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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.

Allogeneic Stem Cell Transplant With Alpha/Beta T AND B Cell Depletion for Hematologic Malignancies (AB-CliniMACs)

Status(es): Recruiting
Study Date(s): Wednesday, October 1, 2014 to Tuesday, October 1, 2024
Disease(s): myelodysplastic syndromes (MDS)
Age Group: Up to 23 years
This is a single arm pilot study for patients using α/β T cell-depleted PSCT in with alternative donor sources with hematologic malignancies receiving alternative donor (unrelated or partially matched related) mobilized peripheral stem cells (PSCs) using the CliniMACS system for T cell depletion plus CD19+ B cell depletion to determine efficacy as determined by engraftment and GVHD, and one year leukemia free survival.

ALXN1210 (Ravulizumab) Versus Eculizumab in Complement Inhibitor Treatment-Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Status(es): Active, not recruiting
Study Date(s): Tuesday, December 20, 2016 to Sunday, January 1, 2023
Disease(s): paroxysmal nocturnal hemoglobinuria (PNH)
Age Group: 18 years and older
The primary purpose of this study was to assess the noninferiority of ravulizumab compared to eculizumab in adult participants with PNH who had never been treated with a complement inhibitor (treatment-naïve).

ALXN1210 Versus Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab

Status(es): Active, not recruiting
Study Date(s): Monday, June 5, 2017 to Monday, March 1, 2021
Disease(s): paroxysmal nocturnal hemoglobinuria (PNH)
Age Group: 18 years and older
The primary purpose of this study was to assess the noninferiority of ravulizumab compared to eculizumab in adult participants with PNH who were clinically stable after having been treated with eculizumab for at least 6 months.

AML/MDS Drug Sensitization by in Vivo Chemotherapy Administration

Status(es): Not yet recruiting
Study Date(s): Friday, February 28, 2020 to Friday, February 28, 2025
Disease(s): acute myeloid leukemia (AML), myelodysplastic syndromes (MDS)
Age Group: 18 years and older
In this study, the investigators will explore the feasibility of ex vivo drug screening to predict sensitivity to chemotherapy resistance and to identify novel synergy between chemotherapies.

An Efficacy and Safety Study of Luspatercept (ACE-536) for the Treatment of Anemia Due to IPSS-R Very Low, Low or Intermediate Risk Myelodysplastic Syndromes (MDS) in Japanese Subjects Who Are Not Requiring Red Blood Cell Transfusion

Status(es): Recruiting
Study Date(s): Monday, May 20, 2019 to Sunday, June 16, 2024
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 20 years and older
The study will be conducted in compliance with the International Council for Harmonisation (ICH) of Technical Requirements for Registration of Pharmaceuticals for Human Use/Good Clinical Practice (GCP) and applicable regulatory requirements. This is a Phase 2, multicenter, single-arm study to evaluate the efficacy, safety and Pharmacokinetics (PK) of luspatercept (ACE-536) for the treatment of anemia due to International prognostic scoring system-Revised (IPSS-R) very low, low or intermediate risk Myelodysplastic syndromes (MDS)in Japanese subjects who are not requiring Red blood cell (RBC)...

An European Platform for Translational Research in Myelodysplastic Syndromes

Status(es): Recruiting
Study Date(s): Monday, September 30, 2019 to Friday, September 30, 2022
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
Rationale Myelodysplastic syndromes (MDS) are rare cancers with unmet medical needs. Study of MDS has been rapidly transformed by genome characterization. The investigators hypothesize that comprehensive analyses of large patient population will allow to correctly estimate the effect of each mutation on clinical outcomes, and that niche factors and immune dysfunctions may influence the development of MDS, clonal evolution and response to treatments

APL-2 Long Term Safety and Efficacy Extension Study

Status(es): Active, not recruiting
Study Date(s): Tuesday, August 28, 2018 to Saturday, August 20, 2022
Disease(s): paroxysmal nocturnal hemoglobinuria (PNH)
Age Group: 18 years and older
This is an Open Label, Non-Randomized, Multi-Center Extension Study. Eligible subjects will have previously completed an APL-2 study.

APR-246 in Combination With Azacitidine for TP53 Mutated AML (Acute Myeloid Leukemia) or MDS (Myelodysplastic Syndromes) Following Allogeneic Stem Cell Transplant

Status(es): Active, not recruiting
Study Date(s): Tuesday, April 30, 2019 to Friday, October 1, 2021
Disease(s): acute myeloid leukemia (AML), myelodysplastic syndromes (MDS)
Age Group: 18 years and older
A multi-center, open label, Phase II clinical trial to assess the safety and efficacy of APR-246 in combination with azacitidine as maintenance therapy after allogeneic HSCT (hematopoietic stem cell transplant) for patients with TP53 mutant AML or MDS. Detailed Description: A multi-center, open label, Phase II clinical trial to assess the safety and efficacy of APR-246 in combination with azacitidine as maintenance therapy after allogeneic HSCT for patients with TP53 mutant AML or MDS. Patients will be prescreened for TP53 mutant AML or MDS before they have a HSCT. In order to proceed with...

Assessing MGTA-145 in Mobilizing Stem Cells for Use in ASTC for Hematological Malignancies

Status(es): Recruiting
Study Date(s): Monday, February 22, 2021 to Tuesday, August 1, 2023
Disease(s): acute myeloid leukemia (AML), aplastic anemia, graft versus host disease (GVHD), myelodysplastic syndromes (MDS)
Age Group: 18 to 65 years
This is a Phase II, open-label, multicenter, prospective study of MGTA-145 + plerixafor mobilized HLA-matched sibling and matched unrelated donor allografts for myeloablative hematopoietic stem cell transplantation (HSCT) in recipients with hematological malignancies. Donors will undergo 1 or 2 days of mobilization and apheresis.

ASTX727 and FT-2102 in Treating IDH1-Mutated Recurrent/Refractory Myelodysplastic Syndrome or Acute Myeloid Leukemia

Status(es): Recruiting
Study Date(s): Thursday, August 22, 2019 to Thursday, March 31, 2022
Disease(s): acute myeloid leukemia (AML), myelodysplastic syndromes (MDS)
Age Group: 18 years and older
This phase Ib/II trial studies the side effects and best dose of FT-2102 when given together with ASTX727 in treating patients with IDH1-mutated myelodysplastic syndrome or acute myeloid leukemia that has come back (recurrent) or does not respond to treatment (refractory). ASTX727 is an oral deoxyribonucleic acid (DNA) methyltransferase (DNMT) inhibitor. DNA methylation is necessary for cell differentiation and development. Changes to the methylation profile can lead to DNA instability which can cause diseases like cancer. DNMT inhibitors target and inhibit these changes. FT-2102 is an...
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