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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06655896




Registration number
NCT06655896
Ethics application status
Date submitted
22/10/2024
Date registered
24/10/2024

Titles & IDs
Public title
Phase 2 Study Evaluating Rapcabtagene Autoleucel in Participants With Diffuse Cutaneous Systemic Sclerosis
Scientific title
A Phase 2, Multi-part, Randomized, Open-label, Assessor-blinded, Active-controlled, Study to Evaluate the Efficacy and Safety of Rapcabtagene Autoleucel Versus Rituximab Treatment in Participants With Severe Refractory Diffuse Cutaneous Systemic Sclerosis
Secondary ID [1] 0 0
2023-510380-34-00
Secondary ID [2] 0 0
CYTB323K12201
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Scleroderma, Diffuse 0 0
Condition category
Condition code
Inflammatory and Immune System 0 0 0 0
Autoimmune diseases
Skin 0 0 0 0
Dermatological conditions
Skin 0 0 0 0
Other skin conditions

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Other - rapcabtagene autoleucel
Treatment: Other - rituximab

Experimental: rapcabtagene autoleucel arm - rapcabtagene autoleucel

Active comparator: rituximab arm - rituximab


Treatment: Other: rapcabtagene autoleucel
single infusion of rapcabtagene autoleucel

Treatment: Other: rituximab
rituximab intravenous infusion (i.v.) as per protocol
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Achievement of a treatment response as per the Revised Composite Response Index in Systemic Sclerosis 50 (rCRISS50) definition at Week 52.
Timepoint [1] 0 0
Week 52
Secondary outcome [1] 0 0
Change from baseline in Forced Vital Capacity (FVC)% predicted at Week 52
Timepoint [1] 0 0
Baseline, Week 52
Secondary outcome [2] 0 0
Change from baseline in modified Rodnan Skin Score (mRSS) at Week 52.
Timepoint [2] 0 0
Baseline, Week 52
Secondary outcome [3] 0 0
Change from baseline in Health Assessment Questionnaire Disability Index (HAQ-DI) at Week 52.
Timepoint [3] 0 0
Baseline, Week 52
Secondary outcome [4] 0 0
Achievement of Week 156 progression-free survival with respect to disease progression criteria.
Timepoint [4] 0 0
Week 156
Secondary outcome [5] 0 0
Incidence and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)
Timepoint [5] 0 0
Week 156

Eligibility
Key inclusion criteria
1. Participant must fulfill the 2013 American College of Rheumatology/ European League Against Rheumatism classification criteria for systemic sclerosis and meet the diffuse cutaneous SSc (dcSSc) subset classification according to LeRoy.
2. Disease onset from the first non-Raynaud symptoms attributable to SSc (e.g., puffy hands, scleroderma, digital ulcers, arthralgia, dyspnea) within 7 years prior to the Screening visit.
3. Severe, progressive systemic sclerosis disease defined by at least one of the following:

* Progressive systemic sclerosis-associated interstitial lung disease
* Severe, progressive systemic sclerosis skin disease
* Clinically significant systemic sclerosis-associated cardiac involvement at Screening
Minimum age
18 Years
Maximum age
65 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Any condition during Screening that could prevent a complete washout of medications as required per protocol or could otherwise make the participant ineligible for anti-CD19 CAR-T therapy and further participation in the study, as judged by the Investigator.
2. Participants with history of hypersensitivity to excipients in rapcabtagene autoleucel or to rituximab.
3. Any participant for whom treatment with rituximab is clinically inappropriate in the opinion of the investigator.
4. Any medical conditions that are not related to SSc that, in the opinion of the Investigator, would jeopardize the ability of the participant to tolerate lymphodepletion and anti-CD19 CAR-T cell therapy.
5. Rheumatic disease other than dcSSc, (except secondary Sjogren's syndrome or scleroderma myopathy), limited cutaneous systemic sclerosis (lcSSc) or sine scleroderma at Screening.
6. Participants with pre-existing pulmonary hypertension.
7. Significant renal pathology at Screening, including:

* Active SSc renal crisis
* Confirmed diagnosis of glomerulonephritis
8. Participants with uncontrolled stage II hypertension at Screening.
9. Vaccination with live attenuated vaccines within 6 weeks prior to randomization.

Other protocol-defined inclusion/exclusion criteria may apply.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?


The people assessing the outcomes
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
29/10/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
17/12/2030
Actual
Sample size
Target
86
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
Novartis Investigative Site - Darlinghurst
Recruitment postcode(s) [1] 0 0
2010 - Darlinghurst
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Illinois
Country [2] 0 0
United States of America
State/province [2] 0 0
Iowa
Country [3] 0 0
France
State/province [3] 0 0
Bordeaux Cedex
Country [4] 0 0
France
State/province [4] 0 0
Rennes
Country [5] 0 0
Germany
State/province [5] 0 0
Jena
Country [6] 0 0
Israel
State/province [6] 0 0
Haifa
Country [7] 0 0
Israel
State/province [7] 0 0
Ramat Gan
Country [8] 0 0
Italy
State/province [8] 0 0
AN
Country [9] 0 0
Italy
State/province [9] 0 0
GE
Country [10] 0 0
Italy
State/province [10] 0 0
RM
Country [11] 0 0
Japan
State/province [11] 0 0
Hokkaido
Country [12] 0 0
Japan
State/province [12] 0 0
Ishikawa
Country [13] 0 0
Singapore
State/province [13] 0 0
Singapore
Country [14] 0 0
Spain
State/province [14] 0 0
Catalunya
Country [15] 0 0
Spain
State/province [15] 0 0
Barcelona
Country [16] 0 0
Spain
State/province [16] 0 0
Madrid

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Novartis Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Novartis Pharmaceuticals
Address 0 0
Novartis Pharmaceuticals
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Novartis Pharmaceuticals
Address 0 0
Country 0 0
Phone 0 0
1-888-669-6682
Fax 0 0
Email 0 0
novartis.email@novartis.com
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
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.
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT06655896