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Systemic Sclerosis (SSc)Phase 1NCT07085676

Phase 1 Study of HBI0101 CAR-T in Refractory B-Cell Autoimmune Diseases

A Phase 1 study testing HBI0101 CART in people with Systemic Sclerosis (SSc) and Idiopathic Inflammatory Myopathy (IIM), run by Polina Stepensky at 1 site in Israel.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial summary

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. Read more about this study

Who is it for?
Ages 18 to 80 years
What is being tested?
HBI0101 CART
Could I receive a placebo?
No, everyone receives an active treatment
How many people
120 participants
Where
1 siteIsrael
Study dates
1 Sep 2024to 1 Nov 2030
Phase
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 80 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to 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) classification criteria, who have severe and progressive disease course reflected by Systemic Lupus Erythematosus Disease Activity Index-2000 (SLEDAI-2K) score of 8 or more. Eligible patients must have failed to at least one of the conventional DMARDs (azathioprine, methotrexate, mycophenolate mofetil or cyclophosphamide), one of the calcineurin inhibitors (tacrolimus or cyclosporin) and one of the biologic agents (belimumab, rituximab or aniflorumab), each administered for a minimum of 3 months, or have a contraindication to, or have experienced toxicity from, any therapy within these categories. The failure is defined as:
  • 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 diffuse or limited cutaneous SSc according to the College of Rheumatology/European Alliance of Associations for Rheumatology (ACR-EULAR) classification criteria, with severe or rapidly progressive disease Eligible patients must meet any of the following criteria:
Show all 62Show fewer
  • 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 15%.
  • 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; each therapy must have been administered for a minimum of 3 months, unless discontinued due to a contraindication or toxicity. The failure is defined as:
  • 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 the 2017 ACR/EULAR Classification Criteria for idiopathic inflammatory myopathies.
  • Eligible patients must have active disease, defined by at least one of the following:
  • 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 therapies and (2) either rituximab or IVIG, each administered for a minimum of 3 months, or have a contraindication to, or have experienced toxicity from, any therapy within these categories. The five non-glucocorticoids therapies considered are azathioprine, MTX, MMF, IVIG, and rituximab The failure is defined as:
  • 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 classification criteria.
  • Eligible patients must exhibit high disease activity (DAS28CRP ≥5.1), and be resistant to at least four conventional synthetic and biologic disease-modifying antirheumatic drugs (DMARD) groups.
  • Each patient must have previously received at least one medication from each of the mentioned groups, including
  • 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).
  • Eligible patients must have experienced at least two relapses in the past 24 months, with at least one occurring in the preceding 12 months before screening Each patients must have previously received with inadequate disease control (one or more clinical attacks, with new MRI activity, despite adequate treatment dosing) at least one immunosuppressant for at least 6 months (azathioprine or mycophenolate mofetil) and/or Rituximab for at least 3 months and/or a targeted therapy (eculizumab, inebilizumab, or satralizumab) or must have a contraindication to such treatment.
  • MS patients: must have confirmed history of diagnosis of primary progressive or secondary progressive MS .
  • Eligible patients must have: Expanded Disability Status Scale (EDSS) score between 3.0 and 8.5 and evidence of disease activity, defined by either clinical progression and/or radiological finding on MRI, such as new, enhancing or enlarging lesions in the last 12 months.
  • Eligible patient must have previously received at least one high efficacy drug and have been offered standard treatments and have experienced disease progression or lack of efficacy despite at least six months of treatment. These will include clinical activity (new relapse[s]), radiological activity (new or enlarging T2 lesions and/or gadolinium-enhancing lesions on MRI), or confirmed disability progression (an increase in least 1 point on the EDSS sustained for ≥6 months).
  • Eligible patient must have history of treatment with anti-CD20 mAb with continuing evidence of worsening physical disability over a period of ≥6 months, with documented clinical disability progression within the year prior to inclusion, irrespective to concurrent MRI activity.
  • MG patients must meet all the following criteria:
  • Confirmed diagnosis of generalized MG, supported by positive autoantibodies (anti-AChR, anti-MuSK).
  • Disease Severity:
  • 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 immunosuppressive or immunomodulatory therapy for at least 6 months for all therapies and 3 months for B cell depletion therapy
  • 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 control but still cannot be weaned off the ventilator due to MG-induced respiratory muscle weakness for more than 14 days.
  • Each patient must receive stable doses of medication prior to enrollment.
  • Refractory APLA patients: must have confirmed history of APLA, based on Sydney 2006 or ACR/EULAR 2023 criteria
  • Eligible patients must have medium to high-titer antiphospholipid antibodies, documented repeatedly (≥2 tests spanning 12 weeks) Eligible patients must have refractory disease: persistent or recurrent arterial thrombosis, venous thrombosis or diffuse alveolar hemorrhage despite conventional therapy with stable treatment with vitamin K antagonist anticoagulation (maintained at therapeutic stable dose keeping INR consistently within target range) and immunosuppression treatment including glucocorticoid, rituximab and Plaquenil
  • 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 oxygen;
  • 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 have a negative serum pregnancy test prior to treatment. All sexually active WCBP and all sexually active male subjects must agree to use effective methods of birth control throughout the study;
  • 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

Who cannot join

  • 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); or total bilirubin test value greater than 3 times the upper limit of normal (ULN); Exceptional: liver function disturbance due to myositis.
  • 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 tachycardia, ventricular fibrillation, high grade ventricular block) in the past 6 months; New York heart function class (NYHA) class III- Level IV or LVEF<45%.
  • Lung disease: patients with chronic lung disease with any of the following: * Oxygen saturation (SpO2) < 90% on room air * FVC≤45% of predicted or DLCO≤40% of predicted at screening. * Evidence of pulmonary hypertension as defined as estimated RVSP> 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, such as inclusion body myositis (IBM), or cancer-associated myositis (myositis diagnosed within 2 years of cancer).
  • 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 controlled and are not related to indicated autoimmune diseases which in the judgment of the investigator may confound study results or place subjects at undue risk.
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  • 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 the 30 days prior to enrollment; see also section 9.1 for the list of restrictions.
  • 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 for at least 3 years prior to screening. A primary malignancy which has been completely resected, or treated, and is in complete remission for at least 5 years prior to screening.
  • 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 subjects, or interfere with the completion of the research procedure and the evaluation of safety and efficacy

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

Polina Stepensky, MD contact

972-2-6778353

Fainak@hadassah.org.il

Israel1 site, 1 recruiting

  • Hadassah MO

    Jerusalem, 9574869

    Polina Stepensky, MD, Professor, +97226776679, Fainak@haddasah.org.il

    Polina Stepensky, MD, Professor

    Recruiting

Other trials in Israel

About this study

Official title: A Phase 1 Dose Escalation and Safety Study of HBI0101 CART in B-cell Mediated Refractory Autoimmune Diseases

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.

Read the detailed description

Up to 120 subjects with B-cell mediated autoimmune rheumatic diseases will be enrolled in a single-arm, open-label, single-site Phase 1 study.

The study includes 2 parts. The first Part A is an establishment of the safety profile followed by a dose ranging, maximum tolerated dose (MTD) study and the second Part B is an extension phase to further evaluate safety at the selected safe dose.

Eligible subjects will undergo leukapheresis procedure to provide starting material for manufacturing of HBI0101 CART investigational product. Each eligible subject will receive a single dose of HBI0101 CART cells. Prior to administration of HBI0101 CART, the study subjects will undergo lymphodepletion. Following administration of HBI0101 CART the subjects will be hospitalized for several days and then will return for routine follow-up periodical visits until 48 months after infusion.

Being tested
HBI0101 CART
Keywords
B-cell mediated autoimmune rheumatic diseases, Systemic Lupus Erythematosus (SLE), Systemic sclerosis (SSC), Idiopathic inflammatory myopathy (IIM), Rheumatoid arthritis (RA), B-cell maturation antigen (BCMA), Autologous CAR-T, Multiple sclerosis (MS) Primary and Secondary progressive, Neuromyelitis Optica Spectrum Disorder (NMOSD), Myasthenia Gravis (MG), refractory antiphospholipid antibody syndrome
Medical subject headings
Scleroderma, Systemic, Myositis, Arthritis, Rheumatoid, Lupus Erythematosus, Systemic, Multiple Sclerosis, Chronic Progressive, Neuromyelitis Optica, Myasthenia Gravis, Antiphospholipid Syndrome

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • HBI0101 CARTBiological

    HBI0101 CART is defined as autologous T cells transduced ex-vivo with anti-BCMA CAR retroviral vector encoding the chimeric antigen receptor (CAR) targeted to human BCMA. The HBI0101 CART may be provided fresh or cryopreserved.

Study groups in NCT07085676
Study groupRoleWhat they receive
BCMA CARTExperimentalEach subject subjects will receive a single dose of 450 x 10^6 or 800 x 10^6 BCMA CART cells
Receives: Biological: HBI0101 CART

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Part A: Establishment of the initial safety profile of HBI0101 CAR T

    Incidence of adverse events (AEs) and abnormal laboratory test results used to determine the dose-limiting toxicities (DLTs).

    Measured: 21 days after infusion

  2. Part B: Confirmation of safety with selected dose

    Incidence, severity and type of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) related to HBI0101 CART at the dose selected in Part A

    Measured: 4 years after infusion

Secondary outcomes (29)

  1. Overall survival

    Evaluation of overall survival

    Measured: 3 months for up to 24 months, every 6 months for up to 48 months

  2. Four-year relapse/progression free survival: All Cohorts

    Relapse-free survival, defined as time from remission to documented relapse. ● Progression-free survival, defined as time from HBI0101 CART infusion to documented disease progression.

    Measured: 4 years following infusion

  3. Disease-specific response/progression endpoints: Systemic Lupus Erythematosus (SLE) cohort

    Number of patients with response in SLEDAI < 4 (no disease activity), time to achieving and maintaining of the response.

    Measured: 4 years following infusion

  4. Disease-specific response/progression endpoints: Systemic Lupus Erythematosus (SLE) cohort

    Number of patients, time to achieving and maintaining Lupus Low Disease Activity State (LLDAS)

    Measured: 4 years following infusion

  5. Disease-specific response/progression endpoints: Systemic Lupus Erythematosus (SLE) cohort

    Number of patients, time to achieving and maintaining remission per DORIS definition

    Measured: 4 years following infusion

  6. Disease-specific response/progression endpoints: Systemic Scleroderma (SSc) cohort

    Number of patients with response in Modified Rodnan skin score (mRSS), time to achieving and maintaining of the response. Response = a decrease of ≥ 5 points on mRSS.

    Measured: 4 years following infusion

  7. Disease-specific response/progression endpoints: Systemic Scleroderma (SSc) cohort

    Number of patients with improvement in Medsger's severity score and time to loss of improvement. Improvement = ≥ 1-point decrease in an affected organ

    Measured: 4 years following infusion

  8. Disease-specific response/progression endpoints: Idiopathic Inflammatory Myopathies (IIM) cohort

    Number of patients with clinically significant response in Myositis disease activity assessment visual analogue scale (MDAAT) score, time to achieving and maintaining of the response. Clinically significant response = ≥ 20% in physician and patient global activity score.

    Measured: 4 years following infusion

  9. Disease-specific response/progression endpoints: Idiopathic Inflammatory Myopathies (IIM) cohort

    Number of patients with clinically significant response in Manual Muscle test (MMT-8), time to achieving and maintaining of the response. Clinically significant response = ≥ 15% improvement

    Measured: 4 years following infusion

  10. Disease-specific response/progression endpoints: Idiopathic Inflammatory Myopathies (IIM) cohort

    Disease related biomarker: Creatine Kinase (CPK)

    Measured: 4 years following infusion

  11. Disease-specific response/progression endpoints: Rheumatoid Arthritis (RA) cohort

    Number of patients, time to achieving and maintaining of remission. Remission = DAS28CRP ≤2.7

    Measured: 4 years following infusion

  12. Disease-specific response/progression endpoints: Rheumatoid Arthritis (RA) cohort

    Number of patients, time to achieving and maintaining of low disease activity. Low disease activity = DAS28CRP ≤3.6

    Measured: 4 years following infusion

  13. Disease-specific response/progression endpoints: Rheumatoid Arthritis (RA) cohort

    Number of patients, time to achieving and maintaining good or moderate response. Good response = DAS28CRP improvement > 1.2 AND current DAS28CRP ≤3.2. Moderate response = DAS28CRP improvement >0.6 but ≤1.2 OR final DAS28CRP is >3.2 but ≤5.1

    Measured: 4 years following infusion

  14. Disease-specific response/progression endpoints: Rheumatoid Arthritis (RA) cohort

    Number of patients, time to achieving and maintaining ACR/EULAR 20, 50 and 70% improvement. ACR20: = ≥20% improvement in tender and swollen joint counts AND similar improvement in three out of five other domains (such as pain, physician and patient global assessment, disability, and an acute phase reactant). ACR50: ≥50% improvement in these domains. ACR70: ≥70% improvement in these domains

    Measured: 4 years following infusion

  15. Disease-specific response/progression endpoints: Neuromyelitis Optica Spectrum Disorder (NMOSD) cohort

    Change from baseline in annual relapse rate (ARR), calculated as the difference between pre-treatment ARR and post-treatment

    Measured: 4 years following infusion

  16. Disease-specific response/progression endpoints: Multiple sclerosis (MS) cohort

    Number of patients with clinically significant improvement in Expanded Disability Status Scale (EDSS), time to achieving and maintaining of the improvement. Improvement = 1.0 point or more for patients with baseline EDSS score of ≤ 5.5 or 0.5 points or more for patients with baseline EDSS score of 6.0 or higher

    Measured: 4 years following infusion

  17. Disease-specific response/progression endpoints: Multiple sclerosis (MS) cohort

    Change from baseline in annual relapse rate (ARR), calculated as the difference between pre-treatment ARR and post-treatment through study assessments

    Measured: 4 years following infusion

  18. Disease-specific response/progression endpoints: Multiple sclerosis (MS) cohort

    Additional MRI response assessment: Change from baseline in T2 lesion volume

    Measured: 4 years following infusion

  19. Disease-specific response/progression endpoints: Multiple sclerosis (MS) cohort

    Additional MRI response assessment: Number of new or enlarging T2 lesions

    Measured: 4 years following infusion

  20. Disease-specific response/progression endpoints: Multiple sclerosis (MS) cohort

    Additional MRI response assessment: Number of T1 gadolinium-enhancing lesions

    Measured: 4 years following infusion

  21. Disease-specific response/progression endpoints: Multiple sclerosis (MS) cohort

    Additional MRI response assessment: Percentage change in brain volume from baseline

    Measured: 4 years following infusion

  22. Disease-specific response/progression endpoints: Myasthenia Gravis (MG) cohort

    Number of patients with clinically significant improvement in Myasthenia Gravis Activities of Daily Living (MG-ADL), time to achieving and maintaining of the improvement. Improvement = ≥2 points improvement in total score or ≥ 1 point improvement in individual MG-ADL items

    Measured: 4 years following infusion

  23. Disease-specific response/progression endpoints: Myasthenia Gravis (MG) cohort

    Percentage of patients reporting a 'Yes' response in Patient Acceptable Symptom State (PASS), compared to baseline

    Measured: 4 years following infusion

  24. Disease-specific response/progression endpoints: Myasthenia Gravis (MG) cohort

    Pace of tapering down prednisone therapy, defined as the average reduction in daily dose (mg/week) from baseline through study assessments, while maintaining ≥2 points MG-ADL improvement

    Measured: 4 years following infusion

  25. Disease-specific response/progression endpoints: Antiphospholipid antibody syndrome (APLA) cohort

    Number of patients with clinical improvement in Adjusted Global Antiphospholipid Syndrome Score (AGAPASS), time to achieving and maintaining of the improvement. Improvement = reduction from "high-risk" category (score ≥7) to a "low risk" category (score ≤ 6)

    Measured: 4 years following infusion

  26. Disease-specific response/progression endpoints: Antiphospholipid antibody syndrome (APLA) cohort

    Number of patients with stabilization in Damage Index for Antiphospholipid Syndrome (DIAPS), time to achieving and maintaining of the stabilization. Stabilization = no increase

    Measured: 4 years following infusion

  27. Disease-specific response/progression endpoints: Antiphospholipid antibody syndrome (APLA) cohort

    Rate of documented thrombotic (venous and arterial) events

    Measured: 4 years following infusion

  28. Disease-specific response/progression endpoints: Antiphospholipid antibody syndrome (APLA) cohort

    Rate of documented minor bleeding (clinically apparent bleeding that does not meet the criteria for major), major and fatal bleeding

    Measured: 4 years following infusion

  29. Disease-specific response/progression endpoints: Antiphospholipid antibody syndrome (APLA) cohort

    Rate of documented other new clinical events

    Measured: 4 years following infusion

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted25 Jul 2025
  2. Study start1 Sep 2024
  3. Primary completion1 Nov 2026 (estimated)
  4. Study completion1 Nov 2030 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
120 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07085676
Sponsor's protocol ID
HBI0101 ARD 001
Status last verified
Apr 2026
First submitted
6 May 2025
Last updated
6 May 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Undecided

Publications

  • Kfir-Erenfeld S, Asherie N, Grisariu S, Avni B, Zimran E, Assayag M, Sharon TD, Pick M, Lebel E, Shaulov A, Cohen YC, Avivi I, Cohen CJ, Stepensky P, Gatt ME. Feasibility of a Novel Academic BCMA-CART (HBI0101) for the Treatment of Relapsed and Refractory AL Amyloidosis. Clin Cancer Res. 2022 Dec 1;28(23):5156-5166. doi: 10.1158/1078-0432.CCR-22-0637.

    PubMed 36107221

  • Asherie N, Kfir-Erenfeld S, Avni B, Assayag M, Dubnikov T, Zalcman N, Lebel E, Zimran E, Shaulov A, Pick M, Cohen Y, Avivi I, Cohen C, Gatt ME, Grisariu S, Stepensky P. Development and manufacture of novel locally produced anti-BCMA CAR T cells for the treatment of relapsed/refractory multiple myeloma: results from a phase I clinical trial. Haematologica. 2023 Jul 1;108(7):1827-1839. doi: 10.3324/haematol.2022.281628.

    PubMed 36200421

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing HBI0101 CART in people with Systemic Sclerosis (SSc) and Idiopathic Inflammatory Myopathy (IIM), run by Polina Stepensky at 1 site in Israel.

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 62 inclusion and 17 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: looking for participants now, as of the sponsor's last update on 6 May 2026. 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 Israel. 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?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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 Polina Stepensky, MD (972-2-6778353, Fainak@hadassah.org.il), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Part A: Establishment of the initial safety profile of HBI0101 CAR T (time frame: 21 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?

On the ClinicalTrials.gov record for NCT07085676, which also lists 2 linked publications.

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 6 May 2026.

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