Descartes-08 treatment for children, teens, and young adults with select autoimmune diseases

Descartes-08 for Children, Adolescents and Young Adults With Childhood-onset Systemic Lupus Erythematosus, ANCA-associated Vasculitis, Juvenile Myasthenia Gravis, and Juvenile Dermatomyositis

This trial will test whether Descartes-08 is safe and can help control disease in children, adolescents, and young adults with childhood-onset lupus, ANCA-associated vasculitis, juvenile myasthenia gravis, or juvenile dermatomyositis.

Quick facts

• Phase: Phase1; Phase2
• Study type: Interventional
• Enrollment: 50 (estimated)
• Ages: 12 Years and up
• Sex: All
• Sponsor: Cartesian Therapeutics (Industry-sponsored)
• Drugs / interventions: chemotherapy
• Locations: 2 sites (Washington D.C., District of Columbia and 1 other locations)
• Trial ID: NCT07089121 on ClinicalTrials.gov

What this trial studies

This is a Phase 1/2 interventional trial testing Descartes-08 in patients aged 12 and older with childhood-onset systemic lupus erythematosus, ANCA-associated vasculitis, juvenile myasthenia gravis, or juvenile dermatomyositis. The trial focuses on safety and tolerability while also looking for early signs of clinical benefit in participants with moderate disease and a history of systemic treatment. Participants must meet specific laboratory and clinical criteria and provide informed consent (or parental/guardian consent as appropriate). Study visits will occur at participating clinical sites in Washington, D.C. and St. Louis and will include medical monitoring and laboratory testing.

Who should consider this trial

Good fit: Ideal candidates are people aged 12 or older with a confirmed diagnosis of childhood-onset lupus, ANCA-associated vasculitis, juvenile myasthenia gravis, or juvenile dermatomyositis who have moderate disease and prior systemic treatment and can provide informed consent.

Not a fit: Patients with mild disease, uncontrolled major chronic illnesses, abnormal coagulation tests or certain anticoagulation needs, or diagnoses other than the listed conditions would not be eligible and are unlikely to benefit from this protocol.

Why it matters

Potential benefit: If successful, Descartes-08 could reduce disease activity and lower the need for other immunosuppressive treatments in these pediatric and young adult autoimmune conditions.

How similar studies have performed: Early-phase cell-based and advanced biologic therapies for autoimmune diseases have shown promising signals in small studies, but these approaches remain experimental and not yet proven in larger trials.

Eligibility criteria

Inclusion Criteria:

* At least age 12
* definitive diagnosis of childhood-onset systemic lupus erythematous, juvenile Myasthenie gravis, juvenile dermatomyositis and AAV
* Signs and symptoms of moderate disease
* History of systemic treatment
* Parent/Guardian/Patient must be able to give written informed consent

Exclusion Criteria:

* Major chronic illness that is not well managed at the time of study entry and in the opinion of the investigator may increase the risk to the patient;
* Abnormal PT/INR or PTT increased \> 1.5-fold or patient is on anticoagulation therapy (except in cases of elevated PTT with documented lupus anticoagulant; or in patients who have been on stable doses of anticoagulation therapy for more than 6 months of VTE diagnosis; or in patients on stable doses of anticoagulation therapy for at least 8 weeks of atrial fibrillation diagnosis; these conditions will not be exclusionary unless, in the investigator's opinion, they make participation in the study unsafe);
* ANC \< 1000 cells/microliter ;
* Hemoglobin \< 8.0 g/dL ;
* Platelets \< 50,000/mm3 (NOTE: platelet transfusions are permissible);
* ALT and/or AST with GGT ≥ 3× upper limit of normal
* Creatine Clearance less than 30mL/min /1.73 m2;
* History of primary immunodeficiency, organ, or allogeneic bone marrow transplant;
* Patients must be seronegative for hepatitis B surface antigen;
* Patients must be seronegative for hepatitis C antibody. If hepatitis C antibody test is positive, then patients must be tested for the presence of viremia by RT-PCR and must be HCV RNA negative;
* History of positive HIV or positive HIV at screening;
* Active tuberculosis or positive QuantiFERON test at screening;
* Any other laboratory abnormality that, in the opinion of the investigator, may jeopardize the subject's ability to participate in the study; 23. Any active significant cardiac or pulmonary disease not related to the primary indication as determined by principal investigator and medical monitor Note: Patients with asthma and COPD controlled with inhaled medications are allowed; 24. Any arterial or venous thromboembolic events in the past 3 months; 25. History of malignancy that required treatment in the past 3 years except for successfully-treated squamous cell and/or basal cell carcinoma of the skin and/or breast or colon cancer that is surgically removed and did not require adjuvant chemotherapy or radiotherapy; 26. Treatment with any investigational agent within 4 weeks of screening or 5 half-lives of the investigational drug (whichever is longer); 27. Receipt of a live vaccination within 4 weeks prior to baseline (Day 1) or intent to receive live vaccination during the study (Note: mRNA-based vaccines such as those against SARS-CoV-2 are not considered live; likewise, the Janssen Covid-19 vaccine is not live); 28. History of significant recurrent infections or any active infection that may interfere with the patient's participation in the opinion of the investigator; 29. Any known psychiatric illness that may interfere with the patient's participation in the study in the opinion of the investigator.

Where this trial is running

Washington D.C., District of Columbia and 1 other locations

• H01- Children's National Hospital — Washington D.C., District of Columbia, United States (Recruiting)
• H03- Washington University School of Medicine — St Louis, Missouri, United States (Recruiting)

Study contacts

• Study coordinator: Cartesian Clinical Trials

Conditions Childhood-onset Systemic Lupus Erythematous; ANCA-Associated Vasculitis; Juvenile Myasthenia Gravis; Juvenile Dermatomyositis