A study to assess safety, cellular kinetics and exploratory efficacy of rapcabtagene autoleucel in rheumatoid arthritis and Sjogren’s disease

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Novartis Pharma AG

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

Trial duration

20 Jun 2025 → ongoing

Decision date (initial)

2025-05-06

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Novartis Pharma AG

External identifiers

EU CT number

2024-514596-18-00

WHO UTN

U1111-1325-1245

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Pharmacokinetic

To assess safety of rapcabtagene autoleucel (XX) in participants with difficult-to-treat rheumatoid arthritis and severe refractory Sjogren's Disease with organ involvement.

Secondary objectives 1

1. To characterize the in vivo cellular kinetics (pharmacokinetics, PK) of rapcabtagene autoleucel in peripheral blood by quantitative polymerase chain reaction (qPCR)

Conditions and MedDRA coding

Difficult to treat rheumatoid arthritis

Regulatory references

Scientific advice from competent authorities

Paul-Ehrlich-Institut

Plan to share IPD

Yes

IPD plan description

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 9

1. Signed informed consent must be obtained prior to participation in the study
2. Men and women aged ≥ 18 years and ≤ 75 years at screening
3. Difficult-to-treat rheumatoid arthritis: Diagnosis of rheumatoid arthritis
4. Difficult-to-treat rheumatoid arthritis: XX
5. Difficult-to-treat rheumatoid arthritis: Treatment failure of standard of care therapies
6. Difficult-to-treat rheumatoid arthritis: Signs suggestive of active, progressive disease
7. Severe refractory Sjogren's Disease: Diagnosis of Sjogren’s disease
8. Severe refractory Sjogren's Disease: XX
9. Severe refractory Sjogren's Disease: Severe systemic, refractory disease with moderate to high disease activity

Exclusion criteria 11

1. Body Mass Index at screening of ≤17 or ≥40 kg/m2
2. Any XX that needs immediate treatment other than XX impossible
3. Clinically significant active, opportunistic, chronic or recurrent infection
4. Sexually active males unwilling to use a condom during intercourse from the time enrollment
5. Women of childbearing potential, unless they are using a highly effective method of contraception starting from the time of enrollment
6. Female participants who are pregnant, breastfeeding or intending to conceive during the course of the study
7. Inadequate organ function during screening
8. History of lymphoproliferative disease or any known malignancy or history of malignancy
9. History of bone marrow/hematopoietic stem cell or solid organ transplantation
10. Any psychiatric condition or disability making compliance with treatment or informed consent impossible
11. Any patients requiring medications prohibited by the protocol

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Safety parameters include vital signs, adverse events, laboratory parameters and ECG evaluation

Secondary endpoints 1

1. Rapcabtagene autoleucel transgene concentrations by qPCR over time in peripheral blood; cellular kinetics parameters (Cmax, AUC, Tmax, T1/2, Clast, Tlast)

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Auxiliary 3

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Novartis Pharma AG

Sponsor organisation

Novartis Pharma AG

Address

Lichtstrasse 35

City

Basel

Postcode

4056

Country

Switzerland

Scientific contact point

Organisation

Novartis Pharma AG

Contact name

Novartis Pharma Arzneimittel GmbH

Public contact point

Organisation

Novartis Pharma AG

Contact name

Novartis Pharma Arzneimittel GmbH

Third parties 11

Locations

5 EU/EEA countries · 19 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 72 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

5 submissions — initial application plus substantial / non-substantial modifications