Clinical trials: Myelodysplastic Disorders
Acute Lymphoblastic Leukemia, Acute Myelogenous Leukemia
Safety Study of Gene Modified Donor T-cells Following Partially Mismatched Stem Cell Transplant
This study will evaluate patients with blood cell cancers who are going to have an allogeneic (donor) blood stem cell transplant from a partially matched relative.
- Conditions
- Acute Lymphoblastic Leukemia, Acute Myelogenous Leukemia, Lymphoma, Myelodysplastic Syndromes
- Trial name
- NCT01744223
- Testing
- BPX-501 dose 1, Rimiducid, BPX-501 dose 2
- Where
- 8 sites in United States
- Volunteers
- 36 people
- Sponsor
- Bellicum Pharmaceuticals
Myelodysplastic Syndromes
Phase 1-2 Study of Low Dose ASTX727 (ASTX727 LD) in Lower Risk MDS
Multicenter, open-label study of various ASTX727 LD doses and schedules to assess safety, pharmacodynamics, pharmacokinetics, and hematologic response in subjects with International Prognostic Scoring System (IPSS) risk category…
- Trial name
- NCT03502668
- Testing
- ASTX727 LD, ASTX727 SD
- Where
- 29 sites in United States, Belgium and 4 more
- Volunteers
- 160 people
- Sponsor
- Taiho Oncology, Inc.
AML, Adult
A Study of APG-2575 in Combination With Azacitidine in Patients With Acute Myeloid Leukemia (AML)
This is a Phase Ib/II, open-label, multi-center study evaluating the safety, tolerability, efficacy and PK of APG-2575 in combination with Azacitidine in the patients with AML/MPAL or MDS/CMML.
- Trial name
- NCT04964518
- Testing
- APG 2575 ramp up arm
- Where
- 9 sites in United States, Australia
- Volunteers
- 24 people
- Sponsor
- Ascentage Pharma Group Inc.
Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia
Transplant-Related Mortality in Patients Undergoing a Peripheral Blood Stem Cell Transplantation or an Umbilical Cord Blood Transplantation
Study CR-AIR-006 is a part of the ATIR clinical development plan and will provide control data for patients treated with ATIR in clinical studies (e.g. study CR-AIR-007).
- Conditions
- Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Myelodysplastic Syndrome
- Trial name
- NCT02188290
- Where
- 9 sites in United States, Belgium and 4 more
- Volunteers
- 178 people
- Sponsor
- Kiadis Pharma
Leukemia, Myeloid, Acute, Myelodysplastic Syndromes
Safety Study of Oral Azacitidine (CC-486) as Maintenance Therapy After Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) in Participants With Acute Myeloid Leukemia (AML) or Myelodysplastic Syndromes (MDS).
The purpose of the study is to determine the maximal tolerated dose and schedule of CC-486, known as oral azacitidine, in patients with AML or MDS after allogeneic hematopoetic stem cell transplant (HSCT).
- Trial name
- NCT01835587
- Testing
- CC-486
- Where
- 5 sites in United States, United Kingdom
- Volunteers
- 31 people
- Sponsor
- Celgene
Myelodysplastic Syndromes
A Study of Darbepoetin Alfa in Anemic Subjects With Low Risk Myelodysplastic Syndrome
The purpose of this study is to assess the effect of 13 weeks of darbepoetin alfa treatment on erythroid response in anemic subjects with low risk Myelodysplastic Syndrome (MDS).
- Trial name
- NCT00095264
- Testing
- Darbepoetin Alfa
- Volunteers
- 209 people
- Sponsor
- Amgen
Leukemia, Myeloid, Acute, Myelodysplastic Syndromes
A Dose-finding Study of CC-90009 in Subjects With Relapsed or Refractory Acute Myeloid Leukemia or Relapsed or Refractory Higher-risk Myelodysplastic Syndromes
CC-90009-AML-001 is a phase 1, open-label, dose escalation and expansion, study in subjects with relapsed or refractory acute myeloid leukemia and relapsed or refractory higher-risk myelodysplastic syndrome.
- Trial name
- NCT02848001
- Testing
- CC-90009
- Where
- 20 sites in United States, Canada and 4 more
- Volunteers
- 101 people
- Sponsor
- Celgene
Myelodysplastic Syndromes, Myelogenous Leukemia, Acute
A Study of Vismodegib in Patients With Relapsed/Refractory Acute Myelogenous Leukemia and Relapsed Refractory High-Risk Myelodysplastic Syndrome
This study will assess the safety and efficacy of vismodegib in patients with relapsed/refractory acute myelogenous leukemia (AML) and relapsed/refractory high-risk myelodysplastic syndrome (MDS).
- Trial name
- NCT01880437
- Testing
- cytarabine, vismodegib
- Where
- 14 sites in United States, Canada and 1 more
- Volunteers
- 38 people
- Sponsor
- Hoffmann-La Roche
Myelodysplastic Syndrome
An Effectiveness and Safety Study of Decitabine in Patients With Myelodysplastic Syndrome
The purpose of this study is to evaluate the effectiveness and safety of decitabine in the treatment of myelodysplastic syndrome (name of a group of conditions that occur when the blood-forming cells in the bone marrow are…
- Trial name
- NCT01751867
- Testing
- Decitabine at 15 mg/m2, Decitabine at 20 mg/m2
- Where
- 8 sites in China
- Volunteers
- 135 people
- Sponsor
- Xian-Janssen Pharmaceutical Ltd.
Myelodysplastic Syndromes
Study of Efficacy and Safety of Eltrombopag in Lower-risk MDS Patients With Platelet Transfusion Dependence
This study is designed to evaluate the efficacy and safety of eltrombopag monotherapy in Japanese adult patients with platelet transfusion-dependent lower-risk Myelodysplastic syndromes (LR-MDS).
- Trial name
- NCT04797000
- Testing
- Eltrombopag
- Where
- 20 sites in Japan
- Volunteers
- 36 people
- Sponsor
- Novartis Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria
Long-term Safety and Tolerability of HRS-5965 in Patients With Paroxysmal Nocturnal Hemoglobinuria
This is a multicenter, single-arm, open-label study. Patients with Paroxysmal Nocturnal Hemoglobinuria who had previously received and completed the HRS-5965 study well included.
- Trial name
- NCT06238544
- Testing
- HRS-5965 tablets, HRS-5965 Capsules
- Where
- 2 sites in China
- Volunteers
- 132 people
- Sponsor
- Chengdu Suncadia Medicine Co., Ltd.
Paroxysmal Nocturnal Hemoglobinuria
Long-term Safety of BCX9930 in Participants With Paroxysmal Nocturnal Hemoglobinuria
This study was designed to provide continued access to BCX9930 for participants with Paroxysmal Nocturnal Hemoglobinuria (PNH) who had benefited from treatment with BCX9930 in another BioCryst-sponsored study for PNH who, in the…
- Trial name
- NCT05741346
- Testing
- BCX9930
- Where
- 11 sites in France, Hungary and 5 more
- Volunteers
- 28 people
- Sponsor
- BioCryst Pharmaceuticals
Myelodysplastic Syndromes, Leukemia, Myelomonocytic, Chronic
Roll-over Study for Patients Who Have Completed a Prior Novartis-sponsored Sabatolimab (MBG453) Study and Are Judged by the Investigator to Benefit From Continued Treatment With Sabatolimab.
This study is intended to collect safety data from participants who completed the parent protocols but are still benefiting from study treatment.
- Trial name
- NCT05201066
- Testing
- decitabine, spartalizumab, sabatolimab
- Where
- 24 sites in United States, Australia and 13 more
- Volunteers
- 33 people
- Sponsor
- Novartis Pharmaceuticals
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
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Study of Efficacy and Safety of Twice Daily Oral Iptacopan (LNP023) in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy
The purpose of this Phase 3 study was to determine whether iptacopan is efficacious and safe for the treatment of Paroxysmal nocturnal hemoglobinuria (PNH) patients who were naïve to complement inhibitor therapy.
- Trial name
- APPOINT-PNH (NCT04820530)
- Testing
- Iptacopan (LNP023)
- Where
- 12 sites in China, France and 6 more
- Volunteers
- 40 people
- Sponsor
- Novartis Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria
A Study to Test if SAR443809 is Tolerated and Safe When Taken as a Single Dose in Healthy Adults
Primary objective The tolerability and safety of SAR443809 Secondary The PK parameters of SAR443809 The PD activity of SAR443809 The immunogenicity of SAR443809
- Trial name
- NCT06326814
- Testing
- Humanized anti-Factor Bb monoclonal antibody
- Where
- 2 sites in United States
- Volunteers
- 54 people, healthy volunteers welcome
- Sponsor
- Sanofi
Acute Myelogenous Leukemia, Chronic Myelogenous Leukemia
A Phase 1/2 Study of SB1518 for the Treatment of Advanced Myeloid Malignancies
This study consists of two phases: the first portion of the study is a Phase 1 dose escalation study to determine the maximum tolerated dose and the dose limiting toxicities of SB1518 when given as a single agent orally once…
- Conditions
- Acute Myelogenous Leukemia, Chronic Myelogenous Leukemia, Chronic Myelomonocytic Leukemia, Myelodysplastic Syndromes
- Trial name
- NCT00719836
- Testing
- SB1518
- Where
- 3 sites in United States
- Volunteers
- 76 people
- Sponsor
- S*BIO
Myelodysplastic Syndrome
Three Dosing Schedules of Oral Rigosertib in MDS Patients
This study will compare the dosing regimen of oral rigosertib, which has been used in other studies of lower risk Myelodysplastic Syndrome (MDS), with 2 new dosing regimens to determine if one of the new regimens gives improved…
- Trial name
- NCT02075034
- Testing
- rigosertib
- Where
- United States
- Sponsor
- Traws Pharma, Inc.
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients
The purpose of this open-label, single arm, multicenter, phase 3 study is to assess the pharmacokinetics of iptacopan in pediatric patients and to assess whether iptacopan is safe and well tolerated when used for the treatment of…
- Trial name
- NCT06934967
- Testing
- LNP023
- Where
- 15 sites in United States, Brazil and 4 more
- Volunteers
- 12 people
- Sponsor
- Novartis Pharmaceuticals
Myelodysplastic Syndrome, Iron Overload
Study of Deferasirox for Treatment of Transfusional Iron Overload in Myelodysplastic Patients
The purpose of this trial is to examine the safety and efficacy of deferasirox in patients with Myelodysplastic Syndrome (MDS) and chronic iron overload from blood transfusions.
- Trial name
- NCT00110266
- Testing
- Deferasirox
- Where
- 48 sites in United States, Canada
- Volunteers
- 176 people
- Sponsor
- Novartis Pharmaceuticals