NCT04167696

Study in Relapsed/Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome Patients to Determine the Recommended Dose of CYAD-02

Open-label, Phase I, Multi-center Study to Determine in Relapsed/Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome Patients the Recommended Dose of CYAD-02 After a Non-myeloablative Preconditioning Chemotherapy Followed by a Potential Consolidation Cycle

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 27 (estimated)
• Sponsor: Celyad Oncology SA · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

An open-label, phase I, multi-center study to determine in relapsed/refractory (r/r) acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) patients the recommended dose of CYAD-02 after a non-myeloablative preconditioning chemotherapy followed by a potential CYAD-02 consolidation cycle for non-progressive patient. A maximum of 27 r/r AML/MDS patients will be evaluated in this study in case of no dose limiting toxicity (DLT) and no replacement of patients.

Detailed description

This open-label phase I, multi-center study aims to determine in relapsed/refractory acute myeloid leukemia or myelodysplastic syndrome patients the recommended dose of CYAD-02 after a non-myeloablative preconditioning chemotherapy followed by a potential CYAD-02 consolidation cycle for non-progressive patients. During dose escalation, three prespecified dose-levels of CYAD-02 will be evaluated in three cohorts. Patient enrollment during dose-escalation will be staggered according to the Fibonacci 3+3 design and extension of cohorts II and III will be done in parallel. The first CYAD-02 infusion will be administered after prior non-myeloablative preconditioning chemotherapy (CYFLU) administered on three consecutive days. Non-progressive patients meeting the criteria specified below may receive a consolidation cycle with three additional CYAD-02 infusions at a 2-week interval without prior preconditioning. For all patients who received at least one CYAD-02 infusion, the overall study duration will be approximately 15 years.

Conditions

• Acute Myeloid Leukemia
• Myelodysplastic Syndrome

Interventions

Type	Name	Description
BIOLOGICAL	CYAD-02	CYAD-02 is a Chimeric Antigen Receptor-T (CAR-T) administered after CYFLU.
DRUG	ENDOXAN	administered as preconditioning chemotherapy
DRUG	Fludara	administered as preconditioning chemotherapy

Timeline

• Start date: 2019-11-25
• Primary completion: 2021-12-01
• Completion: 2035-02-01
• First posted: 2019-11-19
• Last updated: 2020-06-09

Locations

5 sites across 2 countries: United States, Belgium

Regulatory

• FDA-regulated drug study