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. |
|
|
Experimental Part 2: Decartes-08 infusions once weekly for 6 weeks |
Descartes-08 infusions at the maximum tolerated dose level from Part 1. |
|
Recruiting Locations
Children's National and nearby locations
H01- Children's National Hospital
Washington D.C., District of Columbia 20010
Washington D.C., District of Columbia 20010
More Details
- NCT ID
- NCT07089121
- Status
- Recruiting
- Sponsor
- Cartesian Therapeutics