Clinical trial · Interventional
Genetic Ablation of CD33 in HSC to Broaden the Therapeutic Index of CD33-directed Immunotherapy in Patients with AML
Genetic Ablation of CD33 in Hematopoietic Stem Cells to Broaden the Therapeutic Index of CD33-directed Immunotherapy in Patients with Acute Myeloid Leukemia (AML)
NCT05662904CI-TRIAL-00087216GALAXY33not yet recruitingPhase 1ClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Relapsed/Refractory Acute Myeloid Leukemia (AML) | Acute Myeloid Leukemia | CURATED_BROADER | 0.80 |
Interventions
Interventions (2)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Donor-derived CD34+ HSC with CRISPR/Cas9-mediated CD33 deletion | Biological | — | UNRESOLVED |
| Gemtuzumab Ozogamicin | Drug | Gemtuzumab Ozogamicin | ALIAS |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Donor-derived CD33-deleted CD34+ HSC combined with Gemtuzumab-Ozogamicin (GO)
- description
- Patients will be transplanted with CD33-deleted CD34+ HSC derived from the initially matched family donor. Upon HSC engraftment, patients will be treated with escalating doses of the anti-CD33 antibodydrugconjugate Gemtuzumab-Ozogamicin (GO). A conditioning regimen containing GO (d-14, d-11, d-8), Fludarabine 30 mg/m2 (d-6 to d-3) and Melphalan 140mg/m2 (d-2) is used prior to transplantation.
- interventionNames
- Biological: Donor-derived CD34+ HSC with CRISPR/Cas9-mediated CD33 deletion
- Drug: Gemtuzumab Ozogamicin
Primary outcomes (3)
- measure
- engraftement of gene edited CD34+HSC
- timeFrame
- on day 28
- description
- successful engraftement of gene edited CD34+HSC in the bone marrow
- measure
- dose-limiting toxicity
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
Show eligibility criteria text
Key Inclusion Criteria:
* 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
* confirmed CD33 expression on leukemic blasts at current relapse (as detected by flow cytometry)
* adequate organ function:
* Renal function defined as: serum creatinine of ≤ 2x ULN or eGFR ≥ 30 mL/min/1.73 m2
* Liver function defined as:
* ALT ≤ 3 times the ULN for the respective age
* Bilirubin ≤ 2.0 mg/dl with the exception of patients with hyperbilirubinemia explained by Gilbert-Meulengracht syndrome (may be included if total bilirubin is ≤ 3.0 x ULN and direct bilirubin ≤ 1.5 x ULN) or extrahepatic disease (e.g. chronic hemolytic anemia)
* Minimum level of pulmonary reserve defined as ≤ grade 1 dyspnea and pulse oxygenation \> 90% on room air
* Hemodynamic stability and LVEF ≥ 40% as confirmed by echocardiogram
* Absolute lymphocyte count (ALC) ≥ 100/mm3
Key Exclusion Criteria:
* ECOG performance status \>2
* Confirmed CNS involvement
* Acute or chronic Graft versus Host disease (GvHD)
* Availability of other curative standard treatment options
* Prior treatment with GO
* Prior hepatic veno-occlusive disease (VOD) or sinusoidal obstruction syndrome (SOS)
* Uncontrolled active hepatitis B or C
* HIV-positivity
* Uncontrolled bacterial, viral or fungal infection
* Participation in another clinical trial at the time of screening
* Organ dysfunction (liver, kidney, lung, heart) that is a contraindication for conditioning therapy
* Severe concomitant disease (e.g. uncontrolled arterial hypertension, heart failure NYHA III-IV, uncontrolled diabetes mellitus, uncontrolled hyperlipidemia)
* Unstable angina and/or myocardial infarction within 3 months prior to screening
* Pregnant or nursing (lactating) womenReferences
Publications (0)
Data not yet available
No reference posted for this study.