Neuromyelitis Optica Spectrum DisordersEarly Phase 1NCT06485232
Universal CAR-T Cells in Patients with Refractory Autoimmune Diseases of the Nervous System.
A Early Phase 1 study testing Universal BCMA CAR-T, Universal CD19 CAR-T and Universal BCMA CAR-T; Universal CD19 CAR-T in people with Neuromyelitis Optica Spectrum Disorders and Myasthenia Gravis, Generalized, run by Xuanwu Hospital, Beijing at 1 site in China.
Trial summary
This is an open label, single-site, dose-escalation study in up to 25 participants with refractory autoimmune diseases of nervous system. This study aims to evaluate the safety and efficacy of the treatment with universal BCMA and CD19 CART. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Neuromyelitis Optica Spectrum Disorders, Myasthenia Gravis, Generalized, Multiple Sclerosis
- What is being tested?
- Universal BCMA CAR-T, Universal CD19 CAR-T and Universal BCMA CAR-T; Universal CD19 CAR-T
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 1 siteChina
- Study dates
- 28 Feb 2025to 31 Dec 2027
- Phase
- Early Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Aged 18-75 years (for MS patients, 18-55 years); both genders eligible.
- Subjects with refractory neurological autoimmune diseases who have failed standard treatment or lack effective treatment, Including neuromyelitis optica spectrum disorders(NMOSD), generalized myasthenia gravis(gMG), chronic inflammatory demyelinating Polyradiculoneuropathy(CIDP) and multiple sclerosis(MS).
- Anticipated survival of ≥ 12 weeks as judged by the researcher.
- Agrees to use double barrier methods, condoms, oral or injectable contraceptives, or intrauterine devices during the study period and for one year after taking the study medication.
- Provides written informed consent.
Who cannot join
- History of solid organ transplantation.
- Malignant tumor within the last two years.
- Positive for Hepatitis B surface antigen (HBsAg) or Hepatitis B core antibody (HBcAb), with peripheral blood Hepatitis B virus (HBV) DNA detected as positive; positive for Hepatitis C virus antibodies, with peripheral blood Hepatitis C virus RNA detected as positive; positive for Human Immunodeficiency Virus (HIV) antibodies; positive for Cytomegalovirus (CMV) DNA; positive for syphilis.
- Primary immunodeficiency (congenital or acquired).
- Severe cardiac disease.
- History of psychiatric disorders or history of psychotropic drug abuse, with no history of withdrawal.
- Allergic constitution or a history of severe allergies.
- Pregnant or breastfeeding women.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site
Xuanwu Hospital, Capital Medical University
Beijing, Beijing Municipality, 100053
Junwei Hao, MD; PhD, 01083198277, haojunwei@vip.163.com
About this study
Official title: An Exploratory Study on the Safety and Efficacy of Universal CAR-T Cells Targeting BCMA and CD19 in the Treatment of Refractory Autoimmune Diseases of the Nervous System
This is an open label, single-site, dose-escalation study in up to 25 participants with refractory autoimmune diseases of nervous system. This study aims to evaluate the safety and efficacy of the treatment with universal BCMA and CD19 CART.
- Conditions
- Neuromyelitis Optica Spectrum Disorders, Myasthenia Gravis, Generalized, Multiple Sclerosis, Chronic Inflammatory Demyelinating Polyradiculoneuropathy
- Being tested
- Universal BCMA CAR-T, Universal CD19 CAR-T, Universal BCMA CAR-T; Universal CD19 CAR-T
- Keywords
- universal CAR-T, Neuromyelitis Optica Spectrum Disorders, Myasthenia Gravis, Multiple Sclerosis, Chronic Inflammatory Demyelinating Polyradiculoneuropathy
- Medical subject headings
- Neuromyelitis Optica, Myasthenia Gravis, Multiple Sclerosis, Polyradiculoneuropathy, Chronic Inflammatory Demyelinating
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Universal BCMA CAR-TDrug
Universal BCMA CAR-T
Universal CD19 CAR-TDrug
Universal CD19 CAR-T
Universal BCMA CAR-T; Universal CD19 CAR-TDrug
Universal BCMA CAR-T; Universal CD19 CAR-T
| Study group | Role | What they receive |
|---|---|---|
| BCMA CAR-T Group | Experimental | Universal BCMA CAR-T Receives: Drug: Universal BCMA CAR-T |
| CD19 CAR-T Group | Experimental | Universal CD19 CAR-T Receives: Drug: Universal CD19 CAR-T |
| BCMA CAR-T + CD19 CAR-T Group | Experimental | Universal BCMA CAR-T; Universal CD19 CAR-T Receives: Drug: Universal BCMA CAR-T; Universal CD19 CAR-T |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of dose-limiting toxicities(DLTs)
Incidence of dose-limiting toxicities(DLTs)
Measured: First 28 days after infusion
Incidence of adverse events(AEs) and severe adverse
Incidence of adverse events(AEs) and severe adverse events(SAEs).
Measured: Up to 12 months after infusion
Secondary outcomes (8)
Concentrations of UCAR-T cells
The concentration of BCMA UCAR T cells and CD19 UCAR-T cells in peripheral blood after infusion
Measured: 3 months
B cell levels in peripheral blood
The change of B cell levels in peripheral blood after infusion
Measured: 3 months
Changes of pathogenic antibody titers after infusion
The changes of pathogenic antibody titers in peripheral blood or cerebrospinal fluid.
Measured: 1, 3, 6 ,12months
NMOSD: Annualized relapse rate
ARR is defined as the number of relapses divided by the total participant-years after infusion
Measured: 6, 12months
gMG: Changes of Myasthenia Gravis Activities if Daily Living (MG-ADL) Score
MG-ADL scale assesses the impact of gMG on daily functions by measuring 8 signs or symptoms that are commonly affected in gMG. Each item is measured on a 4-point scale, where a score of 0 represents normal function and a score of 3 represents the loss of ability to perform that function. Total scores range from 0 to 24 points, with a higher score showing more severe gMG.
Measured: 1, 3, 6, 12months
CIDP:Changes of Inflammatory Neuropathy Cause and Treatment (INCAT) Score after infusion.
The INCAT score assesses the functionality of the arms and legs by giving a 0-5 score for arms and legs, with 0 representing no disability and 5 representing no arm function or inability to stand/walk.
Measured: 1, 3, 6, 12months
MS: Changes of the number of Gd-enhancing T1 Lesions
The changes of enhancing Lesions as detected by brain Magnetic Resonance Imaging (MRI)
Measured: 6, 12months
MS: Changes of the number of Number of New or Enlarging T2 Lesions
Number of new/enlarging T2 lesions on last available MRI scan compared to baseline.
Measured: 6, 12months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Jul 2024
- Study start28 Feb 2025 (estimated)
- Primary completion31 Dec 2026 (estimated)
- Study completion31 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neuromyelitis Optica Spectrum DisordersStudies of the same condition, open and under way.
- More trials run by Xuanwu Hospital, BeijingThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 25 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Xuanwu Hospital, Beijing
- Sponsor type
- University, hospital or other
- Collaborators
- Bioray Laboratories
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06485232
- Sponsor's protocol ID
- 2024-BRL-302-02
- Status last verified
- Dec 2024
- First submitted
- 20 Jun 2024
- Last updated
- 1 Jan 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Exploratory Study on the Safety and Efficacy of Universal CAR-T Cells Targeting BCMA and CD19 in the Treatment of Refractory Autoimmune Diseases of the Nervous System
- ClinicalTrials.gov ID
- NCT06485232
- Acronym / sponsor ID
- 2024-BRL-302-02
- Conditions
- Neuromyelitis Optica Spectrum Disorders, Myasthenia Gravis, Generalized, Multiple Sclerosis, Chronic Inflammatory Demyelinating Polyradiculoneuropathy
- Keywords
- universal CAR-T, Neuromyelitis Optica Spectrum Disorders, Myasthenia Gravis, Multiple Sclerosis, Chronic Inflammatory Demyelinating Polyradiculoneuropathy
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 25 participants (estimated)
- Lead sponsor
- Xuanwu Hospital, BeijingUniversity, hospital or other
- Collaborators
- Bioray Laboratories
- Study start
- 28 Feb 2025 (estimated)
- Primary completion
- 31 Dec 2026 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 3 Jul 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is an open label, single-site, dose-escalation study in up to 25 participants with refractory autoimmune diseases of nervous system. This study aims to evaluate the safety and efficacy of the treatment with universal BCMA and CD19 CART.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| BCMA CAR-T Group | Experimental | Drug: Universal BCMA CAR-TUniversal BCMA CAR-T |
| CD19 CAR-T Group | Experimental | Drug: Universal CD19 CAR-TUniversal CD19 CAR-T |
| BCMA CAR-T + CD19 CAR-T Group | Experimental | Drug: Universal BCMA CAR-T; Universal CD19 CAR-TUniversal BCMA CAR-T; Universal CD19 CAR-T |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Aged 18-75 years (for MS patients, 18-55 years); both genders eligible.
- Subjects with refractory neurological autoimmune diseases who have failed standard treatment or lack effective treatment, Including neuromyelitis optica spectrum disorders(NMOSD), generalized myasthenia gravis(gMG), chronic inflammatory demyelinating Polyradiculoneuropathy(CIDP) and multiple sclerosis(MS).
- Anticipated survival of ≥ 12 weeks as judged by the researcher.
- Agrees to use double barrier methods, condoms, oral or injectable contraceptives, or intrauterine devices during the study period and for one year after taking the study medication.
- Provides written informed consent.
Key exclusion criteria (8 listed)
- History of solid organ transplantation.
- Malignant tumor within the last two years.
- Positive for Hepatitis B surface antigen (HBsAg) or Hepatitis B core antibody (HBcAb), with peripheral blood Hepatitis B virus (HBV) DNA detected as positive; positive for Hepatitis C virus antibodies, with peripheral blood Hepatitis C virus RNA detected as positive; positive for Human Immunodeficiency Virus (HIV) antibodies; positive for Cytomegalovirus (CMV) DNA; positive for syphilis.
- Primary immunodeficiency (congenital or acquired).
- Severe cardiac disease.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Universal BCMA CAR-TUniversal BCMA CAR-T | Drug | BCMA CAR-T Group |
| Universal CD19 CAR-TUniversal CD19 CAR-T | Drug | CD19 CAR-T Group |
| Universal BCMA CAR-T; Universal CD19 CAR-TUniversal BCMA CAR-T; Universal CD19 CAR-T | Drug | BCMA CAR-T + CD19 CAR-T Group |
Primary outcome
Incidence of dose-limiting toxicities(DLTs)
Time frame: First 28 days after infusion
Incidence of dose-limiting toxicities(DLTs)
Incidence of adverse events(AEs) and severe adverse
Time frame: Up to 12 months after infusion
Incidence of adverse events(AEs) and severe adverse events(SAEs).
8 secondary outcomes
Concentrations of UCAR-T cells
Time frame: 3 months
B cell levels in peripheral blood
Time frame: 3 months
Changes of pathogenic antibody titers after infusion
Time frame: 1, 3, 6 ,12months
NMOSD: Annualized relapse rate
Time frame: 6, 12months
gMG: Changes of Myasthenia Gravis Activities if Daily Living (MG-ADL) Score
Time frame: 1, 3, 6, 12months
CIDP:Changes of Inflammatory Neuropathy Cause and Treatment (INCAT) Score after infusion.
Time frame: 1, 3, 6, 12months
MS: Changes of the number of Gd-enhancing T1 Lesions
Time frame: 6, 12months
MS: Changes of the number of Number of New or Enlarging T2 Lesions
Time frame: 6, 12months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Jan 2025.
- Individual participant dataSharing planned: No
Related trials for Neuromyelitis Optica Spectrum Disorders
Not yet recruiting
Neurogenetics and Multi-omics of Neuroinflammatory Diseases in Multiplex Families to Improve Patients Health
Multiple Sclerosis, Neuromyelitis Optica Spectrum Disorder
Recruiting
Impact of a Structured Wellness Behavioral Intervention on Quality of Life in NMOSD
Neuromyelitis Optica Spectrum Disorder (NMOSD)
RecruitingObservational study
Observational Safety Study in Women With Neuromyelitis Optica Spectrum Disorder (NMOSD) Exposed to UPLIZNA® During Pregnancy
Neuromyelitis Optica Spectrum Disorder, Pregnancy Related
RecruitingPhase 2
Efgartigimod for the Treatment of Acute Optic Neuritis
Optic Neuritis
Active, not recruitingObservational study
Biobank For MS And Other Demyelinating Diseases
Multiple Sclerosis, Transverse Myelitis
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Universal BCMA CAR-T, Universal CD19 CAR-T and Universal BCMA CAR-T; Universal CD19 CAR-T in people with Neuromyelitis Optica Spectrum Disorders and Myasthenia Gravis, Generalized, run by Xuanwu Hospital, Beijing at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 1 Jan 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Junwei Hao, MD;PhD (01083198277, haojunwei@vip.163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of dose-limiting toxicities(DLTs) (time frame: First 28 days after infusion). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Jan 2025.