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CD19 targeted CAR-T cells

Phase 1

SLE (Systemic Lupus) | Monoclonal antibody | Immunology |Precision BioSciences, Inc.|Last Updated: Sep 28, 2023

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
UNCONTROLLEDBiomarker
Total Trials1
Total Enrollment24
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06056921Safety and Efficacy of CD19 Targeted CAR-T Therapy for Refractory Autoimmune DiseasePHASE1 RECRUITING 24Aug 31, 2023Aug 31, 2026Sep 28, 20231 China
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Study Endpoints
Primary Endpoints
Incidence of Adverse events after CD19 CAR-T cells infusion [Safety and Tolerability]
1 month

Therapy-related adverse events were recorded and assessed according to the National Cancer Institute's Common Terminology Criteria for Adverse Events (CTCAE, Version 5.0)

Obtain the maximum tolerated dose of CD19 CAR-T cells[Safety and Tolerability]
1month

Dose-limiting toxicity after cell infusion

Efficacy of CAR-T cell preparations in Refractory systemic lupus erythematosus [Effectiveness]
3 months

The efficacy is assessed by SRI-4 response rate at 3M,SRI-4 response rate at 3M means Percentage of patients who met all three of the following conditions at 3M after refusion: 1. The SLEDAI-2K rating dropped by at least 4 points 2. No deterioration in PGA scores on a 3-point scale (deterioration is defined as an increase of ≥0.3 points from baseline score) 3. No new manifestations of organ system involvement (new system involvement is defined as one or more new symptoms of BILAG grade A or two or more symptoms of BILAG B)

Efficacy of CAR-T cell preparations in Refractory Systemic scleroderma [Effectiveness]
3 months

The efficacy is assessed by Change value of The EULAR Sjögren's syndrome disease activity index(ESSDAI)score at 3M

Efficacy of CAR-T cell preparations in Refractory Sjogren's syndrome [Effectiveness]
3 months

The efficacy is assessed by Change value of modified Rondan skin score(mRSS) at 3M; The evaluator palpated 17 areas of the patient's body. The skin thickness of each area was calculated on a scale of 0 to 3 points. The total score was 51 points, 0 was normal skin with fine lines and no skin thickening. 1 is divided into mild skin thickening, the inspector can easily use two fingers to pinch the skin to form wrinkles, skin fine lines can also exist; 2: moderate skin thickening, more difficult to pinch the skin to form wrinkles, skin fine lines disappear; 3 is classified as severe skin thickening, unable to pinch the skin to form wrinkles.

Efficacy of CAR-T cell preparations in Refractory dermatomyositis [Effectiveness]
3 months

The efficacy is assessed by CSM Total Improvement Score (TIS) 20/40/60 improvement rate at 3M;3M CSM Total Improvement Score (TIS) 20/40/60 Improvement rate: defined as the proportion of patients with a TIS score of 20-40,40-60, and ≥60 at 3M posttransfusion

Efficacy of CAR-T cell preparations in Refractory The antineutrophil cytoplasmic antibody-associated vasculitides [Effectiveness]
3 months

The efficacy is assessed by response rate at 3M; Defined as the percentage of patients whose BVAS score decreased to 0 at 3M after reinfusion in the absence of glucocorticoids and other therapeutic agents

Secondary Endpoints
AUCS of CD19 CAR-T cells [Cell dynamics]
3 months
CMAX of CD19 CAR-T cells [Cell dynamics]
3 months
TMAX of CD19 CAR-T cells[Cell dynamics]
3 months
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
CD19-targeted CAR-TEXPERIMENTALCD19 targeted CAR-T cells treat
Interventions
NameTypeDescription
CD19 targeted CAR-T cellsBIOLOGICALA single infusion of CD19 CAR-T cells will be administered intravenously
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: 1. ≥18 years old, male or female; 2. diagnosed with one of the following diseases: * Systemic Lupus Erythematosus (SLE), EULAR/ACR 2019 standard; * Sjogren's Syndrome (SS), 2016 ACR/EULAR standard; * Systemic Scleroderma (SSc), 2013 ACR/EULAR Standards; * Dermatomyo...

Countries:China
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Recent Changes (Last 90 Days)
LOWMay 24, 2026NCT06056921studyFirstPostDate: changed