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Genetic Ablation of CD33 in HSC to Broaden the Therapeutic Index of CD33-directed Immunotherapy in Patients with AML

ConditionRelapsed/Refractory Acute Myeloid Leukemia (AML)
Trial statusNot yet recruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18+
SponsorGerman Cancer Research Center

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

confirmed AML according to the WHO classification

relapsed disease after allo-SCT from an HLA-identical family donor (≥ 2 months after allo-SCT at time of inclusion)

≤ 29% of bone marrow blasts as detected by cytomorphology or immunohistochemistry

age ≥ 18 years

Disqualifiers

ECOG performance status >2

Confirmed CNS involvement

Acute or chronic Graft versus Host disease (GvHD)

Availability of other curative standard treatment options

Trial design

Design model

Single group

Treatments tested in this trial

  • Donor-derived CD34+ HSC with CRISPR/Cas9-mediated CD33 deletion

    Biological/Vaccine

    CD33-deleted CD34+ hematopoietic stem cells derived from the initially matched family donor

  • Gemtuzumab Ozogamicin

    Drug

    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.

Treatment groups

12 Participants
are divided into 1 treatment group
Group A: Donor-derived CD33-deleted CD34+ HSC combined with Gemtuzumab-Ozogamicin (GO)Experimental treatment 2 interventions

Trial outcomes

Primary outcomes

1

engraftement of gene edited CD34+HSC

successful engraftement of gene edited CD34+HSC in the bone marrow

Time frame
on day 28
2

dose-limiting toxicity

dose-limiting toxicity (DLT) of Gemtuzumab-Ozogamicin

Time frame
until EOS (day 90)
3

toxicities according to the Common Terminology Criteria for Adverse Events (CTCAE v5.0)

frequency and grade of AEs with gene-edited HSC transplantation

Time frame
until EOS (day 90)

Secondary outcomes

1

Anti-tumor efficacy of study treatment in patients with dCD33+ relapsed AML after allo-SCT

overall response rate (ORR), complete response (CR), partial response (PR)) at day 90 (EOS) after last GO application)

Time frame
until EOS (day 90)
2

Time to response

Time to response (at least partial response) after the last GO application

Time frame
until EOS (day 90)
3

Overall response

Duration of overall response (DOR) after the last GO application

Time frame
until EOS (day 90)
4

Progression-free survival

Progression-free survival (PFS) after the last GO application

Time frame
until EOS (day 90)

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

German Cancer Research Center

Lead sponsor

University Hospital Heidelberg

Collaborator

University Hospital Dresden

Collaborator

Source ClinicalTrials.gov

This trial is not recruiting at the moment. You can still explore other options: