Children's National

Descartes-08 for Children, Adolescents, and Young Adults With Autoimmune Disorders

Purpose

Safety, tolerability and efficacy of Descarte-08 in children, adolescents and young adults with childhood-onset systemic lupus erythematosus, ANCA-associated vasculitis, juvenile myasthenia gravis, and juvenile dermatomyositis

Conditions

Childhood-onset Systemic Lupus Erythematous
ANCA-Associated Vasculitis (AAV)
Juvenile Myasthenia Gravis
Juvenile Dermatomyositis

Eligibility

Eligible Ages: Over 12 Years
Eligible Sex: All
Accepts Healthy Volunteers: No

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.

Study Design

Phase: Phase 1/Phase 2
Study Type: Interventional
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Intervention Model Description: Part 1: establish Maximum tolerated dose Part 2: MTD to be given once weekly for 6 weeks
Primary Purpose: Treatment
Masking: None (Open Label)

Arm Groups

Arm	Description	Assigned Intervention
Experimental Part 1: Decartes-08 to establish Maximum tolerated dose	Intra-patient dose escalation arm with three dose levels over the course of six infusions of cell product.	Drug: Descartes-08 In part 1, three different doses will be administered to 3 participants with either disease indication to establish maximum tolerated dose In part 2, the MTD established in Part-1 will be administered as six once-weekly infusions to up to 10 participants per each of four baskets (cSLE, AAV, JDM and JMG) in an outpatient setting.
Experimental Part 2: Decartes-08 infusions once weekly for 6 weeks	Descartes-08 infusions at the maximum tolerated dose level from Part 1.	Drug: Descartes-08 In part 1, three different doses will be administered to 3 participants with either disease indication to establish maximum tolerated dose In part 2, the MTD established in Part-1 will be administered as six once-weekly infusions to up to 10 participants per each of four baskets (cSLE, AAV, JDM and JMG) in an outpatient setting.

Recruiting Locations

Children's National and nearby locations

H01- Children's National Hospital
Washington D.C., District of Columbia 20010

Contact:
Emily Miller
202-476-4130
ejmiller@childrensnational.org

More Details

NCT ID: NCT07089121
Status: Recruiting
Sponsor: Cartesian Therapeutics

Study Contact

Cartesian Clinical Trials
617-231-8102
trials@cartesiantx.com

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.