Clinical trials: Myelodysplastic Syndrome (MDS)
Paroxysmal Nocturnal Hemoglobinuria
Ravulizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With High-Dose Eculizumab
The primary purpose of this study is to assess the safety, efficacy, pharmacokinetics, and pharmacodynamics of ravulizumab in participants who are prescribed and are receiving a higher than approved dose of eculizumab to treat…
- Trial name
- NCT04320602
- Testing
- Eculizumab, Ravulizumab
- Where
- 4 sites in United Kingdom
- Volunteers
- 18 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
VEXAS, VEXAS Syndrome
A Study to Assess the Effectiveness and Safety of Pacritinib in Patients With VEXAS Syndrome (PAXIS)
This trial is to assess the effectiveness and safety of pacritinib in patients with VEXAS (i.e., Vacuoles in myeloid progenitors, E1 ubiquitin-activating enzyme, X-linked, autoinflammatory manifestations, and somatic) syndrome.
- Trial name
- NCT06782373
- Testing
- Pacritinib
- Where
- 39 sites in United States, Canada and 6 more
- Volunteers
- 156 people
- Sponsor
- Swedish Orphan Biovitrum
Myelodysplastic Syndromes
Personalized Adoptive Cellular Therapy Targeting MDS Stem Cell Neoantigens (PACTN)
This study will evaluate the safety of autologous T cells that have been immunized ex vivo with patient-specific MDS stem cell neoantigens in patients with MDS.
- Trial name
- NCT03258359
- Testing
- PACTN
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- PersImmune, Inc
Chronic Myelogenous Leukemia in Blast Crisis, Lymphocytic Leukemia, B Cell, Acute
MK0457 in Patients With Leukemia (0457-003)
In this study participants with relapsed/refractory leukemia will be given MK-0457 in sequential cohorts and with varying treatment duration to determine the maximum tolerated dose (MTD) for MK-0457.
- Conditions
- Chronic Myelogenous Leukemia in Blast Crisis, Lymphocytic Leukemia, B Cell, Acute, Myelodysplastic Syndromes, Myelogenous Leukemia, Chronic
- Trial name
- NCT00111683
- Testing
- MK0457
- Volunteers
- 28 people
- Sponsor
- Merck Sharp & Dohme LLC
Acute Leukemia, Myelodysplastic Syndromes
A Study of ONO-7475 in Patients With Acute Leukemias
[Updated]: To assess the safety and tolerability of ONO-7475 monotherapy in patients with relapsed or refractory acute myeloid leukemia or relapsed or refractory myelodysplastic syndromes and to assess: i) safety and tolerability…
- Trial name
- NCT03176277
- Testing
- ONO-7475 3mg once daily, ONO-7475 6mg once daily, ONO-7475 10mg once daily
- Where
- 16 sites in United States
- Volunteers
- 42 people
- Sponsor
- Ono Pharmaceutical Co., Ltd.
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant
This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially…
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndrome
- Trial name
- NCT02065869
- Testing
- BPX-501 T cells, Rimiducid
- Where
- 4 sites in Italy, United Kingdom
- Volunteers
- 187 people
- Sponsor
- Bellicum Pharmaceuticals
Bone Marrow Suppression
Clinical Study on the Safety and Efficacy of Umbilical Cord Blood Infusion in Treating Bone Marrow Suppression .
The clinical study aims to evaluate the safety and efficacy of umbilical cord blood transfusion in treating bone marrow suppression after chemotherapy in elderly patients with MDS and AML
- Trial name
- NCT07201233
- Testing
- Umbilical cord blood
- Volunteers
- 30 people
- Sponsor
- Shandong Qilu Stem Cells Engineering Co., Ltd.
Myelodysplastic Syndrome, MDS
Controlled Study of Rigosertib Versus Physician's Choice of Treatment in MDS Patients After Failure of an HMA
The study's primary objective [in a population of patients with MDS after failure of treatment with azacitidine (AZA) or decitabine (DAC)], is to compare the overall survival (OS) of patients in the rigosertib group vs the…
- Conditions
- Myelodysplastic Syndrome, MDS, Refractory Anemia With Excess Blasts, RAEB
- Trial name
- INSPIRE (NCT02562443)
- Testing
- rigosertib, Any approved or standard-of-care therapy, best supportive care (BSC)
- Where
- 158 sites in United States, Australia and 20 more
- Volunteers
- 372 people
- Sponsor
- Traws Pharma, Inc.
Hematologic Malignancy, Acute Myeloid Leukemia
A Trial to Evaluate the Potential Impact of Renal Impairment on the Pharmacokinetics and Safety of CPX-351
This study evaluates the pharmacokinetics and safety of CPX-351 in patients with moderate or severe renal impairment.
- Conditions
- Hematologic Malignancy, Acute Myeloid Leukemia, Acute Lymphocytic Leukemia, Acute Lymphoblastic Leukemia
- Trial name
- NCT03555955
- Testing
- CPX-351
- Where
- 13 sites in United States, Canada
- Volunteers
- 21 people
- Sponsor
- Jazz Pharmaceuticals
Acute Myeloid Leukemia, Adult, Myelodysplastic Syndromes
First-in-human Study Aiming to Characterize the Safety, Tolerability, Pharmacokinetic and Preliminary Signs of Activity of ABD-3001 in Refractory or Relapsed AML and High Risk MDS Adult Patients
This First In Human (FIH) study is a prospective, open-label, multicenter, Phase 1 study, with a dose escalation design, followed by an optimized design.
- Trial name
- ODYSSEY (NCT05601726)
- Testing
- ABD-3001
- Where
- 3 sites in France
- Volunteers
- 36 people
- Sponsor
- Advanced BioDesign
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, Myelodysplastic Syndrome
A Biomarker-Directed Phase 2 Trial of Tamibarotene (SY-1425) in Participants With Acute Myeloid Leukemia or Myelodysplastic Syndrome
The purpose of this study is to determine the activity of tamibarotene in participants with relapsed/refractory (R/R) AML (administered as a monotherapy or in combination with azacitidine), R/R higher-risk MDS (HR-MDS)…
- Trial name
- NCT02807558
- Testing
- Tamibarotene, Azacitidine, Daratumumab
- Where
- 26 sites in United States, France
- Volunteers
- 155 people
- Sponsor
- Syros Pharmaceuticals
Leukemia, Myeloid, Acute, Myelodysplastic Syndromes
Study to Evaluate Safety and Tolerability of CC-91633 (BMS-986397) in Participants With Relapsed or Refractory Acute Myeloid Leukemia or Relapsed or Refractory Higher-Risk Myelodysplastic Syndromes
Study CC-91633-AML-001 is a Phase 1, open-label, dose escalation and expansion, first-in-human (FIH) clinical study of CC-91633 (BMS-986397) in participants with relapsed or refractory acute myeloid leukemia (R/R AML) or in…
- Trial name
- NCT04951778
- Testing
- CC-91633
- Where
- 9 sites in United States, Spain
- Volunteers
- 56 people
- Sponsor
- Celgene
Acute Myeloid Leukemia, Myelodysplastic Syndromes
Expanded Access Program (EAP) for Galinpepimut-S (GPS) in Patients Diagnosed With AML or MDS
Single patient expanded access program to provide galinpepimut-S for eligible patients with AML or MDS who have no other treatment option.
- Trial name
- NCT05593185
- Testing
- Galinpepimut-S
- Sponsor
- Sellas Life Sciences Group
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
Advanced Hematologic Malignancies
Oral Histone Deacetylase Inhibitor 4SC-202 in Patients With Advanced Hematologic Malignancies (TOPAS)
The purpose of this study is to determine the Maximum Tolerated Dose, Dose Limiting Toxicities and optimal dosing schedule of 4SC-202 investigating its safety, tolerability and pharmacokinetics.
- Trial name
- NCT01344707
- Testing
- 4SC-202
- Where
- 3 sites in Germany
- Volunteers
- 36 people
- Sponsor
- 4SC AG
Myelodysplastic Syndrome
APG101 in Myelodysplastic Syndrome
It has been shown in preclinical experiments with bone marrow from patients with myelodysplastic syndrome that APG101 rescues erythrocytes from premature cell death.
- Trial name
- APG101 in MDS (NCT01736436)
- Testing
- Treatment with APG101, Bone marrow collection, Blood drawings
- Where
- 2 sites in Germany
- Volunteers
- 20 people
- Sponsor
- Apogenix GmbH
Leukemia, Myelodysplastic Syndrome(MDS)
A Study to Investigate Fadraciclib (CYC065), in Subjects With Leukemia or Myelodysplastic Syndrome (MDS)
This is a 2-part, phase 1/2, open-label, multicenter study designed to evaluate the safety and efficacy of fadraciclib (formerly CYC065) administered orally BID.
- Trial name
- NCT05168904
- Testing
- fadraciclib
- Where
- 2 sites in United States
- Volunteers
- 210 people
- Sponsor
- Cyclacel Pharmaceuticals, Inc.
Leukemia, Myeloid, Acute, Myelodysplastic Syndromes
A Study of ARGX-110 in Combination With Azacytidine in Participants With Newly Diagnosed Acute Myeloid Leukemia (AML) or High Risk Myelodysplatic Syndrome (MDS)
The purpose of this study is to determine the maximum tolerated dose (MTD) of ARGX-110 and/or the recommended Phase II dose (RP2D) in combination with a standard dose of azacytidine (AZA) in Phase 1; and to evaluate efficacy of…
- Trial name
- NCT03030612
- Testing
- ARGX-110, AZA
- Where
- 7 sites in France, Switzerland
- Volunteers
- 38 people
- Sponsor
- OncoVerity, Inc.
Fever, Sweats, and Hot Flashes, Infection
Recombinant Human Mannose-Binding Lectin (MBL) in Treating Young Patients With MBL Deficiency and Fever and Neutropenia
RATIONALE: Recombinant human mannose-binding lectin (MBL) may be effective in preventing infection in young patients with fever and neutropenia receiving chemotherapy for blood disease or cancer.
- Conditions
- Fever, Sweats, and Hot Flashes, Infection, Leukemia, Lymphoma
- Trial name
- NCT00886496
- Testing
- recombinant human mannose-binding lectin
- Where
- 3 sites in United States
- Sponsor
- Enzon Pharmaceuticals, Inc.