A Phase 1 Study of SYNCAR-001 + STK-009 Without Conditioning Chemotherapy (Lymphodepletion) in Subjects With Severe, Refractory Systemic Autoimmune Rheumatic Disease

NCT06544330 | Active Not Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: STK-009-201
• Study Type: interventional
• Target: 48
• Locations: 1 country
• Sites: 5

Brief Summary

This is a phase 1 study of SYNCAR-001 + STK-009 in patients with severe, refractory systemic autoimmune rheumatic disease.

Conditions

Systemic Lupus Erythematosus; Lupus Nephritis; Systemic Sclerosis

Keywords

CAR T; CD19-CAR T; Chimeric antigen receptor; IL-2

Outcome Measures

Primary Outcomes (2)

• Dose-Limiting Toxicities (DLTs)

  Incidence of adverse events (AEs) meeting protocol defined DLT criteria in the dose escalation phase of the study.

  Up to 28 days after SYNCAR-001 infusion

• Adverse Events

  Incidence and severity of AEs including treatment-emergent AEs and serious AEs.

  Up to 96 weeks after SYNCAR-001 infusion

Secondary Outcomes (11)

• Remission rate per Definition of Remission in SLE (DORIS)

  Up to 96 weeks after SYNCAR-001 infusion

• Lupus Low Disease Activity State (LLDAS) attainment rate

  Up to 96 weeks after SYNCAR-001infusion

• Change over time in Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K)

  Up to 96 weeks after SYNCAR-001 infusion

• Change over time in British Isles Lupus Activity Group (BILAG) score

  Up to 96 weeks after SYNCAR-001 infusion

• Change over time in levels of SLE and SSc serum autoantibodies

  Up to 96 weeks after SYNCAR-001 infusion

Study Arms (1)

SYNCAR-001 + STK-009

EXPERIMENTAL

Dose escalation: A single fixed dose of autologous SYNCAR-001 CAR-T intravenously (IV) will be administered in combination with ascending doses of STK-009 subcutaneously (SC) in non-lymphodepleted patients. Dose expansion: A single fixed dose of autologous SYNCAR-001 CAR-T IV will be administered in combination with STK-009 SC at the RP2D in non-lymphodepleted patients.

Drug: SYNCAR-001; Drug: STK-009

Interventions

SYNCAR-001 DRUG

SYNCAR-001 is an autologous CD19-targeted CAR-T with co-expression of hoRb

SYNCAR-001 + STK-009

STK-009 DRUG

STK-009 is a human orthogonal IL-2 cytokine selective for SYNCAR-001 CAR-T cells expressing hoRb

SYNCAR-001 + STK-009

Eligibility Criteria

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

You may qualify if:

• Age ≥18 years at screening.
• Clinical diagnosis of SLE according to the 2019 European League Rheumatism EULAR/ACR classification criteria.
• Subject must be positive for at least one of the following at screening: Anti-dsDNA (above the upper limit of normal \[ULN\]); or anti-Sm (above the ULN); or anti-Chromatin (above the ULN).
• Subjects with active, severe, non-renal SLE or subjects with active proliferative LN
• Classified as SSc according to the ACR/EULAR classification criteria.
• Diffuse cutaneous SSc (dcSSc) or SSc-associated ILD (SSc-ILD; significant or progressive).

You may not qualify if:

• History of or active central nervous system manifestations of autoimmune disease.
• Prior treatment with anti-CD19 adoptive T cell therapy, or any prior gene therapy product (e.g., CAR T cell therapy).
• Rapidly progressive glomerulonephritis.
• End stage renal failure requiring dialysis or most recent renal biopsy with purely chronic lesions (Class III\[C\], IV-S\[C\], or IV-G\[C\]) if isolated renal disease.
• FVC \<50% of predicted or DLCO \<40% of predicted.
• Pulmonary arterial hypertension (PAH) requiring PAH-specific treatment.
• Other protocol-defined criteria apply.

Sponsors & Collaborators

• Synthekine: lead

Study Sites (5)

HonorHealth Research Institute

Scottsdale, Arizona, 85258, United States

Location

Hackensack University Medical Center

Hackensack, New Jersey, 07601, United States

Location

Feinstein Institutes for Medical Research

Manhasset, New York, 11030, United States

Location

The Cleveland Clinic Foundation

Cleveland, Ohio, 44195, United States

Location

Ohio State University Wexner Medical Center

Columbus, Ohio, 43210, United States

Location

MeSH Terms

Conditions

Lupus Erythematosus, Systemic; Lupus Nephritis; Scleroderma, Systemic

Condition Hierarchy (Ancestors)

Connective Tissue Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases; Glomerulonephritis; Nephritis; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Skin Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 25, 2024
• First Posted: August 9, 2024
• Study Start: April 29, 2025
• Primary Completion (Estimated): April 1, 2028
• Study Completion (Estimated): April 1, 2041
• Last Updated: November 4, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (5)