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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 JNJ-75276617 in Participants With Acute Leukemia

Status(es): Not yet recruiting
Study Date(s): Tuesday, March 23, 2021 to Tuesday, December 26, 2023
Disease(s): acute myeloid leukemia (AML)
Age Group: 18 years and older
Acute myeloid leukemia (AML) is a heterogeneous disease characterized by uncontrolled clonal expansion of hematopoietic progenitor cells (myeloid blasts) in the peripheral blood, bone marrow, and other tissues. Acute lymphoblastic leukemia (ALL) is a hematologic malignancy propagated by impaired differentiation, proliferation, and accumulation of lymphoid progenitor cells in the bone marrow and/or extramedullary sites. JNJ-75276617 is an orally bioavailable, potent, and selective protein-protein interaction inhibitor of the binding between histone-lysine N-methyltransferase 2A ([KMT2A], also...

A Study of LB-100 in Patients With Low or Intermediate-1 Risk Myelodysplastic Syndromes (MDS)

Status(es): Recruiting
Study Date(s): Friday, March 22, 2019 to Thursday, July 1, 2021
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 Years and older
The purpose of this study is to test the safety and efficacy (benefits) of an investigational drug LB-100, for treatment of myelodysplastic syndromes. LB-100 has previously been administered to patients with various solid tumors. In this study, LB-100 will be administered as an intravenous infusion over 120 minutes. This study will be conducted in 2 phases. In phase Ib, escalating doses of LB-100 will be administered to patients to study the safety and to determine a safe dose of LB-100. In phase 2, patients will be administered LB-100 at the dose that was found to be safe in phase Ib. The...

A Study of LNK01002 in Patients With Primary (PMF) or Secondary Myelofibrosis (PV-MF, ET-MF) or Acute Myeloid Leukemia

Status(es): Recruiting
Study Date(s): Friday, May 21, 2021 to Monday, October 23, 2023
Disease(s): acute myeloid leukemia (AML)
Age Group: 18 years and older
This is a Phase I, open-label, dose-finding study of the triple kinase inhibitor LNK01002 in patients with myelofibrosis (MF). The study consists of two periods: the dose escalation, main period and a dose expansion period. In the dose escalation period, successive cohorts of patients with MF will be enrolled to establish the maximum tolerated dose. In the dose expansion period (dose-confirmation phase), three cohorts of patients will be enrolled: AML patients with confirmed FLT3-ITD mutations, AML patients without FLT3-ITD mutations, and patients with primary MF or PV/ET-MF. The safety,...

A Study of MBG453 in Combination With Hypomethylating Agents in Subjects With IPSS-R Intermediate, High or Very High Risk Myelodysplastic Syndrome (MDS). (STIMULUS-MDS1)

Status(es): Recruiting
Study Date(s): Tuesday, June 4, 2019 to Thursday, August 3, 2023
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
This Phase II is a multicenter, randomized, two-arm parallel-group, double-blind, placebo-controlled study of MBG453 or placebo added to hypomethylating agents (azacitidine or decitabine) in adult subjects with IPSS-R intermediate, high or very high risk myelodysplastic syndrome (MDS) not eligible for Hematopoietic Stem Cell Transplant (HSCT) or intensive chemotherapy.

A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Status(es): Recruiting
Study Date(s): Wednesday, May 26, 2021 to Monday, April 1, 2024
Disease(s): paroxysmal nocturnal hemoglobinuria (PNH)
Age Group: 12 years to 17 years
This is an open-label study to evaluate pegcetacoplan in people with PNH who are 12-17 years old for up to 5 months (20 weeks). At the completion of the study treatment period, participants will either enter a long-term extension period or a 2-month follow-up period. All eligible study participants will receive pegcetacoplan, administered via subcutaneous infusion twice a week at home. The subcutaneous infusion requires two small needles to be inserted into the fatty layer of tissue under the skin and the investigational medication will flow into the body. Study participants and/or...

A Study of Pevonedistat in Combination With Azacitidine in Participants With Higher-risk Myelodysplastic Syndromes (HR MDS), Chronic Myelomonocytic Leukemia (CMML), or Acute Myelogenous Leukemia (AML) With Severe Renal Impairment or Mild Hepatic Impairmen

Status(es): Recruiting
Study Date(s): Wednesday, January 23, 2019 to Wednesday, October 6, 2021
Disease(s): acute myeloid leukemia (AML), chronic myelomonocytic leukaemia (CMML), myelodysplastic syndromes (MDS)
Age Group: 18 years and older
The drug being tested in this study is called pevonedistat. The study will characterize the PK of pevonedistat, assess the safety, and determine the dose of pevonedistat, in combination with azacitidine, in participants with myelodysplastic syndromes (MDS), CMML and AML who also have severe renal impairment or mild hepatic impairment. The study will enroll approximately 60 participants. The study will be conducted in 2 parts: Part A (PK run-in and dose escalation phase) and Part B. In Part A, participants will be assigned to one of the 3 treatment groups on the basis of their renal and...

A Study of SNDX-5613 in R/R Leukemias Including Those With an MLLr/KMT2A Gene Rearrangement or NPM1 Mutation (AUGMENT-101)

Status(es): Recruiting
Study Date(s): Tuesday, November 5, 2019 to Monday, January 1, 2024
Disease(s): acute myeloid leukemia (AML)
Age Group: 18 years and older
Phase 1 dose escalation will determine the maximum tolerated dose (MTD) and recommended Phase 2 dose (RP2D) of SNDX-5613 in patients with acute leukemia. In Phase 2, patients will be enrolled in 3 indication-specific expansion cohorts to determine the efficacy, short- and long-term safety, and tolerability of SNDX-5613.

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).
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