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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06617793




Registration number
NCT06617793
Ethics application status
Date submitted
26/09/2024
Date registered
27/09/2024

Titles & IDs
Public title
An Open-label Study to Assess the Safety, Efficacy, and Cellular Kinetics of YTB323 in Relapsing Multiple Sclerosis
Scientific title
An Open-label, Multi-center, Phase 1/2 Study to Assess Safety, Efficacy, and Cellular Kinetics of YTB323 in Participants With Relapsing Multiple Sclerosis With Breakthrough Disease Activity During Previous Treatment With a Highly Efficacious Therapy
Secondary ID [1] 0 0
2024-512714-18
Secondary ID [2] 0 0
CYTB323N12101
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Relapsing Multiple Sclerosis 0 0
Condition category
Condition code

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Other - rapcabtagene autoleucel (YTB323)

Experimental: YTB323 Cohort 1 - Participants will receive one dose of YTB323

Experimental: YTB323 Cohort 2 - Participants will recieve one dose of YTB323

Experimental: YTB323 Cohort 3 - Participants will recieve one dose of YTB323

Experimental: YTB323 Cohort 4 - Participants will recieve one dose of YTB323


Treatment: Other: rapcabtagene autoleucel (YTB323)
CAR-T cell suspension for intravenous infusion
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
Timepoint [1] 0 0
Day 1 through Year 2
Secondary outcome [1] 0 0
Measure of Disability: Expanded Disability Status Scale (EDSS).
Timepoint [1] 0 0
Day 1 through Year 2
Secondary outcome [2] 0 0
Measure of Disability: Short Form Health Survey (SF-36 v2)
Timepoint [2] 0 0
Day 1 through Year 2
Secondary outcome [3] 0 0
Measure of Disability: Timed 25 Foot Walk (T25FW)
Timepoint [3] 0 0
Day 1 through Year 2
Secondary outcome [4] 0 0
Measure of Disability: 9 Hole Peg Test (9HPT)
Timepoint [4] 0 0
Day 1 through Year 2
Secondary outcome [5] 0 0
Measure of Disability: Symbol Digit Modalities Test (SDMT)
Timepoint [5] 0 0
Day 1 through Year 2
Secondary outcome [6] 0 0
Measure of Disability: Fatigue Symptoms and Impacts Questionnaire - Relapsing Multiple Sclerosis (FSIQ-RMS)
Timepoint [6] 0 0
Day 1 through Year 2
Secondary outcome [7] 0 0
Number of new and enlarging T2 lesions and Gd-enhancing T1 Lesions
Timepoint [7] 0 0
Day 1 through Year 2
Secondary outcome [8] 0 0
Plasma Pharmacokinetics (PK) of YTB323 - CMAX
Timepoint [8] 0 0
Day 1 through Year 2
Secondary outcome [9] 0 0
Plasma Pharmacokinetics (PK) of YTB323 - AUC
Timepoint [9] 0 0
Day 1 through Year 2
Secondary outcome [10] 0 0
Plasma Pharmacokinetics (PK) of YTB323 - Tmax
Timepoint [10] 0 0
Day 1 through Year 2
Secondary outcome [11] 0 0
Plasma Pharmacokinetics (PK) of YTB323 - Clast
Timepoint [11] 0 0
Day 1 through Year 2
Secondary outcome [12] 0 0
Plasma Pharmacokinetics (PK) of YTB323 - Tlast
Timepoint [12] 0 0
Day 1 through Year 2
Secondary outcome [13] 0 0
Humoral Immunogenicity of YTB323
Timepoint [13] 0 0
Day 1 through Year 2
Secondary outcome [14] 0 0
Cellular Immunogenicity of YTB323
Timepoint [14] 0 0
Day 1 through Year 2
Secondary outcome [15] 0 0
Manufacture success (defined as meeting release specifications and at or above the target dose)
Timepoint [15] 0 0
Day -9 to Day -2

Eligibility
Key inclusion criteria
* Signed informed consent, and able to communicate well with the investigator and comply with the requirements of the study
* Adequate renal, hepatic, cardiac, hematological, and pulmonary function
* Male or female participants, =18 years to =55 years at screening, with diagnosis of RMS according to the 2017 McDonald diagnostic criteria Evidence of recent (i.e. within 1 year) breakthrough disease activity while at least 6 months on a highly efficacious therapy (any of the following): rituximab (Rituxan®), ocrelizumab (Ocrevus®), natalizumab (Tysabri®), ofatumumab (Kesimpta®), ublituximab (Briumvi®) or alemtuzumab (Lemtrada®).

Evidence of breakthrough disease activity is defined as one or more of the following:

1. Confirmed Clinical MS relapse
2. Persistent radiological activity defined by one of the following:

* =2 T1 gadolinium-enhancing lesions on a single MRI scan
* =1 T1 gadolinium-enhancing lesions on two or more separate MRI scans
* =2 new T2 lesions compared to a previous scan within a period =1 year

* Ambulatory patients (EDSS <6.5) (assessed outside of relapse)
* Disease duration less than 10 years
Minimum age
18 Years
Maximum age
55 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Diagnosis of primary progressive multiple sclerosis (PPMS) according to the 2017 revision of the McDonald diagnostic criteria at screening
* History of clinically significant CNS disease (e.g. stroke, traumatic brain or spinal injury, history or presence of myelopathy) or neurological disorders which may mimic MS at screening
* Evidence of clinically significant cardiovascular (such as but not limited to myocardial infarction, unstable ischemic heart disease, New York Heart Association (NYHA) Class III/IV left ventricular failure, arrhythmia and uncontrolled hypertension within 6 months prior to screening), neurological, psychiatric, pulmonary (including, history of or active severe respiratory disease, including Chronic Obstructive Pulmonary Disease, interstitial lung disease or pulmonary fibrosis), renal, hepatic, endocrine, metabolic (e.g. severe hypoproteinemia due to nephrotic syndrome), hematological disorders or gastrointestinal disease that, in the investigator's opinion, would compromise the safety of the participant, interfere with the interpretation of the study results or otherwise preclude participation or protocol adherence of the participant, prior to screening
* Have donated blood or experienced a loss of blood > 400 mL within 3 months prior screening, or longer if required by local regulations
* Any prior stem cell therapy or organ transplantation
* Any contraindications to LP, including but not limited to:

* Known or suspected structural abnormality of the lumbar spine that, in the opinion of the Investigator, may interfere with the performance of the LP, or increase the risk of the procedure for the participant
* Presence of risk for increased or uncontrolled bleeding including, but not limited to, vascular abnormalities or neoplasms at or near the LP site, disorders of the coagulation cascade, platelet function, or platelet count
* Participants on anticoagulants (e.g., warfarin) or antiplatelets [except for low-dose aspirin (100 mg/day or lower) and low-dose ibuprofen (600 mg/day or lower) which are allowable], are not eligible to participate, unless temporal suspension of the anticoagulant or antiplatelet treatment for the purpose of the LP is considered safe for the patient, feasible, and guided by a hematologist
* Patients not willing or able to take MRI scans as per protocol. Unable to undergo MRI due to for example claustrophobia, or presents absolute contraindications to MRI (e.g., metallic implants, metallic foreign bodies, pacemaker, defibrillator)

Other protocol-defined inclusion/exclusion criteria may apply

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised 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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 1
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
16/12/2024
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
18/10/2030
Actual
Sample size
Target
28
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
Novartis Investigative Site - Darlinghurst
Recruitment hospital [2] 0 0
Novartis Investigative Site - Melbourne
Recruitment postcode(s) [1] 0 0
2010 - Darlinghurst
Recruitment postcode(s) [2] 0 0
3004 - Melbourne
Recruitment outside Australia
Country [1] 0 0
Switzerland
State/province [1] 0 0
Bern

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
+41613241111
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 commited 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 expert 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 is currently available according to the 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?
IPD available at link: https://www.clinicalstudydatarequest.com


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/NCT06617793