Stem Cell Treatment Versus Best Available Drugs for Hard-to-Treat Multiple Sclerosis

Plain-English translation of NCT04047628 on ClinicalTrials.gov ↗ · Source last updated January 2026 · Translation generated June 2026 · How we translate trials

Phase 3 — Testing in thousands of people, comparing the treatment against what doctors currently use. This is the last big step before approval.

What is this trial?

This trial tests whether a stem cell transplant procedure can help people with multiple sclerosis whose disease has continued to worsen despite taking strong medications. The study compares autologous hematopoietic stem cell transplantation—a treatment that uses your own stem cells to reset your immune system—against the best available high-strength medications currently approved for MS. Researchers want to find out if this procedure can stop disease activity better than continuing with medication alone.

Multiple sclerosis is an autoimmune disease where the body's immune system attacks nerve fibers in the brain and spinal cord, causing relapses and worsening symptoms over time. While many medications exist to treat MS, some people continue having disease activity despite taking these drugs, and researchers are exploring whether a stem cell transplant might help people whose MS hasn't responded to standard treatment.

Do you qualify?

You likely qualify if…

you are between 18 and 55 years old
you have been diagnosed with relapsing multiple sclerosis according to current diagnostic criteria
you have had at least 2 episodes of MS disease activity in the past 3 years, with at least one occurring in the past year
you have already tried at least one strong FDA-approved MS medication for at least 1 month and still had disease activity
you are a candidate for at least one of several high-strength MS treatments (cladribine, natalizumab, alemtuzumab, ocrelizumab, ofatumumab, ublituximab, or rituximab)
you have completed your COVID-19 vaccination series

You likely don't qualify if…

you have a disability score (EDSS) higher than 6.0, indicating more severe neurological impairment
you have previously been treated with one of the candidate high-strength medications in the past 12 months
you have a medical reason that would make you unsafe to receive one of the candidate high-strength medications
you have active or untreated infection or certain other serious medical conditions that would make transplantation unsafe

What participation looks like

If you join this study, you will be randomly assigned to either receive the stem cell transplant procedure or continue with one of the best available MS medications. The stem cell treatment involves collecting your own stem cells from your blood, giving you high-dose chemotherapy to suppress your immune system, and then reinfusing your stem cells. The entire study lasts 6 years, with visits to monitor your MS symptoms, MRI scans of your brain and spinal cord, and blood tests to track your disease activity and overall health.

AI-generated summary from trial data · Jun 10, 2026 · Not medical advice

Trial locations(22 sites)

United States

Stanford Multiple Sclerosis Center, Palo Alto, California

Rocky Mountain Multiple Sclerosis Center, University of Colorado School of Medicine, Aurora, Colorado

Northwestern University, Evanston, Illinois

University of Massachusetts Memorial Medical Center, Worcester, Massachusetts

University of Minnesota Multiple Sclerosis Center, Minneapolis, Minnesota