Treatment of Participants With Primary or Secondary Progressive Multiple Sclerosis
A Phase 1/2a, Open-Label, Dose-Escalation Study to Evaluate the Safety and Preliminary Efficacy of TRX319 in Subjects With Primary or Secondary Progressive Multiple Sclerosis
Who can join
As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.
- Age
- 18 Years to 65 Years
- Sex
- All sexes
- Healthy volunteers
- Not accepted
- Compensation
- Not stated
Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.
What to expect
No one can promise a trial will help you personally — that is what the research is trying to find out.
What participation involves
Not stated in the study description — ask the study team about visits and procedures.
- Compensation
- Not stated
- Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
- Participants are monitored closely by the research team throughout the study.
- Some participants may receive access to an investigational treatment, which is still being studied.
- Your participation helps researchers learn whether this approach could help future patients.
About this study
In plain language
This study tests TRX319 in adults ages 18 to 65 with primary or secondary progressive multiple sclerosis (MS), forms of MS that get worse over time. It checks safety, the best dose, and whether giving bendamustine first helps TRX319 work better. Participants may receive TRX319 up to 3 times over 1 year, with blood tests, MRI scans, and spinal fluid tests.
The goal of this clinical trial is to treat male and female participants with two types of Multiple Sclerosis (MS) called primary progressive or secondary progressive MS. The main questions the trial aims to answer are the following: * Is TRX319 safe when administered to patients with progressive forms of MS? * At what dose does TRX319 work the best to treat participants with primary and or secondary progressive MS? * Is pre-conditioning (with Bendamustine) needed to allow TRX319 to better treat participants with primary and/or secondary progressive MS? Participants will be asked to be on study for up 1 year and may receive up to 3 total administrations of TRX319. While on study, participants will have blood tests and other assessments (MRI scans and lumbar punctures) done to understand the safety of TRX319 and how it may benefit their multiple sclerosis.
Compensation is read from the study's own description and may be missing — always confirm with the study team.
Eligibility
Who can join
- Have a confirmed diagnosis of primary or secondary progressive MS.
- Have a score of 2.5 to 6.5 on the EDSS, a scale that measures disability from MS.
- Have MS-related disability that has gotten worse in the past 2 years.
- Have spinal fluid tests showing certain signs of immune system activity.
- Be 18 to 65 years old and have organs that work well enough for the study.
- Use birth control during the study. Those who could become pregnant must have a negative pregnancy test.
- Have antibodies against the chickenpox virus, or finish vaccination at least 4 weeks before TRX319.
- Be able to understand and agree to the study, complete all visits and tests, and avoid donating blood for 1 year after TRX319.
Who can't join
- Have MS that is stable on treatment, had an MS flare-up in the past year, or have another disease that explains the first nerve-damage episode.
- Have ever received CAR-T cell treatment or gene therapy, or received mitoxantrone, cladribine, other chemotherapy, or alemtuzumab in the past 2 years.
- Received CD20-lowering antibody drugs within 3 months, or BTK inhibitors or S1P drugs within 1 month of TRX319. These drugs affect the immune system.
- Received or plan to receive a live vaccine within 4 weeks before TRX319, or another vaccine within 2 weeks before it.
- Have active hepatitis B or C, HIV, a history of the brain infection PML, or tuberculosis covered by the study's restrictions, including inactive tuberculosis.
- Have a genetic immune system disorder, had the spleen removed, have significant heart disease or poor heart function, or have current or past cancer.
- Have had an organ or donor stem cell transplant, received blood products within 3 years, or had major surgery within 4 weeks before TRX319 or plan it within 4 weeks after.
- Are pregnant, breastfeeding, or planning pregnancy; have severe allergies or intolerance to TRX319 ingredients; or have seizures or another condition the study doctor considers unsafe or likely to interfere with the study.
Simplified for readability. The official criteria below are what the study team uses.
Official eligibility criteria
Inclusion Criteria: 1. Clinical diagnosis of MS with evidence of PPMS or SPMS according to 2025 McDonald criteria. 2. Expanded Disability Status Scale (EDSS) range ≥ 2.5 to ≤ 6.5. 3. Evidence of clinical disability progression within 2 years prior to enrollment. 4. Documented presence of CSF-restricted OCBs and/or elevated IgG index and/or κ free light chain. 5. Males and females ≥ 18 and ≤ 65 years of age at time of consent. 6. Evidence of adequate organ function 7. Women of child bearing potential have a negative pregnancy test at screening. 8. Contraceptive use by all participants while on study. 9. Participants must be able to understand, consent, and be willing and able to complete all specified procedures and visits. 10. Positive varicella zoster virus titer. Participants who test seronegative for varicella zoster virus IgG antibodies need to complete vaccination ≥ 4 weeks prior to TRX319 infusion. 11. Participants must be willing to refrain from donating blood for 1 year after TRX319 infusion. Exclusion Criteria: 1. MS clinical stability on disease modifying therapy. 2. Clinical relapse of MS in the 1 year prior to study entry. 3. Diseases other than MS to explain the first demyelinating event, including aquaporin 4 IgG or myelin oligodendrocyte glycoprotein-IgG seropositivity. 4. Prior treatment with CAR-T or gene therapy product directed at any target. 5. Prior treatment with mitoxantrone, cladribine (or other chemotherapies), or alemtuzumab within 2 years prior to TRX319 dose. 6. Prior treatment with CD20-depleting antibodies within 3 months and prior treatment with Bruton's tyrosine kinase inhibitor (BTKi) and sphingosine 1 phosphate (S1P) modulators within 1 month of TRX319 dose. 7. Plan to or have received live, attenuated vaccines less than 4 weeks (28 days) prior to TRX319 infusion, and other vaccines less than 2 weeks (14 days) prior to TRX319 infusion. 8. Serologic status reflecting active hepatitis B or C infection. 9. Positive serology for human immunodeficiency virus (HIV). 10. History of progressive multifocal leukoencephalopathy. 11. Untreated active, or active with documented completed treatment but without a negative chest X-ray that shows no evidence of active tuberculosis, or latent tuberculosis. 12. Primary immunodeficiency as defined by a known genetic disorder. 13. History of splenectomy. 14. Impaired cardiac function or clinically significant cardiac disease. 15. Previous or concurrent malignancy. 16. Prior organ transplant, or allogeneic hematopoietic stem cell transplantation or recipient of peripheral blood products \< 3 years prior to TRX319 infusion. 17. Major surgery within 4 weeks prior or planned within 4 weeks after TRX319 administration. 18. History of any other neurologic disorder or medical condition the Investigator considers would increase the risk for the participant, including seizure disorders. 19. Life-threatening allergies, hypersensitivity, or documented intolerance to TRX319 drug product excipients. 20. Subjects that are pregnant, breast feeding or aim to become pregnant during the study period (Subjects must agree to use a highly effective method of contraception). 21. Serious and/or uncontrolled medical condition that, in the Investigator's judgment, would cause unacceptable safety risk, interfere with study procedures or results, or compromise compliance with the protocol.
Study sites (8)
CA
- UC San Diego · La Jolla, CA 92037Vicky Vardouniotis · Study contact · 858-246-1249 · vvardouniotis@health.ucsd.eduJennifer Graves, MD · Principal investigatorMore trials in La Jolla
KS
- University of Kansas Medical Center · Kansas City, KS 66160Sharon Lynch, MD · Study contact · 913-588-6980 · SLYNCH@kumc.eduMore trials in Kansas City
MN
- Mayo Clinic · Rochester, MN 55912Lisa Roemer · Study contact · 507-293-9754 · roemer.lisa@mayo.eduMichel Toledano, MD · Principal investigatorMore trials in Rochester
MO
- Washington University, St. Louis · St Louis, MO 63110Susan Sommer · Study contact · 314-362-3293 · foxs@wustl.eduMore trials in St Louis
NC
- Duke University · Durham, NC 27710Jessica Franceschelli Coordinator · Study contact · 919-613-1107 · jessica.franceschelli@duke.eduSuma Shah · Principal investigatorMore trials in Durham
NY
- Mt. Sinai · New York, NY 10029Sarah Myoung, MBS · Study contact · 2122413391 ext. 43391Fred Lublin, MD · Principal investigatorMore trials in New York
OH
- University of Cincinnati · Cincinnati, OH 45219Kristine Suder Coordinator · Study contact · 513-558-3920 · suderkl@ucmail.uc.eduMore trials in Cincinnati
WA
- Providence Swedish · Seattle, WA 98122Isabella Bueno · Study contact · 206-320-3070 · isabella.bueno1@swedish.orgPavle Repovic, MD, PhD · Principal investigatorMore trials in Seattle