Clinical trials: Myelodysplastic Syndromes, Adult
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.
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
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
Myelodysplastic Syndrome (MDS)
Study Of Venetoclax Tablet With Intravenous or Subcutaneous Azacitidine to Assess Change in Disease Activity In Adult Participants With Newly Diagnosed Higher-Risk Myelodysplastic Syndrome
Myelodysplastic Syndrome (MDS) is a group of disorders that gradually affect the ability of a person's bone marrow (semi-liquid tissue present in many bones like backbones) to produce normal blood cells.
- Trial name
- Verona (NCT04401748)
- Testing
- Venetoclax, Azacitidine
- Where
- 220 sites in United States, Australia and 21 more
- Volunteers
- 531 people
- Sponsor
- AbbVie
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
Paroxysmal Nocturnal Hemoglobinuria
A Study of Multiple Ascending Doses MY008211A in Healthy Adults
This is a single-center, randomized, double-blind, placebo-controlled, dose-escalation, and multiple-dose study to evaluate safety, tolerability, PK and PD of MY008211A Tablets in healthy subjects.
- Trial name
- NCT05828472
- Testing
- MY008211A tablets
- Where
- China
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Wuhan Createrna Science and Technology Co., Ltd
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
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
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.
AML, Adult, Myelodysplastic Syndromes
EPITHINK: Epigenetic Drug Treatment and Therapeutic Immunotherapy With NKR-2
This open-label Phase I study aims to define the recommended dose for further clinical development the NKR-2 treatment administered concurrently with AZA in treatment-naïve AML/MDS patients not candidates for intensive…
- Trial name
- EPITHINK (NCT03612739)
- Testing
- NKR-2 CAR-T Cells
- Sponsor
- Celyad Oncology SA
Myelodysplastic Syndromes
Tamibarotene Plus Azacitidine in Participants With Newly Diagnosed RARA-positive Higher-Risk Myelodysplastic Syndrome
This study compares the efficacy of Tamibarotene in combination with azacitidine to azacitidine in combination with placebo in participants who are Retinoic Acid Receptor Alpha (RARA) positive, and newly diagnosed with…
- Trial name
- NCT04797780
- Testing
- Tamibarotene, Azacitidine
- Where
- 128 sites in United States, Austria and 11 more
- Volunteers
- 246 people
- Sponsor
- Syros Pharmaceuticals
Cancer
BCX-1777 in Treating Patients With Refractory Cancer
RATIONALE: BCX-1777 may stop the growth of cancer cells by blocking the enzymes necessary for their growth. PURPOSE: Phase I trial to study the effectiveness of BCX-1777 in treating patients who have refractory cancer.
- Trial name
- NCT00073944
- Testing
- forodesine hydrochloride
- Where
- United States
- Sponsor
- BioCryst Pharmaceuticals
Acute Myeloid Leukemia, Myelodysplasia
Study of MGTA-117 in Patients With Adult Acute Myeloid Leukemia (AML) and Myelodysplasia-Excess Blasts (MDS-EB)
This research study is designed to selectively deplete CD117-positive cells from participants with AML and MDS-EB.
- Trial name
- NCT05223699
- Testing
- MGTA-117
- Where
- 8 sites in United States
- Volunteers
- 22 people
- Sponsor
- Magenta Therapeutics, Inc.
Paroxysmal Nocturnal Hemoglobinuria (PNH)
ALXN1210 Versus Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab
The primary purpose of this study was to assess the noninferiority of ravulizumab compared to eculizumab in adult participants with PNH who were clinically stable after having been treated with eculizumab for at least 6 months.
- Trial name
- NCT03056040
- Testing
- Ravulizumab, Eculizumab
- Where
- 51 sites in United States, Australia and 9 more
- Volunteers
- 202 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Tumor Lysis Syndrome, Cancer
Rasburicase Versus Allopurinol in Tumor Patients at Risk for Hyperuricemia and Tumor Lysis Syndrome
This is a randomized, multi-center, open-label, parallel group study with three arms: Rasburicase alone Rasburicase followed by Allopurinol Allopurinol alone The primary objective is to compare the adequacy of control of plasma…
- Conditions
- Tumor Lysis Syndrome, Cancer, Hyperuricemia
- Trial name
- NCT00230178
- Testing
- Rasburicase (SR29142), Allopurinol
- Where
- 15 sites in United States
- Volunteers
- 280 people
- Sponsor
- Sanofi
Myelodysplastic Syndromes, Leukemia, Myelomonocytic, Chronic
Guadecitabine (SGI-110) vs Treatment Choice in Adults With MDS or CMML Previously Treated With HMAs
A Phase 3, randomized, open-label, parallel-group, multicenter study designed to evaluate the efficacy and safety of guadecitabine in participants with MDS or CMML who failed or relapsed after adequate prior treatment with…
- Trial name
- NCT02907359
- Testing
- Guadecitabine, Treatment Choice
- Where
- 112 sites in United States, Belgium and 12 more
- Volunteers
- 417 people
- Sponsor
- Astex Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria, PNH
Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Chinese Adults Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
The primary objective of this study is to evaluate the efficacy of ravulizumab in adult participants with PNH.
- Trial name
- NCT06578949
- Testing
- Ravulizumab
- Where
- 8 sites in China
- Volunteers
- 18 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Low and Int 1-risk Myelodysplastic Syndrome
Combination Study of Deferasirox and Erythropoietin in Patients With Low- and Int-1-risk Myelodysplastic Syndrome.
The primary purpose of this trial was is to assess the effect of treatment with deferasirox combined with erythropoietin vs. erythropoietin alone on erythropoiesis in patients with low- and int-1-risk myelodysplastic syndrome.
- Trial name
- NCT01868477
- Testing
- Deferasirox DFX, DT, Erythropoietin alpha, Deferasirox DFX, FCT
- Where
- 30 sites in Algeria, Argentina and 8 more
- Volunteers
- 28 people
- Sponsor
- Novartis Pharmaceuticals
Myelodysplastic Syndromes, Leukemia, Myeloid, Acute
A Study Comparing Treatment Preference Between Oral Decitabine/Cedazuridine and Azacitidine in Myelodysplastic Syndrome, Low-Blast Acute Myeloid Leukemia, or Chronic Myelomonocytic Leukemia
It is hypothesized that significantly more patients would prefer oral decitabine/cedazuridine to subcutaneous (SC) azacitidine (AZA) due to several factors, including improved treatment convenience, the reduced risk of nosocomial…
- Conditions
- Myelodysplastic Syndromes, Leukemia, Myeloid, Acute, Leukemia, Myelomonocytic, Chronic, Patient Preference
- Trial name
- PREFER-HMA (NCT05883956)
- Testing
- Subcutaneous azacitidine, Oral decitabine/cedazuridine
- Where
- 11 sites in Australia, New Zealand
- Volunteers
- 13 people
- Sponsor
- Otsuka Australia Pharmaceutical Pty Ltd