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Multiple MyelomaPhase 2KarMMaNCT03361748

Efficacy and Safety Study of bb2121 in Subjects With Relapsed and Refractory Multiple Myeloma

A Phase 2 study testing bb2121 in people with Multiple Myeloma, run by Celgene at 48 sites in 8 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

This is an open label, single-arm, multicenter, Phase 2 study to evaluate the efficacy and safety of bb2121 in subjects with relapsed and refractory multiple myeloma. A leukapheresis procedure will be performed to manufacture bb2121 chimeric antigen receptor (CAR) modified T cells. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Multiple Myeloma
What is being tested?
bb2121
Could I receive a placebo?
No, everyone receives an active treatment
How many people
149 participants
Where
48 sitesin 8 countries
Study dates
13 Dec 2017to 20 Dec 2023
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

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 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Eligibility is determined prior to leukapheresis. Subjects must satisfy the following criteria to be enrolled in the study:
  • Subject is ≥ 18 years of age at the time of signing the informed consent form (ICF).
  • Documented diagnosis of multiple myeloma
  • Must have received at least 3 prior MM treatment regimens. Note: induction with or without hematopoietic stem cell transplant and with or without maintenance therapy is considered a single regimen.
  • Must have undergone at least 2 consecutive cycles of treatment for each regimen, unless PD was the best response to the regimen.
  • Must have received a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 antibody.
Show all 13Show fewer
  • Must be refractory to the last treatment regimen.
  • Eastern Cooperative Oncology Group (ECOG) performance status 0 or 1.
  • Subjects must have measurable disease, including at least one of the criteria below:
  • Serum M-protein greater or equal to 1.0 g/dL
  • Urine M-protein greater or equal to 200 mg/24 h
  • Serum free light chain (FLC) assay: involved FLC level greater or equal to 10 mg/dL (100 mg/L) provided serum FLC ratio is abnormal
  • Recovery to Grade 1 or baseline of any non-hematologic toxicities due to prior treatments, excluding alopecia and Grade 2 neuropathy.

Who cannot join

  • The presence of any of the following will exclude a subject from enrollment:
  • Subjects with known central nervous system involvement with myeloma.
  • History or presence of clinically relevant central nervous system (CNS) pathology.
  • Subjects with active or history of plasma cell leukemia.
  • Subjects with solitary plasmacytomas or non-secretory myeloma without other evidence of measurable disease
  • Inadequate organ function
Show all 17Show fewer
  • Ongoing treatment with chronic immunosuppressants
  • Previous history of an allogeneic hematopoietic stem cell transplantation or treatment with any gene therapy-based therapeutic for cancer or investigational cellular therapy for cancer or BCMA targeted therapy
  • Evidence of human immunodeficiency virus (HIV) infection.
  • Seropositive for and with evidence of active viral infection with hepatitis B virus (HBV)
  • Seropositive for and with evidence of active viral infection with hepatitis B virus (HBV) and Hepatitis C virus (HCV)
  • Subjects with a history of class III or IV congestive heart failure (CHF) or severe non-ischemic cardiomyopathy, history of stroke, unstable angina, myocardial infarction, or ventricular arrhythmia within the previous 6 months.
  • Subjects with second malignancies in addition to myeloma if the second malignancy has required therapy in the last 3 years or is not in complete remission
  • Pregnant or lactating women.
  • Subject with known hypersensitivity to any component of bb2121 productThe presence of any of the following will exclude a subject from enrollment:
  • 1. Subjects with known central nervous system involvement with myeloma. 2. History or presence of clinically relevant central nervous system (CNS) pathology.
  • 3. Subjects with active or history of plasma cell leukemia. 4. Subjects with solitary plasmacytomas or non-secretory myeloma without other evidence of measurable disease 5. Inadequate organ function 6. Ongoing treatment with chronic immunosuppressants 7. Previous history of an allogeneic hematopoietic stem cell transplantation or treatment with any gene therapy-based therapeutic for cancer or investigational cellular therapy for cancer or BCMA targeted therapy 8. Evidence of human immunodeficiency virus (HIV) infection. 9. Seropositive for and with evidence of active viral infection with hepatitis B virus (HBV) and Hepatitis C virus (HCV) 10. Subjects with a history of class III or IV congestive heart failure (CHF) or severe non-ischemic cardiomyopathy, history of stroke, unstable angina, myocardial infarction, or ventricular arrhythmia within the previous 6 months. 11. Subjects with second malignancies in addition to myeloma if the second malignancy has required therapy in the last 3 years or is not in complete remission 12. Pregnant or lactating women. 13 Subject with known hypersensitivity to any component of bb2121 product, cyclophosphamide, fludarabine, or tocilizumab.

Trial locations and contacts

48 sites in 8 countries.

48 sites in 8 countries

United States18 sites

  • Emory University

    Atlanta, Georgia, 30322

  • Local Institution - 103

    Atlanta, Georgia, 30322

  • Local Institution - 107

    Boston, Massachusetts, 02114

  • Dana Farber Cancer Institute

    Boston, Massachusetts, 02115

  • Massachusetts General Hospital

    Boston, Massachusetts, 02214

  • Local Institution - 106

    Boston, Massachusetts, 02215

  • Local Institution - 104

    Dallas, Texas, 75390

  • University of Texas Southwestern Medical Center

    Dallas, Texas, 75390

  • Hackensack University Medical Center

    Hackensack, New Jersey, 07601

  • Local Institution - 102

    Hackensack, New Jersey, 07601

  • Local Institution - 101

    Nashville, Tennessee, 37203

  • Sarah Cannon Research Institute

    Nashville, Tennessee, 37203

  • Local Institution - 109

    New York, New York, 10029

  • Mt. Sinai Medical Center

    New York, New York, 10029

  • Local Institution - 105

    Rochester, Minnesota, 55905

  • Mayo Clinic

    Rochester, Minnesota, 55905

  • Local Institution - 108

    San Francisco, California, 94143

  • University of California - San Francisco

    San Francisco, California, 94143

Other trials in United States

Japan8 sites

  • Tokai University Hospital

    Isehara, Kanagawa, 259-1193

  • Local Institution - 803

    Isehara City, Kanagawa, 259-1193

  • Japan Red Cross Medical Center

    Shibuya-ku, Tokyo, 150-8935

  • Local Institution - 801

    Shibuya-ku, 150-8935

  • Jichi Medical University Hospital

    Shimotsuke, Tochigi, 3290498

  • Local Institution - 802

    Shimotsuke, 329-0498

  • Local Institution - 804

    Shinjuku, 162-8666

  • Tokyo Women's Medical University Hospital

    Shinjuku, 162-8666

Other trials in Japan

Germany6 sites

  • Universitatsklinikum Heidelberg Medizinische Klinik Krehl-Klinik Haematologie, Onkologie, Rheumato

    Heidelberg, Baden-Wurttemberg, 69120

  • Local Institution - 502

    Heidelberg, 69120

  • University of Tübingen

    Tübingen, Baden-Wurttemberg, 72076

  • Local Institution - 503

    Tübingen, 72076

  • Universitatsklinikum Würzburg

    Würzburg, Bavaria, 97080

  • Local Institution - 501

    Würzburg, 97080

Other trials in Germany

France4 sites

  • Centre Hospitalier Regional Universitaire de Lille-Hopital Calude Huriez Service des Maladies du Sang

    Lille, Hauts-de-France, 59037

  • Local Institution - 402

    Lille, 59037

  • Centre Hospitalier Universitaire de Nantes - Hotel Dieu

    Nantes, Pays de la Loire Region, 44093

  • Local Institution - 401

    Nantes, 44093

Other trials in France

Italy4 sites

  • Local Institution - 602

    Bergamo, 24128

  • Ospedali Riuniti di Bergamo

    Bergamo, 24128

  • Azienda Ospedaliero Universitaria Di Bologna Policlinico

    Bologna, Emilia-Romagna, 40138

  • Local Institution - 601

    Bologna, 40138

Other trials in Italy

Spain4 sites

  • Hospital Universitari Germans Trias i Pujol Can Ruti

    Badalona, Barcelona, 08916

  • Local Institution - 702

    Badalona (Barcelona), 08916

  • Clinica Universidad de Navarra

    Pamplona, Navarre, 31008

  • Local Institution - 701

    Pamplona, 31008

Other trials in Spain

Belgium2 sites

  • Universitaire Ziekenhuizen Leuven

    Leuven, Flemish Brabant, 3000

  • Local Institution - 201

    Leuven, B-3000

Other trials in Belgium

Canada2 sites

  • Local Institution - 301

    Toronto, Ontario, M5G 2M9

  • Princess Margaret Cancer Centre

    Toronto, M5G 2M9

Other trials in Canada

About this study

Official title: A Phase 2, Multicenter Study to Determine the Efficacy and Safety of bb2121 in Subjects With Relapsed and Refractory Multiple Myeloma

This is an open label, single-arm, multicenter, Phase 2 study to evaluate the efficacy and safety of bb2121 in subjects with relapsed and refractory multiple myeloma. A leukapheresis procedure will be performed to manufacture bb2121 chimeric antigen receptor (CAR) modified T cells. Prior to bb2121 infusion subjects will receive lymphodepleting therapy with fludarabine and cyclophosphamide.

Read the detailed description

Anti-myeloma bridging treatment is allowed for disease control while bb2121 is being manufactured.

Being tested
bb2121
Keywords
Multiple Myeloma, Efficacy and Safety, BB2121, CAR T Cell, BCMA, Relapsed and Refractory
Medical subject headings
Multiple Myeloma, Recurrence

What participants receive

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

  • bb2121Biological

    : bb2121 consists of autologous T lymphocytes transduced with an anti-BCMA02 CAR lentiviral vector to express a chimeric antigen receptor targeting the human B cell maturation antigen (anti-BCMA CAR).

Study groups in NCT03361748
Study groupRoleWhat they receive
Administration of bb2121Experimentalbb2121 autologous CAR T cells will be infused at a dose ranging from 15 - 450 x 10^6 CAR+ T cells after receiving lymphodepleting chemotherapy.
Receives: Biological: bb2121

What is being measured

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

  1. Overall Response Rate

    Number of participants who achieved partial response (PR) or better according to IMWG Uniform Response Criteria for Multiple Myeloma as assessed by an independent response committee (IRC).

    Measured: From first dose to 24 Months

Secondary outcomes (20)

  1. Complete Response Rate

    Percentage of participants who achieved CR or sCR according to IMWG Uniform Response Criteria for Multiple Myeloma as assessed by an IRC.

    Measured: From first dose to 24 Months

  2. Time to Response

    Time from first bb2121 infusion to first documentation of response of PR or better.

    Measured: From first dose to initial response (approximately on average 1.2 months, max of 8.8 months)

  3. Duration of Response

    Time from first documentation of response or PR or better to first documentation of disease progression or death from any cause, whichever occurs first.

    Measured: From first dose to 24 months after first dose

  4. Progression Free Survival (PFS)

    Time from first bb2121 infusion to first documentation of progressive disease (PD), or death due to any cause, whichever occurs first.

    Measured: From first dose to 24 months after first dose

  5. Time to Progression (TTP)

    Time from first bb2121 infusion to first documentation of progressive disease (PD), or death due to any cause, whichever occurs first.

    Measured: From first dose to 24 months after first dose

  6. Overall Survival

    Time from first bb2121 infusion to time of death due to any cause.

    Measured: From screening to the end of follow up (approximately 5 years and 2 months)

  7. Number of Participants With Safety Related Events

    Number of participants with adverse events (AEs), adverse events of special interest (AESI), serious adverse events (SAEs), cytokine release syndrome, neurotoxicity, infection and clinically signifcant laboratory abnormalities.

    Measured: From screening to the end of follow up (approximately 5 years and 2 months)

  8. Cmax

    Cmax is defined as the maximum transgene level at Tmax

    Tmax: The time of maximum observed transgene level, obtained directly from the observed transgene level - time.

    Measured: From first dose to the end of follow up (Approximately 5 years)

  9. AUC 0-9M

    The AUC of the transgene level from the time of dosing to 9 months

    Measured: at 9 months post first dose (Approximtately 9 Months)

  10. Tmax

    Tmax: The time of maximum observed transgene level, obtained directly from the observed transgene level - time.

    Measured: From first dose to the end of follow up (Approximately 5 years)

  11. Number of Participants With Anti-CAR-Antibodies

    Number of Participants with Anti-CAR-Antibodies.

    Pre-postive is defined by last value before or on bb2121 infusion date is positive Post-positve is defined by at least one positive value post bb2121 infusion.

    Measured: From first dose to the end of follow up (Approximately 5 years)

  12. Percentage of Participants Who Achieved >= VGPR and MRD Negative Status

    Percentage of participants who achieved ≥ VGPR and MRD negative status at a sensitivity of 10-⁵ at any time point within 3 months prior to achieving at least VGPR until the time of PD/death.

    MRD in the bone marrow will be measured using both next generation sequencing (NGS) techniques measuring immunoglobulin gene rearrangements of the malignant clone.

    MRD will be reported with a sensitivity of 10-⁴, 10-⁵, and 10-⁶ nucleated cells. The primary analysis for MRD negative response will use the sensitivity of 10-⁵.

    MRD = Minimal Residual Disease PD = Progressive Disease VGPR = Very good partial response.

    Measured: From screening to the end of follow up (Approximately 5 years and 2 months)

  13. Mean Change From Baseline on the EORTC QLQ-C30 - Fatigue.

    Mean change from baseline on the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30)

    The QLQ-C30 employs a week recall period for all items. All items will be scored from 0 to 100. The average of the scores will represent the symptoms score. A high score for a symptom scale/item represents a high level of symptomatic problem.

    Measured: At Day 1 and at specific time points up to month 24

  14. Mean Change From Baseline on the EORTC QLQ-C30 - Pain

    Mean change from baseline on the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30)

    The QLQ-C30 employs a week recall period for all items. All items will be scored from 0 to 100. The average of the scores will represent the symptoms score. A high score for a symptom scale/item represents a high level of symptomatic problem.

    Measured: At Day 1 and at specific time points up to month 24

  15. Mean Change From Baseline on the EORTC QLQ-C30 - Physical Functioning

    The QLQ-C30 employs a week recall period for all items. All items will be scored from 0 to 100 and an average will be taken. This average is the overall score. A higher scale score represents a higher level of well-being and better ability of daily functioning. Thus, a high score for a functional scale represents a high/healthy level of functioning.

    Measured: At Day 1 and at specific time points up to month 24

  16. Mean Change From Baseline on the EORTC QLQ-C30 - Cognitive Functioning

    The QLQ-C30 employs a week recall period for all items. All items will be scored from 0 to 100 and an average will be taken. This average is the overall score. A higher scale score represents a higher level of well-being and better ability of daily functioning. Thus, a high score for a functional scale represents a high/healthy level of functioning.

    Measured: At Day 1 and at specific time points up to month 24

  17. Mean Change From Baseline on the EORTC QLQ-C30 - Global Heath/QoL

    Mean change from baseline on the EORTC QLQ-C30

    The EORTC QLQ-C30 is a 30-item scale composed of both multi-item scales and single-item measures. All of the scales and single-item measures range in score from 0 to 100. A higher scale score represents a higher level of well-being and better ability of daily functioning. Thus, a high score for a functional scale represents a high/healthy level of functioning; a high score for the global health status/HRQoL represents a high HRQoL.

    Measured: At Day 1 and at specific time points up to month 24

  18. Mean Change From Baseline on the EORTC QLQ-MY20 - Disease Symptoms

    Mean change from baseline on the EORTC QLQ-MY20

    The EORTC has developed a myeloma module referred to as QLQ- MY20, to be administered alongside the core QLQ-C30. The QLQ-MY20 is a 20-item myeloma module intended for use among patients varying in disease stage and treatment modality.

    All items will be scored from 0 to 100. The average of the scores will represent the symptoms score. A high score for a symptom scale/item represents a high level of symptomatic problem.

    Measured: At Day 1 and at specific time points up to month 24

  19. Mean Change From Baseline on the EORTC QLQ-MY20 - Side Effects

    Mean change from baseline on the EORTC QLQ-MY20

    The EORTC has developed a myeloma module referred to as QLQ- MY20, to be administered alongside the core QLQ-C30. The QLQ-MY20 is a 20-item myeloma module intended for use among patients varying in disease stage and treatment modality.

    All items will be scored from 0 to 100. The average of the scores will represent the symptoms score. A high score for a symptom scale/item represents a high level of symptomatic problem.

    Measured: At Day 1 and at specific time points up to month 24

  20. Mean Change From Baseline on the EQ-5D-5L Index

    The European Quality of Life 5D-5L Scale (EQ-5D-5L) assesses general health-related quality of life. Health is defined in 5 dimensions: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Each dimension has 5 levels: no problems, slight problems, moderate problems, severe problems, and extreme problems. Responses are coded so that a '1' indicates no problem, and '5' indicates the most serious problem. The responses for the 5 dimensions are combined in a 5-digit number. The EQ-5D-5L health utility index (HUI) is assessed using the Crosswalk algorithm for France based on the individual responses to the 5 EQ-5D-5L domains ranging from -0.530 to 1.000. The smallest change considered clinically meaningful, is defined as a score difference of 0.08 points.

    Measured: At Day 1 and at specific time points up to month 24

Timeline

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

  1. First posted5 Dec 2017
  2. Study start13 Dec 2017
  3. Primary completion20 Dec 2023
  4. Study completion20 Dec 2023

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 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
149 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03361748
Sponsor's protocol ID
BB2121-MM-001
Other IDs
U1111-1202-5554, 2017-002245-29, 2017
Acronym
KarMMa
Status last verified
May 2025
First submitted
29 Nov 2017
Last updated
23 May 2025
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • Raje N, Berdeja J, Lin Y, Siegel D, Jagannath S, Madduri D, Liedtke M, Rosenblatt J, Maus MV, Turka A, Lam LP, Morgan RA, Friedman K, Massaro M, Wang J, Russotti G, Yang Z, Campbell T, Hege K, Petrocca F, Quigley MT, Munshi N, Kochenderfer JN. Anti-BCMA CAR T-Cell Therapy bb2121 in Relapsed or Refractory Multiple Myeloma. N Engl J Med. 2019 May 2;380(18):1726-1737. doi: 10.1056/NEJMoa1817226.

    PubMed 31042825

  • Munshi NC, Anderson LD Jr, Shah N, Madduri D, Berdeja J, Lonial S, Raje N, Lin Y, Siegel D, Oriol A, Moreau P, Yakoub-Agha I, Delforge M, Cavo M, Einsele H, Goldschmidt H, Weisel K, Rambaldi A, Reece D, Petrocca F, Massaro M, Connarn JN, Kaiser S, Patel P, Huang L, Campbell TB, Hege K, San-Miguel J. Idecabtagene Vicleucel in Relapsed and Refractory Multiple Myeloma. N Engl J Med. 2021 Feb 25;384(8):705-716. doi: 10.1056/NEJMoa2024850.

    PubMed 33626253

  • Sidana S, Ahmed N, Akhtar OS, Brazauskas R, Oloyede T, Bye M, Hansen D, Ferreri C, Freeman CL, Afrough A, Anderson LD Jr, Dhakal B, Dhanda D, Gowda L, Hashmi H, Harrison MJ, Kitali A, Landau H, Mirza AS, Patwardhan P, Qazilbash M, Usmani S, Patel K, Nishihori T, Ganguly S, Pasquini MC. Standard-of-care idecabtagene vicleucel for relapsed/refractory multiple myeloma. Blood. 2025 Jul 10;146(2):167-177. doi: 10.1182/blood.2024026216.

    PubMed 40198886

  • Delforge M, Otero PR, Shah N, Moshkovich O, Braverman J, Dhanda DS, Lanar S, Devlen J, Miera M, Gerould H, Campbell TB, Munshi NC. Analysis of patient-reported experiences up to 2 years after receiving idecabtagene vicleucel (ide-cel, bb2121) for relapsed or refractory multiple myeloma: Longitudinal findings from the phase 2 KarMMa trial. Leuk Res. 2023 Jun;129:107074. doi: 10.1016/j.leukres.2023.107074. Epub 2023 Apr 3.

    PubMed 37087950

  • Connarn JN, Witjes H, van Zutphen-van Geffen M, de Greef R, Campbell TB, Hege K, Zhou S, Lamba M. Characterizing the exposure-response relationship of idecabtagene vicleucel in patients with relapsed/refractory multiple myeloma. CPT Pharmacometrics Syst Pharmacol. 2023 Nov;12(11):1687-1697. doi: 10.1002/psp4.12922. Epub 2023 Feb 15.

    PubMed 36794354

  • Karampampa K, Zhang W, Venkatachalam M, Cotte FE, Dhanda D. Cost-effectiveness of idecabtagene vicleucel compared with conventional care in triple-class exposed relapsed/refractory multiple myeloma patients in Canada and France. J Med Econ. 2023 Jan-Dec;26(1):243-253. doi: 10.1080/13696998.2023.2173466.

    PubMed 36705644

  • Shah N, Mojebi A, Ayers D, Cope S, Dhanasiri S, Davies FE, Hari P, Patel P, Hege K, Dhanda D. Indirect treatment comparison of idecabtagene vicleucel versus conventional care in triple-class exposed multiple myeloma. J Comp Eff Res. 2022 Jul;11(10):737-749. doi: 10.2217/cer-2022-0045. Epub 2022 Apr 29.

    PubMed 35485211

  • Sharma P, Kanapuru B, George B, Lin X, Xu Z, Bryan WW, Pazdur R, Theoret MR. FDA Approval Summary: Idecabtagene Vicleucel for Relapsed or Refractory Multiple Myeloma. Clin Cancer Res. 2022 May 2;28(9):1759-1764. doi: 10.1158/1078-0432.CCR-21-3803.

    PubMed 35046063

  • Delforge M, Shah N, Miguel JSF, Braverman J, Dhanda DS, Shi L, Guo S, Yu P, Liao W, Campbell TB, Munshi NC. Health-related quality of life with idecabtagene vicleucel in relapsed and refractory multiple myeloma. Blood Adv. 2022 Feb 22;6(4):1309-1318. doi: 10.1182/bloodadvances.2021005913.

    PubMed 34933328

  • Da Via MC, Dietrich O, Truger M, Arampatzi P, Duell J, Heidemeier A, Zhou X, Danhof S, Kraus S, Chatterjee M, Meggendorfer M, Twardziok S, Goebeler ME, Topp MS, Hudecek M, Prommersberger S, Hege K, Kaiser S, Fuhr V, Weinhold N, Rosenwald A, Erhard F, Haferlach C, Einsele H, Kortum KM, Saliba AE, Rasche L. Homozygous BCMA gene deletion in response to anti-BCMA CAR T cells in a patient with multiple myeloma. Nat Med. 2021 Apr;27(4):616-619. doi: 10.1038/s41591-021-01245-5. Epub 2021 Feb 22.

    PubMed 33619368

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing bb2121 in people with Multiple Myeloma, run by Celgene at 48 sites in 8 countries.

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 13 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: finished, as of the sponsor's last update on 23 May 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 48 sites in United States, Japan, Germany, France, Italy and 3 more countries. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 23 May 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Overall Response Rate (time frame: From first dose to 24 Months). 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 NCT03361748, which also lists 10 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 23 May 2025.

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