A phase I/II open-label, multi-center, dose-finding, safety, efficacy study on patients with higher risk myelodysplastic syndrome (MDS) and untreated acute myeloid leukemia (AML) who are ineligible for induction chemotherapy.
The Phase I part of the study consists of a dose exploration administered in 18 patients at a starting dose once every 2 weeks in a 28-day cycle. The dose is escalated and evaluated for the occurrence of dose-limiting toxicities using a standard 3+3 dose-escalation design. This dose exploration phase of the study established the safety and tolerability and the recommended Phase II dose and schedule in adult patients with higher risk MDS.
The Phase II part enrolls 175 patients and employs an open-label 2:1 randomized design to evaluate the effect of treatment at the recommended Phase II dose determined in Phase I.
IDDI as the Biometry Partner to Ensure Data Quality
- IDDI had been selected earlier for the review of previously collected outputs and for the Biostatistics support of the Sponsor’s program including the generation of clinical trial designs, protocols, and statistical analysis plan development. In this particularly important phase, the Sponsor benefited from IDDI’s consultant’s rich experience in oncology, clinical trial design and relationship with regulatory authorities including pre-IND meeting package submission.
- The Sponsor-selected IDDI as the full biometric partner including eClinical tools (EDC & RTSM), data collection, data cleaning and data analysis. It was important to have these tasks executed by one vendor to ensure data quality and coherence with trial design.
- Sponsor-selected another vendor for Clinical and Medical monitoring, ensuring a clear separation of tasks.
Multiple challenges
- Fast access to all collected data including eCRF, lab data, RTSM, external systems, etc.
- Complex protocol design and specific data collection requirements combined to ease site burden
- Covid-19 Implementation
Results
- FAST IMPLEMENTATION OF A COMPLEX CLINICAL TRIAL DESIGN THANKS TO INTEGRATED TECHNOLOGY
- EASY AND PRECISE DETERMINATION OF PHASE II DOSE
- COORDINATED IDENTIFICATION AND RESOLUTION OF MISSING DATA DUE TO COVID-19
- COHERENCE BETWEEN SDTM AND ADaM DATASETS