Clinical trials: Myelodysplastic Syndrome With Gene Mutation
- Condition Myelodysplastic Syndrome With Gene Mutation
- Funder type Industry (pharma, biotech, devices)
Hypereosinophilic Syndrome, Systemic Mastocytosis
Imatinib Mesylate in Patients With Various Types of Malignancies Involving Activated Tyrosine Kinase Enzymes
This trial is for various types of malignancies which may depend on certain enzymes (tyrosine kinases) for growth. The objective of this study is to assess to what extent imatinib mesylate blocks these enzymes and to assess the…
- Conditions
- Hypereosinophilic Syndrome, Systemic Mastocytosis, Chronic Myelomonocytic Leukemia, Dermatofibrosarcoma
- Trial name
- NCT00171912
- Testing
- imatinib mesylate
- Where
- Australia
- Volunteers
- 38 people
- Sponsor
- Novartis Pharmaceuticals
Acute Myeloid Leukemia, Acute Myelogenous Leukemia
Open-label Study of FT-2102 With or Without Azacitidine or Cytarabine in Patients With AML or MDS With an IDH1 Mutation
This Phase 1/2 study will evaluate the safety, efficacy, PK, and PD of FT-2102 (olutasidenib) as a single agent or in combination with azacitidine or cytarabine.
- Conditions
- Acute Myeloid Leukemia, Acute Myelogenous Leukemia, Myelodysplastic Syndrome
- Trial name
- NCT02719574
- Testing
- FT-2102 (olutasidenib), Azacitidine, Cytarabine
- Where
- 60 sites in United States, Australia and 7 more
- Volunteers
- 336 people
- Sponsor
- Forma Therapeutics, Inc.
Hematologic Neoplasms
Phase 1/2 Study of Enasidenib (AG-221) in Adults With Advanced Hematologic Malignancies With an Isocitrate Dehydrogenase Isoform 2 (IDH2) Mutation
The primary objectives of Phase 1 Dose Escalation/Part 1 Expansion are: To assess the safety and tolerability of treatment with enasidenib administered continuously as a single agent dosed orally on Days 1 to 28 of a 28-day cycle…
- Trial name
- NCT01915498
- Testing
- Enasidenib
- Where
- 24 sites in United States, France
- Volunteers
- 345 people
- Sponsor
- Celgene
Paroxysmal Nocturnal Hemoglobinuria (PNH) With Signs of Active Hemolysis
Study of Safety, Efficacy, Tolerability, Pharmacokinetics and Pharmacodynamics of LNP023 in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
This was a Phase 2, open label, single arm, multiple dose study to assess efficacy, safety, pharmacokinetics and pharmacodynamics of iptacopan when administered in addition to Standard of care (SoC) in patients with paroxysmal…
- Trial name
- NCT03439839
- Testing
- iptacopan, Standard of Care
- Where
- 3 sites in France, Germany and 1 more
- Volunteers
- 16 people
- Sponsor
- Novartis Pharmaceuticals
Clonal Cytopenia of Undetermined Significance (CCUS), Low-Risk Myelodysplastic Syndrome (LR-MDS)
Safety and Tolerability of REGN17235 in Adult Participants With Clonal Cytopenia of Undetermined Significance and Low-Risk Myelodysplastic Syndrome With SF3B1 Mutation
This study will test a study drug called REGN17235 (the "study drug") to see if it can help treat Clonal Cytopenia of Undetermined Significance (CCUS) and Low-Risk Myelodysplastic Syndrome (LR-MDS) with a specific genetic…
- Trial name
- NCT07753148
- Testing
- REGN17235
- Where
- 2 sites in Belgium
- Volunteers
- 52 people
- Sponsor
- Regeneron Pharmaceuticals
Leukemia, Myeloid, Acute, Leukemia, Lymphocytic, Acute
A Phase 1/2 Study of Enzomenib (DSP-5336) in Patients With Acute Leukemia (Horizen-1)
A phase 1/2 dose escalation / dose expansion study of Enzomenib (DSP-5336) in patients with acute leukemia.
- Conditions
- Leukemia, Myeloid, Acute, Leukemia, Lymphocytic, Acute, Multiple Myeloma, Myelodysplastic Syndromes
- Trial name
- NCT04988555
- Testing
- Enzomenib, azoles, Venetoclax
- Where
- 104 sites in United States, Belgium and 10 more
- Volunteers
- 606 people
- Sponsor
- Sumitomo Pharma America, Inc.
Acute Myeloid Leukemia (AML), Acute Promyelocytic Leukemia (APL)
Accuracy Testing of the Chromosomal Aberration and Gene Mutation Markers of the AMLProfiler
This clinical study will demonstrate the accuracy of the chromosomal aberration and gene mutation markers of the AMLProfiler molecular diagnostic assay and generate clinical performance data to support a Pre-Market Approval (PMA)…
- Conditions
- Acute Myeloid Leukemia (AML), Acute Promyelocytic Leukemia (APL), Refractory Anemia With Excess of Blasts (RAEB)
- Trial name
- NCT01463410
- Where
- 5 sites in United States, Germany and 2 more
- Volunteers
- 264 people
- Sponsor
- Skyline Diagnostics BV
Clonal Mast Cell Disease, KIT D816V Mutation
Screening Study for KIT D816V Mutated Mast Cell Disease in Select Populations
This is a multicenter screening study to characterize the prevalence of the KIT D816V mutation in participants with suspected clonal mast cell disease.
- Conditions
- Clonal Mast Cell Disease, KIT D816V Mutation, Suspected KITD816V Mutated Clonal Mast Cell Disease
- Trial name
- POLARIS (NCT07143669)
- Testing
- Screening
- Where
- 26 sites in United States, Belgium
- Volunteers
- 750 people
- Sponsor
- Blueprint Medicines Corporation
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
Myelodysplastic Syndromes
Alternative Dosing Regimens of Subcutaneous Azacitidine for Myelodysplastic Syndromes
The purpose of this study is to determine if azacitidine, combined with Best Supportive Care (BSC), is effective in treating myelodysplastic syndromes (MDS) when given according to a different doses and dosing schedules.
- Trial name
- NCT00102687
- Testing
- azacitidine
- Where
- 31 sites in United States
- Volunteers
- 151 people
- Sponsor
- Celgene
Paroxysmal Nocturnal Hemoglobinuria
Efficacy, Safety, Pharmacokinetics and Pharmacodynamics Study, Assessing Multiple LNP023 Doses in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria
This was a Phase II randomized, open-label, multicenter, efficacy, safety, pharmacokinetic and pharmacodynamic study assessing four iptacopan doses in adult Paroxysmal nocturnal hemoglobinuria (PNH) patients with active hemolysis…
- Trial name
- NCT03896152
- Testing
- LNP023
- Where
- 5 sites in Malaysia, Singapore and 2 more
- Volunteers
- 13 people
- Sponsor
- Novartis Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria
First-in-Human Study of BCX9930 in Healthy Volunteers and Patients With PNH
This is a 3-part Phase 1 dose-ranging study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single (Part 1) and multiple (Part 2) ascending doses of BCX9930 in healthy subjects and in subjects with…
- Trial name
- NCT04330534
- Testing
- BCX9930
- Where
- 4 sites in Austria, South Africa and 1 more
- Volunteers
- 168 people, healthy volunteers welcome
- Sponsor
- BioCryst Pharmaceuticals
Acute Myelogenous Leukemia, Myelodysplastic Syndrome
Dose Escalation/ Expansion Study of CA-4948 as Monotherapy in Patients With Acute Myelogenous Leukemia (AML) or Myelodysplastic Syndrome (MDS)
This is a multicenter, open-label, Phase 1/2a dose escalation and expansion study of orally administered emavusertib (CA-4948) monotherapy in adult patients with AML or higher- risk Myelodysplastic Syndrome (hrMDS).
- Trial name
- NCT04278768
- Testing
- Emavusertib, Venetoclax
- Where
- 32 sites in United States, Czechia and 6 more
- Volunteers
- 366 people
- Sponsor
- Curis, Inc.
Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome
Study of Orally Administered AG-881 in Patients With Advanced Hematologic Malignancies With an IDH1 and/or IDH2 Mutation
The purpose of this Phase I, multicenter study is to evaluate the safety, pharmacokinetics, pharmacodynamics and clinical activity of AG-881 in advanced hematologic malignancies that harbor an IDH1 and/or IDH2 mutation
- Conditions
- Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome, Hematologic Malignancies
- Trial name
- NCT02492737
- Testing
- AG881
- Where
- 6 sites in United States, France
- Volunteers
- 46 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Acute Myeloid Leukemia (AML), High-risk Myelodysplastic Syndrome
Safety and Efficacy of RVU120 for Treatment of Relapsed/Refractory AML
The goal of this study is to assess the safety, tolerability, anti-tumor activity (efficacy), pharmacokinetics (PK), and pharmacodynamics (PD) of the agent RVU120 when administered to adult patients with relapsed or refractory…
- Trial name
- RIVER-52 (NCT06268574)
- Testing
- RVU120
- Where
- 41 sites in France, Italy and 2 more
- Volunteers
- 62 people
- Sponsor
- Ryvu Therapeutics SA