Clinical trials: Myelodysplastic Syndrome
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive 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 Syndromes
- Trial name
- NCT03301168
- Testing
- BPX-501 T cells, Rimiducid
- Where
- 10 sites in United States
- Volunteers
- 120 people
- Sponsor
- Bellicum Pharmaceuticals
Advanced MDS, AML
Study of NEROFE, a Novel Hormone-Peptide in Adult Patients With Advanced MDS and AML
This is an open-label Phase 1b study of NEROFE following a traditional 3+3 design to assess safety and to determine the Recommended Phase 2 Dose (RP2D) of NEROFE in patients with MDS or AML.
- Trial name
- NCT04365179
- Testing
- Nerofe
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Immune System Key Ltd
Acute Myeloid Leukemia and Myelodysplastic Syndrome
A Study of QLF32101 in Patients With Acute Myeloid Leukemia and Myelodysplastic Syndrome
This study aimed to evaluate the safety,tolerability and preliminary efficacy of QLF32101 administered intravenously and subcutaneously in patients with R/R, AML.
- Trial name
- NCT05703204
- Testing
- QLF32101
- Where
- China
- Volunteers
- 122 people
- Sponsor
- Qilu Pharmaceutical Co., Ltd.
Acute Lymphocytic Leukaemia, Acute Myeloid Leukaemia Refractory
First-in-human Study of SAR443579 Infusion in Male and Female Children and Adult Participants With Relapsed or Refractory Acute Myeloid Leukemia (R/R AML), B-cell Acute Lymphoblastic Leukemia (B-ALL), High Risk-myelodysplasia (HR-MDS), or Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)
This is an open-label, multicenter, Phase 1/Phase 2, dose escalation and dose expansion study to evaluate the safety, pharmacokinetics, pharmacodynamics and anti-leukemic activity of SAR443579 in various hematological…
- Conditions
- Acute Lymphocytic Leukaemia, Acute Myeloid Leukaemia Refractory, Myelodysplastic Syndromes, Blastic Plasmacytoid Dendritic Cell Neoplasia
- Trial name
- NCT05086315
- Testing
- SAR443579
- Where
- 23 sites in United States, Australia and 3 more
- Volunteers
- 101 people
- Sponsor
- Sanofi
Myelodysplastic Syndromes (MDS)
A Study Evaluating Venetoclax in Combination With Azacitidine in Participants With Treatment-Naïve Higher-Risk Myelodysplastic Syndromes (MDS)
This is a Phase 1b, open-label, non-randomized, multicenter, dose-finding study evaluating venetoclax in combination with azacitidine in participants with treatment-naïve higher-risk MDS comprising a dose-escalation portion and a…
- Trial name
- NCT02942290
- Testing
- Azacitidine, Venetoclax
- Where
- 37 sites in United States, Australia and 5 more
- Volunteers
- 134 people
- Sponsor
- AbbVie
Acute Myelogenous Leukemia, Myelodysplastic Syndromes
A Phase 2a, Open-Label, Two Stage Study of Nerofe or Nerofe With Doxorubicin in Subjects With AML or MDS
This is a Phase 2a, Open-label, one arm study in which the eligible patients will be treated with IV Nerofe, three times a week in 28 days cycles (up to 12 cycles).
- Trial name
- NCT03059615
- Testing
- Nerofe, Doxorubicin
- Where
- 2 sites in Israel
- Sponsor
- Immune System Key Ltd
Myelodysplastic Syndromes
Tipifarnib in Subjects With Myelodysplastic Syndromes
This is a Phase 2 randomized, open-label, two-stage study designed to investigate the antitumor activity of tipifarnib in approximately 36 eligible subjects with MDS who have no known curative treatment.
- Trial name
- NCT02779777
- Testing
- Tipifarnib
- Where
- 2 sites in United States
- Volunteers
- 16 people
- Sponsor
- Kura Oncology, Inc.
Myelodysplastic Syndromes, Refractory Anemia With Excess Blasts
Efficacy and Safety of IV Rigosertib in MDS Patients With Excess Blasts Progressing After Azacitidine or Decitabine
This study will examine the effect intravenously administered rigosertib has on the relationship between bone marrow blasts response and overall survival in myelodysplastic syndromes (MDS) patients who have 5-30% bone marrow…
- Conditions
- Myelodysplastic Syndromes, Refractory Anemia With Excess Blasts, Chronic Myelomonocytic Leukemia, Cytopenia
- Trial name
- NCT01928537
- Testing
- rigosertib sodium
- Where
- 35 sites in United States, Australia and 6 more
- Volunteers
- 67 people
- Sponsor
- Traws Pharma, Inc.
Non-transfusion-dependent Thalassemia, Low Risk Myelodysplastic Syndrome
Study in Beta-thalassaemia or Myelodysplastic Syndrome Patients to Investigate the Safety and Tolerability of SLN124
The primary purpose of this study is to determine the safety and tolerability of SLN124 for the treatment of non-transfusion-dependent (NTD) β-thalassaemia and low risk myelodysplastic syndrome.
- Trial name
- NCT04176653
- Where
- 5 sites in Bulgaria, United Kingdom
- Sponsor
- Silence Therapeutics plc
Advanced or Refractory Leukemia, Myelodysplastic Syndromes
A Study of the Histone-deacetylase Inhibitor JNJ-26481585 in Patients With Advanced or Refractory Leukemia or Myelodysplastic Syndrome
The purpose of this study is to explore the safety, pharmacokinetic (what the body does to the medication), pharmacodynamic (what the medication does to the body), and activity of JNJ-26481585 in patients with advanced or…
- Trial name
- NCT00676728
- Testing
- JNJ-26481585
- Where
- 2 sites in United States
- Volunteers
- 10 people
- Sponsor
- Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
IgA Nephropathy, Paroxysmal Nocturnal Hemoglobinuria
A Clinical Study to Evaluate the Effect of Food on the Pharmacokinetic Profiles of HRS-5965 Capsules in Healthy Subjects
The study is being conducted to evaluate the effect of food on pharmacokinetics of HRS-5965 capsules after oral administration.
- Trial name
- NCT07035665
- Testing
- HRS-5965 Capsules
- Where
- China
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Chengdu Suncadia Medicine Co., Ltd.
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
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
Paroxysmal Nocturnal Hemoglobinuria
A Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Participants With PNH
This is a randomized, double-blind, active-controlled phase 3 study of ABP 959 in participants with paroxysmal nocturnal hemoglobinuria.
- Trial name
- DAHLIA (NCT03818607)
- Testing
- ABP 959, Eculizumab
- Where
- 24 sites in United States, Czechia and 12 more
- Volunteers
- 42 people
- Sponsor
- Amgen
Myelodysplastic Syndromes
Clofarabine for Myelodysplastic Syndrome (MDS) Patients Who Failed Vidaza Treatment (tx)
The investigators hypothesize that, in addition to its apoptotic effect, clofarabine induces DNA hypomethylation. If the investigators' hypothesis is correct, findings from the present proposal will not only contribute to…
- Trial name
- NCT00700011
- Testing
- Clofarabine
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Texas Oncology Cancer Center
Myelodysplastic Syndrome (MDS)
Rabbit Anti-thymocyte Globulin in the Treatment of Patients With Low to Intermediate-1 Risk Myelodysplastic Syndrome
This is a Phase II, single-arm, open-label, multinational, multicenter study of rATG in patients with low or intermediate-1 risk MDS who have either failed 1 prior treatment with growth factor(s), hypomethylating agents…
- Trial name
- RISE (NCT00542828)
- Testing
- Thymoglobulin®, Rabbit Anti-thymocyte Globulin (rATG)
- Where
- 7 sites in France, Germany and 2 more
- Volunteers
- 16 people
- Sponsor
- Genzyme, a Sanofi Company
Chronic Iron Overload
Extended Evaluation of Deferasirox Film-coated Tablet (FCT) Formulation
Extend evaluation of deferasirox film-coated tablet (FCT) formulation
- Trial name
- NCT02720536
- Testing
- Deferasirox
- Where
- 14 sites in Austria, Greece and 1 more
- Volunteers
- 53 people
- Sponsor
- Novartis Pharmaceuticals
MDS
A Clinical Study of KRN321 in Adult Subjects With Myelodysplastic Syndrome
This is a multicenter, Randomized, Open-Label, Parallel, Comparative, Dose-Response Study to Evaluate the Efficacy and Safety study of KRN321 of subcutaneous injection in Adult Subjects with Low- or Intermediate-1-Risk…
- Trial name
- NCT01497145
- Testing
- KRN321
- Where
- 2 sites in Japan, South Korea
- Volunteers
- 45 people
- Sponsor
- Kyowa Kirin Co., Ltd.
Myelodysplastic Syndrome, Myelofibrosis
Blinded Cross-Over Bioequivalence (BE) Trial of Luitpold Azacitidine vs Vidaza
The purpose of this study is to assess the bioequivalence of subcutaneous Vidaza® and subcutaneous Luitpold Azacitidine pharmacokinetics and to assess the comparative safety of subcutaneous Vidaza® versus subcutaneous Luitpold…
- Conditions
- Myelodysplastic Syndrome, Myelofibrosis, Chronic Myeloid Leukemia, Chronic Lymphocytic Leukemia
- Trial name
- NCT01290302
- Testing
- Luitpold Azacitidine, Vidaza®
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- American Regent, Inc.
Recurrent Adult Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia
Clinical Trial of BP1001 (L-Grb-2 Antisense Oligonucleotide) in CML, AML, ALL & MDS
The first goal of this clinical research study is to find the highest safe dose of BP1001, a liposomal Growth Factor Receptor Bound Protein-2 antisense oligodeoxynucleotide (L-Grb2 AS), for patients with Philadelphia Chromosome…
- Conditions
- Recurrent Adult Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Myelodysplastic Syndrome, Ph1 Positive CML
- Trial name
- NCT01159028
- Testing
- BP1001, BP1001 in combination with LDAC
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Bio-Path Holdings, Inc.