Short summary

The aim of this study is to assess the safety and efficacy of Venetoclax in a reduced dosis of 400 mg daily in combination with dexamethasone in patients with relapsed or refractory multiple myeloma who have translocation t(11;14). This is an open label, phase II, multicenter study that will be conducted in Denmark.

Rationale

Venetoclax-dexamethasone, although currently not an approved regimen in MM, seems to be a safe and effective treatment option in patients with relapsed and refractory MM, especially in patients who are positive for t(11;14). The aim of this phase II trial is to provide results about the safety and efficacy of this regimen specifically in the subgroup of patients with t(11:14). One of the objectives of this study is to assess the humoral immunodeficiency of subjects and to minimize the risk of pneumonia by anti-pneumococcal vaccination and infection prophylaxis.

Description of the cohort

Adults with relapsed or refractory multiple myeloma with t(11;14)

Data and biological material

Primary end-point

• Overall response rate

Secondary end-points

• Progression-free survival

• Clinical benefit rate

• Time to next treatment

• Overall survival

• Time to response

• Duration of response

• Safety and tolerability

• Discontinuation rate

• Quality of life

• Number of serious adverse events due to infections

• Duration of hospital admissions due to infections

Exploratory end-points

• Estimation of humoral immunodeficiency of subjects at baseline by measuring serum anti-pneumococcal polysaccharide IgG, IgA, IgM antibodies

• Assessment of the relation of humoral immunodeficiency to infections

• Assessment of pneumococcal vaccination response and its relation to infections

• Estimation of Bcl-2 overexpression by immunohistochemistry Assessment of the relation of Bcl-2 overexpression to the efficacy of the study treatment

Collaborating researchers and departments

Department of Hematology, Odense University Hospital

• Niels Abildgaard

Department of Haematology, Zealand Hospital, Roskilde

• Emil Hermansen