sabatolimab for Myelodysplastic Syndromes (MDS)

Phase-Based Estimates
1
Effectiveness
2
Safety
City of Hope National Medical Center Medical Oncology & Therapeutic, Duarte, CA
+3 More
sabatolimab - Drug
Eligibility
18+
All Sexes
Eligible conditions
Myelodysplastic Syndromes (MDS)

Study Summary

A Study of Sabatolimab in Combination With Azacitidine and Venetoclax in High or Very High Risk MDS Participants

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Eligible Conditions

  • Preleukemia
  • Myelodysplastic Syndromes
  • Syndrome
  • Myelodysplastic Syndromes (MDS)

Treatment Effectiveness

Effectiveness Estimate

1 of 3

Compared to trials

Study Objectives

This trial is evaluating whether sabatolimab will improve 2 primary outcomes and 16 secondary outcomes in patients with Myelodysplastic Syndromes (MDS). Measurement will happen over the course of Continuously collected for patients during treatment with sabatolimab up to 150 days after last treatment.

Day 150
Anti-drug Antibody (ADA) prevalence at baseline and ADA incidence on-treatment by dose level
Peak Serum Concentration (Cmax) MBG453
Trough Serum Concentration (Cmin) MBG453
Year 3
Duration of transfusion independence
Percentage of participants who are RBC/platelets transfusion independent
Year 3
Overall Survival (OS)
Day 28
Incidence of dose limiting toxicities (DLTs) (Safety run-in patients only)
Year 3
Duration of CR/mCR
Duration of complete remission (CR)
Duration of response for participants who achieved hematologic improvement (HI) or better
Event-Free Survival (EFS)
Leukemia-Free Survival (LFS)
Overall Response Rate (ORR) of participants who achieved hematologic improvement (HI) or better as best response
Percentage of subjects achieving a complete remission (CR) + morphologic complete remission (mCR): Safety run-in (Part 1) and Expansion (Part 2)
Progression-Free Survival (PFS)
Time to complete remission(CR)/marrow complete remission (mCR)
Year 3
Percentage of participants (receiving 800mg sabatolimab) achieving complete remission (CR) per investigator assessment
Year 3
Changes in fatigue (Part 2 - Expansion)

Trial Safety

Safety Estimate

2 of 3
This is better than 68% of similar trials

Compared to trials

Trial Design

2 Treatment Groups

Control
sabatolimab + azacitidine + venetoclax

This trial requires 76 total participants across 2 different treatment groups

This trial involves 2 different treatments. Sabatolimab is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 2 and have already been tested with other people.

sabatolimab + azacitidine + venetoclaxPart 1: Safety run-in consists of 2 subsequent cohorts of a lower dose (cohort 1) and s higher dose (cohort 2) of sabatolimab in combination with fixed dose of venetoclax and azacitidine. Cohort 2 will be open only after the review of safety data from cohort 1 indicates the regimen is safe. If the regimen using sabatolimab at the lower dose is not safe, the study will be stopped. Subsequently, if the review of safety data from participants enrolled in cohort 2 indicates that the regimen is safe, then Part 2 will be opened. Otherwise, if the regimen at the higher dose is not safe, the study will be also stopped. Part 2: Expansion will enroll additional participants to further investigate the regimen including sabatolimab at the higher dose, azacitidine and venetoclax. Participants data from Part 1 and Part 2 treated with the higher dose will be combined to determine the complete remission rate.
ControlNo treatment in the control group
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Azacitidine
FDA approved
Venetoclax
FDA approved

Trial Logistics

Trial Timeline

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

Closest Location

City of Hope National Medical Center Medical Oncology & Therapeutic - Duarte, CA

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. There are 6 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Signed informed consent must be obtained prior to participation in the study
Age ≥ 18 years at the date of signing the informed consent form (ICF)
Very high (> 6 points)
High (> 4.5-6 points)
Not immediately eligible for hematopoietic stem-cell transplantation (HSCT) or intensive chemotherapy at the time of screening due to individual clinical factors such as age, comorbidities and performance status, donor availability (de Witte et al 2017)
Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2

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 is myelodysplastic syndromes (mds)?

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Recent findings shows that myelodysplastic syndromes are a heterogeneous group of neutropenia-related disorders with a dismal prognosis. However, most patients with MDS have features of acute myeloid leukemia. Myelodysplasia is frequently the sole finding in patients referred for a bone marrow transplant. All subjects with MDS should be evaluated for disease progression or MDS in the setting of new symptomatic finding.

Unverified Answer

Can myelodysplastic syndromes (mds) be cured?

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In children with MDS, complete cytogenetic and cytogenetic/molecular transformation, or the presence of a bona fide JAK2 gene mutation is associated with an increased risk of recurrence and/or progression to acute myeloid leukemia. However, the development of a true curative treatment approach awaits more detailed identification of the genetic pathways that lead to MDS and associated leukemogenesis.

Unverified Answer

What causes myelodysplastic syndromes (mds)?

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Some MDS and CMML may occur spontaneously, but the majority are caused by specific types of treatment, such as chemotherapy or radiation therapy, used to deal with hematologic malignancies.\n

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

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The number of new cases of MDS per year in the U.S. remains low. However, the number of new MDS cases that are [not first in their family(s)] appears to be growing. This is important to know, because research on MDS is a U.S. National Cancer Institute priority. Since it is possible for people to [get MDS] who appear to be of normal health who [later] develop MDS, the number of new cases of MDS seems to be growing, even in people who were [not first] in their family(s).

Unverified Answer

What are the signs of myelodysplastic syndromes (mds)?

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Decreased counts of red blood cells (anemia) and/or white blood cells (leukopenia) in the blood (a "cut and a run") is an indication of low, progressively increasing blood loss over time. This can be associated with bleeding diathesis, such as increased sensitivity of platelets to aggregation (thrombocytosis) and decreased platelet count (thrombocytopenia). On examination, these can often be felt in the bone marrow as red-pink to pinkish-yellow to pinkish-gray discoloration.

Unverified Answer

What are common treatments for myelodysplastic syndromes (mds)?

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As MDS progresses to acute myelogenous leukemia, patients with MDS often require intensive chemotherapy with fludarabine or related drugs and are usually treated with radiotherapy.

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What are the chances of developing myelodysplastic syndromes (mds)?

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For MDS patients aged 60 years or older, the prognosis is very poor (<10%) without intensive treatment. For younger MDS patients, the prognosis is also very poor (<10%); therefore, for these patients this kind of therapy is not indicated. These patients are frequently and erroneously erroneously treated or cured, while this therapy was not effective. Therefore, the treatment should be discontinued for these patients.

Unverified Answer

Is sabatolimab safe for people?

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Data from a recent study suggest that sabatolimab is well tolerated in a range of clinical settings and is an acceptable treatment option for patients with MDS and for patients to whom autologous stem cell infusions are contraindicated.

Unverified Answer

How does sabatolimab work?

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Even if the role of TNF-α in MDS has been supported by several independent studies, it should be stressed that the mechanisms by which MDS is a TNF-α dependent process are still unknown and the use of TNF blockers should be viewed as a second line therapy. Sabatolimab may be effective in reducing transfusional requirements in patients having an underlying MDS. It seems justified, because a higher rate of patients with MDS received allogeneic blood transfusion in this trial. TNF-α may play a role in the early steps of MDS development. Further studies in a bigger number of patients with MDS appear justified.

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Is sabatolimab typically used in combination with any other treatments?

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Sabatolimab is being typically used in combination with other therapies, but there is a high rate of serious infections (13%) associated with use of sabatolimab in combination with other agents and/or therapies. This rate is likely higher with higher-risk patients such as patients on chemotherapy.

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What is the survival rate for myelodysplastic syndromes (mds)?

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The overall DSS at 5 yr was 38% (36% for < or =65 yrs; 62% for > or =65 yrs), decreasing to 21% at 10 yr and 12% at 25 yr. The 5-y DSS varied by MDS type, % marrow blasts, number of prior cytogenetic abnormalities, and transfusions. There was a progressive decline of DSS over time for MDS patients.

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What are the latest developments in sabatolimab for therapeutic use?

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The most recent clinical trial showed that the therapeutic use of the new mAbs did not produce a clinically significant effect on the [prognosis of patients with MDS] (https://clinicaltrials.gov/ct2/show/WJ00299927?NCI_TAN_LIS) after 2 years.

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