).

The names of the study drugs involved in this study are/is:

* BXCL701

This is a multi-center, single-arm, Phase I research study for the study drug, BXCL701, for participants with refractory or relapsed Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome with Excess Blasts-2 (MDS-EB-2). Phase I clinical trials test the safety and effectiveness of an investigational drug to learn whether the drug works in treating a specific disease. "Investigational" means the drug is being studied. BXCL701 is a synthetic dipeptide that is in tablet form and is taken by mouth.

The U.S. Food and Drug Administration (FDA) has not approved BXCL701 as a treatment for any disease.

Research study procedures include screening for eligibility and study treatment including evaluations, blood collections, bone marrow biopsies, and follow up visits.

Participation in this study will last approximately 3 years.

It is expected that about 24 people will participate in this study.

BioXcel is supporting this research study by providing the study drug and funding research tests and procedures.

BXCL701 Phase 1 R/R Acute Myeloid Leukemia or Myelodysplastic Syndrome

A Phase 1 Study of BXCL701 in Relapsed/Refractory Acute Myeloid Leukemia or Relapsed/Refractory Myelodysplastic Syndrome With Excess Blasts - 2

The Dana-Farber / Harvard Cancer Center encourages and supports the responsible and ethical sharing of data from clinical trials. De-identified participant data from the final research dataset used in the published manuscript may only be shared under the terms of a Data Use Agreement. Requests may be directed to: \[contact information for Sponsor Investigator or designee\]. The protocol and statistical analysis plan will be made available on Clinicaltrials.gov only as required by federal regulation or as a condition of awards and agreements supporting the research.

Defined as the highest dose at which fewer than one-third of patients experience a dose-level toxicity (DLT). MTD will be used to inform recommended Phase II dose. If no DLTs are observed, the MTD is not reached.

Assessed using NCI Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.

Proportion of patients who have the response, criteria are based upon International Working Group (IWG) and European LeukemiaNet (ELN) criteria for Acute Myeloid Leukemia (APPENDIX D) and Myelodysplastic Syndrome (APPENDIX F).

Proportion of patient who has the response, criteria are based upon International Working Group (IWG) and European LeukemiaNet (ELN) criteria for Acute Myeloid Leukemia (APPENDIX D) and Myelodysplastic Syndrome (APPENDIX F).

Based upon International Working Group (IWG) and European LeukemiaNet (ELN) criteria for AML (APPENDIX D) and MDS (APPENDIX F). Defined as decrease by \>= 50% in blast percentage to 5-25% or to \<= 5% with auer rods present in marrow; normalization neutrophil count \>= 1000μL and platelet count \>= 100000μL.

Evaluated only in AML patients per the 2017 ELN criteria (Dohner, Blood 2017, PMID: 27895058), defined as: Bone marrow blasts \<5%; absence of blasts with Auer rods; absence of extramedullary disease; no hematologic recovery required.

OS is based on the Kaplan-Meier method, defined as the time from study entry to death or censored at date last known alive.

Defined as the time of first morphologic response until the earlier of progression of disease or death due to any cause.

Defined as the maximum concentration of BCXL701 over the collection period.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
BXCL701 and Pembrolizumab in Patients With Metastatic Pancreatic Ductal Adenocarcinoma	NCT05558982	Metastatic Panc...	BXCL701 Pembrolizumab	18 Years -	Georgetown University
BXCL701 Phase 1 R/R Acute Myeloid Leukemia or Myelodysplastic Syndrome	NCT05703542	Acute Myeloid L... Myelodysplastic...	BXCL701	18 Years -	Dana-Farber Cancer Institute
BXCL701 Phase 1 R/R Acute Myeloid Leukemia or Myelodysplastic Syndrome	NCT05703542	Acute Myeloid L... Myelodysplastic...	BXCL701	18 Years -	Dana-Farber Cancer Institute

Spots Global Cancer Trial Database for bxcl701

Every month we try and update this database with for bxcl701 cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Spots Global Cancer Trial Database for bxcl701

Every month we try and update this database with for bxcl701 cancer trials from around the world to help patients and their families find trials that might be right for them. We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Every month we try and update this database with for bxcl701 cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.