JNJ-64619178 for Myelodysplastic Syndromes

Phase-Based Estimates
1
Effectiveness
1
Safety
Hadassah Medical Center, Jerusalem, Israel
Myelodysplastic Syndromes+3 More
JNJ-64619178 - Drug
Eligibility
18+
All Sexes
Eligible conditions
Myelodysplastic Syndromes

Study Summary

This study is evaluating whether a drug may help treat lymphoma and other cancers.

See full description

Eligible Conditions

  • Myelodysplastic Syndromes
  • Cancer
  • Solid Tumors, Adult
  • Neoplasms
  • Non-Hodgkin's Lymphoma (NHL)

Treatment Effectiveness

Effectiveness Estimate

1 of 3

Study Objectives

This trial is evaluating whether JNJ-64619178 will improve 1 primary outcome and 19 secondary outcomes in patients with Myelodysplastic Syndromes. Measurement will happen over the course of Approximately 3 years.

Approximately 3 years
Part 1 and Part 2: Accumulation Index (RA)
Part 1 and Part 2: Apparent Total Systemic Clearance of Drug (CL/F) after Extravascular Administration
Part 1 and Part 2: Area Under the Plasma Concentration Versus Time Curve From Time Zero to End of Dosing Interval (AUCtau)
Part 1 and Part 2: Maximum Plasma Concentration (Cmax) of JNJ-64619178
Part 1 and Part 2: Minimum Plasma Concentration (Cmin)
Part 1 and Part 2: Number of Participants with AE by Severity
Part 1 and Part 2: Number of Participants with Abnormal Vital Signs
Part 1 and Part 2: Number of Participants with Adverse Events (AE)
Part 1 and Part 2: Number of Participants with Dose-limiting Toxicities (DLTs)
Part 1 and Part 2: Number of Participants with Electrocardiogram (ECG) Abnormalities
Part 1 and Part 2: Number of Participants with Laboratory Abnormalities
Part 1 and Part 2: Plasma Concentration of Symmetric Dimethyl-Arginine (SDMA)
Part 1 and Part 2: Plasma Decay Half-Life (t1/2)
Part 1 and Part 2: Volume of Distribution at Steady-State Influenced by the Fraction Absorbed (Vss/F)
Part 1: Clinical Benefit Rate
Part 1: Duration of Response
Part 1: Percentage of Participants with B cell non-Hodgkin lymphoma (NHL) Showing Overall Response of Partial Response (PR) or Better
Part 1: Percentage of Participants with Solid Tumors Showing Overall Response of PR or Better
Part 2: Overall Improvement Rate
Part 2: Red Blood Cell (RBC) Transfusion Independence (TI) Rate

Trial Safety

Safety Estimate

1 of 3

Trial Design

2 Treatment Groups

No Control Group
Part 1: Dose escalation and RP2D Selection

This trial requires 114 total participants across 2 different treatment groups

This trial involves 2 different treatments. JNJ-64619178 is the primary treatment being studied. Participants will be divided into 2 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.

Part 1: Dose escalation and RP2D Selection
Drug
Participants with solid tumors or non-Hodgkin lymphoma (NHL) will receive JNJ-64619178 orally as per the assigned sequential cohorts and doses will be escalated based on the review of all available data including, but not limited to, pharmacokinetic, pharmacodynamic, safety, and clinical activity. One or more recommended Phase 2 dose(s) (RP2Ds) may be determined for further exploration.
Part 2:Dose Confirmation and Expansion
Drug
Participants with myelodysplastic syndromes (MDS) will receive JNJ-64619178 at a dose less than or equal to the RP2D selected in Part 1 for 24 weeks, or longer if there is evidence of clinical benefit. The dose level of JNJ-64619178 may be adjusted based on observed toxicities.

Trial Logistics

Trial Timeline

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

Closest Location

Massachusetts General Hospital - Boston, MA

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 3 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:
Women of childbearing potential must have a negative highly sensitive serum (beta-human chorionic gonadotropin [beta-hCG]) at screening and prior to the first dose of study drug. Women must agree not to donate eggs (ova, oocytes) for the purposes of assisted reproduction during the study and for a period of 90 days after receiving the last dose of study drug
B cell non-Hodgkin lymphoma (NHL) or solid tumors, or lower risk MDS
At least 1 measurable site of disease for B cell-NHL and solid tumors
Eastern Cooperative Oncology Group (ECOG) performance status score of 0 or 1
Adequate organ function

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 common side effects of jnj-64619178?

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The most common side effects were fever and pain in the arm or back. The most frequent side effects were back pain, muscle pain, fever, and arthralgia. For some side effects, the highest rates were seen in patients receiving the 2 mg JND-64619178 dose as opposed to the 0.5, 1.0, or 1.5 mg dose.

Unverified Answer

Can myelodysplastic syndromes be cured?

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The treatments listed above do not cure myelodysplastic syndromes but can reduce their symptoms and reduce the number of blood transfusions needed. The benefits on survival from these agents should be carefully balanced with their side effects, patient resistance and the financial cost of these agents. For MDS patients eligible for HSCT, consideration must be given to choosing between the benefit of HSCT versus the risks of chemotherapy for both the patients and the donor. An individualized plan must be discussed and carefully weighed.

Unverified Answer

What are common treatments for myelodysplastic syndromes?

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The treatment of myelodysplastic syndromes includes various medications in addition to supportive care. The most common is the antiproliferative agent thalidomide and protease inhibitor. Immunosuppressants, such as thymoglobulin, can be employed in patients who do not respond to other medication, but immunosuppressive agents are associated with high morbidity.

Unverified Answer

What is myelodysplastic syndromes?

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Myelodysplastic syndromes are a diverse group of neoplastic disorders, all characterized by abnormal production of red blood cells, white blood cells and/or platelets by normal hematopoietic cells. However, the etiopathology and genetic predisposition to myelodysplastic syndromes are distinct between cases.

Unverified Answer

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

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As many as 100,000 people per year in the United States can be diagnosed with a MDS. Approximately 40,000 of these patients have a myelodysplastic crisis, which implies that myelodysplastic syndrome patients in the United States suffer one-third of all myelodysplastic crises described in the current literature.

Unverified Answer

What are the signs of myelodysplastic syndromes?

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Patients who present with symptoms of MDS generally do not have conclusive criteria to support the diagnosis of MDS. The diagnosis of MDS is bolstered by finding cytopenias, marrow blast ≥ 10%, and pancytopenias by ≥ 8% on peripheral smear examination. The finding of dysplastic megakaryocytes on a bone-marrow aspirate (BMAs) confirms the diagnosis of MDS. The BMAs may be normal (5%), minimal changes defects (6%), dysplastic megakaryocytes (36%), and dysplastic megakaryocytes with ring-out (20%), similar to what is seen in MDS with ring-out.

Unverified Answer

What causes myelodysplastic syndromes?

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Both a genetic mutation and environmental factors are involved in the development of MDS. There is a significant risk of MDS in solid-organ transplant recipients. There is also a significant risk of acute myeloid leukemia in non-Hodgkin's lymphoma survivors. There has not been a consistent genetic signature for MDS and AML, but recent studies have identified risk loci consistent with previous linkage and association studies. MDS may have a common genetic abnormality, a chromosomal abnormality, or both. It is estimated that 10% of MDS patients will eventually require allogeneic hematopoietic stem cell transplantation (HSCT), with the likelihood increasing with older age and blast transfusion at diagnosis.

Unverified Answer

What are the latest developments in jnj-64619178 for therapeutic use?

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JUBIO is currently undertaking a Phase III trial in the USA to evaluate the safety and efficacy of low-dose JUBIO plus bendamustine in relapsed or refractory chronic myeloid leukemia that has received two prior generations of therapy. This trial is scheduled to start in early 2017 and is expected to be completed in 2 yr.

Unverified Answer

How does jnj-64619178 work?

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As we progress into understanding the pathogenesis of myelodysplastic syndrome, researchers are beginning to explore possible targets in the NF-κB pathway to stop the disease. However, until now, no effective therapy has been discovered that can modify the NF-κB pathway to control the disease progression. By looking at the effects of NF-κB pathway inhibitors on myelodysplastic syndromes, we hope to find an avenue to treat these disorders.

Unverified Answer

What is the latest research for myelodysplastic syndromes?

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Despite advances in understanding of the pathophysiology and biology of MDS, we have a significant lack of effective treatments. A more detailed understanding of the molecular mechanisms underlying the pathophysiology of MDS may allow the identification of specific molecular therapeutic targets that may be of therapeutic value and might assist in the optimization protocols of existing MDS-associated therapeutic regimes.

Unverified Answer

Who should consider clinical trials for myelodysplastic syndromes?

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Patients with low or intermediate risk, newly diagnosed MDS should consider clinical trial enrollment. Patients with an advanced-stage disease or with a dismal prognosis who wish to stay well for longer on less efficacious treatment should consider clinical trial enrollment as well. These patients may benefit from a trial and can achieve a higher OS over time.

Unverified Answer

What does jnj-64619178 usually treat?

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The studies of jnj-64619178 as anti-tumor agent showed that its mechanism of action is more complex than an inhibition of growth of BCR-ABL (JNJ-64619178) BCR, c-myb and c-kit (JNJ-64619178) oncogenic B cells. JNJ-64619178 acts through an anti-apoptotic mechanism enhancing, for example, the apoptotic response of BCR-ABL-positive oncogenic B cells to imatinib (Gleevec) and inhibits BCR activation.

Unverified Answer
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