Clinical Trials | Page 5 | Aplastic Anemia & MDS International Foundation

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 ASTX030 (Cedazuridine in Combination With Azacitidine) in MDS, CMML, or AML

Status(es): Not yet recruiting
Study Date(s): Wednesday, April 1, 2020 to Saturday, April 1, 2023
Disease(s): acute myeloid leukemia (AML), chronic myelomonocytic leukaemia (CMML), myelodysplastic syndromes (MDS), myeloproliferative neoplasms (MPN)
Age Group:
Study ASTX030-01 is designed to move efficiently from Phase 1 to Phase 3. Phase 1 consists of an open-label Dose Escalation Stage (Stage A) using multiple cohorts at escalating dose levels of oral cedazuridine and azacitidine (only one study drug will be escalated at a time) followed by a Dose Expansion Stage (Stage B) of ASTX030. Phase 2 is a randomized open-label crossover study to compare oral ASTX030 to subcutaneous (SC) azacitidine. Phase 3 is a randomized open-label crossover study comparing the final oral ASTX030 tablet to SC azacitidine. The duration of the study is expected to be...

A Study of ASTX660 as a Single Agent and in Combination With ASTX727 in Subjects With Relapsed/Refractory Acute Myeloid Leukemia (AML)

Status(es): Not yet recruiting
Study Date(s): Friday, May 1, 2020 to Friday, October 1, 2021
Disease(s): acute myeloid leukemia (AML)
Age Group: 18 years and older
This is a three-part dose escalation and dose expansion Phase 1 study of ASTX660 alone and in combination with ASTX727 in adults with R/R AML. Part 1 is an open-label, single arm, dose escalation with ASTX660 in combination with ASTX727 at the standard fixed dose combination (FDC). Part 2 is an open-label, randomized, dose escalation intended to evaluate ASTX660 as a monotherapy and ASTX660 in combination with ASTX727 FDC. Part 3 is an exploratory single arm dose expansion to further expand the number of participants treated with ASTX660 in combination with ASTX727 FDC.

A Study of BLEX 404 Oral Liquid in Patients With International Prognostic Scoring System (IPSS) Intermediate-1, Intermediate-2 or High-Risk Myelodysplastic Syndrome (MDS) and Chronic Myelomonocytic Leukemia (CMML)

Status(es): Not yet recruiting
Study Date(s): Thursday, October 1, 2020 to Wednesday, December 1, 2021
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 20 years and older
This is a clinical trial to determine the safety, recommended dose level (RDL), and infection control of BLEX 404 Oral Liquid in combination with azacitidine in patients with International Prognostic Scoring System (IPSS) intermediate-1 (int-1), intermediate-2 (int-2) or high-risk myelodysplastic syndrome (MDS) and Chronic Myelomonocytic Leukemia (CMML).

A Study of Brequinar in Subjects With Relapsed/Refractory Acute Myeloid Leukemia

Status(es): Recruiting
Study Date(s): Friday, December 13, 2019
Disease(s): acute myeloid leukemia (AML)
Age Group: 18 years and older
A Phase 1b/2a multi-center, open-label, non-randomized study to assess the safety, tolerability and efficacy of dose-adjusted brequinar in adult subjects with acute myeloid leukemia (AML).

A Study of BTX-A51 in People With Relapsed or Refractory Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome

Status(es): Recruiting
Study Date(s): Monday, January 6, 2020 to Tuesday, February 28, 2023
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
This is an open-label, dose escalation study to evaluate the safety, toxicity, and pharmacokinetics (PK) as well as preliminary efficacy of BTX-A51 capsules in participants with relapsed or refractory acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS). The study will be done in two phases. Phase 1a of this study is designed to determine the dose-limiting toxicities (DLTs) and maximum tolerated dose (MTD) of orally administered BTX-A51 in up to 35 participants who are evaluable for toxicity. Once the MTD is determined, it is planned that an additional 15 participants...

A Study of Cusatuzumab in Combination With Azacitidine Compared With Azacitidine Alone in Patients With Higher-risk Myelodysplastic Syndrome (MDS) or Chronic Myelomonocytic Leukemia (CMML) and Who Are Not Candidates for Hematopoietic Stem Cell Transplanta

Status(es): Not yet recruiting
Study Date(s): Wednesday, April 1, 2020 to Tuesday, April 19, 2022
Disease(s): chronic myelomonocytic leukaemia (CMML), myelodysplastic syndromes (MDS)
Age Group: 18 years and older
The purpose of the study is to compare overall response rate (ORR) between treatment groups in participants with higher-risk Myelodysplastic Syndrome (MDS) or Chronic Myelomonocytic Leukemia (CMML) who are not eligible for Hematopoietic Stem Cell Transplantation (HSCT).   Condition or disease  Intervention/treatment  Phase  Myelodysplastic SyndromesLeukemia, Myelomonocytic, Chronic Drug: AzacitidineDrug: Cusatuzumab Phase 2 Detailed Description: Cusatuzumab (also known as JNJ-74494550 and ARGX-110) is a humanized monoclonal antibody of camelid origin that binds with high affinity...

A Study of E6201 for the Treatment of Advanced Hematologic Malignancies With FLT3 Mutation (AML)

Status(es): Recruiting
Study Date(s): Wednesday, April 1, 2015 to Sunday, October 1, 2017
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years or older
This is a Phase 1/2a dose-escalation study of E6201, a dual MEK1 and FLT3 inhibitor, in subjects with advanced hematologic malignancies with documented FLT3 mutation. The Phase1 portion of the study will be a safety run-in to establish a recommended Phase 2 dose (RP2D). The Ph. 2a portion of the study will evaluate two specific patients groups: Cohort 1 will enroll up to 26 patients with relapsed or refractory AML and confirmed FLT3 mutation without prior exposure to a FLT3 inhibitor while Cohort 2 will enroll up to 12 patients with relapsed or refractory AML and confirmed FLT3 mutation with...

A Study of Engineered Donor Grafts (TregGraft) in Recipients Undergoing Allogeneic Transplantation for Hematologic Malignancies

Status(es): Recruiting
Study Date(s): Wednesday, July 10, 2019 to Friday, September 30, 2022
Disease(s): acute myeloid leukemia (AML), myelodysplastic syndromes (MDS)
Age Group: 18 years to 65 years
This study will evaluate the safety, tolerability, and efficacy of an engineered donor graft ("TregGraft", a T-cell-Depleted Graft With Additional Infusion of Conventional T Cells and Regulatory T Cells) in participants undergoing myeloablative allogeneic hematopoietic cell transplant transplantation for hematologic malignancies.

A Study of Idasanutlin With Cytarabine Versus Cytarabine Plus Placebo in Participants With Relapsed or Refractory Acute Myeloid Leukemia (AML) (MIRROS)

Status(es): Recruiting
Study Date(s): Wednesday, December 30, 2015 to Wednesday, May 26, 2021
Disease(s): acute myeloid leukemia (AML)
Age Group: 18 years and older
This is a multicenter, double-blind, randomized, placebo-controlled study designed to compare overall survival in participants with relapsed or refractory AML treated with idasanutlin in combination with cytarabine versus participants treated with placebo and cytarabine. Participants will receive induction treatment with idasanutlin/placebo and cytarabine (Cycle 1). Responding participants may continue to receive a maximum of further two cycles of consolidation (Cycle 2 and Cycle 3). Complete remission (CR), CR with incomplete platelet count recovery (CRp), overall remission rate (ORR),...

A Study of JNJ-67571244 in Participants With Relapsed or Refractory Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS)

Status(es): Recruiting
Study Date(s): Thursday, March 28, 2019 to Tuesday, January 25, 2022
Disease(s): acute myeloid leukemia (AML), myelodysplastic syndromes (MDS)
Age Group: 18 years and older
The main purpose of this study are to determine the recommended Phase 2 dose(s) (RP2D), schedule and the maximum tolerated dose (MTD) in Part 1 and to determine the safety and tolerability of JNJ-67571244 at the RP2D regimen(s) and to evaluate the preliminary clinical activity of JNJ-67571244 in Part 2.