SY-1425 + Azacitidine for Myelodysplastic Syndromes

2
Effectiveness
3
Safety
Hopital Mignot, Le Chesnay, France
Myelodysplastic Syndromes+2 More
SY-1425 + Azacitidine - Drug
Eligibility
18+
All Sexes
Eligible conditions
Myelodysplastic Syndromes

Study Summary

This study is evaluating whether a drug may help treat a type of blood cancer.

See full description

Eligible Conditions

  • Myelodysplastic Syndromes
  • Syndrome
  • Preleukemia

Treatment Effectiveness

Effectiveness Estimate

2 of 3
This is better than 85% of similar trials

Study Objectives

This trial is evaluating whether SY-1425 + Azacitidine will improve 1 primary outcome and 9 secondary outcomes in patients with Myelodysplastic Syndromes. Measurement will happen over the course of Up to 5 Years.

Up to 5 Years
Duration of Complete Response
Duration of Event Free Survival
Duration of Overall Response
Duration of Overall Survival
Proportion of Participants Who Achieve Objective Response
Proportion of Participants Who Achieve Transfusion Independence
Proportion of Participants with Complete Remission
Proportion of participants with Adverse Events and Serious Adverse Events
Time to Complete Remission
Time to Initial Response

Trial Safety

Safety Estimate

3 of 3
This is better than 85% of similar trials

Trial Design

4 Treatment Groups

Tamibarotene Matched Placebo + Azacitidine
Tamibarotene + Azacitidine
Placebo group

This trial requires 190 total participants across 4 different treatment groups

This trial involves 4 different treatments. SY-1425 + Azacitidine is the primary treatment being studied. Participants will be divided into 2 treatment groups. Some patients will receive a placebo treatment. The treatments being tested are in Phase 3 and have had some early promising results.

Tamibarotene + Azacitidine
Drug
Tamibarotene: 6 mg administered orally twice per day (BID) on Days 8 through 28 of each 28-day treatment cycle. Azacitidine: 75 mg/m^2 administered intravenously or subcutaneously each day on Days 1 through 7 of each 28-day treatment cycle.
SY-1425 + Azacitidine
Drug
SY-1425: 6 mg administered orally twice per day (BID) on Days 8 through 28 of each 28-day treatment cycle. Azacitidine: 75 mg/m^2 administered intravenously or subcutaneously each day on Days 1 through 7 of each 28-day treatment cycle.
Tamibarotene Matched Placebo + Azacitidine
Drug
Placebo: Tamibarotene-matching tablets administered orally BID on Days 8 through 28 of each 28-day treatment cycle. Azacitidine: 75 mg/m^2 administered intravenously or subcutaneously each day on Days 1 through 7 of each 28-day treatment cycle.
SY-1425 Matched Placebo + Azacitidine
Drug
Placebo: SY-1425-matching tablets administered orally BID on Days 8 through 28 of each 28-day treatment cycle. Azacitidine: 75 mg/m^2 administered intravenously or subcutaneously each day on Days 1 through 7 of each 28-day treatment cycle.

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: up to 5 years
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly up to 5 years for reporting.

Closest Location

University of Utah Huntsman Cancer Institute - Salt Lake City, UT

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. You must have received newly diagnosed for Myelodysplastic Syndromes or one of the other 2 conditions listed above. There are 5 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
People taking part in the study must be 18 or older when they sign the consent form. show original
People who want to participate in the study must have a positive result on the investigational assay. show original
MDS is diagnosed according to the World Health Organization classification, which is then used to determine the patient's risk category using the Revised International Prognostic Scoring System show original
Requirements for participation include having an Eastern Cooperative Oncology Group (ECOG) Performance Status of ≤2. show original
Key

Patient Q&A Section

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

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

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The average age of diagnosis of myelodysplastic syndrome in the USA is 67.0 years for males and 58.8 years for females. The yearly incidence is 22 new cases per 100,000 inhabitants aged 18 years with a mean duration that is 7.7 years ago of diagnosis of myelodysplastic syndrome. The most frequent clinical sub-types are: chronic myeloid leukemia (7.5% of new cases), monoclonal gammopathy of undetermined significance (2.4%) and refractory anemia, chronic (1.1%).

Unverified Answer

Can myelodysplastic syndromes be cured?

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The overall 5-year survival of patients with MDS is 63%, but when a complete remission of myelodysplasia can be reached, the 5-year overall survival is up to 95%.

Unverified Answer

What is myelodysplastic syndromes?

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MDS involves impaired blood cell production that can progress to MDS Type I and Type II. MDS-I is a specific disease involving defects in the marrow. It can progress to leukaemia. Patients with MDS Type III disease can suffer from other types of cancer. Patients should be monitored for changes in the marrow that can potentially lead to worsening symptoms.

Unverified Answer

What causes myelodysplastic syndromes?

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Inherited mutations in cell cycle regulators, such as TP53 can cause myelodysplastic syndromes. Genetic variation in the 5-aminosalicylate biosynthetic pathway lead to increased production of AMP by MDS-derived precursors and subsequent oncogenic effects.

Unverified Answer

What are the signs of myelodysplastic syndromes?

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Signs of myelodysplastic syndromes will evolve over time; therefore, an individual must be aware of their signs. For example, they may not have as much blood in their stool as expected (anemia) or they may find it hard to urinate (dysuria).

Unverified Answer

What are common treatments for myelodysplastic syndromes?

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Most patients with MDS receive thalidomide as a part of their disease. A larger percentage of patients with a good response receive cladribine. Other agents used include alkylating drugs, azacitidine, and cytarabine. Patients with good quality of life can often be treated with supportive care to prolong survival.

Unverified Answer

Does sy-1425 + azacitidine improve quality of life for those with myelodysplastic syndromes?

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Sy-1425 + TAC regimen can significantly improve the D-PANSS and QOL for patients with myelodysplastic syndromes compared with the TAC regimen alone. Findings from a recent study are consistent with those from other trials in myelodysplastic syndromes and pave the way forward for future trials to investigate the role of adjuvant azacitidine combined with other novel agents for myelodysplastic syndromes.

Unverified Answer

What is the primary cause of myelodysplastic syndromes?

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Although certain mutations have been identified in MDS, their prevalence in this disorder is small. The pathologic mechanism(s) of the disorder are still unknown. The clinical presentation and response to therapy of MDS vary greatly among individuals.

Unverified Answer

Have there been any new discoveries for treating myelodysplastic syndromes?

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Although the most effective agents are still under investigation, some of the drugs already approved for other indications have been found to be active for improving the quality of life in patients with MDS.

Unverified Answer

How does sy-1425 + azacitidine work?

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Data from a recent study demonstrate that sy-1425 is efficacious in altering the epigenome and warranting further investigations into how sy-1425 + azacitidine might be used as a treatment for myelodysplastic syndromes (MDS).

Unverified Answer

What is the survival rate for myelodysplastic syndromes?

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The average survival rate of myelodysplastic syndromes is approximately 3 years. Patients who are in the MDS phase of life typically have the longer survival rate than patients who are older. This is because patients who are older are likely to have other health problems that affect their quality of life. However, patients who are in the MDS phase are more likely to be overweight as well as have a white blood cell count >2 x 10 but less than 10 x 10/uL in which case a bone marrow biopsy may be conducted. However, the bone marrow biopsy has an 85% sensitivity and 92% specificity for distinguishing among different types of MDS and detecting progression towards acute myeloid leukemia.

Unverified Answer

Does myelodysplastic syndromes run in families?

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Patients with secondary MDSs carry more clinically significant familial abnormalities in complex segregation and the familial clustering of specific mutations suggests an important genetic component, including one probably due to a common disorder.

Unverified Answer
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