Clinical trials: Myelodysplastic Syndrome
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Study of Safety and Efficacy of MY008211A in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
The main purpose of this study is to evaluate the efficacy of MY008211A in adult patients with PNH, showing signs of active hemolysis.
- Trial name
- NCT06134414
- Testing
- MY008211A tablets
- Volunteers
- 40 people
- Sponsor
- Wuhan Createrna Science and Technology Co., Ltd
PNH - Paroxysmal Nocturnal Hemoglobinuria
A Phase II Study Evaluating the Efficacy and Safety of XH-S003 Capsules in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
This is a multicenter, randomized, single-blind Phase II trial to evaluate the efficacy and safety of XH-S003 capsules in PNH patients.
- Trial name
- XH-S003-II-101 (NCT06978699)
- Testing
- XH-S003 Capsule
- Where
- China
- Volunteers
- 24 people
- Sponsor
- S-INFINITY Pharmaceuticals Co., Ltd
AML and High Risk MDS
A Safety and Efficacy Study of LGH447 in Patients With Acute Myeloid Leukemia (AML) or High Risk Myelodysplastic Syndrome (MDS)
This study will assess the safety and preliminary efficacy of escalating doses of LGH447 monotherapy in AML and MDS and LGH447 in combination with midostaurin in AML.
- Trial name
- NCT02078609
- Testing
- LGH447, LGH447 + midostaurin
- Where
- 9 sites in United States, Australia and 5 more
- Volunteers
- 70 people
- Sponsor
- Novartis Pharmaceuticals
Myelodysplastic Syndromes
ELEMENT-MDS: A Study to Compare the Efficacy and Safety of Luspatercept in Participants With Myelodysplastic Syndrome (MDS) and Anemia Not Receiving Blood Transfusions
The purpose of the study is to compare the efficacy and safety of Luspatercept vs epoetin alfa in the treatment of anemia in adults due to IPSS-R very low, low, intermediate-risk MDS in ESA-naïve participants who are…
- Trial name
- ELEMENT-MDS (NCT05949684)
- Testing
- Luspatercept, Epoetin Alfa
- Where
- 144 sites in United States, Argentina and 17 more
- Volunteers
- 402 people
- Sponsor
- Bristol-Myers Squibb
Leukemia, Myelodysplastic Syndrome(MDS)
A Study to Investigate Fadraciclib (CYC065), in Subjects With Leukemia or Myelodysplastic Syndrome (MDS)
This is a 2-part, phase 1/2, open-label, multicenter study designed to evaluate the safety and efficacy of fadraciclib (formerly CYC065) administered orally BID.
- Trial name
- NCT05168904
- Testing
- fadraciclib
- Where
- 2 sites in United States
- Volunteers
- 210 people
- Sponsor
- Cyclacel Pharmaceuticals, Inc.
Complement Mediated Diseases
First-In-Human Clinical Study of the C3 Complement Inhibitor AMY- 101 in Healthy Male Volunteers
Safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of a Single Ascending Dose (SAD) and a Multiple Dose (MD) of the complement inhibitor AMY-101.
- Trial name
- NCT03316521
- Testing
- AMY-101
- Where
- United States
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Amyndas Pharmaceuticals S.A.
Myelodysplastic Syndromes, Lymphoma, Mantle-Cell
A Non-interventional Study of REVLIMID® (Lenalidomide) Treatment of IPSS Low- or Intermediate-1-risk Myelodysplastic Syndromes Associated With a Deletion 5q or Refractory/Relapsed Mantle Cell Lymphoma in Korea
The Drug Use Examination (DUE) is planned and designed for the safety evaluation of new indications after the approval of a new drug in Korea.
- Conditions
- Myelodysplastic Syndromes, Lymphoma, Mantle-Cell, Lymphoma, Follicular
- Trial name
- RevlimidPMS (NCT04036448)
- Testing
- REVLIMID®
- Where
- 26 sites in South Korea
- Volunteers
- 28 people
- Sponsor
- Celgene
Hematologic Neoplasms
Phase 1/2 Study of Enasidenib (AG-221) in Adults With Advanced Hematologic Malignancies With an Isocitrate Dehydrogenase Isoform 2 (IDH2) Mutation
The primary objectives of Phase 1 Dose Escalation/Part 1 Expansion are: To assess the safety and tolerability of treatment with enasidenib administered continuously as a single agent dosed orally on Days 1 to 28 of a 28-day cycle…
- Trial name
- NCT01915498
- Testing
- Enasidenib
- Where
- 24 sites in United States, France
- Volunteers
- 345 people
- Sponsor
- Celgene
Hematological Neoplasms, Non-Hodgkin's Lymphoma
Bioequivalence & Food Effect Study in Patients With Solid Tumor or Hematologic Malignancies
This is a Phase 1, open-label, multicenter, randomized, 2-stage crossover study consisting of 2 phases: Stage I - Pharmacokinetics (Bioequivalence), with an Extension Stage II - Pharmacokinetics (Food Effect) with an Extension…
- Conditions
- Hematological Neoplasms, Non-Hodgkin's Lymphoma, Hodgkin's Lymphoma, Lymphoma
- Trial name
- NCT02223052
- Testing
- CC-486, Vidaza
- Where
- 10 sites in United States
- Volunteers
- 89 people
- Sponsor
- Celgene
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
Paroxysmal Nocturnal Hemoglobinuria
Effect of Food on Pharmacokinetics of MY008211A Tablets in Healthy Adult Subjects
This was a single-center, single-dose, open-label clinical study. 12 subjects were randomly assigned in a 1:1 ratio to one of the following dosing sequences (sequence 1: AB; Sequence 2: BA).
- Trial name
- NCT05828485
- Testing
- MY008211A tablets
- Where
- China
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Wuhan Createrna Science and Technology Co., Ltd
Myelodysplastic Syndrome
Expanded Access for ACE-011
This is an expanded access program (EAP) for eligible participants designed to provide access to ACE-011.
- Trial name
- NCT03724227
- Testing
- ACE-011
- Where
- United States
- Sponsor
- Celgene
Paroxysmal Nocturnal Hemoglobinuria (PNH)
BCX9930 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Participants Not Receiving Other Complement Inhibitor Therapy
The purpose of this study was to determine the efficacy and safety of BCX9930 monotherapy for the treatment of adult participants with PNH not currently receiving complement inhibitor therapy.
- Trial name
- REDEEM-2 (NCT05116787)
- Testing
- BCX9930 monotherapy
- Where
- 5 sites in Malaysia, South Africa and 1 more
- Volunteers
- 12 people
- Sponsor
- BioCryst Pharmaceuticals
Acute Lymphoblastic Leukaemias (ALL), Acute Myeloid Leukaemias (AML)
Treosulfan-based Conditioning in Paediatric Patients With Haematological Malignancies
The primary goal of this study is to evaluate an alternative myeloablative, but reduced toxicity conditioning regimen in children, to describe the safety and efficacy of intravenous (i.v.) Treosulfan administered as part of a…
- Conditions
- Acute Lymphoblastic Leukaemias (ALL), Acute Myeloid Leukaemias (AML), Myelodysplastic Syndromes (MDS), Juvenile Myelomonocytic Leukaemias (JMML)
- Trial name
- NCT02333058
- Testing
- Treosulfan
- Where
- 24 sites in Austria, Czechia and 4 more
- Volunteers
- 70 people
- Sponsor
- medac GmbH
AML, Adult Recurrent, MDS
A Phase 1 Study With LYT-200 in Patients With Relapsed/Refractory Acute Myeloid Leukemia (AML), or With Relapsed/Refractory, High-risk Myelodysplastic Syndrome (MDS)
A Phase 1 Open-label, Multi-center Study of the Safety, Pharmacokinetics (PK), and Anti-tumor Activity of LYT- 200 in Patients with Relapsed/Refractory Acute Myeloid Leukemia (AML), or with Relapsed/refractory, High-risk…
- Trial name
- NCT05829226
- Testing
- LYT-200, Venetoclax, Azacitidine
- Where
- 9 sites in United States
- Volunteers
- 101 people
- Sponsor
- PureTech
Acute Myeloid Leukemia (AML), High-risk Myelodysplastic Syndrome (MDS)
HDM201 in Combination With MBG453 or Venetoclax in Patients With Acute Myeloid Leukemia (AML) or High-risk Myelodysplastic Syndrome (MDS)
This was a phase 1b, multi-arm, open-label study of HDM201 in combination with MBG453 or venetoclax in subjects with AML or high-risk MDS.
- Trial name
- NCT03940352
- Testing
- HDM201, MBG453, Venetoclax
- Where
- 9 sites in United States, Australia and 5 more
- Volunteers
- 52 people
- Sponsor
- Novartis Pharmaceuticals
Acute Myeloid Leukemia, Myelodysplastic Syndrome
A Study of BTX-A51 in People With Relapsed or Refractory Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome
This is an open-label, dose escalation study to evaluate the safety, toxicity, and pharmacokinetics (PK) as well as preliminary efficacy of BTX-A51 capsules in participants with relapsed or refractory acute myeloid leukemia (AML)…
- Trial name
- NCT04243785
- Testing
- BTX-A51, Azacitidine
- Where
- 3 sites in United States
- Volunteers
- 80 people
- Sponsor
- Edgewood Oncology Inc.
Myelodysplastic Syndromes
A Study to Compare the Efficacy and Safety of Luspatercept (ACE-536) Versus Epoetin Alfa for the Treatment of Anemia Due to IPSS-R Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) Participants Who Require Red Blood Cell Transfusions and Are ESA Naïve
The purpose of this study is to determine the effectiveness of luspatercept (ACE-536) compared to epoetin alfa on red blood cell (RBC) transfusion independence (for at least 12 weeks) with a concurrent hemoglobin increase of at…
- Trial name
- COMMANDS (NCT03682536)
- Testing
- Luspatercept, Epoetin alfa
- Where
- 226 sites in United States, Australia and 24 more
- Volunteers
- 363 people
- Sponsor
- Celgene
Hemoglobinuria, Paroxysmal
Post-authorization Safety Study of Iptacopan in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Using Data From the IPIG PNH Registry
This is an observational single-arm descriptive cohort study based on the secondary use of data collected on iptacopan-treated patients with paroxysmal nocturnal hemoglobinuria (PNH) through the International PNH Interest Group…
- Trial name
- NCT06903234
- Testing
- Iptacopan
- Where
- Switzerland
- Volunteers
- 200 people
- Sponsor
- Novartis Pharmaceuticals
Myelodysplastic Syndrome
Safety Study of Mocetinostat in Combination With Azacitidine in Subjects With MDS
Mocetinostat is an orally administered histone deacetylase (HDAC) inhibitor. Azacitidine is a hypomethylating agent (HMA) used to treat MDS.
- Trial name
- NCT02018926
- Testing
- Mocetinostat, Azacitidine
- Where
- 10 sites in United States
- Volunteers
- 18 people
- Sponsor
- Mirati Therapeutics Inc.