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Clinical Trials

Clinical research is at the heart of all medical advances, identifying new ways to prevent, detect or treat disease. If you have a bone marrow failure disease, you may want to consider taking part in a clinical trial, also called a research study.

Venetoclax With Combination Chemotherapy in Treating Patients With Newly Diagnosed or Relapsed or Refractory Acute Myeloid Leukemia

Status(es): Recruiting
Study Date(s): Tuesday, July 11, 2017 to Saturday, September 30, 2023
Disease(s): acute myeloid leukemia (AML), myelodysplastic syndromes (MDS)
Age Group: 18 years or older
This phase Ib/II trial studies the best dose and side effects of venetoclax and how well it works when given with combination chemotherapy in treating patients with newly diagnosed acute myeloid leukemia or acute myeloid leukemia that has come back or does not respond to treatment. Venetoclax may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as fludarabine phosphate, cytarabine, filgrastim and idarubicin hydrochloride, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping...

Venetoclax, Busulfan, Cladribine, and Fludarabine for the Treatment of High-Risk Acute Myeloid Leukemia or Myelodysplastic Syndrome

Status(es): Not yet recruiting
Study Date(s): Wednesday, January 13, 2021 to Friday, December 31, 2021
Disease(s): acute myeloid leukemia (AML), myelodysplastic syndromes (MDS)
Age Group: 18 years to 70 years
This phase II trial studies the effect of venetoclax together with busulfan, cladribine, and fludarabine in treating patients with high-risk acute myeloid leukemia or myelodysplastic syndrome who are undergoing stem cell transplant. Chemotherapy drugs, such as venetoclax, busulfan, cladribine, and fludarabine, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Adding venetoclax to the current standard of care stem cell transplant regimen of busulfan, fludarabine, and cladribine may help...

Vorinostat Dose-escalation After Allogeneic Hematopoietic Cell Transplantation

Status(es): Recruiting
Study Date(s): Monday, February 18, 2019 to Saturday, May 1, 2021
Disease(s): acute myeloid leukemia (AML), myelodysplastic syndromes (MDS)
Age Group: 1 year to 21 years
The objective of this study is to evaluate the maximum tolerated (MTD) of vorinostat used in combination with low-dose azacitidine after allogeneic hematopoietic cell transplantation (alloHCT) for prevention of relapse of childhood myeloid malignancies.

Whole Genome Sequencing (ChromoSeq) as an Adjunct to Conventional Genomic Profiling in AML and MDS

Status(es): Active, not recruiting
Study Date(s): Tuesday, August 3, 2021 to Friday, August 30, 2024
Disease(s): acute myeloid leukemia (AML), myelodysplastic syndromes (MDS)
Age Group: 18 years and older
This is a single institution, prospective study of the whole genome sequencing assay, ChromoSeq. Using prospectively collected patient data, coupled with physician surveys, the investigators seek to determine the feasibility of implementing ChromoSeq in addition to standard genomic testing, for patients with the diagnoses of acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS).

WU-NK-101 in Relapsed/Refractory AML and MDS

Status(es): Not yet recruiting
Study Date(s): Thursday, May 20, 2021 to Wednesday, February 28, 2024
Disease(s): acute myeloid leukemia (AML), myelodysplastic syndromes (MDS)
Age Group: 18 years and older
This is a phase 2 study with a lead-in cohort of WU-NK-101, a cytokine-induced memory-like NK cell product derived from leukapheresed allogeneic donor NK cells activated ex vivo using HCW-9201, a GMP-grade fusion cytokine comprising IL-12, IL-15, and IL-18. Patients with relapsed/refractory acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) will receive lymphodepleting chemotherapy (Flu/Cy) and two infusions of WU-NK-101 at the previously defined maximum tolerated dose (MTD), fourteen days apart. Low dose rhIL-2 will be administered to patients for in vivo expansion following...
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