Clinical trials: Myelodysplastic Syndrome (MDS)
Hemoglobinuria
Paroxysmal Nocturnal Hemoglobinuria (PNH), Level of CD59 on Red and White Blood Cells in Bone Marrow Failure Syndromes
Examine red and white blood cells of PNH patients with bone marrow failure syndromes.
- Trial name
- EXPLORE (NCT01192425)
- Volunteers
- 5,580 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria
To Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Escalating Single and Multiple Doses of KP104
The purpose of this study is to evaluate safety, tolerability, immunogenicity, pharmacokinetics, pharmacodynamics, and efficacy of KP104 in healthy volunteers.
- Trial name
- NCT05490017
- Testing
- KP104
- Where
- Australia
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Kira Pharmacenticals (US), LLC.
Paroxysmal Nocturnal Hemoglobinuria, PNH
Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: Analysis of IPIG-Registry Data
This is a noninterventional registry-based cohort study utilizing data collected on danicopan-treated patients with paroxysmal nocturnal hemoglobinuria (PNH) through the International PNH Interest Group (IPIG) PNH registry.
- Trial name
- NCT07413250
- Testing
- Danicopan, Soliris/Ultomiris
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Aggressive B-Cell Non-Hodgkin's Lymphoma (NHL), Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma (CLL/SLL)
A Study of PRT2527 as Monotherapy and in Combination With Zanubrutinib or Venetoclax in Participants With R/R Hematologic Malignancies
This is a Phase 1 dose-escalation study of PRT2527, a potent and highly selective cyclin-dependent kinase (CDK) 9 inhibitor, in participants with select relapsed or refractory (R/R) hematologic malignancies.
- Conditions
- Aggressive B-Cell Non-Hodgkin's Lymphoma (NHL), Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma (CLL/SLL), Mantle Cell Lymphoma (MCL), Richter's Syndrome
- Trial name
- NCT05665530
- Testing
- PRT2527, Zanubrutinib, Venetoclax
- Where
- 24 sites in United States, Australia and 8 more
- Volunteers
- 86 people
- Sponsor
- Prelude Therapeutics Incorporated
Acute Leukemia
Clinical Study of PM01183 in Patients With Acute Leukemia or Relapsed/Refractory Myelodysplastic Syndrome
Phase I Study of PM01183 in Patients with Advanced Acute Leukemia to determine the maximum tolerated dose (MTD) and the recommended dose (RD) of PM01183.
- Trial name
- NCT01314599
- Testing
- PM01183 1 mg Powder for concentrate for solution for infusion and PM01183 4 mg Powder for concentrate for solution for infusion
- Where
- 2 sites in United States
- Volunteers
- 45 people
- Sponsor
- PharmaMar
Paroxysmal Nocturnal Hemoglobinuria (PNH), Persistent Anemia
A First-in-Human Safety and Efficacy Study of ALN-CFB, a Small Interfering RNA (siRNA) Targeting Complement Factor B, in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria With Persistent Anemia on a C5 Inhibitor
This study is researching an experimental drug called ALN-CFB. The study is focused on people with Paroxysmal Nocturnal Hemoglobinuria (PNH) who are currently taking a complement component C5 inhibitor ("C5-inhibitor") and…
- Trial name
- NCT07187401
- Testing
- ALN-CFB
- Where
- 8 sites in Canada, South Korea and 1 more
- Volunteers
- 24 people
- Sponsor
- Regeneron Pharmaceuticals
Myelodysplastic Syndromes
A Study of Darbepoetin Alfa in Anemic Subjects With Low Risk Myelodysplastic Syndrome
The purpose of this study is to assess the effect of 13 weeks of darbepoetin alfa treatment on erythroid response in anemic subjects with low risk Myelodysplastic Syndrome (MDS).
- Trial name
- NCT00095264
- Testing
- Darbepoetin Alfa
- Volunteers
- 209 people
- Sponsor
- Amgen
Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia
Safety and Efficacy of Donor T-lymphocytes Depleted ex Vivo of Host Alloreactive T-cells (ATIR) in Patients With a Hematologic Malignancy Who Received a Hematopoietic Stem Cell Transplantation From a Haploidentical Donor
The purpose of this study is to determine whether ATIR is safe and effective in reducing transplant-related mortality and improving overall survival, when infused in patients with a hematologic malignancy following a T-cell…
- Conditions
- Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Myelodysplastic Syndrome
- Trial name
- NCT01794299
- Testing
- ATIR
- Where
- 8 sites in Belgium, Canada and 2 more
- Volunteers
- 31 people
- Sponsor
- Kiadis Pharma
PNH - Paroxysmal Nocturnal Hemoglobinuria
A Phase Ib, Multicenter, Open-Label Study of Multiple-Dose EA5 in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH)
This is a multicenter, open-label, Phase Ib clinical trial designed to evaluate the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of multiple doses of the humanized monoclonal antibody EA5 in adult patients with…
- Trial name
- NCT07256301
- Testing
- EA5
- Where
- 2 sites in China
- Volunteers
- 24 people
- Sponsor
- Shanghai Lanyi Therapeutics Co., Ltd.
Leukemia, Myeloid, Acute, Myelodysplastic Syndromes
A Study of H3B-8800 (RVT-2001) in Participants With Lower Risk Myelodysplastic Syndromes
A Phase 1, an Open-label, Multicenter Phase 1 Trial to Evaluate the Safety, Pharmacokinetics and Pharmacodynamics of Splicing Modulator H3B-8800 (RVT-2001) for Subjects With Myelodysplastic Syndromes, Acute Myeloid Leukemia, and…
- Conditions
- Leukemia, Myeloid, Acute, Myelodysplastic Syndromes, Leukemia, Myelomonocytic, Chronic
- Trial name
- Encore-MDS (NCT02841540)
- Testing
- H3B-8800 (RVT-2001)
- Where
- 49 sites in United States, Belgium and 5 more
- Volunteers
- 127 people
- Sponsor
- Hemavant Sciences GmbH
Acute Leukemia, Acute Lymphoblastic Leukemia
A First-in-Human Study of HLA-Partially to Fully Matched Allogenic Cryopreserved Deceased Donor Bone Marrow Transplantation for Patients With Hematologic Malignancies
The goal of this clinical trial is to determine the safety and feasibility of allogeneic transplantation with bone marrow from a deceased donor in patients with acute and chronic leukemias, myelodysplastic syndrome, and certain…
- Conditions
- Acute Leukemia, Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Acute Biphenotypic Leukemia
- Trial name
- NCT05589896
- Testing
- Ossium HPC Marrow, Bone Marrow Transplant, Pre-transplant conditioning - Myeloablative (MAC), Pre-transplant conditioning - Reduced Intensity (RIC)
- Where
- 9 sites in United States
- Volunteers
- 12 people
- Sponsor
- Ossium Health, Inc.
Myelodysplastic Syndromes(MDS)
A Phase 3 Clinical Study to Evaluate Lemzoparlimab for Injection in Combination With Azacitidine (AZA) Versus AZA Monotherapy in Treatment-naïve Patients With Higher-risk Myelodysplastic Syndrome (MDS)
This phase3 study is a randomized, controlled, open-label, multicenter study to evaluate the efficacy and safety of Lemzoparlimab for injection in combination with AZA versus AZA monotherapy as first-line therapy in…
- Trial name
- NCT05709093
- Testing
- Lemzoparlimab+Azacitidine (AZA), Azacitidine (AZA)
- Where
- 44 sites in China
- Volunteers
- 10 people
- Sponsor
- TJ Biopharma Co., Ltd.
Clonal Cytopenia of Undetermined Significance (CCUS), Low-Risk Myelodysplastic Syndrome (LR-MDS)
Safety and Tolerability of REGN17235 in Adult Participants With Clonal Cytopenia of Undetermined Significance and Low-Risk Myelodysplastic Syndrome With SF3B1 Mutation
This study will test a study drug called REGN17235 (the "study drug") to see if it can help treat Clonal Cytopenia of Undetermined Significance (CCUS) and Low-Risk Myelodysplastic Syndrome (LR-MDS) with a specific genetic…
- Trial name
- NCT07753148
- Testing
- REGN17235
- Where
- 2 sites in Belgium
- Volunteers
- 52 people
- Sponsor
- Regeneron Pharmaceuticals
Myelodysplastic Syndrome
Efficacy and Safety of ON 01910.Na in Myelodysplastic Syndrome (MDS) Patients With Trisomy 8 or Classified as Intermediate-1, -2 or High Risk
This study will explore the efficacy and safety of a regimen of ON 01910.Na as a 48-hour continuous intravenous infusion once a week for 3 weeks of a 4-week cycle in MDS patients with Trisomy 8 or classified as Intermediate-1, -2…
- Trial name
- NCT00906334
- Testing
- ON 01910.Na
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Traws Pharma, Inc.
Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome
Study of Orally Administered AG-881 in Patients With Advanced Hematologic Malignancies With an IDH1 and/or IDH2 Mutation
The purpose of this Phase I, multicenter study is to evaluate the safety, pharmacokinetics, pharmacodynamics and clinical activity of AG-881 in advanced hematologic malignancies that harbor an IDH1 and/or IDH2 mutation
- Conditions
- Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome, Hematologic Malignancies
- Trial name
- NCT02492737
- Testing
- AG881
- Where
- 6 sites in United States, France
- Volunteers
- 46 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome (MDS)
Antigen-specific T Cell Therapy for AML or MDS Patients With Relapsed Disease After Allo-HCT
This Research study is being done to characterize the safety, tolerability, and preliminary antitumor activity of the NEXI-001 T cell product (a new experimental therapy), which contains populations of CD8+ T cells targeting…
- Trial name
- NCT04284228
- Testing
- Infusion of NEXI-001 T Cells
- Where
- 5 sites in United States
- Volunteers
- 22 people
- Sponsor
- NexImmune Inc.
AML, MDS
A Study of E6201 for the Treatment of Advanced Hematologic Malignancies With FLT3 and/or Ras Mutations
This is a Phase 1/2a dose-escalation study of E6201, a dual mitogen-activated protein kinase/extracellular-signal regulated kinase 1 (MEK1) and FMS-like tyrosine kinase 3 (FLT3) inhibitor, in subjects with advanced hematologic…
- Conditions
- AML, MDS, CMML
- Trial name
- NCT02418000
- Testing
- E6201
- Where
- 4 sites in United States
- Volunteers
- 27 people
- Sponsor
- Spirita Oncology, LLC
Hematological Malignancies, Myelodysplasia
A Phase 1 Study of MKC-1 in Patients With Refractory Hematologic Malignancies
The main objectives of this study are to evaluate the side effects of MKC-1 and to determine a safe dose of MKC-1 for future studies in patients with hematological malignancies
- Conditions
- Hematological Malignancies, Myelodysplasia, Agnogenic Myeloid Metaplasia
- Trial name
- NCT00506402
- Testing
- MKC-1
- Where
- Canada
- Volunteers
- 30 people
- Sponsor
- CASI pharmaceuticals, Inc.
MDS
A Study Evaluating the Safety and Efficacy of IBI188 in Combination With AZA in Subjects With Newly Diagnosed MDS
This is an open-label, phase 1b study to evaluate the safety, tolerability and preliminary efficacy of IBI188 in combination with Azacitidine in newly diagnosed higher risk MDS patients.
- Trial name
- NCT04511975
- Testing
- IBI188, Azacitidine
- Where
- 3 sites in United States
- Volunteers
- 32 people
- Sponsor
- Innovent Biologics (Suzhou) Co. Ltd.
Paroxysmal Nocturnal Hemoglobinuria
Proof of Concept Study to Assess the Efficacy, Safety of HRS-5965 in Patients With Paroxysmal Nocturnal Hemoglobinuria
This is a multicenter, randomized, open-label phase II clinical trial. A total of 24 patients with paroxysmal nocturnal hemoglobinuria naïve to complement inhibitor therapy were included.
- Trial name
- NCT06051357
- Testing
- HRS-5965 tablets
- Where
- 2 sites in China
- Volunteers
- 26 people
- Sponsor
- Chengdu Suncadia Medicine Co., Ltd.