ClinicalTrials.gov record NCT06547866
Study Evaluating the Efficacy and Tolerance of a Zanubrutinib and BGB-11417 Combination in Patients Previously Treated for Waldenström Macroglobulinemia
Open Label Phase 2 Study Evaluating the Efficacy and Tolerance of a Zanubrutinib and BGB-11417 Combination in Patients Previously Treated for Waldenström Macroglobulinemia. A FILO Study
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AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This open-label Phase 2 study tests zanubrutinib combined with BGB-11417 in people aged 18 and older with previously treated Waldenström macroglobulinemia who require therapy. Treatment lasts twenty 28-day cycles (about 18 months): cycle 1 is zanubrutinib alone, cycle 2 adds a ramp-up of BGB-11417, and cycles 3-20 give both drugs at full dose; participants are then followed for three years. The study tracks how many reach deep responses (complete or very good partial response), safety, and whether the monoclonal IgM (M‑protein) rebounds after stopping treatment.
What it is studying. Whether a time-limited combination of zanubrutinib plus BGB-11417 produces deep responses (CR or VGPR) and is tolerated in previously treated (relapsed/refractory) Waldenström macroglobulinemia, and to measure M‑protein rebound after treatment cessation.
What the researchers hope to learn. To measure the proportion of patients achieving Complete Response (CR) or Very Good Partial Response (VGPR); to assess overall and major response rates; to measure time to response and progression-free survival; to evaluate safety and tolerability; and to determine the incidence and severity of serum M‑protein (IgM) rebound after planned treatment cessation.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
This is a French multicenter open label non-randomized Phase II trial evaluating the efficacy and tolerance of a combination of oral zanubrutinib and BGB-11417 in subjects aged 18 years and older with previously treated Waldenström macroglobulinemia (WM) who require therapy according to the consensus panel criteria from the Second International Workshop on Waldenström's macroglobulinemia. population : Patients with previously treated Waldenstrom macroglobulinemia The investigational medicinal products (IMP) are Zanubrutinib (BGB- 3111) and BGB-11417.Treatment will be administered for a total of twenty 28 day cycles:
- Cycle 1 with zanubrutinib only
- Cycle 2 with zanubrutinib plus BGB-11417 ramp-up
- cycle 2, day 1 : 10mg
- cycle 2, day 2 : 20 mg
- cycle 2, day 3 : 40mg
- cycle 2, day 4-7 : 80md daily
- cycle 2, day 8 and beyond : 160 mg daily
- Cycles 3-20 with zanubrutinib plus BGB-11417 full dose
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- zanubrutinib + BGB-11417
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- Be ≥ 18-year-old.
- Have received at least 1 prior line of treatment (excluding treatment with any BTKi or Bcl-2 antagonist, see non-inclusion criteria).
- Provide written informed consent.
- Have an Eastern Cooperative Oncology Group (ECOG) performance status ≤ 3.
- Have adequate renal function defined as creatinine clearance ≥ 50 mL/min as determined by the Cockroft-Gault equation.
- Have adequate hepatic function defined as:
- total serum bilirubin ≤ 1.5 × ULN, unless bilirubin rise is due to Gilbert's syndrome or non-hepatic cause.
- alanine aminotransferase (ALAT) < 2 × ULN
- aspartate aminotransferase (ASAT) < 2 × ULN,
- Have adequate BM function defined as:
- absolute neutrophil count ≥ 1x109/L
- platelet count ≥ 75 x109/L
- For women of childbearing potential, a negative pregnancy test must be documented prior to enrollment.
- A woman is considered of childbearing potential, ie, fertile, following menarche and until becoming postmenopausal unless permanently sterile. Permanent sterilization methods include hysterectomy, bilateral salpingectomy, and bilateral oophorectomy.
- A post-menopausal state is defined as no menses for 12 months without an alternative medical cause.
- Agree to use a highly effective form of contraception with sexual partners throughout study participation (for female and male patients who are fertile). Patients using hormonal contraceptives (eg, birth control pills or devices) must use a barrier method of contraception (eg, condoms) as well.
- Ability to comply with study procedures, in the Investigator's opinion.
- Patient covered by any social security system
Exclusion criteria
- Have previously been treated with a BTK inhibitor.
- Have been previously treated with a bcl-2 antagonist.
- Have active central nervous system (CNS) disease as evidenced by cytology or pathology. In the absence of clinical signs of CNS disease, a lumbar puncture is not mandatory.
- Have significant or active cardiovascular disease:
- stage III to IV congestive heart failure (CHF) as determined by the New York Heart Association (NYHA) classification system for heart failure and/or with left ventricular ejection fraction < 50%
- myocardial infarction within 6 months before study treatment.
- unstable angina within 6 months before study treatment.
- uncontrolled atrial arrhythmia.
- history of clinically significant ventricular arrhythmias (e.g sustained ventricular tachycardia, ventricular fibrillation, torsades de pointe).
- uncontrolled hypertension.
- history of stroke or intracranial hemorrhage within 180 days before the first dose of study drugs
- QTcF interval > 450 ms on screening electrocardiogram (ECG) evaluation.
- Have a history of stroke or intracranial hemorrhage within 6 months before first dose of study drug, have a history of a severe bleeding disorder such as hemophilia A, hemophilia B, von Willebrand disease, or history of spontaneous bleeding requiring blood transfusion or other medical intervention:
- patients with constitutional hemophilia or von Willebrand's disease will be excluded.
- patients with acquired hemophilia will be excluded.
- Requires ongoing treatment with warfarin or warfarin derivatives.
- patients with acquired von Willebrand's disease related to WM can be included. i) if bleeding manifestations are considered as non-clinically significant (i.e.grade 2 or below) ii) or if bleeding manifestations have been corrected by plasma exchange
- Have received live vaccine within 4 weeks of inclusion.
- Receive other concomitant investigational therapy.
- Have a history of renal, neurologic, psychiatric, endocrinologic, metabolic, immunologic, or hepatic condition that, in the opinion of the Investigator, would adversely affect a subject's participation in the study.
- Have currently active, clinically significant Child-Pugh Class B or C hepatic impairment.
- Present an inability or difficulty swallowing capsules/tablets, malabsorption syndrome, or any disease or medical condition significantly affecting gastrointestinal function.
- Have a known allergy to either xanthine oxidase inhibitors or rasburicase or zanubrutinib (patients at risk for G6PD deficiency may be screened before enrolment).
- Are pregnant or lactating. Women of childbearing potential must agree to use highly effective contraception from the time of signing informed consent until end-of-treatment visit,
- ≥90 days after last dose of BGB-11417-101 and Zanubrutinib. Male patients must be abstinent, vasectomized, or agree to the use of barrier contraception in combination with other methods.
- Have a history of other active malignancies requiring treatment within 3 years of study entry, with exception of (1) localized basal cell or squamous cell carcinoma of the skin, (2), adequately treated in situ endometrial carcinoma, (3) incidental histology finding of prostate carcinoma, (4) previous malignancy confined and treated locally (surgery or other modality) with curative intent.
- Be known to be positive for HIV.
- Present evidence of other clinically significant uncontrolled condition(s) including, but not limited to:
- uncontrolled and/or active systemic infection (viral, bacterial or fungal) including COVID-19
- chronic hepatitis B virus (HBV) or hepatitis C (HCV) requiring treatment. Note: subjects with serologic evidence of prior vaccination to HBV (i.e. hepatitis B surface [HBs] antigen negative-, anti-HBs antibody positive and anti-hepatitis B core [c] antibody negative) or positive anti-HBc antibody from intravenous immunoglobulins (IVIG) may participate. Patients with serologic evidence of prior resolved infection can be included according to recommendations .
- Suffer from any condition or illness that, in the opinion of the Investigator or medical monitor, would compromise patient safety or interfere with the evaluation of the safety of the study drugs.
- Have received or consumed any of the following within 3 days prior to the first dose of study drugs:
- grapefruit or grapefruit products.
- Seville oranges (including marmalade containing Seville oranges).
- star fruit.
- Have received a treatment with any of the following prior to the first dose of study drugs:
- ≤7 days steroid therapy with anti-neoplastic intent.
- ≤ 7 days or 5 half-lives (whichever is longer) of any moderate or strong CYP3A4 inhibitor and ≤ 14 days or 5 half-lives, (whichever is longer) of moderate or strong CYP3A4 inducer before the first dose of study drugs.
- allogeneic or autologous stem cell transplantation or CAR-T cell therapy less than 3 months before the first dose of study drugs.
- Severe or debilitating pulmonary disease.
- Major surgery within 4 weeks of the first dose of study drug.
- Active and/or ongoing autoimmune anemia and/or autoimmune thrombocytopenia (eg, idiopathic thrombocytopenia purpura).
- Ongoing alcohol or drug addiction or any psychiatric condition(s) which would compromise ability to comply with study procedures.
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Show the detailed description from the registry
study design : Open label, multicenter phase 2 trial population : Patients with previously treated Waldenstrom macroglobulinemia Primary objective : To evaluate the efficacy of a combination of zanubrutinib and BGB-11417 given for a limited duration of time in Refractory/Relapsing (R/R) WM by the proportion of subjects achieving either a Complete Response (CR) or Very Good Partial Responses (VGPR).duration of time in Refractory/relapsing (R/R) Waldenstrom macroglobulinemia (WM) Secondary objectives:
- To further evaluate the efficacy of a combination of zanubrutinib and BGB-11417 given for a limited duration of time in R/R WM
- To determine the incidence and severity of serum M-protein (monoclonal IgM) rebound after planned cessation of the combination of zanubrutinib and BGB-11417-101 in R/R WM
- To evaluate the safety and tolerability of a combination of zanubrutinib and BGB-11417 given for a limited duration of time in R/R WM
Sample size : 102 patients Length of study: Inclusion period: 24 months Treatment duration: 18 months (twenty 28-days cycles) Follow-up period: 3 years Study treatment : The investigational medicinal products (IMP) are Zanubrutinib (BGB- 3111) and BGB-11417.Treatment will be administered for a total of twenty 28 day cycles:
- Cycle 1 with zanubrutinib only
- Cycle 2 with zanubrutinib plus BGB-11417 ramp-up
- Cycle 2 with zanubrutinib plus BGB-11417 ramp-up
*cycle 2, day 1 : 10mg
- cycle 2, day 2 : 20 mg
- cycle 2, day 3 : 40mg
- cycle 2, day 4-7 : 80md daily
- cycle 2, day 8 and beyond : 160 mg daily
- Cycles 3-20 with zanubrutinib plus BGB-11417 full dose
All patient will receive both drugs. Study procedures: Screening period: Assessments may be done up to 28 days before the treatment start and will include: Clinical assessments
- Medical history, WM history including prior treatment lines, previous IPSS WM score, histology, known cytogenetics and molecular features including MYD88, CXCR4 and TP53 mutational status
- Concomitant therapies
- Complete physical examination, including weight, height, vital signs, ECOG performance status, B symptoms, detailed evaluation of lymph nodes, liver and spleen and infection monitoring (including COVID-19 nasal test)
- IPSS WM score
Biological assessments
- Standard hematology tests including CBC (hemoglobin, WBC count, absolute differential count, platelet count)
- Blood coagulation tests including: prothrombin time, activated cephalin time, fibrinogen, von Willebrand factor (vWF; including vWF antigen [vWF:Ag] and vWF:Activity [WF:Act]; evaluation according to ABO blood group) and FVIII* *If acquired Willebrand syndrome according to hemorrhagic risk assessment, further analysis is encouraged.
- Blood chemistry tests including sodium, potassium, chloride, bicarbonate, fasting glucose, phosphate, blood urea nitrogen (BUN), creatinine, calcium, phosphate, magnesium, total, direct and indirect bilirubin, total protein, albumin, alanine aminotransferase (ALAT), aspartate aminotransferase (ASAT), lactate dehydrogenase (LDH), alkaline phosphatase (PAL), uric acid, C-reactive protein (CRP), beta2microglobulin and pregnancy test
- Troponin; NT-proBNP will be performed on blood
- Serum "M-protein" (monoclonal IgM) quantification based on protein electrophoresis or alternate methods (according to appendix 3), immunofixation, nephelometry, free light chains kappa and lambda, cryoglobulinemia
- IgG, IgA and IgM levels
- Serologies for HBV, HCV and HIV
Cardiovascular assessments:
- Cardiovascular evaluation by investigator:
o 12-lead ECG and QTc calculation o blood pressure after 5 minutes rest, average of 3 measurements separated by ≥ 1 minute.
- Cardiovascular evaluation by cardiologist*:
- 12-lead ECG with QTc calculation
- echocardiography
- 24h Holter ECG monitoring
- 24h Ambulatory blood pressure monitoring (ABPM)
Imaging assessments: -Whole-Body CT scan (neck, thorax, abdomen, pelvis) with tumor measurements Specific assessments:
- BM biopsy* (local laboratory).
*This can be omitted if already done within the past 3 months.
- BM aspirate for oLocal laboratory for Cytogenetic analysis and FISH for 17p oFILOthèque central laboratory, storage for future analysis: unsorted and CD19+ sorted cells for mutational profile.
- Blood MRD assessment on plasmatic cell-free tumor DNA (FILOthèque central laboratory).
Treatment period Clinical assessment:
- Concomitant therapies and adverse events, severe adverse events and adverse events of special interest (AE, SAE and AESI)
- Complete physical examination, including evaluation of lymph nodes, liver and spleen
Cardiovascular assessments: -Cardiovascular evaluation by investigator after 1, 3, 6, 9, 12, 15 and 18 cycles: o12-lead ECG and QTc calculation* oblood pressure after a 5 minute rest, average of 3 measurements separated by ≥ 1 minute.
- The 12-lead ECG by investigator is not mandatory if already done or planned during the cardiology consultation within one month (at cycle 3, 6, 12, and 18)
-Cardiovascular evaluation by cardiologist after 3, 6, 12, and 18 cycles: ocardiology consultation o24h Holter ECG monitoring* o24h ABPM** oEchocardiography***
- 24h Holter ECG will be repeated only at cycles 3, 6 and 18 (only risk patient)
- 24h ABMP will be repeated only at cycles 3, 12 and 18
- Echocardiography will be repeated at cycle 18.
Biological assessments (at day 1 +/- 7 days of each cycle):
- Standard hematology tests including CBC (hemoglobin, WBC count, absolute differential count, platelet count)
- Blood chemistry: including sodium, potassium, chloride, bicarbonate, fasting glucose, phosphate, BUN, creatinine, calcium, phosphate, magnesium, total, direct and indirect bilirubin, total protein, albumin, ALAT, ASAT, LDH, PAL, uric acid, CRP and pregnancy test.
During ramp up period: serum creatinine, sodium, potassium, chloride, bicarbonate, calcemia, phosphoremia, LDH is mandatory before initiation of any ramp up dose of BGB-11417, and 8 and 24 hours after drug intake.
- Troponin and NT-proBNP at cycles 1, 3, 6, 9, 12, 15 and 18
- Serum "M-protein" (monoclonal IgM) quantification based on protein electrophoresis or alternate methods (according to appendix 3) with additional immunofixation (to confirm CR)
- IgG, IgA and IgM levels
Imaging assessments: -Whole-body CT scan (neck, thorax, abdomen, pelvis) every 24 weeks (6 cycles) i.e. after cycles 3, 6, 12 and 18 in all subjects who had measurable nodal/extranodal disease at screening. Specific assessments: -FILOthèque central laboratory: Blood on specific tube for MRD assessment on plasmatic cell-free tumor DNA (at cycles 3, 6, 12 and 18), storage. Response evaluation: Throughout the treatment period, especially before "end of treatment (EOT) + 3 months (EVAL 2 ) evaluation":
- if a CR is suspected (negative IF), an unscheduled CT scan should be done followed by biological evaluations including BM biopsy (local laboratory) to confirm CR
- If a VGPR is suspected (≥90% reduction in serum M-protein (monoclonal IgM) level from baseline (but still detectable) and decreased lymphadenopathy/ splenomegaly (if present at baseline), an unscheduled CT scan should be done to confirm VGPR.
Evaluation 1 (EVAL 1): 1 month after the end of treatment This evaluation takes place 1 month after EOT either after completion of cycle 20 or after the last cycle in case of early discontinuation. This evaluation may detect M-protein rebound with or without other features of early progression. Clinical assessments:
- Concomitant therapies
- AE, SAE and AESI
- Complete physical examination including evaluation of lymph nodes, liver and spleen
Cardiovascular assessments: -Cardiovascular evaluation by investigator: o12-lead ECG and QTc calculation oBlood pressure after a 5 minute rest, average of 3 measurements separated by ≥ 1 minute Biological assessments:
- Standard hematology tests including CBC (hemoglobin, WBC count, absolute differential count, platelet count)
- Blood chemistry: sodium, potassium, chloride, bicarbonate, fasting glucose, phosphate, BUN, creatinine, calcium, phosphate, magnesium, total, direct and indirect bilirubin, total protein, albumin, ALAT, ASAT, LDH, PAL, uric acid, CRP, and pregnancy test.
- Serum "M-protein" (monoclonal IgM) quantification based on protein electrophoresis or alternate methods (according to appendix 3) with additional immunofixation (to confirm a CR)
- IgG, IgA and IgM levels
Evaluation 2 (=EVAL 2): 3 months after the end of treatment This evaluation takes place 3 months after EOT either after completion of cycle 20 or after the last cycle in case of early discontinuation. This evaluation may detect M-protein rebound with or without other features of early progression. Clinical assessments:
- Concomitant therapies
- AE, SAE and AESI
- Complete physical examination, including evaluation of lymph nodes, liver and spleen
Cardiovascular assessments: -Cardiovascular evaluation by investigator: o12-lead ECG and QTc calculation oBlood pressure after 5 minutes rest, average of 3 measurements separated by ≥ 1 minute -Cardiovascular evaluation by a cardiologist: oCardiology consultation o24h Ambulatory blood pressure monitoring (ABPM) oEchocardiography Biological assessments:
- Standard hematology tests including CBC (hemoglobin, WBC count, absolute differential count, platelet count)
- Blood chemistry: sodium, potassium, chloride, bicarbonate, fasting glucose, phosphate, BUN, creatinine, calcium, phosphate, magnesium, total, direct and indirect bilirubin, total protein, albumin, ALAT, ASAT, LDH, PAL, uric acid, CRP
- NT-proBNP
- Serum "M-protein" (monoclonal IgM) quantification based on protein electrophoresis or alternate methods (according to appendix 3) with additional immunofixation (to confirm a CR)
- IgG, IgA and IgM levels
Imaging and BM assessments:
- Whole body CT scan
- BM biopsy (local laboratory) if not performed during the treatment period for CR or progression.
- BM aspirate (FILOthèque central laboratory) for storage and analysis for molecular studies without CD19 sorting.
- Blood collected on specific tube (FILOthèque central laboratory) for assessment of plasmatic cell-free tumor DNA
Evaluation in case of suspected CR At any time throughout the study, especially before "EOT evaluation" when a CR is suspected based on a negative immunofixation, an additional evaluation should be realized in order to confirm the CR. This additional evaluation includes primarily:
- Repeated screening for cryoglobulinemia, in patients known to have significant cryoglobulinemia at screening.
- CT scan
Then, if both immunofixation and CT scan are consistent with CR, a biological assessment should be scheduled with: -BM biopsy (local laboratory) Evaluation in case of suspected VGPR At any time throughout the study, especially before "EOT evaluation", when a VGPR is suspected based on ≥90% reduction in M-protein (monoclonal IgM) level from baseline (but still detectable) and decreased lymphadenopathy/splenomegaly (if present at baseline), an additional evaluation should be realized in order to confirm the VGPR. This additional evaluation includes:
- Repeated screening for cryoglobulinemia, in patients known to have significant cryoglobulinemia at screening.
- CT scan: to confirm the reduction of deep-seated lymphadenopathy and splenomegaly (if present at baseline)
Evaluation in case of progression during treatment and follow-up period
- BM biopsy optional (local laboratory)
- BM aspirate, indicated according to study only in case of progression for :
- local laboratory: cytogenetics and FISH for 17p
- FILOthèque central laboratory: storage of unsorted and CD19+ sorted cells for molecular analyzes.
- Blood collected on specific tube for assessment of plasmatic cell-free tumor DNA (FILOthèque central laboratory).
Post-treatment follow-up period after EVAL 2 Two follow-up evaluations scheduled at 1 month (EVAL 1) and at 3 months (EVAL 2) have been already detailed above. Thereafter, all patients enrolled in the study will be followed until progression or death every 3 months the first year (FU6, FU9 and FU12), then every 6 months for a total follow up period of three years (FU18, FU24, FU30 and FU36). Clinical assessments:
- Concomitant therapies
- AE, SAE and AESI
- Complete physical examination, evaluation of lymph nodes, liver and spleen.
Cardiovascular assessments:
- Cardiovascular evaluation by investigator o12-lead ECG and QTc calculation oblood pressure after 5 minutes rest, average of 3 measurements separated by ≥ 1 minute
- Cardiovascular evaluation by a cardiologist (only at FU36) ocardiology consultation o24h ABPM oEchocardiography
Biological assessments:
- Standard hematology tests including CBC (hemoglobin, WBC count, absolute differential count, platelet count).
- Blood chemistry: sodium, potassium, chloride, bicarbonate, fasting glucose, phosphate, BUN, creatinine, calcium, phosphate, magnesium, total, direct and indirect bilirubin, total protein, albumin, ALAT, ASAT, LDH, PAL, uric acid, CRP
- NT-proBNP (only FU36)
- Serum "M-protein" (monoclonal IgM) quantification based on protein electrophoresis or alternate methods (according to appendix 3) with additional immunofixation (to confirm a CR)
- IgG, IgA and IgM levels
Imaging and BM assessments:
- Whole body CT scan if clinically indicated, especially in case of progression.
- BM biopsy (local laboratory)
- BM aspirate, indicated according to study, only in case of progression
Premature end of treatment (PEOT) A visit will be performed 4 weeks (EVAL 1) and 3 months (EVAL 2) after the last treatment day for patients who discontinue treatment prematurely and for patients withdrawing their consent (if possible), In case of PEOT for disease progression, patients will be evaluated End of study (EOS) The end of study becomes effective after the end of the last study visit of the last patient enrolled once the FU36 visit has been achieved. The end of study visit corresponds to the last follow-up visit (FU36). For patients withdrawing consent during the follow up period, no end of study visit is required. Observational study For all patients if they consent, the survival data will be collected every year for an observational study, in the e-CRF (Survival date and/or event date) until death, for OS analysis.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
Participants will take two oral study drugs over up to twenty 28‑day cycles: zanubrutinib alone in cycle 1, then zanubrutinib plus BGB‑11417 with a ramp‑up in cycle 2 to a 160 mg daily dose and both drugs at full dose in cycles 3–20. They will have frequent clinic visits with blood tests each cycle, ECGs and cardiology assessments (including some 24‑hour Holter and ambulatory blood‑pressure monitoring and echocardiography), and CT scans about every 24 weeks. Bone‑marrow biopsies are required at screening and may be repeated to confirm complete response or at progression; blood samples for minimal residual disease (MRD) testing and other laboratory monitoring are collected periodically. After treatment there are evaluations at 1 and 3 months, then regular follow‑up every 3 months for the first year and every 6 months up to a total follow‑up of 3 years.
How the study is structured. Open-label, non-randomized Phase 2 interventional trial in a single treatment group. Everyone receives zanubrutinib and BGB-11417: cycle 1 zanubrutinib alone, cycle 2 with a BGB-11417 ramp-up to 160 mg, then cycles 3–20 with both drugs at full dose (total 20 × 28‑day cycles). No placebo or comparison arm. Safety, blood tests, imaging, bone marrow sampling, and long-term follow-up visits are scheduled during and after treatment.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- The registry says recruitment has not started. When is it expected to open, and can I register interest now?
- The listed sites are in France. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on Waldenström macroglobulinemia
Important dates
- Study start
- Dec 1, 2025 (estimated)
- Primary completion
- Dec 31, 2028 (estimated)
- Study completion
- Dec 31, 2031 (estimated)
- Target enrollment
- 102 participants (estimated)
- Last updated on registry
- Nov 26, 2025
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- French Innovative Leukemia Organisation
- Collaborators
- BeiGene
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Adeline Lhermitte 📞 +33787888464 ✉ a.lhermitte@filo-leucemie.org
- Valérie Rouillé 📞 +33247473798 ✉ v.rouille@filo-leucemie.org
Participating sites (37)
- AMIENS - CH Amiens Picardie Site Sud
Amiens, 80054 · France
- Angers Chu
Angers, 49933 · France
- ANNECY - CH Annecy Genevois
Annecy, 74374 · France
- ARGENTEUIL - Centre hospitalier Victor Dupouy
Argenteuil · France
- BAYONNE - CH de la Côte Basque - Hématologie
Bayonne, 64109 · France
- BESANCON - Hôpital Jean Minjoz
Besançon, 25000 · France
- Bordeaux-Institut Bergonié
Bordeaux, 33076 · France
- CAEN - CHU Caen - IHBN
Caen, 14033 · France
- Clermont-Ferrand - Chu Estaing
Clermont-Ferrand, 63000 · France
- CRETEIL - CHU Henri Mondor
Créteil, 94000 · France
Terms used on this page · explanations by MedicalUpdates.ai
- open label
- No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
- randomized
- Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
- investigational
- A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
- inclusion criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- written informed consent
- The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
- ECOG
- A scale from 0 to 5 that describes how a person's condition affects daily activity. 0 means fully active; 1 means restricted in strenuous activity; 2 means able to self-care but unable to work; higher numbers mean more limitation.
- creatinine clearance
- A measure of kidney function, calculated from a blood creatinine level together with age, weight and sex.
- × ULN
- The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
- absolute neutrophil count
- The number of neutrophils, a type of white blood cell that fights infection, in a blood sample. Studies often require it to be above a set level.
- women of childbearing potential
- Studies often ask participants who could become pregnant, or could father a child, to use contraception during the study and for a period afterwards, because the effect of the treatment on a pregnancy is unknown.
- enrollment
- The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
- lumbar puncture
- A procedure in which a needle is placed between the bones of the lower back to collect spinal fluid or to give a treatment.
- QTcF
- A recording of the heart's electrical activity. QTc is one measurement from it; some drugs are avoided if it is prolonged.
- 5 half-lives
- The time it takes for the amount of a drug in the body to fall by half. 'Five half-lives' is often used as the point at which a previous drug has essentially left the body.
- CAR-T
- A treatment in which a person's own immune cells (T cells) are collected, modified in a laboratory to recognise a target, and returned to the body.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
- Primary objective
- The main result the study is designed to measure and report on. Other results, called secondary outcomes, are also collected.
- Refractory
- A condition that has not responded to a treatment. 'Relapsed' means it responded and then came back; 'refractory' means it did not respond in the first place.
- Complete Response
- No sign of the condition can be found with the tests used in the study. It does not always mean the condition is gone permanently, and the definition depends on the condition and the study.
- VGPR
- A response category used in some blood conditions meaning the disease marker has fallen a great deal but not disappeared. The exact definition depends on the condition and the study.
- Follow-up
- The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
- Screening period
- The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
- prior treatment
- Treatment a person has already had for the condition. A 'line of therapy' is one course of treatment; studies often require, or limit, the number of prior lines.
- free light chains
- Small pieces of antibody protein made by plasma cells and measured in a blood test. In some conditions, such as AL amyloidosis, abnormal free light chains are the source of the disease.
- echocardiography
- An ultrasound scan of the heart. A transesophageal echocardiogram is done through a probe passed into the food pipe for a closer view.
- CT scan
- An X-ray scan that builds detailed cross-section images of the body. 'High-resolution' CT (HRCT) is used to look closely at the lungs.
- biopsy
- Removal of a small piece of tissue so it can be examined under a microscope. 'Biopsy-proven' or 'histologically confirmed' means the diagnosis was confirmed by examining tissue under a microscope.
- MRD
- Very small amounts of disease that remain after treatment and can only be found with highly sensitive tests. 'MRD-negative' means none was detected at the test's limit. The exact test and threshold depend on the condition and the study.
- adverse events
- Any unwanted medical problem that happens during a study, whether or not it was caused by the treatment. A 'serious' adverse event is one that meets specific criteria, such as causing death, being life-threatening, requiring or prolonging hospital admission, causing significant disability, or another medically important outcome.
- baseline
- Measurements taken at the start of the study, before any study treatment, so that later results can be compared with them.
- Observational study
- A study in which researchers do not assign participants to a treatment or other intervention. They observe or measure what happens, which may include study visits, tests, questionnaires or a review of medical records.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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