Clinical trials: Myelodysplastic Neoplasm
Paroxysmal Nocturnal Hemoglobinuria (PNH)
ALXN1210 (Ravulizumab) Versus Eculizumab in Complement Inhibitor Treatment-Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
The primary purpose of this study was to assess the noninferiority of ravulizumab compared to eculizumab in adult participants with PNH who had never been treated with a complement inhibitor (treatment-naïve).
- Trial name
- NCT02946463
- Testing
- Ravulizumab, Eculizumab
- Where
- 120 sites in United States, Argentina and 24 more
- Volunteers
- 272 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Relapsed/Refractory Advanced Solid Tumors, Relapsed/Refractory Diffuse Large B-cell Lymphoma
A Study of PRT543 in Participants With Advanced Solid Tumors and Hematologic Malignancies
This is a Phase 1 cohort, dose-escalation, dose-expansion study of PRT543 in patients with advanced cancers who have exhausted available treatment options.
- Conditions
- Relapsed/Refractory Advanced Solid Tumors, Relapsed/Refractory Diffuse Large B-cell Lymphoma, Relapsed/Refractory Myelodysplasia, Relapsed/Refractory Myelofibrosis
- Trial name
- NCT03886831
- Testing
- PRT543
- Where
- 23 sites in United States
- Volunteers
- 232 people
- Sponsor
- Prelude Therapeutics Incorporated
MDS
APR-246 & Azacitidine for the Treatment of TP53 Mutant Myelodysplastic Syndromes (MDS)
A Phase III, multicenter, randomized study to compare the rate of complete response (CR) and duration of CR, in patients with TP53-mutated MDS who will receive APR-246 and azacitidine or azacitidine alone.
- Trial name
- NCT03745716
- Testing
- APR-246 + azacitidine, Azacitidine
- Where
- 27 sites in United States, France
- Volunteers
- 154 people
- Sponsor
- Aprea Therapeutics
Myelodysplastic Neoplasms
First-Line Luspatercept in Transfusion-Dependent Lower-Risk Myelodysplastic Neoplasms
This study will observe adults with lower-risk myelodysplastic neoplasms (MDS) who have anemia requiring regular red blood cell transfusions and who are prescribed first-line luspatercept as part of routine medical care.
- Trial name
- LUNIS (NCT07752121)
- Testing
- Luspatercept
- Where
- Germany
- Volunteers
- 190 people
- Sponsor
- Bristol-Myers Squibb
Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia
Assessment of Chimerism and Relapse Post Bone Marrow/Hematopoietic Cell Transplant (HCT) Using AlloHeme Test
AlloHeme is a chimerism test service that utilizes NGS technology to analyze SNP loci to quantify donor and recipient cells by measuring genomic DNA.
- Conditions
- Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Myelodysplastic Syndrome
- Trial name
- ACROBAT (NCT04635384)
- Where
- 10 sites in United States
- Volunteers
- 307 people
- Sponsor
- CareDx
Myelodysplastic Syndrome
Study of AR-67 (DB-67) in Myelodysplastic Syndrome (MDS)
The purpose of this study is to determine if AR-67 is effective in the treatment for patients with MDS.
- Trial name
- NCT00956787
- Testing
- AR-67 (20S)-7-t-Butyldimethylsilyl-10-hydroxycamptothecin)
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Arno Therapeutics
Acute Myeloid Leukemia, Myelodysplastic Syndrome
Continuation Study of B1371019(NCT03416179) and B1371012(NCT02367456) Evaluating Azacitidine With Or Without Glasdegib In Patients With Previously Untreated AML, MDS or CMML
An open-label study available to all eligible participants from Study B1371019 and participants originating from Study B1371012 continuing on study intervention with azacitidine with or without glasdegib.
- Conditions
- Acute Myeloid Leukemia, Myelodysplastic Syndrome, Chronic Myelomonocytic Leukemia
- Trial name
- NCT04842604
- Testing
- Glasdegib, Azacitidine
- Where
- 18 sites in Austria, Canada and 9 more
- Volunteers
- 14 people
- Sponsor
- Pfizer
Myelodysplastic Syndrome
MORE - Monitoring Revlimid - Collecting of Patient Information From Myelodysplastic Syndrome (MDS) Italian National Registry
The purpose of this study is to collect clinical and laboratory data for patients affected by intermediate 1 and low risk Myelodysplastic Syndrome (MDS) associated by deletion 5q who were prescribed Revlimid from 31October 2008…
- Trial name
- MORE (NCT01347944)
- Testing
- lenalidomide
- Where
- 45 sites in Italy
- Volunteers
- 149 people
- Sponsor
- Celgene
Paroxysmal Nocturnal Hemoglobinuria
A Study of Single-dose MY008211A in Healthy Adults
The trial is the first human trial. The safety, tolerability, PK and PD of MY008211A Tablets will be evaluated in healthy subjects.
- Trial name
- NCT05642585
- Testing
- MY008211A tablets
- Where
- China
- Volunteers
- 63 people, healthy volunteers welcome
- Sponsor
- Wuhan Createrna Science and Technology Co., Ltd
Leukemia
A Study to Investigate BAY2402234, a Dihydroorotate Dehydrogenase (DHODH) Inhibitor, in Myeloid Malignancies
The primary objective is to determine the safety, tolerability, pharmacokinetics, maximum tolerated dose (MTD), or pharmacological active dose (PAD) of BAY2402234 in patients with acute myeloid leukemia (AML), myelodysplastic…
- Trial name
- NCT03404726
- Testing
- BAY2402234
- Where
- 5 sites in United States, France
- Volunteers
- 40 people
- Sponsor
- Bayer
Acute Myelogenous Leukemia, Myelodysplastic Syndrome
Safety and Tolerance Study of Oral Doses of CT53518 to Treat Patients With Acute Myelogenous Leukemia (AML)
This is the first study of the drug CT53518 when given to humans. The purpose of this study is to determine the highest dose of CT53518 that can safely be given to patients with Acute Myelogenous Leukemia (AML) and to identify…
- Trial name
- NCT00064584
- Testing
- CT53518
- Where
- 5 sites in United States
- Volunteers
- 60 people
- Sponsor
- Millennium Pharmaceuticals, Inc.
AML, MDS
CYC065 CDK Inhibitor and Venetoclax Study in Relapsed/Refractory AML or MDS
A Phase I Combination Study of CYC065 and Venetoclax for Relapsed or Refractory AML or MDS
- Trial name
- NCT04017546
- Testing
- CYC065, Venetoclax
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Cyclacel Pharmaceuticals, Inc.
Myelodysplastic Syndrome, MDS
Efficacy and Safety of Oral Rigosertib in Transfusion-dependent, Low or Int-1 or Trisomy 8 Int-2 Myelodysplastic Syndrome
The primary objectives of this study are to determine if rigosertib sodium, given orally in the form of soft gel capsules, is safe and is associated with a reduction in the number of blood transfusion units that are needed in…
- Conditions
- Myelodysplastic Syndrome, MDS, Trisomy 8
- Trial name
- ONTARGET (NCT01584531)
- Testing
- rigosertib
- Where
- 5 sites in United States
- Volunteers
- 82 people
- Sponsor
- Traws Pharma, Inc.
Myelodysplastic Syndrome, Leukemia, Myelomonocytic, Chronic
Pevonedistat Plus Azacitidine Versus Single-Agent Azacitidine as First-Line Treatment for Participants With Higher-Risk Myelodysplastic Syndromes (HR MDS), Chronic Myelomonocytic Leukemia (CMML), or Low-Blast Acute Myelogenous Leukemia (AML)
The purpose of this study is to determine whether the combination of pevonedistat and azacitidine improves event-free survival (EFS) when compared with single-agent azacitidine.
- Conditions
- Myelodysplastic Syndrome, Leukemia, Myelomonocytic, Chronic, Leukemia, Myeloid, Acute
- Trial name
- PANTHER (NCT03268954)
- Testing
- Azacitidine, Pevonedistat
- Where
- 242 sites in United States, Australia and 18 more
- Volunteers
- 454 people
- Sponsor
- Takeda
Cancer, MDS-EB
A Phase I Open-Label Dose Escalation Study of Intravenous INKmune In Patients With MDS or AML
INMB-INB16-002 is a Phase I open-label, dose escalation study of INKmune therapy in subjects with myelodysplastic syndrome (MDS) with excess blasts without Auer rods (EB-1 or 2, or CMML 1 or 2) or subjects with acute myeloid…
- Conditions
- Cancer, MDS-EB, Myelodysplastic Syndromes, Acute Myeloid Leukemia
- Trial name
- LAUREL (NCT05933070)
- Testing
- INKmune
- Where
- 3 sites in Greece, United Kingdom
- Volunteers
- 2 people
- Sponsor
- Inmune Bio, Inc.
Myelodysplastic Syndrome, Leukemia, Myelomonocytic, Juvenile
Study With Azacitidine in Pediatric Subjects With Newly Diagnosed Advanced Myelodysplastic Syndrome (MDS) and Juvenile Myelomonocytic Leukemia (JMML)
Indication Treatment of pediatric subjects with newly diagnosed advanced myelodysplastic syndrome (MDS) or juvenile myelomonocytic leukemia (JMML) prior to hematopoietic stem cell transplantation (HSCT).
- Trial name
- NCT02447666
- Testing
- Azacitidine
- Where
- 39 sites in Austria, Belgium and 11 more
- Volunteers
- 28 people
- Sponsor
- Celgene
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
PK/Efficacy Bridging Study of ASTX727 in Chinese Subjects With Myelodysplastic Syndromes
This is an Open-Label, Crossover, Pharmacokinetic and Efficacy Bridging Study of Oral ASTX727 versus IV Decitabine in Chinese Subjects with Myelodysplastic Syndromes
- Trial name
- NCT06091267
- Testing
- IV Decitabine, Decitabine and cedazuridine, only Decitabine and cedazuridine
- Where
- China
- Volunteers
- 72 people
- Sponsor
- Otsuka Beijing Research Institute
AML, Adult, Myelodysplastic Syndromes
A Phase I Study of CYC140, a PLK-1 Inhibitor, in Advanced Leukemias or MDS
A Phase I Pharmacologic Study of CYC140, a polo-like kinase 1 inhibitor, in Patients with Advanced Leukemias or Myelodysplastic Syndromes
- Conditions
- AML, Adult, Myelodysplastic Syndromes, ALL, Adult, CML, Refractory
- Trial name
- NCT03884829
- Testing
- CYC140
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Cyclacel Pharmaceuticals, Inc.
Hemoglobinuria, Paroxysmal
Study Using Eculizumab in Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients
The primary objective is to evaluate the safety and efficacy of eculizumab in transfusion dependent patients with hemolytic PNH.
- Trial name
- NCT00122330
- Testing
- eculizumab
- Where
- 43 sites in United States, Australia and 10 more
- Volunteers
- 75 people
- Sponsor
- Alexion Pharmaceuticals, Inc.