Clinical trials: Myelodysplastic Neoplasm
Paroxysmal Nocturnal Hemoglobinuria (PNH)
ALXN1210 Versus Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab
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.
- Trial name
- NCT03056040
- Testing
- Ravulizumab, Eculizumab
- Where
- 51 sites in United States, Australia and 9 more
- Volunteers
- 202 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Acute Myeloid Leukemia, Myelodysplastic Syndromes
Study of Cell Therapies for the Treatment of Patients With Relapsed or Refractory Acute Myeloid Leukemia or High-risk Myelodysplastic Syndrome.
Master Protocol: The main goal of this master clinical trial study is to assesses the safety, tolerability, pharmacokinetic (PK), Pharmacodynamic (PD) in participants with relapsed or refractory acute myeloid leukemia (AML) or…
- Trial name
- NCT05457010
- Testing
- SPRX002, ARC-T Cells, Cyclophosphamide
- Where
- 5 sites in United States
- Volunteers
- 24 people
- Sponsor
- Kite, A Gilead Company
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study
This is a long-term follow up study evaluating the safety of BPX-501 T cells (rivogenlecleucel) and infused in pediatric patients previously enrolled on the BP-004 study.
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndromes
- Trial name
- NCT03733249
- Testing
- Rimiducid, rivogenlecleucel
- Where
- Italy
- Volunteers
- 187 people
- Sponsor
- Bellicum Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria
Real Life Use of Ravulizumab in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria
This study will collect clinical response data on participants who were already treated with eculizumab for at least 26 weeks and who started ravulizumab treatment as a specific therapeutic strategy as per ordinary clinical…
- Trial name
- NCT05274633
- Testing
- Ravulizumab
- Where
- 6 sites in Italy
- Volunteers
- 120 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Investigation of Pharmacokinetics,Safety,and Pharmacodynamics of HSK39297 in Subjects With Hepatic Impairment
The study is being conducted to compare the pharmacokinetics, safety, and pharmacodynamics of HSK39297 in subjects with mild to moderate hepatic impairment and normal hepatic function
- Trial name
- NCT07152288
- Testing
- HSK39297 tablets
- Where
- China
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Haisco Pharmaceutical Group Co., Ltd.
Paroxysmal Nocturnal Hemoglobinuria
Proof of Concept Study to Assess the Efficacy, Safety and Pharmacokinetics of LFG316 in Patients With Paroxysmal Nocturnal Hemoglobinuria
The purpose of this study was to determine whether LFG316 can induce a hematological response, as measured by reduction in hemolytic activity, in patients with paroxysmal nocturnal hemoglobinuria (PNH).
- Trial name
- NCT02534909
- Testing
- LFG316, LNP023
- Where
- 7 sites in Czechia, Japan and 1 more
- Volunteers
- 10 people
- Sponsor
- Novartis Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria
A Study of the Efficacy and Safety of FXS6837 Capsules Compared to Eculizumab for 24 Weeks in Patients With PNH.
An open label study to evaluate the efficacy and safety of FXS6837 capsules compared to eculizumab in naive PNH patients. About 90 PNH patients who are naive to complement inhibitor therapies will be enrolled to take FXS6837…
- Trial name
- NCT07747090
- Testing
- FXS6837 Capsule, Eculizumab infusion
- Where
- China
- Volunteers
- 90 people
- Sponsor
- Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.
Acute Myeloid Leukemia, Chronic Myelomonocytic Leukemia
Study to Evaluate the Safety and Tolerability of EP0042
A research study looking at a new treatment for patients with advanced cancer, to investigate different doses of the experimental study drug, EP0042, in order to determine a dose, which is safe, well-tolerated and likely to be…
- Conditions
- Acute Myeloid Leukemia, Chronic Myelomonocytic Leukemia, Myelodysplastic Syndromes
- Trial name
- NCT04581512
- Testing
- EP0042, Venetoclax, Azacitidine (AZA)
- Where
- 6 sites in Australia, Netherlands and 1 more
- Volunteers
- 70 people
- Sponsor
- Ellipses Pharma
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.
Relapsed / Refractory AML, Relapsed / Refractory MDS
Safety, Pharmacokinetics, and Preliminary Efficacy of BYON4413 in Acute Myeloid Leukemia and Myelodysplastic Neoplasms.
This is the first-in-human trial with BYON4413 to evaluate safety, PK, immunogenicity, and anti-leukemia activity of BYON4413 in patients with AML or MDS.
- Trial name
- NCT06359002
- Testing
- BYON4413
- Where
- 9 sites in Belgium, Netherlands and 1 more
- Volunteers
- 16 people
- Sponsor
- Byondis B.V.
Tumor Lysis Syndrome, Cancer
Rasburicase Versus Allopurinol in Tumor Patients at Risk for Hyperuricemia and Tumor Lysis Syndrome
This is a randomized, multi-center, open-label, parallel group study with three arms: Rasburicase alone Rasburicase followed by Allopurinol Allopurinol alone The primary objective is to compare the adequacy of control of plasma…
- Conditions
- Tumor Lysis Syndrome, Cancer, Hyperuricemia
- Trial name
- NCT00230178
- Testing
- Rasburicase (SR29142), Allopurinol
- Where
- 15 sites in United States
- Volunteers
- 280 people
- Sponsor
- Sanofi
Myelodysplastic Syndromes
A Study of Tebapivat (AG-946) in Participants With Anemia Due to Lower-Risk Myelodysplastic Syndromes (LR-MDS)
This purpose of this study is to establish proof of concept of tebapivat in participants with LR-MDS in Phase 2a and to evaluate the effect of tebapivat on transfusion independence (TI) in participants with LR-MDS in phase 2b.
- Trial name
- NCT05490446
- Testing
- Tebapivat
- Where
- 45 sites in United States, Australia and 11 more
- Volunteers
- 87 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Myelodysplastic Syndrome
A Phase I/II Study of OPN-305 in Second-line Lower Risk Myelodysplastic Syndrome
The dose-confirming part of this study, comprising at least 10 patients is designed as a single center, prospective, single arm, open label in patients who have failed or are unresponsive to Azacitidine (AZA) or Decitabine (they…
- Trial name
- NCT02363491
- Testing
- OPN-305
- Where
- 4 sites in United States
- Volunteers
- 96 people
- Sponsor
- Opsona Therapeutics Ltd.
Leukemia, Myeloid, Acute, Myelodysplastic Syndromes
Allogeneic Engineered Hematopoietic Stem Cell Transplant (HCT) Lacking the CD33 Protein, and Post-HCT Treatment With Mylotarg, for Patients With CD33+ AML or MDS
This is a Phase 1/2a, multicenter, open-label, first-in-human (FIH) study of VOR33 in participants with AML or MDS who are undergoing human leukocyte antigen (HLA)-matched allogeneic hematopoietic cell transplant (HCT).
- Trial name
- NCT04849910
- Testing
- VOR33, Mylotarg
- Where
- 15 sites in United States, Canada
- Volunteers
- 67 people
- Sponsor
- Vor Biopharma
Myelodysplastic Syndromes, Leukemia, Myelomonocytic, Chronic
Guadecitabine (SGI-110) vs Treatment Choice in Adults With MDS or CMML Previously Treated With HMAs
A Phase 3, randomized, open-label, parallel-group, multicenter study designed to evaluate the efficacy and safety of guadecitabine in participants with MDS or CMML who failed or relapsed after adequate prior treatment with…
- Trial name
- NCT02907359
- Testing
- Guadecitabine, Treatment Choice
- Where
- 112 sites in United States, Belgium and 12 more
- Volunteers
- 417 people
- Sponsor
- Astex Pharmaceuticals, Inc.
Hematologic Malignancies
Safety Study of MultiStem® in Patients With Acute Leukemia, Chronic Myeloid Leukemia, or Myelodysplasia
The purpose of this study is to determine if MultiStem® can safely be given to patients with acute leukemia, chronic myeloid leukemia, or myelodysplasia after they have received hematopoietic stem cell transplantation.
- Trial name
- NCT00677859
- Testing
- MultiStem®
- Where
- 6 sites in United States, Belgium
- Volunteers
- 36 people
- Sponsor
- Healios K.K.
Paroxysmal Nocturnal Hemoglobinuria, PNH
Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Chinese Adults Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
The primary objective of this study is to evaluate the efficacy of ravulizumab in adult participants with PNH.
- Trial name
- NCT06578949
- Testing
- Ravulizumab
- Where
- 8 sites in China
- Volunteers
- 18 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Myelodysplastic Syndromes
A Study to Evaluate the MNV-201 in Patients With Low Risk MDS
Myelodysplastic syndromes (MDS) are a group of bone marrow failures that occur when the blood-forming cells in the bone marrow become abnormal leading to an abnormal differentiation and production of one or more blood cell types.
- Trial name
- NCT06465160
- Testing
- MNV-201 (Autologous CD34+ Cells Enriched with allogenic Placenta Derived Mitochondria)
- Where
- Israel
- Volunteers
- 15 people
- Sponsor
- Minovia Therapeutics Ltd.
Myelodysplastic Syndromes, Beta-thalassemia
A Study to Evaluate Luspatercept Utilization in Patients With Lower Risk Myelodysplastic Syndromes or Beta-thalassemia
The purpose of this study is to understand the patient profile, utilized treatments, treatment patterns, resulting outcomes, and associated costs relating to luspatercept treatment in patients with lower-risk myelodysplastic…
- Trial name
- NCT07681440
- Testing
- Luspatercept
- Where
- Germany
- Volunteers
- 30 people
- Sponsor
- Bristol-Myers Squibb
B-cell Lymphoma, Acute Myeloid Leukemia
A Study of XS-04 in Patients With Relapsed or Refractory Hematologic Malignancies
Evaluation of the safety, tolerability, pharmacokinetics, and preliminary efficacy of XS-04 in patients with relapsed or refractory hematologic malignancies
- Conditions
- B-cell Lymphoma, Acute Myeloid Leukemia, Myelodysplastic Syndrome
- Trial name
- NCT06820268
- Testing
- XS-04 tablet
- Where
- 4 sites in China
- Volunteers
- 168 people
- Sponsor
- Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.