ION251 for Multiple Myeloma
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment called ION251 for individuals with multiple myeloma, a type of blood cancer. The goal is to determine the safest and most effective dose of ION251 for future studies. Participants will receive the treatment through an IV infusion over 28-day cycles. The trial seeks individuals whose multiple myeloma has returned or stopped responding to other treatments. Participants should require additional treatment and have tried other available options without success. As a Phase 1 trial, this research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new treatment.
Will I have to stop taking my current medications?
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial team or your doctor.
Is there any evidence suggesting that ION251 is likely to be safe for humans?
Research shows that ION251 is still undergoing testing to determine its safety for people. Initial studies aim to find the appropriate dosage for patients with multiple myeloma, assessing how much of the drug can be administered without causing serious issues. As this is an early trial, limited information is available about its safety in humans. However, similar treatments for other cancer types have generally been well-tolerated, with most patients not experiencing severe side effects. While this is encouraging, it may not apply to everyone. Prospective participants should discuss any concerns with their doctor.12345
Why do researchers think this study treatment might be promising?
Researchers are excited about ION251 for multiple myeloma because it offers a novel approach compared to current treatments like proteasome inhibitors, immunomodulatory drugs, and monoclonal antibodies. Unlike these standard therapies, ION251 utilizes a unique mechanism by targeting specific proteins involved in the disease process through RNA-based technologies. This approach could potentially lead to more precise and effective treatment outcomes, with the hope of improving patient responses and reducing side effects commonly seen with traditional therapies.
What evidence suggests that ION251 might be an effective treatment for multiple myeloma?
Research has shown that ION251, an experimental medicine studied in this trial, has shown promise in early animal studies. It reduced the disease and myeloma cells in mice, suggesting it might target and eliminate cancer stem cells, which play a crucial role in multiple myeloma. Although limited information exists from human studies, this approach offers hope for patients whose multiple myeloma has returned or is not responding to treatment. Early results are encouraging, but further research is needed to confirm its effectiveness in people.12467
Are You a Good Fit for This Trial?
This trial is for adults over 18 with relapsed/refractory multiple myeloma who have not responded to, cannot tolerate, or are ineligible for established therapies. Participants must have measurable MM and be in good physical condition (ECOG status of 0 or 1). They can't join if they have low blood counts, uncontrolled high blood pressure, certain other cancers or bleeding disorders, poor kidney function, or significantly elevated liver enzymes.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Dose Escalation
Part 1 involves a 3+3 dose-escalation scheme to determine the maximum-tolerated dose (MTD) during repeated 28-day treatment cycles
Treatment
Part 2 involves administering the recommended Phase 2 dose (RP2D) of ION251 for further assessments in safety, tolerability, and anti-myeloma activity
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- ION251
Trial Overview
The study aims to find the safest and most effective dose of a new drug called ION251 for patients with multiple myeloma that has come back after treatment or hasn't responded to previous treatments. The focus is on determining the maximum-tolerated dose and the recommended Phase 2 dose.
How Is the Trial Designed?
1
Treatment groups
Experimental Treatment
In Part 1, the dose escalation phase, increased amounts of ION251 will be administered at multiple time points by intravenous (IV) infusion during 28-day cycles. In Part 2, the determined RP2D of ION251 will be administered at multiple time points by IV infusion.
Find a Clinic Near You
Who Is Running the Clinical Trial?
Ionis Pharmaceuticals, Inc.
Lead Sponsor
Dr. Brett P. Monia
Ionis Pharmaceuticals, Inc.
Chief Executive Officer since 2020
PhD in Pharmacology from the University of Pennsylvania, BSc in Molecular Biology and Analytical Chemistry from Stockton State College
Dr. Eric Bastings
Ionis Pharmaceuticals, Inc.
Chief Medical Officer
MD
Citations
A Study of ION251 Administered to Patients With Relapsed/ ...
The purpose of this study is to determine the maximum-tolerated dose (MTD) and recommended Phase 2 dose (RP2D) of ION251 in patients with relapsed/refractory ...
ION251 for Multiple Myeloma · Info for Participants
Trial Overview The study aims to find the safest and most effective dose of a new drug called ION251 for patients with multiple myeloma that has come back after ...
A Study to Evaluate ION251 to Treat Patients with ...
The purpose of this study is to determine the maximum-tolerated dose (MTD) and recommended Phase 2 dose (RP2D) of ION251 in patients with ...
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today.ucsd.edu
today.ucsd.edu/story/designer-dna-therapeutic-wipes-out-cancer-stem-cells-treats-multiple-myeloma-in-miceDesigner DNA Therapeutic Wipes Out Cancer Stem Cells ...
The oligonucleotide — an investigational antisense medicine developed by Ionis and known as ION251 — lowered disease burden, reduced myeloma ...
Multiple myeloma: patient outcomes in real‐world practice
These real‐world data provide an insight into patient outcomes and treatment decisions being made in clinical practice. Keywords: multiple myeloma, real‐world ...
A Study of ION251 Administered to Patients With Relapsed/ ...
Safety, tolerability and molecular correlates of the IRF4-targeted antisense oligonucleotide frenlosirsen in patients with relapsed/refractory multiple myeloma.
Next-Generation Therapies for Multiple Myeloma - PMC
The clinical data for these trials are not mature, but results from similar trials in RR large B cell lymphoma or follicular lymphoma suggest a tolerable safety ...
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