Clinical trials: Myelodysplastic Neoplasm
Hemoglobinuria, Paroxysmal
The Paroxysmal Nocturnal Hemoglobinuria Early Access Treatment Protocol
The primary objective is to provide access to eculizumab for PNH patient pending commercial availability.
- Trial name
- EMBRACE (NCT00438789)
- Testing
- eculizumab
- Where
- United States
- Sponsor
- Alexion Pharmaceuticals, Inc.
Myelodysplastic Syndrome
Study to Determine the Safety and Preliminary Efficacy of CC-1088 in the Treatment of Myelodysplastic Syndromes
The primary objective of the study is to assess the safety of CC-1088 to patients with myelodysplastic syndromes (MDS).
- Trial name
- NCT00045786
- Testing
- CC-1088
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Celgene
Paroxysmal Nocturnal Hemoglobinuria
Danicopan PMS in Korea
As part of a post-approval commitment, the Korean health authority requests a study to characterize safety and effectiveness in patients who are treated with Danicopan as an add-on to ravulizumab or eculizumab in normal clinical…
- Trial name
- NCT07457151
- Where
- 4 sites in South Korea
- Volunteers
- 27 people
- Sponsor
- AstraZeneca
Myelodysplastic Syndrome (MDS)
Study Of Venetoclax Tablet With Intravenous or Subcutaneous Azacitidine to Assess Change in Disease Activity In Adult Participants With Newly Diagnosed Higher-Risk Myelodysplastic Syndrome
Myelodysplastic Syndrome (MDS) is a group of disorders that gradually affect the ability of a person's bone marrow (semi-liquid tissue present in many bones like backbones) to produce normal blood cells.
- Trial name
- Verona (NCT04401748)
- Testing
- Venetoclax, Azacitidine
- Where
- 220 sites in United States, Australia and 21 more
- Volunteers
- 531 people
- Sponsor
- AbbVie
Myelodysplastic Syndrome
A Study of JNJ-30979754 (Decitabine) in Patients With Myelodysplastic Syndrome
The purpose of this study is to to determine the recommended dose level of JNJ-30979754 (decitabine) as well as to assess the safety and effectiveness in patients with Myelodysplastic Syndrome (MDS).
- Trial name
- NCT00796003
- Testing
- JNJ-30979754 15 mg/m2, JNJ-30979754 20 mg/m2
- Where
- 7 sites in Japan
- Volunteers
- 39 people
- Sponsor
- Janssen Pharmaceutical K.K.
Myelodysplastic Syndromes, Acute Myeloid Leukemia
A Multi-phase Study of ASTX030 (Azacitidine and Cedazuridine) in Myeloid Neoplasm Alone or in Combination With Venetoclax in AML (AZTOUND Study)
Study ASTX030-01 is a multi-phase study comprising of Phases 1-3 Monotherapy arms, and Phase 1 and Phase 2 Combination Therapy arms.
- Conditions
- Myelodysplastic Syndromes, Acute Myeloid Leukemia, Myelodysplastic Syndrome/Neoplasm, Chronic Myelomonocytic Leukemia
- Trial name
- NCT04256317
- Testing
- Azacitidine, ASTX030 (cedazuridine + azacitidine), Cedazuridine
- Where
- 73 sites in United States, Canada and 8 more
- Volunteers
- 316 people
- Sponsor
- Taiho Oncology, Inc.
Paroxysmal Nocturnal Hemoglobinuria
A Phase III Study of NTQ5082 Capsules in the Treatment of Paroxysmal Nocturnal Hemoglobinuria Patients
NTQ5082 capsules are a small molecule CFB inhibitor. This study is a multicenter, randomized, open-label, active-controlled Phase III clinical trial designed to evaluate the efficacy and safety of NTQ5082 capsules in the…
- Trial name
- NCT07177859
- Testing
- NTQ5082 capsules 200 mg, Eculizumab Injection
- Where
- China
- Volunteers
- 78 people
- Sponsor
- Nanjing Chia-tai Tianqing Pharmaceutical
Higher-risk Myelodysplastic Syndromes
A Study of AK117 in Combination With Azacitidine in Patients With Myelodysplastic Syndromes
This is a Phase 2 randomized, double-blind, placebo-controlled, multicenter study evaluating the efficacy and safety of AK117 or placebo, combined with azacitidine in patients with newly diagnosed higher-risk myelodysplastic…
- Trial name
- NCT06196203
- Testing
- AK117, Azacitidine
- Where
- 15 sites in United States, China
- Volunteers
- 90 people
- Sponsor
- Akeso
Myelodysplastic Syndrome
Phase 2 Study of Pracinostat With Azacitidine in Patients With Previously Untreated Myelodysplastic Syndrome
The purpose of this randomized, double-blind, placebo-controlled study is to determine the safety and efficacy of pracinostat compared to placebo when combined with azacitidine, and FDA approved treatment for Myelodysplastic…
- Trial name
- NCT01873703
- Testing
- pracinostat, Azacitidine
- Where
- 24 sites in United States
- Volunteers
- 102 people
- Sponsor
- Helsinn Healthcare SA
Myelodysplastic Syndromes
A Study to Assess Luspatercept in Lower-risk Myelodysplastic Syndrome Participants
The purpose of this study is to evaluate the efficacy and safety of Luspatercept when administered at the maximum approved dose in low-risk Myelodysplastic Syndrome participants who require red blood cell transfusions.
- Trial name
- MAXILUS (NCT06045689)
- Testing
- Luspatercept
- Where
- 52 sites in United States, Belgium and 7 more
- Volunteers
- 106 people
- Sponsor
- Bristol-Myers Squibb
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
REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate Its Long Term Safety, Efficacy and Tolerability.
The primary objective of the study is to evaluate the long-term safety, tolerability, and effect on intravascular hemolysis of REGN3918 in patients with paroxysmal nocturnal hemoglobinuria (PNH).
- Trial name
- NCT04162470
- Testing
- REGN3918
- Where
- 14 sites in Hong Kong, Hungary and 4 more
- Volunteers
- 24 people
- Sponsor
- Regeneron Pharmaceuticals
AML/MDS
Study of Mana 312 (Multi Tumor-Associated Antigen T Cells) in Adults With AML/MDS After HSCT
This is a Phase 1, open-label, non-randomized, single and multiple dose escalation study designed to evaluate the safety and preliminary efficacy of administering Mana 312 to subjects with AML/MDS after allogeneic HSCT.
- Trial name
- NCT04679194
- Testing
- Mana 312
- Where
- 8 sites in United States
- Volunteers
- 11 people
- Sponsor
- Mana Therapeutics
MDS, AML
Non-interventional Study With Azacitidin (Vidaza®)
The purpose of this non-interventional study is to collect data on the efficiency and safety of Azacitidin in the routine application.
- Conditions
- MDS, AML, CMMOL
- Trial name
- Piaza (NCT01192945)
- Where
- Germany
- Volunteers
- 150 people
- Sponsor
- iOMEDICO AG
Myelodysplastic Syndromes (MDS), Chronic Myelomonocytic Leukemia (CMML)
Safety, Pharmacokinetics, and Pharmacodynamics of Oral Azacitidine in Subjects With Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia and Acute Myelogenous Leukemia
The purpose of this study is to determine whether a tablet form of azacitidine that taken by mouth is safe. This Phase I study will also look at different doses and different treatment schedules in order to better understand the…
- Conditions
- Myelodysplastic Syndromes (MDS), Chronic Myelomonocytic Leukemia (CMML), Acute Myelogenous Leukemia (AML)
- Trial name
- NCT00528983
- Testing
- Subcutaneous (SC) Azacitidine, Oral Azacitidine
- Where
- 18 sites in United States
- Volunteers
- 133 people
- Sponsor
- Celgene
Myelodysplastic Syndromes, Higher Risk Myelodysplastic Syndromes
Study of REM-422 in Patients With AML or Higher Risk MDS
The goal of this study is to determine the safety and antitumor effects of REM-422, a MYB mRNA degrader, in people with Higher Risk MDS and relapsed/refractory AML
- Conditions
- Myelodysplastic Syndromes, Higher Risk Myelodysplastic Syndromes, Acute Myeloid Leukemia, Acute Myeloid Leukemia Refractory
- Trial name
- NCT06297941
- Testing
- REM-422
- Where
- 9 sites in United States, France
- Volunteers
- 100 people
- Sponsor
- Remix Therapeutics
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
Acute Myeloid Leukemia Refractory, Acute Myeloid Leukemia, in Relapse
AB8939 in Patients With Relapsed/Refractory Acute Myeloid Leukemia
The primary objective is to define the safety and tolerability of AB8939 in patients with AML by determining the dose-limiting toxicities, the maximum tolerated dose, and the recommended dose for dose expansion study.
- Conditions
- Acute Myeloid Leukemia Refractory, Acute Myeloid Leukemia, in Relapse, Myelodysplastic Syndrome Acute Myeloid Leukemia
- Trial name
- NCT05211570
- Testing
- AB8939, Venetoclax
- Where
- 10 sites in United States, France and 2 more
- Volunteers
- 78 people
- Sponsor
- AB Science
Leukemia, Myeloid, Acute
A Phase 1, Open Label Study of Intravenous GSK3745417 to Evaluate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Determine RP2D & Schedule in Participants With Relapsed or Refractory Myeloid Malignancies Including AML and HR MDS
This is a Phase 1, open label, two-part study to determine recommended phase 2 dose (RP2D) and schedule of GSK3745417 administration in participants with relapsed/refractory AML or HR-MDS.
- Trial name
- NCT05424380
- Testing
- GSK3745417
- Where
- 10 sites in Canada, Germany and 3 more
- Volunteers
- 18 people
- Sponsor
- GlaxoSmithKline
Life Threatening Diseases
Phase II Study of Imatinib Mesylate in Patients With Life Threatening Malignant Rare Diseases
Exploratory study to examine the effect(s) of Imatinib mesylate treatment on life threatening rare diseases with known associations to one or more Imatinib mesylate -sensitive tyrosine kinases, and to identify the contribution of…
- Trial name
- NCT00154388
- Testing
- Imatinib mesylate
- Volunteers
- 185 people
- Sponsor
- Novartis Pharmaceuticals