CovidResearchTrials by Shray Alag


CovidResearchTrials Covid 19 Research using Clinical Trials (Home Page)


Report for D009190: Myelodysplastic Syndromes NIH

(Synonyms: Myelody, Myelodys, Myelodysp, Myelodyspl, Myelodyspla, Myelodysplasti, Myelodysplastic, Myelodysplastic S, Myelodysplastic Sy, Myelodysplastic Syn, Myelodysplastic Syndr, Myelodysplastic Syndrom, Myelodysplastic Syndrome, Myelodysplastic Syndromes)

Developed by Shray Alag
Clinical Trial MeSH HPO Drug Gene SNP Protein Mutation


Correlated Drug Terms (6)


Name (Synonyms) Correlation
drug360 Biosensors Wiki 0.58
drug83 ASP7517 Wiki 0.58
drug72 ALX148 Wiki 0.58
drug260 Azacitidine Wiki 0.58
drug1163 Ibrutinib Wiki 0.41
drug332 Best Practice Wiki 0.33

Correlated MeSH Terms (10)


Name (Synonyms) Correlation
D011289 Preleukemia NIH 0.82
D000741 Anemia, Aplastic NIH 0.58
D010265 Paraproteinemias NIH 0.58
D008998 Monoclonal Gammopathy of Undetermined Significance NIH 0.58
D008218 Lymphocytosis NIH 0.58
D007951 Leukemia, Myeloid, NIH 0.33
D015470 Leukemia, Myeloid, Acute NIH 0.29
D007938 Leukemia, NIH 0.24
D009369 Neoplasms, NIH 0.13
D013577 Syndrome NIH 0.13

Correlated HPO Terms (7)


Name (Synonyms) Correlation
HP:0002863 Myelodysplasia HPO 1.00
HP:0100827 Lymphocytosis HPO 0.58
HP:0012133 Erythroid hypoplasia HPO 0.58
HP:0012324 Myeloid leukemia HPO 0.33
HP:0004808 Acute myeloid leukemia HPO 0.29
HP:0001909 Leukemia HPO 0.16
HP:0002664 Neoplasm HPO 0.13

There are 3 clinical trials

Clinical Trials


1 A Phase 1/2 Open-label Study Investigating the Safety, Tolerability and Efficacy of ASP7517 in Subjects With Relapsed/Refractory Acute Myeloid Leukemia (AML) and Relapsed/Refractory Higher Risk Myelodysplastic Syndrome (MDS)

The purpose of this study is to evaluate the safety and tolerability and to determine the recommended phase 2 dose (RP2D) and/or the maximum tolerated dose (MTD) of ASP7517. This study will also evaluate the clinical response of ASP7517 as well as other measures of anticancer activity of ASP7517.

NCT04079296 Acute Myeloid Leukemia (AML) Myelodysplastic Syndrome Biological: ASP7517
MeSH:Leukemia Leukemia, Myeloid Leukemia, Myeloid, Acute Preleukemia Myelodysplastic Syndromes Syndrome
HPO:Acute megakaryocytic leukemia Acute myeloid leukemia Leukemia Myelodysplasia Myeloid leukemia

Primary Outcomes

Description: A DLT is defined as any of the following events that occur within 28 days starting with the first dose on cycle 1 day 1 (C1D1) and that is considered to be related to investigation product (IP). The severity of AEs will be assessed according to National Cancer Institute's Common Terminology Criteria for Adverse Events (NCI-CTCAE), version 5.0. DLT is defined as follows: non-hematologic AEs that are ≥ grade 3; confirmed Hy's law case; new onset of grade 4 thrombocytopenia (with minimum of 2 grade worsening from baseline) within 24 hours of dosing; prolonged myelosuppression, defined as absolute neutrophil count (ANC) < 500/μL for more than 28 days off therapy and in the absence of evidence of active leukemia or MDS in the marrow or blood, will be considered as a DLT.

Measure: Incidence of dose limiting toxicities (DLTs)

Time: 28 days

Description: An AE is any untoward medical occurrence in a participant administered a study drug, and which does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of a medicinal product whether or not considered related to the medicinal product. AEs will be graded using NCI-CTCAE guidelines, version 5.0.

Measure: Number of participants with adverse events (AEs)

Time: Up to 2 years

Description: An AE is considered "serious" if the event: results in death; is life-threatening (An AE is considered "life-threatening" if its occurrence places the subject at immediate risk of death; it does not include an AE that, had it occurred in a more severe form, might have caused death.); results in persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions; results in congenital anomaly, or birth defect; requires inpatient hospitalization (except for planned procedures as allowed per study) or leads to prolongation of hospitalization (except if prolongation of planned hospitalization is not caused by an AE); other medically important events.

Measure: Number of participants with serious adverse events (SAEs)

Time: Up to 2 years

Description: Number of participants with potentially clinically significant laboratory values.

Measure: Number of participants with laboratory value abnormalities and/or AEs

Time: Up to 2 years

Description: Routine 12-lead ECGs will be taken after the subject has been resting in the supine position for at least 5 minutes. Routine 12-lead ECGs will be taken in triplicate.

Measure: Number of participants with 12-lead electrocardiogram (ECG) abnormalities and/or AEs

Time: Up to 2 years

Description: Number of participants with potentially clinically significant vital sign values.

Measure: Number of participants with vital sign abnormalities and/or AEs

Time: Up to 2 years

Description: Number of participants with potentially clinically significant physical exam values.

Measure: Number of participants with physical exam abnormalities and/or AEs

Time: Up to 2 years

Description: The ECOG scale will be used to assess performance status. Grades range from 0 (fully active) to 5 (dead). Negative change scores indicate an improvement. Positive scores indicate a decline in performance.

Measure: Number of participants with Eastern Cooperative Oncology Group (ECOG) performance status

Time: Up to 2 years

Description: CRc rate is defined for AML subjects as the number of participants who achieve the best response of CRc (complete response [CR], complete remission with incomplete platelet recovery [CRp] or complete remission with incomplete hematological recovery [CRi]) divided by the number of participants in the analysis population.

Measure: Composite complete remission (CRc) rate for participants with R/R AML (phase 2)

Time: Up to 2 years

Description: Complete response + bone marrow complete response + partial response (CR + BM CR + PR) rate for participants with R/R higher risk MDS (phase 2) [ Time Frame: Up to 2 years ] CR + BM CR + PR rate is defined for MDS participants as the number of participants who achieve the best response of CR + BM CR + PR divided by the number of subjects in the analysis population.

Measure: Complete response + bone marrow complete response + partial response (CR + BM CR + PR) rate for participants with R/R higher risk MDS (phase 2)

Time: Up to 2 years

Secondary Outcomes

Description: Duration of remission for participants with AML includes duration of CRc, duration of CR/complete remission with partial hematologic recovery (CRh), duration of CRh, duration of CR, and duration of response (i.e., CRc + PR).

Measure: Duration of remission for participants with AML

Time: Up to 2 years

Description: Duration of remission for MDS includes duration of CR and duration of response (i.e., CR + PR).

Measure: Duration of remission for participants with MDS

Time: Up to 2 years

Description: EFS is defined as the time from the date of first dose until the date of documented relapse, treatment failure or death from any cause within 30 days after the last dose of study drug (whichever occurs first earliest of [relapse date, treatment failure date, death date] - first dose date + 1).

Measure: Number of participants with event-free survival (EFS)

Time: Up to 2 years

Description: OS is defined as the time from the date of first dose until the date of death from any cause (death date - first dose date + 1).

Measure: Duration of overall survival (OS)

Time: Up to 2 years

Description: CR rate is defined as the number of participants who achieve CR at any of the postbaseline visits divided by the number of participants in the analysis population.

Measure: CR rates for participants with R/R AML

Time: Up to 2 years

Description: Best response rate is defined as the number of subjects who achieve CRc or PR at any of the postbaseline visits divided by the number of subjects in the analysis population.

Measure: Best response (CRc + PR) rates for participants with R/R AML

Time: Up to 2 years

Description: CRh rate is defined as the number of participants who achieve CRh at any of the postbaseline visits divided by the number of participants in the analysis population.

Measure: CRh rates for participants with R/R AML

Time: Up to 2 years

Description: CR rate is defined as the number of participants who achieve CR at any of the postbaseline visits divided by the number of participants in the analysis population.

Measure: CR rates for participants with R/R higher risk MDS

Time: Up to 2 years

Description: HI requires 1 measurement of erythroid or platelets or neutrophils maintained at a specified level for at least 8 weeks without ongoing cytotoxic therapy

Measure: Hematologic improvement (HI) rates for participants with R/R higher risk MDS

Time: Up to 2 years

Description: Objective response (CR + BM CR + PR + HI) rates (ORR) for participants with R/R higher risk MDS [Time Frame: Up to 2 years] ORR is defined as the number of participants who achieve CR or BM CR or PR or HI at any of the postbaseline visits divided by the number of participants in the analysis population.

Measure: Objective response (CR + BM CR + PR + HI) rates (ORR) for participants with R/R higher risk MDS

Time: Up to 2 years

2 A Phase 1/2 Study of ALX148 in Combination With Azacitidine in Patients With Higher Risk Myelodysplastic Syndrome (MDS)

This Phase 1/2 clinical study will evaluate ALX148 in combination with azacitidine for the treatment of patients with higher risk myelodysplastic syndrome (MDS).

NCT04417517 Higher Risk Myelodysplastic Syndromes Drug: ALX148 Drug: Azacitidine
MeSH:Preleukemia Myelodysplastic Syndromes Syndrome
HPO:Myelodysplasia

Primary Outcomes

Description: Number of participants with a DLT

Measure: Phase 1: Dose Limiting Toxicities (DLT)

Time: Up to 28 days

Description: Number of participants achieving a response per International Working Group (IWG) criteria

Measure: Phase 2: Objective response rate (ORR)

Time: Approximately 6 months

3 Randomized Double-Blind Phase 2 Trial of Ibrutinib Versus Standard Treatment for COVID-19 Illness Requiring Hospitalization With Safety Lead-In

This phase Ib/II trial studies the side effects and best dose of ibrutinib and how well it works in treating patients with COVID-19 requiring hospitalization. Ibrutinib may help improve COVID-19 symptoms by lessening the inflammatory response in the lungs, while preserving overall immune function. This may reduce the need to be on a ventilator to help with breathing.

NCT04439006 Aplastic Anemia Hematopoietic and Lymphoid Cell Neoplasm Malignant Solid Neoplasm Monoclonal B-Cell Lymphocytosis Monoclonal Gammopathy of Undetermined Significance Myelodysplastic Syndrome Symptomatic COVID-19 Infection Laboratory-Confirmed Other: Best Practice Drug: Ibrutinib
MeSH:Laboratory Infection Myelodysplastic Syndromes Anemia, Aplastic Paraproteinemias Monoclonal Gammopathy of Undetermined Significance Lymphocytosis Neoplasms
HPO:Aplastic anemia Erythroid hypoplasia Hypoplastic anemia Lymphocytosis Myelodysplasia Neoplasm

Primary Outcomes

Description: Associations between baseline characteristics and the primary endpoint will be evaluated with logistic regression, adjusting for arm. These analyses will be largely descriptive, as a result of a limited sample size.

Measure: Proportion of patients with diminished respiratory failure and death

Time: During hospitalization for COVID-19 infection or within 30 days of registration

Measure: Death

Time: During hospitalization for COVID-19 infection or within 30 days of registration

Secondary Outcomes

Description: Fever-free will be assessed by a temperature of < 100.5 degrees Fahrenheit orally. Will be estimated for each arm using the method of Kaplan-Meier. Medians estimates and/or estimates at specific time points will be provided with 95% confidence intervals.

Measure: Time from study initiation to 48 hours fever-free

Time: Up to 14 days

Description: Will be estimated for each arm using the method of Kaplan-Meier. Medians estimates and/or estimates at specific time points will be provided with 95% confidence intervals.

Measure: Duration of hospitalization

Time: Up to 14 days

Measure: Time in intensive care unit (ICU)

Time: Up to 14 days

Measure: Time to ICU admission

Time: Up to 14 days

Measure: Number of days requiring supplemental oxygen

Time: Up to 14 days

Measure: Total days of mechanical ventilation

Time: Up to 14 days

Measure: Time to mechanical ventilation

Time: Up to 14 days

Measure: Shock and need for pressure support

Time: Up to 14 days

Measure: Incidence of any infection (viral, fungal, bacterial)

Time: Up to 14 days

Measure: Time to clinical resolution

Time: Up to 14 days

Description: Adverse events will be summarized by grade, type, and attribution (regardless of attribution and treatment-related) for each arm.

Measure: Incidence of grade 3 or higher adverse events

Time: Up to 12 months

Description: The proportion of patients with viral clearance at the time of hospital discharge will be estimated with 95% confidence intervals for each arm.

Measure: At the end of therapy (day 14)

Time: Up to 14 days

Description: Will be estimated for each arm using the method of Kaplan-Meier. Medians estimates and/or estimates at specific time points will be provided with 95% confidence intervals.

Measure: Time to viral clearance

Time: Up to 12 months

Description: Patients will be followed for up to 12 months or until death or withdrawal of study consent for further follow-up. Following hospitalization, study visits will be telephone or video encounters.

Measure: Survival

Time: Up to12 months


HPO Nodes