Feasibility of a Novel Academic BCMA-CART (HBI0101) for the Treatment of Relapsed and Refractory AL Amyloidosis.
Shlomit Kfir-Erenfeld, Nathalie Asherie, Sigal Grisariu...
https://pubmed.ncbi.nlm.nih.gov/36107221Actively Recruiting
Phase 1
Age: 18Years - 80Years
All Genders
ID07085676
A Phase 1 Dose Escalation and Safety Study of HBI0101 CAR-T Therapy in B-cell Mediated Refractory Autoimmune Diseases
Led by Polina Stepensky · Updated on 2026-05-06
120
Participants Needed
1
Research Sites
208 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating HBI0101 BCMA-CART therapy in adults aged 18 to 80 with B-cell mediated autoimmune rheumatic diseases that have been resistant to standard treatments. This Phase 1, open-label study aims to assess the safety of this investigational CAR-T cell therapy and identify the highest dose that can be safely given to patients. The trial includes diseases such as systemic lupus erythematosus, systemic sclerosis, idiopathic inflammatory myopathies, rheumatoid arthritis, multiple sclerosis, neuromyelitis optica spectrum disorder, myasthenia gravis, and antiphospholipid antibody syndrome. The study has two parts: Part A focuses on establishing safety and determining the maximum tolerated dose through dose escalation, while Part B is an extension phase to further evaluate safety at the selected dose. Eligible participants will undergo a leukapheresis procedure to collect cells needed to manufacture the HBI0101 CART cells. Before receiving a single dose of either 450 million or 800 million BCMA CART cells, participants will undergo lymphodepletion. After receiving the CAR-T cell infusion, they will be hospitalized for several days and then attend routine follow-up visits for up to 48 months. Participants will be closely monitored through clinical exams, disease-specific assessments, and survival tracking for up to four years after infusion. Researchers will evaluate safety outcomes in the first 21 days and confirm safety over the longer term. Disease progression and response will be tracked using measures specific to each autoimmune condition. Participants will also be required to adhere to study visit schedules and protocols, including regular laboratory tests and clinical evaluations. This long-term follow-up aims to understand the safety profile and disease effects of HBI0101 CART therapy.
CONDITIONS
Brief Title
Phase 1 Study of HBI0101 CAR-T in Refractory B-Cell Autoimmune Diseases
Who Can Participate
Age: 18Years - 80Years
All Genders
Eligibility Criteria
You may qualify if you...
- Age between 18 and 80 years; patients aged 75 or older require geriatric assessment and endorsement
- Diagnosis of B-cell mediated autoimmune rheumatic diseases including SLE, SSc, IIM, RA, NMOSD, MS, MG, or APLA
- Disease must be severe, progressive, and refractory to standard treatments as defined for each disease type
- Adequate liver and cardiopulmonary function
- No active infections
- Women of child-bearing potential must have a negative pregnancy test and agree to use effective contraception
- Ability and willingness to follow study visit schedule and protocol requirements
- Voluntary participation with signed informed consent
You will not qualify if you...
- History of central nervous system tumors or progressive neurological disorders unrelated to autoimmune diseases
- Abnormal liver function beyond specified limits except for myositis-related changes
- Recent unstable cardiovascular events or severe cardiac dysfunction
- Chronic lung disease with severe impairment or hypoxia
- Severe muscle atrophy or other muscle diseases not related to the autoimmune disease
- Uncontrolled acute diseases or infections
- Recent biologic therapy including rituximab or plasma exchange within specified timeframes
- Participation in other clinical studies within 3 months
- Previous or concurrent malignancy with some exceptions for treated cancers
- History of vital organ or stem cell transplantation
- Disease relapse within 30 days for MS/NMO patients
- Known HIV positive status
- Active hepatitis B, C, or CMV infection
- Pregnant or lactating women
- Inability to understand or comply with study requirements
- Any other significant medical condition that may pose safety risks or interfere with the study outcomes
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
1
2
3
Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Treatment
Duration - Several days hospitalization plus initial treatment period
Participants undergo leukapheresis to collect cells for manufacturing HBI0101 CAR-T therapy, followed by lymphodepletion and a single infusion of HBI0101 CAR-T cells. Participants are hospitalized for several days after infusion for monitoring and care.
1 leukapheresis visit, 1 lymphodepletion visit, 1 CAR-T infusion visit, hospitalization for several days
Follow-up
Duration - Up to 48 months
Participants return for routine follow-up visits to monitor safety and disease status for up to 48 months after CAR-T infusion.
Periodic visits over 48 months, including visits at 3 months, every 6 months up to 48 months
Trial Site Locations
Total: 1 location
1
Hadassah MO
Jerusalem, Israel, 9574869
Actively Recruiting
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Research Team
P
Polina Stepensky, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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Published Research Related To This Trial
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Nathalie Asherie, Shlomit Kfir-Erenfeld, Batia Avni...
https://pubmed.ncbi.nlm.nih.gov/36200421