BCMA-directed mRNA CAR-T cell therapy for myasthenia gravis: exploratory biomarker analysis of a placebo-controlled phase 2b trial.
Renee R Fedak, Rachel N Ruggerie, Yufei Shan...
https://pubmed.ncbi.nlm.nih.gov/41514039Actively Recruiting
Phase 2
Age: 18Years +
All Genders
ID07391605
A Randomized Double-Blind Placebo-Controlled Study to Evaluate Efficacy, Safety, and Tolerability of Descartes-08 in Patients With Dermatomyositis and Antisynthetase Syndrome
Led by Cartesian Therapeutics · Updated on 2026-04-23
60
Participants Needed
2
Research Sites
26 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating a new treatment called Descartes-08, which uses a patient's own T-cells modified to target a specific protein (BCMA), in adults with autoantibody-mediated myositis, including antisynthetase syndrome and dermatomyositis. This phase 2 study is randomized, double-blind, and placebo-controlled to assess the treatment's effects on disease improvement, safety, and tolerability. Participants will be randomly assigned to receive either Descartes-08 or a placebo called Plasma-Lyte. The study compares these two groups to see how well Descartes-08 works and how safe it is. The treatment involves autologous T-cell therapy targeting B-cell maturation antigen, aiming to address muscle and skin involvement in these autoimmune conditions. During the study, participants will be monitored over 24 weeks for major improvement using a specific total improvement score. Researchers will assess safety, tolerability, and clinical responses. Participants must provide informed consent and comply with study visits, contraception, and vaccinations as part of the study procedures. The study excludes those with severe lung disease without muscle or skin involvement and other serious conditions, ensuring participants meet strict health criteria.
CONDITIONS
Brief Title
Descartes-08 in Autoantibody Myositis
Who Can Participate
Age: 18Years +
All Genders
Eligibility Criteria
You may qualify if you...
- Confirmed diagnosis of dermatomyositis with a probability score 65% on the 2017 EULAR/ACR criteria or antisynthetase syndrome based on ACR/EULAR classification criteria
- Dermatomyositis or antisynthetase syndrome with muscle and/or skin involvement
- Refractory or intolerance to standard therapy
- Stable background immunosuppressive therapy for at least 8 weeks
- Adequate hematologic, kidney, liver, and lung function with oxygen saturation 62% on room air
- Informed consent provided and compliance with visits, contraception, and vaccinations
You will not qualify if you...
- Isolated interstitial lung disease without muscle or skin involvement
- Severe irreversible muscle damage or advanced weakness such as being wheelchair-bound
- Interstitial lung disease requiring oxygen or with severe pulmonary impairment or pulmonary hypertension
- Other inflammatory myopathies like polymyositis, immune-mediated necrotizing myopathy, inclusion body myositis, cancer- or drug-induced myositis, or overlap myositis except Sj�f6gren's syndrome
- Other severe neuromuscular, heart, lung, or systemic autoimmune diseases requiring immunosuppression
- Significant uncontrolled chronic illnesses or psychiatric conditions interfering with participation
- Pregnancy or breastfeeding
- Recent use of prohibited immunosuppressants, biologics, investigational agents within washout periods
- Live vaccination within 4 weeks prior to study
- History of primary immunodeficiency, organ or bone marrow transplant
- Active or uncontrolled infections such as hepatitis B or C, HIV, tuberculosis, or recurrent/severe infections
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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2
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Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
screening and enrollment visit
Treatment
Duration - 24 weeks
Participants receive either Descartes-08 or placebo to evaluate efficacy, safety, and tolerability over the treatment period.
Multiple visits during the 24-week treatment period
Trial Site Locations
Total: 2 locations
1
T13
Chapel Hill, North Carolina, United States, 27599
Actively Recruiting
2
T23
Austin, Texas, United States, 78759
Actively Recruiting
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Research Team
V
Vera MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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