CLINICAL TRIAL

ASTX727 LD for Myelodysplastic Syndromes

Low Risk
Recruiting · 18+ · All Sexes · Rochester, MN

This study is evaluating whether ASTX727 may help treat low-risk or intermediate-1 myelodysplastic syndrome.

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About the trial for Myelodysplastic Syndromes

Eligible Conditions
Myelodysplastic Syndromes · Preleukemia · Syndrome

Treatment Groups

This trial involves 3 different treatments. ASTX727 LD is the primary treatment being studied. Participants will be divided into 3 treatment groups. There is no placebo group. The treatments being tested are in Phase 1 & 2 and have already been tested with other people.

Experimental Group 1
ASTX727 LD
DRUG
+
ASTX727 SD
DRUG
Experimental Group 2
ASTX727 LD
DRUG
Experimental Group 3
ASTX727 LD
DRUG

Eligibility

This trial is for patients born any sex aged 18 and older. There are 8 eligibility criteria to participate in this trial as listed below.

Inclusion & Exclusion Checklist
Mark “yes” if the following statements are true for you:
The patient must have a ANC of less than five hundred million cells per liter in at least two blood counts prior to randomization. show original
The individual has a red blood cell transfusion dependence of 2 or more units of red blood cells or a hemoglobin level of less than 8.5 grams per deciliter in at least 2 blood counts prior to randomization. show original
People who have a platelet count of less than 50 in at least two blood tests prior to the start of the study are not eligible to participate in the study. show original
The Eastern Cooperative Oncology Group (ECOG) performance status is 0 to 2. show original
The text discusses the importance of organ function and how it is necessary for adequate health. show original
Women who could potentially become pregnant are not allowed to participate in the clinical trial, and they must have a negative pregnancy test at the beginning of the study. show original
The subject is able to understand the study procedures, the risks involved in the study, and provide written informed consent before the first study-specific procedure. show original
Women who could get pregnant must take birth control for six months after finishing treatment; men must use birth control and not father a child for at least three months after finishing treatment. show original
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Odds of Eligibility
Unknown<50%
Be sure to apply to 2-3 other trials, as you have a low likelihood of qualifying for this one.Apply To This Trial
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Approximate Timelines

Please note that timelines for treatment and screening will vary by patient
Screening: ~3 weeks
Treatment: varies
Reporting: 18-24 months
Screening: ~3 weeks
Treatment: Varies
Reporting: 18-24 months
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: 18-24 months.
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Trial Expert
Connect with the researchersHop on a 15 minute call & ask questions about:
- What options you have available- The pros & cons of this trial
- Whether you're likely to qualify- What the enrollment process looks like

Measurement Requirements

This trial is evaluating whether ASTX727 LD will improve 2 primary outcomes and 9 secondary outcomes in patients with Myelodysplastic Syndromes. Measurement will happen over the course of 18-24 months.

Hematologic response (Phase 1 only) based on normalization of conversion of any baseline cytopenia or anemia (hemoglobin response, neutrophil response, platelet response, transfusion independence)
18-24 MONTHS
Phase 1: Efficacy
18-24 MONTHS
Leukemia-free survival
18-24 MONTHS
Number of days from the date of randomization to the date when bone marrow or peripheral blood blasts reach ≥20%, or death from any cause
18-24 MONTHS
Time to bone marrow blasts >5%
18-24 MONTHS
Number of days from the date of randomization to the date when bone marrow blasts are >5% and increased by ≥50%.
18-24 MONTHS
Half life (t1/2)
18-24 MONTHS
pharmacokinetics parameter
18-24 MONTHS
Time to reach maximum concentration (Tmax)
18-24 MONTHS
pharmacokinetics parameter
18-24 MONTHS
Area under the curve (AUC)
18-24 MONTHS
pharmacokinetics parameter
18-24 MONTHS
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Patient Q & A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What are the signs of myelodysplastic syndromes?

Myelodysplastic syndrome is characterized by marrow failure and pancytopenia. Patients with myelodysplastic syndrome have a broad spectrum of clinical and hematological presentations, varying from an uncomplicated picture with anemia and thrombocytopenia to life-threatening acute leukemia.

Anonymous Patient Answer

What causes myelodysplastic syndromes?

It is clear that there is a combination of environmental and genetic factors that underlie myelodysplastic syndromes. Inadequate red blood cell production is the likely underlying defect that leads to the typical pancytopenia.

Anonymous Patient Answer

Can myelodysplastic syndromes be cured?

Currently, the treatment of patients with myelodysplastic syndromes is based on conventional modalities, particularly myelosuppressive induction agents. New data suggest that the curative potential of azacitidine, the histone deacetylase inhibitor, has been underestimated. Since these agents can be used in patients with a wide spectrum of myelodysplastic syndrome, the potential risk-benefit ratio of these agents may be better. On the basis of these data, we propose and report the concept of a multimodality approach in managing patients with myelodysplastic syndromes that is based on this novel concept.

Anonymous Patient Answer

What are common treatments for myelodysplastic syndromes?

It is important that the health professionals consider the clinical features, prognosis of each MDS subtype and the side effects of therapy for these patients.

Anonymous Patient Answer

What is myelodysplastic syndromes?

This article outlines the history and current clinical diagnostic definition of MDS, including recent clinical and genetic diagnostic criteria. It illustrates recent major advances in MDS cytogenetics and molecular cytogenetics, with particular emphasis on MDS with t(5;17) translocation that can result in chimera formation, leukemias, and the development of intramedullary iron stores.

Anonymous Patient Answer

How many people get myelodysplastic syndromes a year in the United States?

Data from a recent study indicates that about 1 in 40,000 is diagnosed a year and 1 in 2,300 live with the disease at the time of death. In most studies of the prevalence of these diseases, mortality data were not collected.

Anonymous Patient Answer

What is the survival rate for myelodysplastic syndromes?

This is the first study to demonstrate the survival rate of MM and smMDS. The MM survival rate was 67% and smMDS survival rate was 33% at 5 years. Although there are no previous publications on the OS of smMDS, the present study showed a similar survival as MM (67%). However, it is unclear why the OS of smMDS is lower than MM because this result did not reach statistical significance.

Anonymous Patient Answer

What are the latest developments in astx727 ld for therapeutic use?

This review summarizes recent studies on the use of ASXL7L and its biological aspects in cell biology, and also shows that further development might be undertaken at different levels.

Anonymous Patient Answer

What is the primary cause of myelodysplastic syndromes?

[With this article] there was a study that shows what genes in myelodysplastic syndromes maybe a problem, for example if a new study with patients suffering from myelodysplastic syndromes is done, and the genes will be analysed, that could help in the future. Also a [study] found that the mutations of ATM gene were found more often in leukemia. ATM is involved in the DNA damage response. And [a study] found that a low activity ATM leads to more cancer then a high activity ATM.

Anonymous Patient Answer

How does astx727 ld work?

In our study, we could not identify any statistically significant differences between those patients who experienced clinical adverse reactions and the patients who did not. The low incidence of allergic reactions supports the view that the Astx727 ld used was not associated with the development of allergenicity.

Anonymous Patient Answer

Is astx727 ld typically used in combination with any other treatments?

Astx727 is a potent inhibitor of the NF-κB pathway, and the combination of it with other treatments is an effective approach to managing multiple myeloma, including in the context of standard therapies.

Anonymous Patient Answer

Have there been any new discoveries for treating myelodysplastic syndromes?

While there are a few treatment options for MDS, there is not a single conclusive solution. It currently can't be given as a standalone treatment for MDS. It is important to focus on the symptoms of a patient to help treat. More research is needed to determine if there is any real benefit to specific treatments.

Anonymous Patient Answer
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