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OncoMatch/Clinical Trials/NCT05099471

Efficacy of Venetoclax in Combination With Rituximab in Waldenström's Macroglobulinemia

Is NCT05099471 recruiting? Yes, currently enrolling (May 2026). This Phase 2 trial studies multiple treatments including Venetoclax; Rituximab and DRC for waldenstrom macroglobulinemia.

Phase 2RecruitingChristian BuskeNCT05099471Data as of May 2026

Treatment: Venetoclax; Rituximab · DRCIn Waldenström's macroglobulinemia (WM) chemotherapy induces only low CR/VGPR rates and response duration is limited. In addition, WM patients are often elderly, partly not tolerating chemotherapy related toxicities. Thus, innovative approaches are needed which combine excellent activity and tolerability in WM. Chemotherapy-free approaches are highly attractive for this patient group. Based on its high activity and favorable toxicity profile in indolent B-NHL such as CLL, Venetoclax was approved for the treatment of this diseases by the FDA and the European Medicines Agency (EMA). First data in relapsed/refractory WM have documented high activity and low toxicity of Venetoclax also in WM, including patients with prior Ibrutinib treatment or patients carrying CXCR4 mutations. Ibrutinib itself has high activity and a relatively low toxicity profile in WM, but has also major disadvantages: the main disadvantage is the need to apply this drug continuously. Furthermore, Ibrutinib efficacy depends largely on the genotype with a substantial drop in major responses and PFS in the presence of CXCR4 mutations and non-mutated MYD88. In particular the need of continuous treatment for Ibrutinib has prevented that Ibrutinib has become the standard of care outcompeting conventional Rituximab/chemotherapy. This is reflected in current guidelines such as the NCCN and the ESMO guidelines, which still see immunochemotherapy as a backbone of treatment, largely because of the advantage of a timely fixed application. Data in CLL in the relapsed as well as in the first line setting have convincingly shown that in contrast to Ibrutinib Venetoclax is highly efficient also when used in a timely defined application scheme over 12 months in combination with the anti-CD20 antibody Rituximab. Data documented deep responses including molecular responses and a highly significant advantage over immunochemotherapy in large international Phase III trials, changing the standard of care in this disease. Based on this the hypothesis is that timely fixed application of the combination of Venetoclax and Rituximab induces significantly superior treatment outcomes compared to chemotherapy and Rituximab (DRC) in patients with treatment naïve WM, regardless of the genotype. A first indication for this assumption in the proposed trial will allow the performance of confirmatory phase 3 trials that might change the standard of care in WM.

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Extracted eligibility criteria

Cancer type

Non-Hodgkin Lymphoma

Biomarker criteria

Allowed: MYD88 any tested

determination of the mutational status of MYD88 ... prior to randomization if the mutational status hasn't been determined before

Allowed: CXCR4 any tested

determination of the mutational status of ... CXCR4 prior to randomization if the mutational status hasn't been determined before

Performance status

ECOG 0–2(Ambulatory, capable of self-care)

World Health Organization (WHO) / ECOG performance status ≤ 2

Prior therapy

No prior treatment (treatment-naive required)
Max 0 prior lines

Cannot have received: chemotherapy

Chemotherapy with approved or investigational anticancer therapeutic within 21 days prior to start of therapy

Cannot have received: glucocorticoid therapy

Exception: ≤ 160 mg dexamethasone or equivalent for anti-neoplastic intent within 14 days prior to therapy

Glucocorticoid therapy within 14 days prior to therapy that exceeds a cumulative dose of 160 mg of dexamethasone or equivalent dose of other corticosteroids given for anti-neoplastic intent.

Lab requirements

Blood counts

Baseline platelet count ≥ 50x10^9/L, absolute neutrophil count ≥ 0.75x10^9/L (if not due to BM infiltration by the lymphoma)

Kidney function

creatinine clearance ≥ 30 mL/min; calculated by Cockcroft Gault or measured by 24h urine collection

Liver function

AST and ALT < 3.0 x ULN; Bilirubin ≤1.5 x ULN (unless due to Gilbert's syndrome or of non-hepatic origin)

Cardiac function

Left ventricular ejection fraction ≥ 40% by TTE

Adequate hepatic function per local laboratory reference range as follows: AST and ALT < 3.0 x ULN; Bilirubin ≤1.5 x ULN (unless bilirubin rise is due to Gilbert's syndrome or of non-hepatic origin). Subject must have adequate renal function as demonstrated by a creatinine clearance ≥ 30 mL/min; calculated by the Cockcroft Gault formula or measured by 24 hours urine collection. Baseline platelet count ≥ 50x10^9/L, absolute neutrophil count ≥ 0.75x10^9/L (if not due to BM infiltration by the lymphoma). Left ventricular ejection fraction ≥ 40% as assessed by transthoracic echocardiogram (TTE).

Structured fields extracted by AI. May contain errors — verify against the official protocol.

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