Genetic Ablation of CD33 in Hematopoietic Stem Cells to Broaden the Therapeutic Index of CD33-directed Immunotherapy in Patients with Acute Myeloid Leukemia (AML)

Official Summary

The study "GALAXY33" is an open-label, prospective, nonrandomized, one arm phase I clinical trial in which patients with relapsed AML after allogeneic hematopoietic stem cell transplantation will be transplanted with CD33-deleted CD34+ HSC derived from the initially matched family donor.

Study Design

• Study Type: INTERVENTIONAL
• Allocation: NA
• Model: SINGLE_GROUP
• Masking: NONE
• Enrollment: 12 participants

Interventions

• BIOLOGICAL: Donor-derived CD34+ HSC with CRISPR/Cas9-mediated CD33 deletion — CD33-deleted CD34+ hematopoietic stem cells derived from the initially matched family donor
• DRUG: Gemtuzumab Ozogamicin — Intrapatient intra-individual dose escalation Level 0: GO day 1 Level 1: GO day 1, day 4 Level 2: GO day 1, day 4, day 7 with repetition after 21 to 28 days up to 84 days.

Primary Outcomes

• engraftement of gene edited CD34+HSC (on day 28)
• dose-limiting toxicity (until EOS (day 90))
• toxicities according to the Common Terminology Criteria for Adverse Events (CTCAE v5.0) (until EOS (day 90))

Secondary Outcomes

• Anti-tumor efficacy of study treatment in patients with dCD33+ relapsed AML after allo-SCT (until EOS (day 90))
• Time to response (until EOS (day 90))
• Overall response (until EOS (day 90))
• Progression-free survival (until EOS (day 90))
• Overall survival (until EOS (day 90))

Trial Locations

• University Hospital Dresden, Department of Medicine I, Dresden, Germany
• University Hospital Heidelberg, Internal Medicine V, Heidelberg, Germany

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