Myelodysplastic Syndromes — Expanded Access Programs
Overview
Right2Hope maintains a directory of 42 expanded access programs, clinical trials, and compassionate use pathways listed for Myelodysplastic Syndromes. Each entry below was sourced from sponsor disclosures and public clinical-trial registries. Use this hub to understand the breadth of ongoing research, identify sponsors with myelodysplastic syndromes programs, and link out to specific protocols for eligibility, contact, and request details. Of these, 15 programs are currently active or recruiting, with the remaining 27 in other lifecycle stages. Sponsors actively running programs in this area include Masonic Cancer Center, University of Minnesota, Case Comprehensive Cancer Center, Center for International Blood and Marrow Transplant Research, M.D. Anderson Cancer Center, National Cancer Institute (NCI), and others.
By recruiting status
Snapshot of where every Myelodysplastic Syndromes program currently sits in its lifecycle.
- Active / Recruiting15
- Inactive27
Top sponsors
Organisations running the most Myelodysplastic Syndromes programs in the directory.
- Masonic Cancer Center, University of Minnesota3 programs
- Case Comprehensive Cancer Center2 programs
- Center for International Blood and Marrow Transplant Research2 programs
- M.D. Anderson Cancer Center2 programs
- National Cancer Institute (NCI)2 programs
- Taiho Oncology, Inc.2 programs
- University of Florida2 programs
- University of Illinois at Chicago2 programs
- Abhay Singh, MD MPH1 program
- Ascentage Pharma Group Inc.1 program
Myelodysplastic Syndromes programs (42)
Sorted by recruiting status, then alphabetically by program name. Click any program to view eligibility, locations, and sponsor contact details.
- Phase 2 Study of Olutasidenib for Patients with IDH1-mutated Blood Disorders — M.D. Anderson Cancer Center
- Study for Participants Continuing ASTX727 Treatment from Previous Clinical Trials — Taiho Oncology, Inc.
- Study on Abatacept with Cord Blood Transplant for Preventing Acute Graft Versus Host Disease — Leland Metheny
- Study on Combining Unmodified Haplo-Identical Graft with Cord Blood for Transplant Patients — Case Comprehensive Cancer Center
- Cord Blood Transplantation for Children and Young Adults with Blood Cancer — Memorial Sloan Kettering Cancer Center
- Study on CPX-351 for Treating Older Patients with Acute Myeloid Leukemia (AML) and Myelodysplastic Syndromes (MDS) — Case Comprehensive Cancer Center
- Study on Helical Tomotherapy, Fludarabine, and Melphalan Followed by Donor Stem Cell Transplant for Blood Cancers — City of Hope Medical Center
- Study on Targeted Radiation and Chemotherapy Before Stem Cell Transplant for High-Risk Leukemia and Myelodysplastic Syndrome — University of Illinois at Chicago
- Study on Using KIR-Favorable Donors for Transplants in Children with Certain Blood Cancers — Michael Pulsipher
- Study on Low Dose Weekly Decitabine and Venetoclax for Treating MDS and AML — Montefiore Medical Center
- Study on Using Reduced Intensity Conditioning and Stem Cell Transplant for Treating Blood Diseases — Masonic Cancer Center, University of Minnesota
- Pre-emptive Therapy With DEC-C to Improve Outcomes in MDS Patients With Measurable Residual Disease Post Allogeneic Hematopoietic Cell Transplant — Washington University School of Medicine
- Study on Reduced Intensity or Nonmyeloablative Conditioning with Orca-T for Acute Myelogenous Leukemia and Myelodysplastic Syndrome — Orca Biosystems, Inc.
- Study on Gene-Modified Donor T-Cells After Stem Cell Transplant for Blood Disorders — Bellicum Pharmaceuticals
- Study on Using Intensity Modulated Total Marrow Irradiation with Fludarabine/Busulfan for Transplant in High-Risk AML and Myelodysplastic Syndromes — University of Illinois at Chicago
- A Phase II Study of Cladribine and Low Dose Cytarabine in Combination With Venetoclax, Alternating With Azacitidine and Venetoclax, in Patients With Higher-risk Myeloproliferative Chronic Myelomonocytic Leukemia or Higher-risk Myelodysplastic Syndromes With Excess Blasts — M.D. Anderson Cancer Center
- Pilot Study on Using Decitabine and Filgrastim After Stem Cell Transplant in Children and Young Adults with Blood Cancers — Franziska Wachter
- Study on APG-115 Alone or Combined with Azacitidine for Patients with AML, CMML, or MDS — Ascentage Pharma Group Inc.
- Study of ASTX030 (Cedazuridine and Azacitidine) for MDS, CMML, or AML — Taiho Oncology, Inc.
- A Study of BGB-11417 in Participants With Myeloid Malignancies — BeiGene
- A Study of CFI-400945 With or Without Azacitidine in Patients With AML, MDS or CMML — Treadwell Therapeutics, Inc
- Study on CTX-712 for Treating Relapsed or Hard-to-Treat Acute Myeloid Leukemia and High-Risk Myelodysplastic Syndromes — Chordia Therapeutics, Inc.
- Study on LB-100 for Patients with Low or Intermediate-1 Risk Myelodysplastic Syndromes (MDS) — Lixte Biotechnology Holdings, Inc.
- Study on the Safety and Effectiveness of Bexmarilimab Combined with Standard Treatment for Blood Cancers — Faron Pharmaceuticals Ltd
- Study on Using Reduced Intensity Conditioning and Post-Transplant Cyclophosphamide for Blood Disorders — Masonic Cancer Center, University of Minnesota
- Study on Stem Cell Transplant Without CD33 Protein and Post-Transplant Treatment with Mylotarg for Patients with CD33+ AML or MDS — Vor Biopharma
- Study on Depleting Alpha/Beta T Cells and CD19+ B Cells in Stem Cell Transplants for Patients with Blood Cancers — University of Florida
- Study on Eltanexor (KPT-8602) and Inqovi for High-Risk Myelodysplastic Syndromes — National Cancer Institute (NCI)
- Eltrombopag as a New Treatment for Low-risk MDS and CMML with TET2 Mutations — Abhay Singh, MD MPH
- Expanded Access Program for Imetelstat Treatment — Geron Corporation
- Expanded Access Program for Galinpepimut-S in Patients with AML or MDS — Sellas Life Sciences Group
- First-in-Human Study of SAR443579 Infusion in Children and Adults with Certain Types of Blood Cancer — Sanofi
- Study on HLA-Mismatched Unrelated Donor Stem Cell Transplant with Post-Transplant Cyclophosphamide — Center for International Blood and Marrow Transplant Research
- Study on HLA-Mismatched Unrelated Donor Stem Cell Transplant with Reduced Cyclophosphamide for Graft Versus Host Disease Prevention — Center for International Blood and Marrow Transplant Research
- Using Interferon-γ and Donor Leukocyte Infusion to Treat Relapsed Acute Myeloid Leukemia and Myelodysplastic Syndromes After Stem Cell Transplant — Sawa Ito, MD
- Study on Leflunomide and Decitabine for Treating Relapsed or Hard-to-Treat Myelodysplastic Syndromes — West Virginia University
- Study on MBG453 for Treating Lower Risk Myelodysplastic Syndromes (MDS) — Massachusetts General Hospital
- Study on Pacritinib for Treating Blood Disorders in Adults and Children Aged 12 and Older — National Cancer Institute (NCI)
- Study on PTCy, Sirolimus, and VIC-1911 to Prevent GVHD in Stem Cell Transplant Patients — Masonic Cancer Center, University of Minnesota
- Continuation Study for Patients Benefiting from Sabatolimab Treatment in Previous Novartis Trials — Novartis Pharmaceuticals
- Study Comparing Cladribine and Low-Dose Cytarabine with Decitabine vs. Standard Treatment for AML or High-Grade MDS in Patients Unable to Undergo Intensive Therapy — University of Florida
- Vorinostat for Graft vs Host Disease Prevention in Children, Adolescents and Young Adults Undergoing Allogeneic Blood and Marrow Transplantation — University of Michigan Rogel Cancer Center
About this hub
Expanded access programs (sometimes called compassionate use, named-patient programs, or pre-approval access) allow patients with serious or life-threatening conditions such as Myelodysplastic Syndromes to receive an investigational therapy outside of a controlled clinical trial when no comparable approved option is available. Eligibility, location, and the application process differ for each program — review the individual program pages below for sponsor contact information, inclusion criteria, and the request workflow. Right2Hope is a 501(c)(3) nonprofit and provides this directory free of charge for patients, caregivers, and treating physicians.
Looking for a different condition? Browse the full Cancer Type hub directory or search the full expanded access program catalog.