Myelodysplastic Syndrome — Expanded Access Programs
Overview
Right2Hope maintains a directory of 49 expanded access programs, clinical trials, and compassionate use pathways listed for Myelodysplastic Syndrome. 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 syndrome programs, and link out to specific protocols for eligibility, contact, and request details. Of these, 13 programs are currently active or recruiting, with the remaining 36 in other lifecycle stages. Sponsors actively running programs in this area include M.D. Anderson Cancer Center, Fred Hutchinson Cancer Center, National Cancer Institute (NCI), City of Hope Medical Center, Mayo Clinic, and others.
By recruiting status
Snapshot of where every Myelodysplastic Syndrome program currently sits in its lifecycle.
- Active / Recruiting13
- Draft1
- Inactive35
Top sponsors
Organisations running the most Myelodysplastic Syndrome programs in the directory.
- M.D. Anderson Cancer Center19 programs
- Fred Hutchinson Cancer Center6 programs
- National Cancer Institute (NCI)5 programs
- City of Hope Medical Center4 programs
- Mayo Clinic2 programs
- Orca Biosystems, Inc.2 programs
- Thomas Jefferson University2 programs
- Children's Oncology Group1 program
- Curis, Inc.1 program
- Medical College of Wisconsin1 program
Myelodysplastic Syndrome programs (49)
Sorted by recruiting status, then alphabetically by program name. Click any program to view eligibility, locations, and sponsor contact details.
- Study on Azacitidine With or Without Lenalidomide or Vorinostat for Treating Higher-Risk Myelodysplastic Syndromes or Chronic Myelomonocytic Leukemia — National Cancer Institute (NCI)
- Study on Clofarabine and Melphalan Before Donor Stem Cell Transplant for Patients with Myelodysplasia, Acute Leukemia in Remission, or Chronic Myelomonocytic Leukemia — City of Hope Medical Center
- Study on Chemotherapy and Radiation Therapy Followed by Donor Cell Transplant for Treating Older Adults with High-Risk Acute Myeloid Leukemia or Other Severe Blood Cancers — Fred Hutchinson Cancer Center
- Study on Adding Pomalidomide to Standard Chemotherapy for Newly Diagnosed Acute Myeloid Leukemia with Myelodysplastic Syndrome-Related Changes — National Cancer Institute (NCI)
- Study on Guadecitabine and Atezolizumab for Treating Advanced Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia — University of Southern California
- Study on Using Fludarabine, Cytarabine, Idarubicin, and Venetoclax for Treating Acute Myeloid Leukemia — OHSU Knight Cancer Institute
- Study on Nivolumab and/or Ipilimumab With or Without Azacitidine for Treating Myelodysplastic Syndrome — M.D. Anderson Cancer Center
- Study on Orca-T for Patients Undergoing Stem Cell Transplant for Blood Cancers — Orca Biosystems, Inc.
- Study on Using Geriatric Assessment to Plan Treatment for Older Adults with Blood Disorders — Memorial Sloan Kettering Cancer Center
- Home-Based Cancer Treatment Program for Patients in the Florida Panhandle and Nearby Areas — Mayo Clinic
- Study on Two-Step ATG Treatment to Prevent Acute GVHD After Stem Cell Transplant — University of Alabama at Birmingham
- Study on Vedolizumab, Cyclophosphamide, and Tacrolimus to Prevent Graft Versus Host Disease in Patients Undergoing Stem Cell Transplant — City of Hope Medical Center
- Study on Venetoclax and Chemotherapy Before Donor Stem Cell Transplant for Treating Acute Myelogenous Leukemia or Myelodysplastic Syndrome — M.D. Anderson Cancer Center
- Orca-T Expanded Access Program Study for Patients With Advanced Hematologic Malignancies — Orca Biosystems, Inc.
- Phase II Study of Canakinumab for Patients with Low or Intermediate-Risk Myelodysplastic Syndromes and Chronic Myelomonocytic Leukemia — M.D. Anderson Cancer Center
- Study on Using Hyperbaric Oxygen to Improve Stem Cell Transplant from Umbilical Cord Blood — University of Rochester
- Study on ASTX727, Venetoclax, and Gilteritinib for Treating FLT3-Mutated Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome — M.D. Anderson Cancer Center
- At-Home Cancer Treatment Compared to In-Clinic Treatment for Patients with Advanced Cancer — Mayo Clinic
- Study on Azacitidine and Quizartinib for Treating Myelodysplastic Syndrome or Myelodysplastic/Myeloproliferative Neoplasm with FLT3 or CBL Mutations — M.D. Anderson Cancer Center
- Study on Cladribine, Idarubicin, Cytarabine, and Venetoclax for Treating Acute Myeloid Leukemia, High-Risk Myelodysplastic Syndrome, or Blastic Phase Chronic Myeloid Leukemia — M.D. Anderson Cancer Center
- Study on Cord Blood Transplant with Chemotherapy and Radiation for Treating High-Risk Blood Diseases — Fred Hutchinson Cancer Center
- Study on CPX-351 and Ivosidenib for Treating IDH1 Mutated Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome — M.D. Anderson Cancer Center
- Study on Cytokine-Treated Veto Cells for Patients with Blood Cancers After Stem Cell Transplant — M.D. Anderson Cancer Center
- Study on Decitabine with Ruxolitinib, Fedratinib, or Pacritinib for Treating Advanced Myeloproliferative Neoplasms — University of Washington
- Donor Stem Cell Transplant with Treosulfan, Fludarabine, and Total-Body Irradiation for Treating Blood Cancers — Fred Hutchinson Cancer Center
- Study on CA-4948 as a Single Treatment for Patients with Acute Myelogenous Leukemia (AML) or Myelodysplastic Syndrome (MDS) — Curis, Inc.
- Study on Cladribine-Based Chemotherapy for Treating Relapsed or Hard-to-Treat Acute Myeloid Leukemia and High-Risk Myelodysplastic Syndrome — Medical College of Wisconsin
- Study on Enasidenib and Azacitidine for Treating Recurrent or Resistant Acute Myeloid Leukemia with IDH2 Gene Mutation — M.D. Anderson Cancer Center
- Study on Navitoclax for Patients with Aggressive Myelodysplastic Syndrome After Standard Treatments Have Failed — Thomas Jefferson University
- Graft Versus Host Disease-Reduction Strategies for Donor Blood Stem Cell Transplant Patients With Acute Leukemia or Myelodysplastic Syndrome (MDS) — Fred Hutchinson Cancer Center
- Study on Using Expanded Cord Blood Cells with Single Cord Blood Transplant for Treating Blood Cancers — Fred Hutchinson Cancer Center
- Study on Ivosidenib and Venetoclax With or Without Azacitidine for Treating Blood Cancers with IDH1 Mutation — M.D. Anderson Cancer Center
- Liposome-encapsulated Daunorubicin-Cytarabine and Gemtuzumab Ozogamicin in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML) or High Risk Myelodysplastic Syndrome — M.D. Anderson Cancer Center
- Comparing Stem Cell Transplant Options for Young People with Acute Leukemia or Myelodysplastic Syndrome — Children's Oncology Group
- MYELOMATCH: Screening Study for Myeloid Cancer Treatment Options — National Cancer Institute (NCI)
- Study on Off-the-shelf NK Cells and Stem Cell Transplant for Myeloid Blood Cancers — M.D. Anderson Cancer Center
- Personalized NK Cell Therapy After Chemotherapy and Cord Blood Transplant for Blood Cancers — M.D. Anderson Cancer Center
- Study on Quizartinib, Decitabine, and Venetoclax for Treating Untreated or Returning Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome — M.D. Anderson Cancer Center
- Study on Reduced-Intensity Conditioning to Lower Treatment-Related Deaths in Patients Undergoing Stem Cell Transplant — Thomas Jefferson University
- Study on Ruxolitinib with Tacrolimus and Methotrexate to Prevent Graft Versus Host Disease in Young Patients Undergoing Bone Marrow Transplant for Leukemia or Myelodysplastic Syndrome — City of Hope Medical Center
- Study on Seclidemstat and Azacitidine for Treating Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia — M.D. Anderson Cancer Center
- Study on Adding Enasidenib to Standard Treatment for Higher-Risk Myelodysplastic Syndrome with IDH2 Mutation — National Cancer Institute (NCI)
- Study on Total Marrow and Lymphoid Irradiation Before Stem Cell Transplant for Patients with Myelodysplastic Syndrome or Acute Leukemia — City of Hope Medical Center
- Study Comparing Treosulfan-Based and Clofarabine-Based Preparations Before Stem Cell Transplant for Treating Myelodysplastic Syndromes or Acute Myeloid Leukemia — Fred Hutchinson Cancer Center
- Study on Venetoclax and Azacitidine for Treating High-Risk Myelodysplastic Syndrome That Has Returned or Is Not Responding to Treatment — M.D. Anderson Cancer Center
- Study on Venetoclax and Decitabine for Treating Relapsed or Hard-to-Treat Acute Myeloid Leukemia and High-Risk Myelodysplastic Syndrome — M.D. Anderson Cancer Center
- Study on Venetoclax and ASTX727 for Treating Chronic Myelomonocytic Leukemia and Other Related Blood Disorders — National Cancer Institute (NCI)
- Study on Venetoclax and ASTX727 for Untreated High-Risk Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia — M.D. Anderson Cancer Center
- Study on Venetoclax to Improve Outcomes of Chemotherapy in Patients with High-Risk Acute Myeloid Leukemia and Myelodysplastic Syndrome — M.D. Anderson 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 Syndrome 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.
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