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New treatment option for Systemic Sclerosis (SSc)
Official title Phase 1 Study of HBI0101 CAR-T in Refractory B-Cell Autoimmune Diseases
ClinicalTrials.gov ID: NCT07085676
What this study is testing
What is HBI0101 CART?
HBI0101 CART is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for systemic sclerosis (ssc).
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- A Phase 1 study of HBI0101 BCMA-CART in B-Cell Mediated Autoimmune Rheumatic Diseases. The goal of the study is evaluation of safety and identification of the maximum HBI0101 CART dose that may be administered safely to patients with B-cell mediated autoimmune disease.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 80
You may be able to join if
- Age: 18\~80 years old; for patients aged ≥ 75 years, geriatric assessment and endorsement are required;
- Diagnosis of B-cell mediated ARDs listed below: SLE patients: individuals diagnosed with SLE according to American College of Rheumatology (ACR)...
- lack of response per SLEDAI-2k (\<4 points reduction from baseline) or no improvement in BILAG domains, or
- disease flare per SLEDAI-2k (≥4 points increase from baseline) or new BILAG A or ≥2 new BILAG B organ scores, or intolerance or discontinuation due...
- Progression of skin thickening ≥ 12 over the past 6 months or Modified Rodnan skin score (mRSS) ≥15
You likely can't join if
- CNS disease- History of CNS or spinal cord tumor, metabolic or infectious cause of myelopathy, genetically inherited progressive CNS disorder...
- Abnormal liver function: aspartate transaminase (AST) or alanine transaminase (ALT) or glutamyl transpeptidase (GGT) or alkaline phosphatase (ALP)...
- Cardiovascular disease: Unstable angina or myocardial infarction or coronary artery bypass graft (CABG) within 6 months prior to leukapheresis/...
- Lung disease: patients with chronic lung disease with any of the following: \ Oxygen saturation (SpO2) \ 50 mmHg.
- Muscle disease: evidence of any of the following: \ Severe proximal muscle atrophy of upper or lower extremity on MRI or clinical examination. \...
- Other uncontrolled diseases: acute diseases (such as acute pneumonia or other infection, pulmonary embolism, diabetic ketoacidosis, acute...
See the full eligibility criteria
Who can join
- Age: 18\~80 years old; for patients aged ≥ 75 years, geriatric assessment and endorsement are required;
- Diagnosis of B-cell mediated ARDs listed below: SLE patients: individuals diagnosed with SLE according to American College of Rheumatology (ACR) and/or Systemic lupus international collaborating clinics (SLICC)...
- lack of response per SLEDAI-2k (\<4 points reduction from baseline) or no improvement in BILAG domains, or
- disease flare per SLEDAI-2k (≥4 points increase from baseline) or new BILAG A or ≥2 new BILAG B organ scores, or intolerance or discontinuation due to adverse effects. SSc patients: Patients who were diagnosed with...
- Progression of skin thickening ≥ 12 over the past 6 months or Modified Rodnan skin score (mRSS) ≥15
- Any Medsger Disease Severity Score grade 3-4 in one major organ or grade ≥2 in two organs
- Progressive interstitial lung disease evidenced by HRCT or FVC \<80% or DLCO \<80%, or evidence of pulmonary function decline, defined as an absolute FVC decline of ≥ 10% , or FVC decline of 5% -9% combined with DLCO...
- Other internal organ involvement. Eligible patients must have failed to at least two state-of-the-art immunosuppressive therapies including MTX, MMF, cyclophosphamide, azathioprine, nintedanib, tocilizumab or rituximab...
- lack of response in skin per mRSS (≤20% relative and ≤5 point absolute reduction from baseline) or no clinically meaningful improvement in FVC, DLCO, or other organ involved assessed by Medsger DSS, or
- disease flare in skin per mRSS (increase in mRSS ≥20% and ≥5 points from baseline) or decline in FVC ≥10% predicted or in DLCO ≥15% predicted from baseline, other major SSc complication, or
- intolerance or discontinuation due to adverse effects IIM, including dermatomyositis, anti-synthetase syndrome, immune mediated necrotizing myopathy, and polymyositis: patients must be diagnosed with IIM according to...
- CPK ≥4xULN
- Loss of muscle strength in the weakest muscle group for less than 80% per MMT8
- Evidence on MRI of active myositis within last 6 months
- Evidence on EMG of active myositis within last 6 months
- Muscle biopsy evidence of active myositis within last 6 months Only patients with refractory disease will be recruited, defined as previous failure to (1) at least two of five non-glucocorticoids immunosuppressive...
- lack of response in muscle strength per MMT-8 or CPK (\<20% relative improvement) or per MRI or
- disease flare in muscle strength per MMT-8 (≥30% decline) or CPK (≥30% rise) or objective worsening of other organ involvement per Myositis Disease Activity Assessment Tool or
- intolerance or discontinuation due to adverse effects RA patients: Seropositive RA patients (positive for anti-cyclic citrullinated peptide and rheumatoid factor), diagnosed according to the 2010 ACR/EULAR...
- JAK-STAT inhibitors
- Anti-TNF agents
- Anti-IL6 drugs
- Anti-CTLA4-Ig treatments, each administered for a minimum of 3 months, or have a contraindication to, or have experienced toxicity from, any therapy within these categories Drug resistance is defined as:
- Inadequate clinical response measured by composite score DAS28CRP (\> 3.6) or failure to reach ACR20 or
- Disease progression measured by DAS28CRP (≥20% increase) or radiographic progression or
- Intolerance or discontinuation due to adverse effects NMOSD: Patients must be diagnosis of AQP4-IgG-positive NMOSD based on the 2015 International Panel for NMOSD Diagnosis (IPND) criteria (Wingerchuk et al., 2015)...
- Myasthenia Gravis Activities of Daily Living (MG-ADL) score of ≥6, with ocular symptoms constituting less than 50% of the total score.
- Myasthenia Gravis Foundation of America (MGFA) clinical classification of II to IV. Eligible patients must have Refractory Status and meet at least one of the following criteria:
- No improvement or worsening myasthenic symptoms, with failure to achieve minimal manifestation status or meaningful improvement in MGFA class or MG-ADL score, or recurrent myasthenic exacerbations despite adequate...
- Improvement in Post-Intervention Status (PIS), but with an MG-ADL score ≥6 persisting for at least 4 months.
- Remission or improvement in PIS, but with ≥1 episode of disease exacerbation (MG-ADL ≥6) per year during tapering of immunotherapy medications.
- After experiencing a myasthenic crisis, patients who undergo multiple immunotherapies (e.g., intravenous immunoglobulin, plasma exchange, high-dose intravenous methylprednisolone), thymectomy, and active infection...
- AST/ALT below 5 times the upper limit of normal, blood bilirubin below 3 times the upper limit of normal ;
- Cardiopulmonary function is basically normal, echocardiography indicates that the ejection fraction is \>45%, normal to mild pulmonary hypertension, and the oxygen saturation is above 93% in the resting state without...
- No obvious active infection;
- There are no contraindications for blood collection;
- Women of child-bearing potential (WCBP), defined as a sexually mature woman who has not undergone a hysterectomy or tubal ligation or who has not been naturally postmenopausal for at least 24 consecutive months, must...
- Voluntary participation and informed consent signed by the patient or his/her legal/authorized representative.
- Ability and willingness to adhere to the study visit schedule and all protocol requirements
What rules you out
- CNS disease- History of CNS or spinal cord tumor, metabolic or infectious cause of myelopathy, genetically inherited progressive CNS disorder, sarcoidosis, non-autoimmune progressive neurologic condition or PML
- Abnormal liver function: aspartate transaminase (AST) or alanine transaminase (ALT) or glutamyl transpeptidase (GGT) or alkaline phosphatase (ALP) detection value is greater than 5 times the upper limit of normal (ULN)...
- Cardiovascular disease: Unstable angina or myocardial infarction or coronary artery bypass graft (CABG) within 6 months prior to leukapheresis/ moderate- severe pulmonary hypertension/ severe arrhythmia (ventricular...
- Lung disease: patients with chronic lung disease with any of the following: \ Oxygen saturation (SpO2) \ 50 mmHg.
- Muscle disease: evidence of any of the following: \ Severe proximal muscle atrophy of upper or lower extremity on MRI or clinical examination. \ Finding of muscular inflammation or myopathy other than the indication...
- Other uncontrolled diseases: acute diseases (such as acute pneumonia or other infection, pulmonary embolism, diabetic ketoacidosis, acute pancreatitis, etc.) that are clinically unstable or have not been effectively...
- Biologics therapy: Received rituximab within 4 months of expected CAR T treatment: No plasma exchange or immunoglobulin treatment within 4 weeks prior to screening. MS patients: No high dose corticosteroid treatment in...
- Participated in any clinical study within 3 months prior to enrollment, or participate in other clinical investigations during the study period.
- Previous or concurrent malignancy with the following exceptions: Adequately treated basal cell or squamous cell carcinoma, in situ carcinoma of the cervix or breast, treated curatively and without evidence of recurrence...
- Transplantation: History of vital organ transplantation (e.g., heart, lung, kidney, liver) or hematopoietic stem cell/or bone marrow transplantation.
- Disease-specific criteria: MS/NMO patients: No disease relapse in the 30 days prior to enrollment
- Known HIV positive status.
- Active hepatitis B or C infection.
- Active CMV infection
- Pregnant or lactating women.
- Inability to understand or follow the research protocol subject requirements.
- Have any other clinically significant disease history or current disease that, in the judgment of the research physician, may pose a risk to the safety of the people, or interfere with the completion of the research...
The study team makes the final eligibility decision.
Where it's taking place
- Jerusalem, Israel
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 80 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Jerusalem, Israel. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.