NIS793 for Myelodysplastic Syndromes

Phase-Based Estimates
1
Effectiveness
1
Safety
Novartis Investigative Site, Barcelona, Spain
Myelodysplastic Syndromes+2 More
NIS793 - Drug
Eligibility
18+
All Sexes
Eligible conditions
Myelodysplastic Syndromes

Study Summary

This study is evaluating whether a set of drugs can be safely given to people with low risk myelodysplastic syndromes.

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Eligible Conditions

  • Myelodysplastic Syndromes
  • Preleukemia
  • Syndrome

Treatment Effectiveness

Effectiveness Estimate

1 of 3

Compared to trials

Study Objectives

This trial is evaluating whether NIS793 will improve 4 primary outcomes and 14 secondary outcomes in patients with Myelodysplastic Syndromes. Measurement will happen over the course of 30 months.

30 Months
Characterize pharmacokinetics for single agents and combinations: Cmax
Characterize pharmacokinetics for single agents and combinations: Ctrough
Characterize pharmacokinetics for single agents and combinations: Tmax
Characterize the prevalence of immunogenicity
Dose intensity
Dose interruption reduction
Efficacy of single agents and combinations in patients who are transfusion independent: Best Overall Response (BOR)
Efficacy of single agents and combinations in patients who are transfusion independent: Duration of Response (DOR)
Efficacy of single agents and combinations in patients who are transfusion independent: Overall Response Rate (ORR)
Efficacy of single agents and combinations in patients who are transfusion independent: Progression Free Survival (PFS)
Efficacy of single agents and combinations in patients who are transfusion independent: Time to Progression (TTP)
Efficacy of single agents and combinations on transfusion dependent patients: Best Overall Response (BOR)
Efficacy of single agents and combinations on transfusion dependent patients: Duration of Response (DOR)
Efficacy of single agents and combinations on transfusion dependent patients: Overall Response Rate (ORR)
Efficacy of single agents and combinations on transfusion dependent patients: Progression free survival (PFS)
Efficacy of single agents and combinations on transfusion dependent patients: Time to progression (TTP)
Incidence of DLTs
30 months
AE and SAE indicence

Trial Safety

Safety Estimate

1 of 3

Compared to trials

Trial Design

5 Treatment Groups

No Control Group
Arm 5: MBG453 + canakinumab combination

This trial requires 90 total participants across 5 different treatment groups

This trial involves 5 different treatments. NIS793 is the primary treatment being studied. Participants will be divided into 5 treatment groups. There is no placebo group. The treatments being tested are in Phase 1 and are in the first stage of evaluation with people.

Arm 5: MBG453 + canakinumab combinationTreatment with MBG453 + canakinumab combination Q4W to confirm safety and tolerability of combination RD.
Arm 1: MBG453 single agent
Drug
Treatment with MBG453 single agent Q4W to confirm safety and tolerability of RD.
Arm 2: NIS793 single agent
Drug
Treatment with NIS793 single agent Q3W to establish RD in this indication and confirm safety and tolerability.
Arm 4: MBG453 + NIS793 combinationTreatment with combination of MBG453 and NIS793 Q3W to confirm safety and tolerability of combination RD.
Arm 3: canakinumab single agent
Drug
Treatment with single agent canakinumab Q4W to confirm safety and tolerability of RD.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
NIS793
2017
Completed Phase 1
~120
Canakinumab
FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 30 months
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly 30 months for reporting.

Closest Location

The Ohio State University Wexner Medical Center - Columbus, OH

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. You must have received 1 prior treatment for Myelodysplastic Syndromes or one of the other 2 conditions listed above. There are 10 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Anemia that is causing symptoms and has a hemoglobin level of less than 10 grams per deciliter, and has not responded to or has stopped responding to erythropoietin stimulators (ESA) therapy, or the patient is not able to tolerate ESA therapy. show original
) People who have symptomatic anemia and a hemoglobin level of less than 10 g/dL and have never been treated with ESA therapy and have an EPO level of at least 500/uL are candidates for EPO therapy. show original
The patient has neutropenia with an ANC of <500 or 1000/ µL and recurrent and/or severe infections show original
or pomalidomide are potential candidates for carfilzomib For patients who are not responding to, intolerant of, or ineligible/ unable to receive standard of care therapeutic options including lenalidomide or pomalidomide, carfilzomib may be a potential treatment option. show original
This text is about a condition called thrombocytopenia, which is a decrease in the number of platelets in the blood show original
Before you take part in the study, you must agree to give your informed consent show original
You must be at least 18 years old in order to sign the informed consent form. show original
Eastern Cooperative Oncology Group (ECOG) performance status (PS) ≤2
The patient must be eligible for serial bone marrow aspirate and/or biopsy according to the study's guidelines, and be willing to undergo a bone marrow aspirate and/or biopsy at screening, during and at the end of therapy. 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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Between 0.5%-1.5% of all newly diagnosed cases of AML are MDSs. This is consistent with reports from the UK and Australia and supports the hypothesis that there is a genetic anomaly common in the population which leads to a high incidence of MDSs (MDP syndrome/GATA1 deficiency syndrome) in the United States.

Unverified Answer

What are common treatments for myelodysplastic syndromes?

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Most patients with myelodysplastic syndromes receive supportive intensive care and blood cell transfusion support or chemotherapy (e.g., cytostatics and anthracycline derivatives) in combination with supportive intensive care and blood cell transfusion support. It should be stressed that these supportive intensive-care therapies are not always indispensable and that a relatively small number of patients with myelodysplastic syndromes may be treated successfully with curative intent without intensive-care therapy.

Unverified Answer

What are the signs of myelodysplastic syndromes?

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There are a number of signs that characterize malignant myelodysplastic syndromes. The presence of a non-isolated clonal chromosomal abnormality and/or blast ("blastic") morphology in a neoplastic

Unverified Answer

What causes myelodysplastic syndromes?

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Causes of MDS are not well understood but they arise mostly from a combination of genetic and environmental factors affecting the maturation and/or survival of one or more types of blood cell.

Unverified Answer

Can myelodysplastic syndromes be cured?

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Recent findings we have shown that patients with de novo MDS in the absence of a predisposing disease can be treated with a durable response. Patients may derive significant benefit from treatments which target the maturation of myeloid lineage and the eradication of aberrant cell proliferation and differentiation.

Unverified Answer

What is myelodysplastic syndromes?

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Myelodysplastic syndromes are a group of diseases characterized by a gradual maturation of cellular blood cell precursors. They are usually associated with anemia; they do not cause cancer; and when cancer occurs, it usually has a poor response to treatment.\n

Unverified Answer

What are the latest developments in nis793 for therapeutic use?

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The current developments in nis793-based clinical trials have produced new data, some of which may provide a better understanding of the biologic and clinical efficacy of this agent. Further studies should follow with a better understanding of the biologic mechanisms and potential advantages of nis793 treatment compared with bortezomib or MM.

Unverified Answer

How serious can myelodysplastic syndromes be?

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The mortality rate of patients is lower than that in other groups of hematology patients owing to the improvement of patient treatment and survival of such patients. An increasing number of patients are surviving MDS for more than 10 years, and will hopefully reach the age range of average lifespan of a person. This leads to a higher social cost for treating myelodysplastic syndromes.

Unverified Answer

What is the survival rate for myelodysplastic syndromes?

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Myelodysplastic syndromes carry an extremely poor prognosis. Median survival can be as short as 7 weeks in MDS with myelosuppression and a marrow blast percentage higher than 50%. We offer a series of survival tables which will be useful as a basis for estimating survival.

Unverified Answer

How quickly does myelodysplastic syndromes spread?

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When using PCR methods to diagnose the presence of MDS-related cytogenetic changes, a high proportion of patients can be identified with such changes prior to the development of overt disease. This is a significant finding in supporting the use of cytogenetic study in the early diagnosis of this disease.

Unverified Answer

Have there been any new discoveries for treating myelodysplastic syndromes?

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The new [drugs] that have been approved or are approved for use and/or that have been found to be effective are listed in the table. Please note that these are only [newer medicines], but many old medicines still have an [effective] role to play, because they are still used or are [effective] in certain types of MDS or MDS with certain subtypes. This is because patients have different genetic and other characteristics which influence response and toxicity to a particular drug.

Unverified Answer

Is nis793 safe for people?

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Nis793 was well tolerated in people over the age of 70 years. Although the data from early clinical studies support its safety in older people, further work is required to explore the longer term effects.

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