Pfizer COVID-19 vaccine appointments are available to our patients. Sign up for Connect today to schedule your vaccination.

A Phase 1 Study of ION251 Administered by Intravenous Infusion to Patients with Relapsed/Refractory Multiple Myeloma

Clinical Trial Details

ION251 is an investigational drug that inhibits IRF4 (Interferon Regulatory Factor 4), a key regulator of multiple genes controlling the survival of multiple myeloma (MM) tumor cells. This protein is overexpressed in MM, and even limited reduction of IRF4 can lead to rapid MM tumor cell death.  

ION251 is considered investigational, meaning the drug has not been approved by the U.S. Food and Drug Administration (FDA) or other regulatory agencies for use outside research studies like this one and is therefore considered experimental. This is the first time this drug is being tested in humans.

The primary purpose of this study is to evaluate the safety and tolerability of multiple doses of ION251 when administered by intravenous (IV) infusion. The secondary purpose is to measure the amount of study drug present in the blood at various points in time and to see how long the study drug stays in the body.

This study has two parts. Part 1 is intended to find the maximum tolerated dose and recommended phase 2 dose and Part 2 consists of dosing subjects at the recommended phase 2 dose found in Part 1 for a period of time to assess longer term safety, tolerability, anti-tumor activity, and IRF4 expression in tumor and non-tumor cells.

Currently, we are enrolling Part 1 of the study. In Part 1, participants will be assigned to one of seven (7) sequential dose groups, with doses of ION251 starting at 100 mg and up to 940 mg. These doses can change at any time depending on the safety information discovered during the duration of the study.

In Part 1, each treatment cycle will last 28 days. ION251 will be administered by 2-hour intravenous infusions on day 1, 4, 8, 11, 15, and 22 in the first cycle and weekly after the first cycle. Safety and tolerability will be assessed at each dose by collecting labs and physical examinations. Some other procedures that participants will have to complete throughout the study include imaging, electrocardiograms, and bone marrow biopsies.

Treatment will be given in repeated 28-day cycles and will continue until either unacceptable toxicity or disease progression, judgement of the treating physician, or participants  choose to withdraw consent at any point in the study.

Key Eligibility: 

Inclusion Criteria:

  • Over 18 years of age at the time of informed consent
  • Diagnosis of multiple myeloma (MM)
  • In need of systemic treatment for MM and either is refractory to, or is not otherwise a candidate for other established therapies. Refractory to treatment is defined as documented MM disease progression while on or within 60 days from the last dose (LD) of treatment

Exclusion Criteria:

  • Abnormal laboratory values
  • History of or current plasma cell leukemia, POEMS (polyneuropathy, organomegaly, endocrinopathy, and skin changes) syndrome, myelodysplastic syndrome or a myeloproliferative neoplasm
  • Uncontrolled hypertension (systolic pressure ≥ 160 mm of mercury (mm Hg) and/or diastolic pressure ≥ 100 mm Hg)
  • Presence of a bleeding disorder or an underlying disease state associated with active bleeding.

Detailed eligibility reviewed when you contact the study team

Study contact by location

Upper East Side - Manhattan


Kathleen Pogonowski, RN
(646) 962-6500

Primary Investigator(s)

Protocol ID(s)

Weill Cornell Medicine IRB #:






Open to Enrollment

Age Group