Clinical trials: Myelodysplastic Neoplasm
Myelodysplastic Syndromes, Acute Myeloid Leukemia
A Study of Sabatolimab and Magrolimab-based Treatment in AML or Higher Risk MDS Participants
This study is to determine the safety and preliminary efficacy of sabatolimab in combination with magrolimab and azacitidine in adult participants with 1L unfit Acute Myeloid Leukemia (AML) or with 1L higher risk Myelodysplastic…
- Trial name
- NCT05367401
- Testing
- Sabatolimab, Magrolimab, Azacitidine
- Sponsor
- Novartis Pharmaceuticals
Myelodysplastic Syndrome (MDS)
STIMULUS MDS-US : Sabatolimab Added to HMA in Higher Risk MDS
The main objective of this study was to assess the safety profile of MBG453 (sabatolimab) in combination with FDA approved hypomethylating agents (HMAs) of investigator's choice (IV Decitabine or Azacitidine /SC Azacitidine /Oral…
- Trial name
- NCT04878432
- Testing
- MBG453, Azacitidine, Decitabine
- Where
- 15 sites in United States
- Volunteers
- 39 people
- Sponsor
- Novartis Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria
Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients
A study to assess the safety and efficacy of eculizumab in Japanese patients with hemolytic PNH.
- Trial name
- NCT01192399
- Testing
- Eculizumab
- Volunteers
- 29 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria, C3 Glomerulopathy
A Study of Iptacopan in Korean Patients With Paroxysmal Nocturnal Hemoglobinuria or C3 Glomerulopathy
This is a post-marketing surveillance study conducted as part of the Risk Management Plan (RMP) for South Korea, to evaluate the safety and effectiveness of iptacopan in real-world clinical settings for the treatment of either…
- Trial name
- NCT07416162
- Where
- 3 sites in South Korea
- Volunteers
- 21 people
- Sponsor
- Novartis Pharmaceuticals
Clonal Mast Cell Disease, KIT D816V Mutation
Screening Study for KIT D816V Mutated Mast Cell Disease in Select Populations
This is a multicenter screening study to characterize the prevalence of the KIT D816V mutation in participants with suspected clonal mast cell disease.
- Conditions
- Clonal Mast Cell Disease, KIT D816V Mutation, Suspected KITD816V Mutated Clonal Mast Cell Disease
- Trial name
- POLARIS (NCT07143669)
- Testing
- Screening
- Where
- 26 sites in United States, Belgium
- Volunteers
- 750 people
- Sponsor
- Blueprint Medicines Corporation
AML, Acute Myeloid Leukemia
CER-1236 in Patients With Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome (MDS), and Myelofibrosis (MF)
This is a first in human, multi center, open label, phase 1/1b study to evaluate the safety and preliminary efficacy of CER-1236 in patients with relapsed/refractory (R/R), measurable residual disease (MRD) positive acute myeloid…
- Conditions
- AML, Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia
- Trial name
- CertainT-1 (NCT06834282)
- Testing
- CER-1236, Cyclophosphamide, Fludarabine
- Where
- 5 sites in United States
- Volunteers
- 18 people
- Sponsor
- CERo Therapeutics Holdings, Inc.
Advanced Malignancies That Harbor IDHR132 Mutations
A Study of IDH305 in Patients With Advanced Malignancies That Harbor IDH1R132 Mutations
A Phase I study of IDH305 in patients with advanced malignancies that harbor IDH1R132 mutations.
- Trial name
- NCT02381886
- Testing
- IDH305
- Where
- 16 sites in United States, Australia and 6 more
- Volunteers
- 166 people
- Sponsor
- Novartis Pharmaceuticals
Hematology, MDS
Evaluating the Safety of Long Term Dosing of Romiplostim (Formerly AMG 531) in Thrombocytopenic Subjects With Myelodysplastic Syndromes (MDS)
This is an open label extension study of romiplostim for treatment of thrombocytopenia (platelet count ≤ 50 x 10^9/L) in MDS subjects.
- Conditions
- Hematology, MDS, Myelodysplastic Syndromes, Thrombocytopenia
- Trial name
- NCT00472290
- Testing
- Romiplostim (formerly AMG 531)
- Volunteers
- 72 people
- Sponsor
- Amgen
Acute Leukemia, Chronic Myeloproliferative Disease
A Phase I Trial of ZIO-101 in Hematologic Cancers
This study uses a new investigational (not yet approved by the FDA for widespread use) drug called ZIO-101, an organic arsenical.
- Conditions
- Acute Leukemia, Chronic Myeloproliferative Disease, Chronic Lymphoproliferative Disease, Multiple Myeloma
- Trial name
- NCT00592046
- Testing
- ZIO-101 (Darinaparsin)
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Alaunos Therapeutics
Paroxysmal Nocturnal Hemoglobinuria
A Study to Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Whose Current Treatment is Not Working Efficiently
This study is researching a treatment combination with two experimental drugs called pozelimab and cemdisiran referred to as "study drugs".
- Trial name
- NCT07154745
- Testing
- Pozelimab, Cemdisiran
- Where
- 20 sites in Brazil, Canada and 5 more
- Volunteers
- 35 people
- Sponsor
- Regeneron Pharmaceuticals
Anemia, Myelodysplastic Syndromes
Study of Sotatercept for the Treatment of Anemia in low-or Intermediate-1 Risk Myelodysplastic Syndromes (MDS) or Non-proliferative Chronic Myelomonocytic Leukemia (CMML)
The primary objective of this study is to determine a safe, tolerable and effective dose of sotatercept that results in the greatest frequency of improvement of anemia in patients diagnosed with low- or intermediate-1 risk…
- Conditions
- Anemia, Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, Low to Intermediate-1 MDS
- Trial name
- NCT01736683
- Testing
- Sotatercept
- Where
- 20 sites in United States, France
- Volunteers
- 74 people
- Sponsor
- Merck Sharp & Dohme LLC
Paroxysmal Nocturnal Hemoglobinuria
Long-term Safety and Tolerability of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria
This is a multicenter, open-label study to evaluate the long-term safety, tolerability and efficacy of HSK39297. Adult patients with PNH who had previously received and completed HSK39297 study treatment will be included.
- Trial name
- NCT06745622
- Testing
- HSK39297 tablets
- Where
- China
- Volunteers
- 47 people
- Sponsor
- Haisco Pharmaceutical Group Co., Ltd.
Essential Thrombocythemia (ET), Myelodysplastic/Myeloproliferative Neoplasms (MDS/MPN)
Phase 1/ Phase 2 Study to Assess Safety and Efficacy of Orally Administered JBI-802 in Subjects With Myeloproliferative Neoplasms (MPN) and Myelodysplastic/Myeloproliferative Neoplasms (MDS/MPN) With Thrombocytosis
This study aims to assess the safety and efficacy of orally administered JBI-802 in subjects with Myeloproliferative Neoplasms (MPN) and Myelodysplastic/ Myeloproliferative Neoplasms (MDS/MPN) with Thrombocytosis.
- Conditions
- Essential Thrombocythemia (ET), Myelodysplastic/Myeloproliferative Neoplasms (MDS/MPN), Myeloproliferative Neoplasms (MPN)
- Trial name
- NCT07612280
- Testing
- JBI-802
- Where
- 7 sites in Australia
- Volunteers
- 30 people
- Sponsor
- Jubilant Therapeutics Inc.
Paroxysmal Nocturnal Hemoglobinuria
Study of Safety and Efficacy of HS-10542 in Patients With Paroxysmal Nocturnal Hemoglobinuria
This was a phase 1b/2,open label, multi-center study to assess efficacy and safety of HS-10542 in adulte patients with paroxysmal nocturnal hemoglobinuria (PNH) with signs of active hemolysis.
- Trial name
- NCT07470762
- Testing
- HS-10542
- Where
- China
- Volunteers
- 50 people
- Sponsor
- Jiangsu Hansoh Pharmaceutical Co., Ltd.
Hematologic Malignancy
Safety/PK Study of Gene Modified Donor T Cell Infusion in Children With Recurrent Hem Malignancies After Allo Transplant
Phase I, open-label, non-randomized study of safety, pharmacokinetics and efficacy of donor BPX-501 T cell infusion in children with recurrent or minimal residual disease (MRD) hematologic malignancies post-allogeneic transplant.
- Trial name
- NCT03459170
- Testing
- BPX-501 T cells, rimiducid
- Where
- 3 sites in Italy
- Volunteers
- 15 people
- Sponsor
- Bellicum Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria
A Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors
A study designed to evaluate the non-inferiority of crovalimab compared with eculizumab in participants with PNH who have not been previously treated with complement inhibitor therapy.
- Trial name
- COMMODORE 2 (NCT04434092)
- Testing
- Crovalimab, Eculizumab
- Where
- 63 sites in Argentina, Brazil and 21 more
- Volunteers
- 210 people
- Sponsor
- Hoffmann-La Roche
Paroxysmal Nocturnal Hemoglobinuria
A Study to Evaluate the Efficacy and Safety of Oral HRS-5965 in Adult Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Who Are Naive to Complement Inhibitor Therapy
A study of the efficacy and safety of HRS-5965 capsules compared to eculizumab for 24 weeks in patients with PNH.
- Trial name
- NCT06593938
- Testing
- HRS-5965 capsule, Eculizumab Injection
- Where
- 2 sites in China
- Volunteers
- 76 people
- Sponsor
- Chengdu Suncadia Medicine Co., Ltd.
Non-transfusion-dependent Thalassemia, Low Risk Myelodysplastic Syndrome
A Study Investigate the Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Response of SLN124 in Adults With Alpha/Beta-thalassaemia and Very Low- and Low-risk Myelodysplastic Syndrome
This study will investigate the safety and tolerability of SLN124 in patients with Thalassaemia or patients with Very Low- and Low-risk Myelodysplastic Syndrome (MDS) after single ascending s.c.
- Conditions
- Non-transfusion-dependent Thalassemia, Low Risk Myelodysplastic Syndrome, Very-Low Risk Myelodysplastic Syndrome
- Trial name
- NCT04718844
- Testing
- SLN124
- Where
- 20 sites in Germany, Israel and 5 more
- Volunteers
- 44 people
- Sponsor
- Silence Therapeutics plc
Acute Myeloid Leukemia, Acute Lymphoid Leukemia
Precision-T: A Randomized Study of Orca-T in Recipients Undergoing Allogeneic Transplantation for Hematologic Malignancies
This study will evaluate the safety, tolerability, and efficacy of Orca-T, an allogeneic stem cell and T-cell immunotherapy biologic manufactured for each patient (transplant recipient) from the mobilized peripheral blood of a…
- Conditions
- Acute Myeloid Leukemia, Acute Lymphoid Leukemia, Mixed Phenotype Acute Leukemia, Undifferentiated Leukemia
- Trial name
- Orca-T (NCT05316701)
- Testing
- Orca-T, Standard-of-Care
- Where
- 19 sites in United States
- Volunteers
- 187 people
- Sponsor
- Orca Biosystems, Inc.
Myelodysplastic Syndrome
The Efficacy and Safety of Oral Azacitidine Plus Best Supportive Care Versus Placebo and Best Supportive Care in Subjects With Red Blood Cell (RBC) Transfusion-Dependent Anemia and Thrombocytopenia Due to International Prognostic Scoring System (IPSS) Low Risk Myelodysplastic Syndrome (MDS)
Evaluation of the Efficacy and Safety of Oral Azacitidine plus Best Supportive care versus Placebo and Best Supportive care in subjects with red blood cell (RBC) transfusion-dependent anemia and thrombocytopenia due to…
- Trial name
- NCT01566695
- Testing
- Oral Azacitidine, Best Supportiv Care (BSC)
- Where
- 196 sites in United States, Australia and 21 more
- Volunteers
- 216 people
- Sponsor
- Celgene