Randomization in phase II trials: No exemption based on sample size.

ABSTRACT: Conducting randomized clinical trials (RCTs) is challenging. For this reason, in earlier phases of drug development and rare diseases with limited sample sizes, there is a tendency to omit control groups and to replace them with historical information for perceived feasibility arguments. This has significant implications for interpretation of trial results. We present headline results from a trial in stem cell transplantation, where fludarabine and clofarabine were investigated for induction chemotherapy in high-risk acute myeloid leukaemia or advanced myelodysplastic syndrome. The trial was initially designed as single-arm trial (SAT) and is discussed here from the planning and assessment perspective. Changes in the treatment setting or patient selection may lead to 'promising results' in

SUBMITTER: Framke T 

PROVIDER: S-EPMC12381617 | biostudies-literature | 2025 Sep

REPOSITORIES: biostudies-literature