Clinical trials: MDS
Beta-thalassemia, Myelodysplastic Syndromes
Study of Deferasirox in Iron Overload From Beta-thalassemia Unable to be Treated With Deferoxamine or Chronic Anemias
The purpose of this study is to determine the effects of the oral iron chelator Deferasirox on liver iron content after one year of treatment in patients with iron overload from repeated blood transfusions.
- Conditions
- Beta-thalassemia, Myelodysplastic Syndromes, Fanconi Syndrome, Anemia, Diamond-Blackfan
- Trial name
- NCT00061763
- Testing
- Deferasirox
- Where
- 6 sites in United States
- Volunteers
- 175 people
- Sponsor
- Novartis Pharmaceuticals
High-risk Myelodysplastic Syndrome, Chronic Myelomonocytic Leukemia (CMML)
A Study of CBX-250 in Participants With Relapsed or Refractory Myeloid Leukemias
Study CBX-250-001 is a Phase 1, open-label, dose-escalation study of CBX-250 in participants with relapsed/refractory AML, HR-MDS, CMML, and CML. Participants aged ≥ 12 years are planned to be enrolled.
- Conditions
- High-risk Myelodysplastic Syndrome, Chronic Myelomonocytic Leukemia (CMML), AML - Acute Myeloid Leukemia, Chronic Myeloid Leukemia
- Trial name
- CROSSCHECK-001 (NCT06994676)
- Testing
- CBX-250
- Where
- 11 sites in United States
- Volunteers
- 72 people
- Sponsor
- Crossbow Therapeutics, Inc.
Paroxysmal Nocturnal Hemoglobinuria
Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients
A study to assess the safety and efficacy of eculizumab in Japanese patients with hemolytic PNH.
- Trial name
- NCT01192399
- Testing
- Eculizumab
- Volunteers
- 29 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
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 (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
Paroxysmal Nocturnal Hemoglobinuria
A Real-world, Multi-center, Prospective, Observational Study for PNH in China
As a rare disease listed in the First Catalogue of Rare Diseases in China (National Health Commission of the People's Republic of China, 2019), PNH is poorly studied in China subse-quently leading to the inadequate elucidation of…
- Trial name
- ReWoPNH (NCT06154512)
- Where
- 40 sites in China
- Volunteers
- 724 people
- Sponsor
- AstraZeneca
Paroxysmal Nocturnal Hemoglobinuria
Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry
This study is a collection of data to evaluate safety and characterize progression of Paroxysmal Nocturnal Hemoglobinuria (PNH).
- Trial name
- NCT01374360
- Where
- United States
- Volunteers
- 6,061 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Acute Myeloid Leukemia, in Relapse, Acute Myeloid Leukemia, in Relapsed or Refractory
A Long-term Follow-up Study of Patients With ARD103 CAR-T Cell Therapies
This study will evaluate the long-term safety of ARD103 cellular therapies
- Conditions
- Acute Myeloid Leukemia, in Relapse, Acute Myeloid Leukemia, in Relapsed or Refractory, MDS (Myelodysplastic Syndrome)
- Trial name
- NCT06906380
- Testing
- Non interventional study
- Where
- 3 sites in United States
- Volunteers
- 49 people
- Sponsor
- ARCE Therapeutics, Inc.
Paroxysmal Nocturnal Hemoglobinuria
A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibition
This study will enroll participants aged 12 years or older with a body weight ≥ 40 kilograms (kg) diagnosed with PNH who have not been previously treated with complement inhibitor therapy.
- Trial name
- COMMODORE 3 (NCT04654468)
- Testing
- Crovalimab
- Where
- 5 sites in China
- Volunteers
- 51 people
- Sponsor
- Hoffmann-La Roche
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
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
Hematological Malignancies, Acute Lymphoblastic Leukemia (ALL)
Stem Cell Transplantation With NiCord® (Omidubicel) vs Standard UCB in Patients With Leukemia, Lymphoma, and MDS
This study is an open-label, controlled, multicenter, international, Phase III, randomized study of transplantation of NiCord® versus transplantation of one or two unmanipulated, unrelated cord blood units in patients with acute…
- Conditions
- Hematological Malignancies, Acute Lymphoblastic Leukemia (ALL), Acute Myelogenous Leukemia (AML), Chronic Myelogenous Leukemia (CML)
- Trial name
- NCT02730299
- Testing
- NiCord® (omidubicel), Cord Blood Unit
- Where
- 52 sites in United States, Brazil and 8 more
- Volunteers
- 125 people
- Sponsor
- Gamida Cell ltd
Paroxysmal Nocturnal Hemoglobinuria
A Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors
A study designed to evaluate the safety of crovalimab with eculizumab in participants with PNH currently treated with complement inhibitors. This study will enroll approximately 190 participants.
- Trial name
- COMMODORE 1 (NCT04432584)
- Testing
- Crovalimab, Eculizumab
- Where
- 80 sites in United States, Belgium and 22 more
- Volunteers
- 190 people
- Sponsor
- Hoffmann-La Roche
Chronic Anemia, Transfusional Hemosiderosis
Efficacy and Safety of Deferasirox in Patients With Chronic Anemia and Transfusional Hemosiderosis
The overall purpose of this trial is to further evaluate the efficacy and safety of deferasirox, dosed initially according to the transfusional iron intake, in patients with transfusion dependant anemia related to disorders other…
- Trial name
- NCT00631163
- Testing
- Deferasirox (ICL670)
- Where
- 31 sites in Japan, Poland and 3 more
- Volunteers
- 102 people
- Sponsor
- Novartis Pharmaceuticals
Advanced MDS, Acute Myeloid Leukemia
A Study of ARRY-520 in Patients With Advanced Myeloid Leukemia
This is a 2-phase study during which patients with select myeloid leukemias or advanced myelodysplastic syndrome (MDS), who have failed, refused or are not eligible for standard treatment, will receive investigational study drug…
- Trial name
- NCT00637052
- Testing
- ARRY-520, KSP(Eg5) inhibitor; intravenous
- Where
- 2 sites in United States
- Volunteers
- 36 people
- Sponsor
- Pfizer
Advanced Hematologic Malignancy, Relapsed Acute Myeloid Leukemia
FHD-286 as Monotherapy or Combination Therapy in Subjects With Advanced Hematologic Malignancies
This Phase 1, multicenter, open-label, dose escalation study is designed to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary clinical activity of FHD-286 administered orally as…
- Conditions
- Advanced Hematologic Malignancy, Relapsed Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia, Relapsed Myelodysplastic Syndromes
- Trial name
- NCT04891757
- Testing
- FHD-286, Low Dose Cytarabine, Decitabine
- Where
- 5 sites in United States
- Volunteers
- 144 people
- Sponsor
- Foghorn Therapeutics Inc.
Acute Myeloid Leukemia, Myelodysplastic Syndromes
A Phase 1 Study of Orca-Q in Recipients Undergoing Allogeneic Transplantation for Hematologic Malignancies
This study will evaluate the safety, tolerability, and efficacy of engineered donor grafts ("OrcaGraft"/"Orca-Q") in participants undergoing allogeneic hematopoietic cell transplant (alloHCT) transplantation for hematologic…
- Conditions
- Acute Myeloid Leukemia, Myelodysplastic Syndromes, Mixed Phenotype Acute Leukemia, Acute Lymphoblastic Leukemia
- Trial name
- NCT03802695
- Testing
- OrcaGraft (Orca-Q)
- Where
- 12 sites in United States
- Volunteers
- 300 people
- Sponsor
- Orca Biosystems, Inc.
Leukemia, Myelodysplastic Syndromes
Chemotherapy in Treating Patients Who Have Hematologic Cancer
RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. PURPOSE: Phase I trial to study the effectiveness of DX-8951f in treating patients who have hematologic…
- Trial name
- NCT00004047
- Testing
- exatecan mesylate
- Where
- United States
- Sponsor
- Daiichi Sankyo
Myelodysplastic Syndromes
AVIDA The Vidaza® (Azacitidine) Patient Registry
This registry is a prospective, multi-center, observational study compiling data on the natural history and management of patients receiving Vidaza, and will provide unique insights into the management of myelodysplastic…
- Trial name
- AVIDA (NCT00481273)
- Testing
- Azacitidine
- Where
- United States
- Volunteers
- 479 people
- Sponsor
- Celgene
Graft Versus Host Disease, Leukemia
Treatment of Bone Marrow to Prevent Graft-Versus-Host Disease in Patients With Acute or Chronic Leukemia Undergoing Bone Marrow Transplantation
RATIONALE: Bone marrow that has been treated to remove certain white blood cells may reduce the chance of developing graft-versus-host disease following bone marrow transplantation.
- Conditions
- Graft Versus Host Disease, Leukemia, Myelodysplastic Syndromes
- Trial name
- NCT00004255
- Testing
- anti-thymocyte globulin, filgrastim, cyclophosphamide
- Where
- 15 sites in United States
- Sponsor
- Chimeric Therapies