Rheumatoid Arthritis (RA) and Sjögren's Disease (SjD)
Conditions
Keywords
CAR-T,, YTB323,, rapcabtagene autoleucel,, D2T RA,, srSjD
Brief summary
A study to assess safety, cellular kinetics and exploratory efficacy of rapcabtagene autoleucel in rheumatoid arthritis and Sjogren's disease
Detailed description
The study is intended to assess safety, cellular kinetics and any early sign for efficacy of rapcabtagene autoleucel in participants with difficult-to-treat rheumatoid arthritis (D2T RA) and severe, refractory Sjogren's disease (srSjD) with organ involvement.
Interventions
BIOLOGICALRapcabtagene autoleucel
Single infusion of Rapcabtagene autoleucel
Sponsors
Novartis Pharmaceuticals
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
* Signed informed consent must be obtained prior to participation in the study * Men and women aged ≥ 18 years and ≤ 75 years at Screening For RA only * Diagnosis of rheumatoid arthritis * Treatment failure of standard of care therapies * Active disease For SjD only * Diagnosis of Sjogren's disease * Active disease
Exclusion criteria
* BMI at Screening of ≤17 or ≥ 40 kg/m2 * Clinically significant active, opportunistic, chronic or recurrent infection * Sexually active males unwilling to use a condom during intercourse from the time enrollment * Women of childbearing potential, unless they are using a highly effective method of contraception starting from the time of enrollment * Female participants who are pregnant, breastfeeding or intending to conceive during the course of the study * Inadequate organ function during screening * History of lymphoproliferative disease or any known malignancy or history of malignancy * History of bone marrow/hematopoietic stem cell or solid organ transplantation * Any psychiatric condition or disability making compliance with treatment or informed consent impossible Other protocol-defined inclusion/
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants with Adverse Events as a Measure of Safety and Tolerability | 24 months | Safety parameters include vital signs, adverse events, laboratory parameters and ECG evaluation |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Pharmacokinetic (PK) parameter: Cmax | 24 months | CAR transgene levels by quantitative polymerase chain reaction (qPCR) in blood (Maximum observed blood concentration Cmax). |
| PK parameter: Tmax | 24 months | CAR transgene levels by quantitative polymerase chain reaction (qPCR) in blood (Time to reach maximum concentration Tmax). |
| PK parameter: T1/2 | 24 months | CAR transgene levels by quantitative polymerase chain reaction (qPCR) in blood (Terminal elimination half-life T1/2). |
| PK parameter: Clast | 24 months | CAR transgene levels by quantitative polymerase chain reaction (qPCR) in blood (Last measurable concentration Clast). |
| PK parameter: Tlast | 24 months | CAR transgene levels by quantitative polymerase chain reaction (qPCR) in blood (Time to reach last measurable concentration Tlast). |
| PK parameter: AUC | 24 months | CAR transgene levels by quantitative polymerase chain reaction (qPCR) in blood (Area under plasma concentration -time AUC). |
Countries
France, Germany, Singapore, Spain
Outcome results
None listed