Clinical trials: Myelodysplastic Syndrome
Myelodysplastic Syndrome (MDS)
A Study of Single and Multiple Dose Administration of LP-001 in Healthy Subjects
The purpose of this study is to evaluate safety, tolerability, immunogenicity, pharmacokinetics, pharmacodynamics, and efficacy of LP-001 in healthy volunteers.
- Trial name
- NCT06294275
- Testing
- LP-001 Dose 1 (Single), LP-001 Dose 2 (Single), LP-001 Dose 3 (Single)
- Where
- China
- Volunteers
- 68 people, healthy volunteers welcome
- Sponsor
- Longbio Pharma
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
The purpose of this study was to determine the safety and efficacy of ACH-0144471 (also known as danicopan and ALXN2040) in currently untreated participants with PNH.
- Trial name
- NCT03053102
- Testing
- Danicopan
- Where
- 5 sites in Italy, New Zealand and 2 more
- Volunteers
- 10 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Myelodysplastic Syndrome
Post Marketing Surveillance to Collect and Evaluate the Safety and Efficacy Information of Korean MDS Patients Treated With VIDAZA®, After Approval of Marketing Authorization for New Drug in Korea
The main purpose of VIDAZA® DUE (Drug Use Examination) is to collect and evaluate the safety information of Korean MDS patients treated with VIDAZA®(SC or IV) according to the approved package insert, after approval of marketing…
- Trial name
- NCT02137629
- Testing
- Vidaza®
- Where
- 48 sites in South Korea
- Volunteers
- 511 people
- Sponsor
- Celgene
Chronic Myelogenous Leukemia, Ph1-Positive, Acute Myeloid Leukemia
Clinical Trial of BP1001 (Liposomal Grb2 Antisense Oligonucleotide) in Combination With Dasatinib in Patients With Ph + CML Who Have Failed TKI, Ph+ AML, Ph+ MDS
The primary objective of the Phase Ib study is to determine the dose-limiting toxicity (DLT) and maximal tolerated dose (MTD) of BP1001 in combination with dasatinib in patients with with Philadelphia Chromosome Positive (Ph+)…
- Conditions
- Chronic Myelogenous Leukemia, Ph1-Positive, Acute Myeloid Leukemia, Myelodysplastic Syndrome
- Trial name
- NCT02923986
- Testing
- BP1001 (varying dose), BP1001 (fixed dose), Dasatinib
- Where
- United States
- Sponsor
- Bio-Path Holdings, Inc.
Myelodysplastic Syndrome (MDS)
Study of TLK199 Tablets in Myelodysplastic Syndrome (MDS)
The purpose of this study is to determine the safety and efficacy of TLK199 Tablets in patients with Myelodysplastic Syndrome (MDS)
- Trial name
- NCT00280631
- Testing
- Ezatiostat Hydrochlorine
- Where
- 3 sites in United States
- Volunteers
- 49 people
- Sponsor
- Telik
Leukemia, Myelodysplastic Syndromes
Arsenic Trioxide in Treating Patients With Myelodysplastic Syndromes
RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. PURPOSE: Phase II trial to study the effectiveness of arsenic trioxide in treating patients who have…
- Conditions
- Leukemia, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms
- Trial name
- NCT00020969
- Testing
- arsenic trioxide
- Where
- 8 sites in United States
- Sponsor
- CTI BioPharma
Paroxysmal Nocturnal Hemoglobinuria
Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria
The primary objective of the study is: To evaluate the effect of pozelimab and cemdisiran combination therapy on hemolysis, as assessed by lactate dehydrogenase (LDH), after 36 weeks of treatment, in patients with PNH who switch…
- Trial name
- ACCESS 2 (NCT05131204)
- Testing
- Cemdisiran, Eculizumab, Pozelimab
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Regeneron Pharmaceuticals
Beta Thalassemia Major Anemia, Myelodysplastic Syndrome
A Trial Testing SP-420 in Subjects With Transfusion-dependent β-thalassemia or Low-risk Myelodysplastic Syndromes
The goal of this clinical trial is to learn about SP-420 ability to remove iron from organs in subjects with transfusion-dependent β-thalassemia or transfusion-dependent low-risk myelodysplastic syndrome.
- Trial name
- NCT05693909
- Testing
- SP-420
- Where
- Denmark
- Volunteers
- 90 people
- Sponsor
- Pharmacosmos A/S
Relapsed Acute Myeloid Leukemia (AML), Refractory Acute Myeloid Leukemia (AML)
Phase 1 Study of AUTX-703 in Relapsed/Refractory AML and MDS
This Phase 1, multicenter, open-label, dose escalation and dose optimization study is designed to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary clinical activity of AUTX-703…
- Conditions
- Relapsed Acute Myeloid Leukemia (AML), Refractory Acute Myeloid Leukemia (AML), Relapsed/Refractory AML, Relapsed Myelodysplastic Syndromes
- Trial name
- NCT06846606
- Testing
- AUTX-703
- Where
- 9 sites in United States
- Volunteers
- 69 people
- Sponsor
- Auron Therapeutics, Inc.
Myelodysplastic Syndrome
Combination Therapy of SyB C-1101 and Azacytidine in Patients With Myelodysplastic Syndrome
This is a Phase 1 clinical trial to evaluate the tolerability of a combination therapy of SyB C-1101 (rigosertib sodium) and Azacytidine and to determine the recommended dose of SyB C-1101for Phase 2 trial in patients with…
- Trial name
- NCT02783547
- Testing
- SyB C-1101 and Azacytidine
- Where
- 4 sites in Japan
- Sponsor
- SymBio Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria
Dose Escalation of BCX10013 in Participants with Paroxysmal Nocturnal Hemoglobinuria (PNH)
This is a multicenter, open-label, intra-subject, dose escalation study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and therapeutic potential of BCX10013 in participants with PNH.
- Trial name
- NCT06100900
- Testing
- BCX10013
- Where
- 4 sites in Malaysia, South Africa
- Volunteers
- 8 people
- Sponsor
- BioCryst Pharmaceuticals
Myelodysplastic Syndromes(MDS)
Collection of Samples From Patients With MDS
The purpose of this study is to collect information and bone marrow, blood, saliva, cheek cells and skin to be used in the laboratory to assist the sponsor in identifying a new way of treating MDS.
- Trial name
- NCT03072498
- Where
- 2 sites in United States
- Volunteers
- 24 people
- Sponsor
- PersImmune, Inc
AML, MDS
Study of APVO436 in Patients With AML or MDS
The primary objective of the Phase 1 part of the study is to determine the recommended dose of APVO436 administered intravenously to patients with AML or MDS.
- Trial name
- NCT03647800
- Testing
- APVO436
- Where
- 12 sites in United States
- Volunteers
- 136 people
- Sponsor
- Aptevo Research and Development LLC
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 Syndromes
An Efficacy and Safety Study of Luspatercept (ACE-536) for the Treatment of Anemia Due to IPSS-R Very Low, Low or Intermediate Risk Myelodysplastic Syndromes (MDS) in Japanese Subjects Who Are Not Requiring Red Blood Cell Transfusion
The study will be conducted in compliance with the International Council for Harmonisation (ICH) of Technical Requirements for Registration of Pharmaceuticals for Human Use/Good Clinical Practice (GCP) and applicable regulatory…
- Trial name
- NCT03900715
- Testing
- Luspatercept
- Where
- 19 sites in Japan
- Volunteers
- 21 people
- Sponsor
- Celgene
Low- or Intermediate-1-risk Myelodysplastic Syndrome (MDS)
A Pharmacokinetic And Pharmacodynamic Study Of Oral Lenalidomide (Revlimid) In Subjects With Low- Or Intermediate-1-Risk Myelodysplastic Syndromes
The purpose of this study is to assess pharmacokinetic and pharmacodynamic characteristics of oral lenalidomide monotherapy administered to patients with Low- or Intermediate-1-risk Myelodysplastic Syndrome (MDS).
- Trial name
- NCT00910858
- Testing
- Lenalidomide, Recombinant human erythropoietin
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Celgene Corporation
Low Risk Myelodysplastic Syndromes
RVU120 for Treatment of Anemia in Patients With Lower-risk Myelodysplastic Neoplasms
This study will evaluate orally administered RVU120, a novel small molecule Cyclin-dependent Kinase (CDK) 8/19 inhibitor, in terms of erythroid hematologic improvement (HI-E) and safety in participants with lower-risk…
- Trial name
- (MDS) (NCT06243458)
- Testing
- RVU120 (SEL120)
- Where
- 25 sites in France, Germany and 3 more
- Volunteers
- 41 people
- Sponsor
- GCP-Service International West GmbH
Acute Lymphoblastic Leukaemia, Acute Myeloid Leukaemia
Study of AZD3632 Monotherapy or in Combination With Anticancer Agents in Participants With Advanced Haematologic Malignancies With KMT2Ar, NPM1m, or Other Genotypes Associated With HOX Overexpression
The purpose of this study is to understand the safety, tolerability, efficacy, pharmacokinetic (PK), pharmacodynamic (PD), and preliminary efficacy of orally administered AZD3632 in participants with advanced haematologic…
- Conditions
- Acute Lymphoblastic Leukaemia, Acute Myeloid Leukaemia, Higher-risk Myelodysplastic Syndromes
- Trial name
- MOMENTUM (NCT07155226)
- Testing
- AZD3632, Posaconazole
- Where
- 30 sites in United States, Australia and 7 more
- Volunteers
- 85 people
- Sponsor
- AstraZeneca
Paroxysmal Nocturnal Hemoglobinuria
Prospective Observational Study of Long-term Pathogenic Treatment of Elizaria®
This is prospective observational study of long-term pathogenic treatment of Elizaria® in patients with paroxysmal nocturnal hemoglobinuria.
- Trial name
- NAP (NCT04671810)
- Testing
- Elizaria®
- Where
- 8 sites in Russia
- Volunteers
- 44 people
- Sponsor
- AO GENERIUM
Leukemia, Leukemia, Myeloid
Study of PDR001 and/or MBG453 in Combination With Decitabine in Patients With AML or High Risk MDS
To characterize the safety and tolerability of 1) MBG453 as a single agent or in combination with PDR001 or 2) PDR001 and/or MBG453 in combination with decitabine or azacitidine in AML and intermediate or high- risk MDS patients…
- Conditions
- Leukemia, Leukemia, Myeloid, Leukemia, Myeloid, Acute, Myelodysplastic Syndromes
- Trial name
- NCT03066648
- Testing
- Decitabine, PDR001, MBG453
- Where
- 11 sites in United States, Australia and 6 more
- Volunteers
- 241 people
- Sponsor
- Novartis Pharmaceuticals