ION251 for Multiple Myeloma

Recruiting at 14 trial locations

Overseen By Ionis Pharmaceuticals

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Ionis Pharmaceuticals, Inc.

Disqualifiers: Plasma cell leukemia, POEMS syndrome, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: 28 days per cycle

23 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing ION251, a new drug, in patients with multiple myeloma. The goal is to determine the appropriate dosage by starting with small amounts and increasing them over time.

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.

Eligibility Criteria

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

You have multiple myeloma and have not responded well to or cannot tolerate the standard treatments for this condition.

You should be able to perform daily activities without any assistance or with a little help.

You have multiple myeloma that can be measured.

Exclusion Criteria

Your body has a very low number of a type of white blood cell called neutrophils.

Screen laboratory results as follows, or any other clinically significant abnormalities in screen laboratory values that would render a participant unsuitable for inclusion

Your total bilirubin levels are higher than 1.3 times the upper limit of normal.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Dose Escalation

Part 1 involves a 3+3 dose-escalation scheme to determine the maximum-tolerated dose (MTD) during repeated 28-day treatment cycles

28 days per cycle

Treatment

Part 2 involves administering the recommended Phase 2 dose (RP2D) of ION251 for further assessments in safety, tolerability, and anti-myeloma activity

28 days per cycle

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

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.

Participant Groups

Group I: ION251 Experimental Treatment 1 Intervention

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.

Who Is Running the Clinical Trial?

Ionis Pharmaceuticals, Inc.

Lead Sponsor

Trials

151

Recruited

27,800+

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