Clinical Trials | Page 2 | 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.

Personalized Adoptive Cellular Therapy Targeting MDS Stem Cell Neoantigens (PACTN)

Status(es): Recruiting
Study Date(s): Thursday, February 1, 2018 to Tuesday, December 1, 2020
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
Patients with higher risk myelodysplastic syndromes (MDS) who fail current approved therapy or become refractory, have few therapeutic options other than bone marrow transplant, which may not be an option due to age, infirmity or lack of a donor. This is a Phase 1 open label, dose escalation clinical trial of Personalized, Adoptive immunotherapy by Cytotoxic T cells that are targeted to patient-specific cancer cell Neoantigens (PACTN) for such patients. It’s primary goal is to learn the safety and tolerability of PACTN infusion and secondarily, if PACTN show signs of being effective. The...

Study of PTC299 in Relapsed/Refractory Acute Leukemias

Status(es): Recruiting
Study Date(s): Monday, October 29, 2018 to Wednesday, November 25, 2020
Disease(s): acute myeloid leukemia (AML)
Age Group: 18 years and older
This is an open-label, non-randomized, Phase 1b study to evaluate the safety, pharmacokinetics (PK) profiles, and preliminary evidence of antitumor activity of PTC299 and the metabolite, O-desmethyl PTC299, in participants with relapsed/refractory acute myeloid leukemia (AML) who have exhausted standard available therapies known to provide clinical benefit. The study is designed as a series of cohort-based dose escalations. For each cohort, a minimum of 3 evaluable participants with PK and safety data will be assessed. Additional participants will be recruited if additional PK data are...

The National Myelodysplastic Syndromes Natural History Study

Status(es): Recruiting
Study Date(s): Friday, April 1, 2016 to Wednesday, September 1, 2021
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
Multi-center study enrolling patients suspected or newly diagnosed with myelodysplastic syndromes (MDS), myelodysplastic syndromes/myeloproliferative neoplasms (MDS/MPN) overlap disorder, or idiopathic cytopenia of undetermined significance (ICUS).  Participants will be followed long term.  Clinical data, blood, and tissue samples will be collected to establish a biorepository to facilitate the study of the natural history of MDS.

Trial of the Combination of Bortezomib and Clofarabine in Adults With Relapsed Solid Tumors

Status(es): Recruiting
Study Date(s): Wednesday, August 6, 2014 to Friday, August 31, 2018
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
This is an open-label Phase I trial. The starting dose of clofarabine will be 1 mg/m2 administered intravenously on days 1 through 5 of a 21-day cycle; bortezomib will be administered at 0.8 mg/m2 subcutaneously on days 1 and 4 of a 21-day cycle. Dose escalation will follow a 3+3 design, with dose limiting toxicities defined during cycle 1. Dose escalation will proceed in cohorts comprised of two separate groups of patients (one group of patients with solid tumor/lymphoma and one group of patients with MDS), with at least 1 from each group, until hematologic DLT or the second grade 2...

211At-BC8-B10 Followed by Donor Stem Cell Transplant in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, or Myelodysplastic Syndrome

Status(es): Recruiting
Study Date(s): Wednesday, March 20, 2019 to Sunday, September 1, 2024
Disease(s): acute myeloid leukemia (AML), chronic myelomonocytic leukaemia (CMML), myelodysplastic syndromes (MDS)
Age Group: 18 years and older
  This phase I/II trial studies the side effects and best dose of a radioactive agent linked to an antibody (211At-BC8-B10) followed by donor stem cell transplant in treating patients with acute myeloid leukemia, or acute lymphoblastic leukemia, or myelodysplastic syndrome that has come back or isn't responding to treatment. Monoclonal antibodies, such as 211At-BC8-B10, may interfere with the ability of cancer cells to grow and spread. Giving chemotherapy and total body irradiation before a stem cell transplant helps stop the growth of cells in the bone marrow, including normal blood-forming...

211^At-BC8-B10 Before Donor Stem Cell Transplant in Treating Patients With High-Risk Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Myelodysplastic Syndrome, or Mixed-Phenotype Acute Leukemia

Status(es): Recruiting
Study Date(s): Tuesday, October 24, 2017 to Friday, March 22, 2024
Disease(s): acute myeloid leukemia (AML), myelodysplastic syndromes (MDS)
Age Group: 18 years and older
This phase I/II trial studies the side effects and best dose of 211^astatine(At)-BC8-B10 before donor stem cell transplant in treating patients with high-risk acute myeloid leukemia, acute lymphoblastic leukemia, myelodysplastic syndrome, or mixed-phenotype acute leukemia. Radioactive substances, such as astatine-211, linked to monoclonal antibodies, such as BC8, can bind to cancer cells and give off radiation which may help kill cancer cells and have less of an effect on healthy cells before donor stem cell transplant.

5-Azacitidine and Decitabine Epigenetic Therapy for Myeloid Malignancies

Status(es): Not yet recruiting
Study Date(s): Wednesday, April 1, 2020 to Friday, December 1, 2023
Disease(s): myelodysplastic syndromes (MDS), myeloproliferative neoplasms (MPN)
Age Group: 18 years and older
Another term for myelodysplastic syndrome is bone marrow failure. The bone marrow is where components of blood such as red cells, platelets and white cells are made. In bone marrow failure, the ability for bone marrow to make these cells is decreased. In myelodysplastic syndrome, this decreased bone marrow function is believed to result from abnormalities that prevent the normal maturation process by which bone marrow cells develop into red blood cells, white blood cells and platelets. In myelodysplastic syndrome, these abnormal bone marrow cells occupy space in the bone marrow and prevent...

8-Chloro-Adenosine in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia

Status(es): Recruiting
Study Date(s): Tuesday, September 1, 2015 to Saturday, September 1, 2018
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
This phase I/II trial studies the side effects and best dose of 8-chloro-adenosine and how well it works in treating patients with acute myeloid leukemia that has returned after a period of improvement (relapsed) or has not responded to previous treatment (refractory). Drugs used in chemotherapy, such as 8-chloro-adenosine, 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.

A Biomarker-Directed Phase 2 Trial of SY-1425 in Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome

Status(es): Recruiting
Study Date(s): Monday, August 1, 2016 to Friday, December 31, 2021
Disease(s): acute myeloid leukemia (AML), myelodysplastic syndromes (MDS)
Age Group: 18 years and older
The purpose of this study is to determine the activity of SY-1425 in relapsed/refractory acute myeloid leukemia (AML) patients (SY-1425 administered as a monotherapy or in combination with azacitidine), relapsed/refractory higher-risk myelodysplastic syndrome (MDS) patients (SY-1425 administered as a monotherapy or in combination with daratumumab), newly diagnosed treatment naïve AML patients who are unlikely to tolerate standard intensive chemotherapy (SY-1425 administered as a monotherapy or in combination with azacitidine), or lower-risk myelodysplastic syndrome (MDS) patients (SY-1425...

A Dose Escalation Study to Investigate the Safety, Pharmacokinetics (PK), Pharmacodynamics (PD) and Clinical Activity of GSK525762 in Subjects With Relapsed, Refractory Hematologic Malignancies

Status(es): Recruiting
Study Date(s): Thursday, December 19, 2019
Disease(s): acute myeloid leukemia (AML), myeloproliferative neoplasms (MPN)
Age Group: 18 years and older
This is an open-label repeat dose, multicenter, 2-part study to determine the maximum tolerated dose (MTD) and the recommended Phase 2 dose (RP2D) for GSK525762 given once-daily (QD) orally. Part 1 of the study is a dose escalation phase to select the recommended Part 2 dose (RP2D) based on the safety, PK, and PD profiles observed after oral administration of GSK525762. Eligible subjects with select relapsed refractory hematological malignancies (acute myeloid leukemia [AML], non-Hodgkin's Lymphoma [NHL]and multiple myeloma [MM]), will be enrolled in the QD and/or BID dosing cohorts until a...