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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.

A Study of the Effect of Blood Stem Cell Transplant After Chemotherapy Alone in Patients With Fanconi Anemia

Status(es): Active, not recruiting
Study Date(s): Tuesday, May 15, 2018 to Tuesday, May 31, 2022
Disease(s): acute myeloid leukemia (AML), myelodysplastic syndromes (MDS)
Age Group: 1 month and older
The goal of this study is to see if the study therapy can decrease the chemotherapy-related side effects while maximizing the effectiveness of disease control. The physicians will also be studying the effect of removing T-cells from the donor‟s stem cells before transplant. T-cells are a type of white blood cell that may help cause a serious side effect of transplant called Graft versus Host Disease (GVHD). The way it removes the T-cells from the donor stem cells is actually by selecting only the stem cells (called CD34 cells) by using a device called CliniMACS. This process is called CD34...

A Study of the IDH1 Inhibitor AG-120 in Combination With the Checkpoint Blockade Inhibitor, Nivolumab, for Patients With IDH1 Mutated Relapsed/Refractory AML and High Risk MDS

Status(es): Recruiting
Study Date(s): Wednesday, February 12, 2020 to Tuesday, September 12, 2023
Disease(s): acute myeloid leukemia (AML), myelodysplastic syndromes (MDS)
Age Group: 19 years and older
In this trial the investigators aim to evaluate safety and efficacy of combination Ivosidenib (AG-120) and nivolumab in the context of adult patients with Isocitrate dehydrogenase-1 (IDH1) mutated acute myeloid leukemias (AML) or Myelodysplastic syndromes (MDS).

A Study of Venetoclax and Alvocidib in Patients With Relapsed/Refractory Acute Myeloid Leukemia

Status(es): Recruiting
Study Date(s): Wednesday, February 21, 2018 to Tuesday, September 28, 2021
Disease(s): acute myeloid leukemia (AML)
Age Group: 18 years and older
An open-label, dose-escalation study to assess the safety and pharmacokinetics (PK), to determine the dose limiting toxicity (DLT) and the recommended Phase 2 dose (RPTD), and to assess the preliminary efficacy of alvocidib with venetoclax when co-administered in participants with relapsed or refractory (R/R) acute myeloid leukemia (AML).

A Study of Venetoclax and Dinaciclib (MK7965) in Patients With Relapsed/Refractory Acute Myeloid Leukemia

Status(es): Recruiting
Study Date(s): Monday, July 23, 2018 to Tuesday, October 5, 2021
Disease(s): acute myeloid leukemia (AML)
Age Group: 18 years and older
  An open-label, dose-escalation study to assess safety, pharmacokinetics and efficacy as well as determine the recommended Phase 2 doses of co-administered therapy of dinaciclib and venetoclax for patients with relapsed or refractory Acute Myeloid Leukemia (R/R AML).

A Study of Venetoclax in Combination With Cobimetinib and Venetoclax in Combination With Idasanutlin in Patients Aged >/= 60 Years With Relapsed or Refractory Acute Myeloid Leukemia Who Are Not Eligible for Cytotoxic Therapy

Status(es): Recruiting
Study Date(s): Wednesday, March 9, 2016 to Friday, May 27, 2022
Disease(s): acute myeloid leukemia (AML)
Age Group: 60 years and older
The primary objective for this study is to assess the safety and tolerability as well as preliminary efficacy of venetoclax in combination with cobimetinib, and venetoclax in combination with idasanutlin in patients >/= 60 years of age with relapsed or refractory acute myeloid leukemia (R/R) AML who are not eligible for cytotoxic therapy.

A Study to Assess Efficacy and Safety of PF-06462700 in Japanese Participants With Aplastic Anemia

Status(es): Completed
Study Date(s): Saturday, July 25, 2020 to Tuesday, August 3, 2021
Disease(s): aplastic anemia
Age Group: 2 years and older
The purpose of the study is to assess the efficacy and safety of PF-06462700 administered intravenously at 40 mg/kg/day for 4 days in Japanese participants with moderate and above aplastic anemia for making an approval application in Japan.

A Study to Compare T-Guard vs Ruxolitinib for Treatment of Steroid-Refractory Acute Graft-Versus-Host Disease (SR-aGVHD) (2002)

Status(es): Not yet recruiting
Study Date(s): Tuesday, June 22, 2021 to Wednesday, January 1, 2025
Disease(s): graft versus host disease (GVHD)
Age Group: 18 years and older
Graft-vs-Host Disease (GVHD) is a complication that affects many hematopoietic stem cell transplant (HSCT) patients; it occurs when the new cells from a transplant attack the recipient's body. Acute GVHD (aGVHD) typically develops within the first three months after HSCT and is typically treated with steroid therapy. A significant fraction of the aGVHD population (10-50%) fail to respond to treatment and are deemed steroid-refractory (SR). Participants that develop Grade III or IV SR aGVHD will be randomized to receive T-Guard or ruxolitinib and will be followed for approximately 180 days....

A Telehealth Advance Care Planning Intervention for Older Patients With Acute Myeloid Leukemia and Myelodysplastic Syndrome

Status(es): Not yet recruiting
Study Date(s): Tuesday, February 9, 2021 to Tuesday, May 31, 2022
Disease(s): acute myeloid leukemia (AML), myelodysplastic syndromes (MDS)
Age Group: 18 years and older
Older adults with AMLamd MDS are more likely to receive aggressive care and less likely to utilize hospice at the end-of-life. Advance care planning (ACP) intervention delivered through telehealth may improve patient-reported outcomes and end-of-life care in this population. This pilot study seeks to evaluate the usability and feasibility of a telehealth-delivered advance care planning intervention. We will adapt the Serious Illness Care Program.

Accelerated Dose Schedule of Cytarabine Consolidation Therapy for Patients With Acute Myeloid Leukemia (AML) in Complete Remission

Status(es): Not yet recruiting
Study Date(s): Friday, June 4, 2021 to Sunday, December 1, 2024
Disease(s): acute myeloid leukemia (AML)
Age Group: 61 years to 99 years
This phase 2, open label, non-randomized study will evaluate the safety of administering high dose cytarabine (HiDAC) consolidation therapy on days 1-3 of each cycle, as compared to standard administration on days 1, 3, and 5 of each cycle, in patients 61 years and older with de novo acute myeloid leukemia (AML).

Administration of Donor Multi TAA-Specific T Cells for AML or MDS (ADSPAM)

Status(es): Recruiting
Study Date(s): Wednesday, February 17, 2016 to Friday, February 28, 2025
Disease(s): acute myeloid leukemia (AML), myelodysplastic syndromes (MDS)
Age Group: All ages
This research study uses special blood cells called multiple tumor-associated antigen (TAA)-specific T cells (a new experimental therapy) to treat patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) which has come back, or may come back, or has not gone away after standard treatment, including an allogeneic hematopoietic stem cell transplant (HSCT). The investigators have previously used this sort of therapy to treat Hodgkin or non-Hodgkin lymphomas that are infected with Epstein-Barr virus (EBV). EBV is found in cancer cells of up to half of all patients with...
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