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

NCT07089121 | Recruiting Phase 1 FDA Drug

• 1 other identifier: HELIOS-001
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 2

Brief Summary

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

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

Outcome Measures

Primary Outcomes (2)

• Maximum tolerated dose in Part 1, type and frequency of treatment related SAE's in Part 2

  The primary endpoint for Part-1 is the Maximum Tolerated Dose (MTD), defined as the Dose Level at which no more than 20% of the patients treated have shown Dose-Limiting Toxicity (DLT), i.e. at which 3 patients received all 6 weekly infusions without a DLT by Day 50; or 6 patients received 3 weekly infusions with no more than 1 patient having a DLT by Day 50. The primary endpoint for Part-2 is the type and frequency of treatment-related SAEs. This will be assessed on Days 22, 50 and Months 3,6,9,12

  Days 22 and 50 for Part1 , Days 22, 50 and Months 3,6,9 and 12 for part 2

• Maximum Dose Tolerated

  The primary endpoint for Part-1 is the Maximum Tolerated Dose (MTD), defined as the Dose Level at which no more than 20% of the patients treated have shown Dose-Limiting Toxicity (DLT), i.e. at which 3 patients received all 6 weekly infusions without a DLT by Day 50; or 6 patients received 3 weekly infusions with no more than 1 patient having a DLT by Day 50. The primary endpoint for Part-2 is the type and frequency of treatment-related SAEs.

  Days 2250 for Part 1, Days 11,50, month 3 for part 2 in addition to months, 4,6,9,12

Study Arms (2)

Part 1: Decartes-08 to establish Maximum tolerated dose

EXPERIMENTAL

Intra-patient dose escalation arm with three dose levels over the course of six infusions of cell product.

Drug: Descartes-08

Part 2: Decartes-08 infusions once weekly for 6 weeks

EXPERIMENTAL

Descartes-08 infusions at the maximum tolerated dose level from Part 1.

Drug: Descartes-08

Interventions

Descartes-08 DRUG

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.

Part 1: Decartes-08 to establish Maximum tolerated dose; Part 2: Decartes-08 infusions once weekly for 6 weeks

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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;
• 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.

Sponsors & Collaborators

• Cartesian Therapeutics: lead

Study Sites (2)

H01- Children's National Hospital

Washington D.C., District of Columbia, 20010, United States

RECRUITING

H03- Washington University School of Medicine

St Louis, Missouri, 63110, United States

RECRUITING

MeSH Terms

Conditions

Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis; Dermatomyositis

Condition Hierarchy (Ancestors)

Systemic Vasculitis; Vasculitis; Vascular Diseases; Cardiovascular Diseases; Skin Diseases, Vascular; Skin Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases; Polymyositis; Myositis; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Connective Tissue Diseases

Central Study Contacts

Cartesian Clinical Trials

CONTACT

617-231-8102; trials@cartesiantx.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Part 1: establish Maximum tolerated dose Part 2: MTD to be given once weekly for 6 weeks

Study Record Dates

• First Submitted: July 20, 2025
• First Posted: July 28, 2025
• Study Start: January 14, 2026
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): December 1, 2028
• Last Updated: April 8, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)