NCT01020539

Allogeneic Stem Cell Transplantation Followed By Targeted Immune Therapy In Average Risk Leukemia

Allogeneic Stem Cell Transplantation Followed By Targeted Immune Therapy In Average Risk Acute Myelogenous Leukemia/Myelodysplastic Syndrome/Juvenile Myelomonocytic Leukemia (AML/MDS/JMML)

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 18 (actual)
• Sponsor: Columbia University · Academic / Other
• Sex: All
• Age: 1 Month – 30 Years
• Healthy volunteers: Not accepted

Summary

Allogeneic stem cell transplantation (AlloSCT) followed by targeted immune therapy Gemtuzumab Ozogamicin patients with acute myeloid leukemia (AML)/juvenile myelomonocytic leukemia (JMML)/myelodysplastic syndromes (MDS) will be safe and well tolerated.

Detailed description

Gemtuzumab Ozogamicin (CMA-676) is a chemotherapeutic agent consisting of a recombinant humanized anti-CD33 antibody conjugated with calicheamicin, a highly potent cytotoxic antitumor antibiotic. The antibody portion of Gemtuzumab binds specifically to the CD33 antigen, a sialic acid-dependent adhesion protein expressed on the surface of leukemic blasts, normal and leukemic myeloid colony-forming cells, including leukemic clonogenic precursors, but excluding pluripotent hematopoietic stem cells and nonhematopoietic cells. This results in formation of a complex that is internalized, upon which the calicheamicin derivative is released within the lysosomes of the myeloid cell. The free calicheamicin derivative then binds to deoxyribonucleic acid (DNA), resulting in DNA double strand breaks and consequential cell death. Over 80% of AML patients possess myeloid blast cells with CD33 surface antigen expression.

Conditions

• Acute Myelogenous Leukemia
• Myelodysplastic Syndrome
• Juvenile Myelomonocytic Leukemia

Interventions

Type	Name	Description
DRUG	Fludarabine	Conditioning Regimen
DRUG	Busulfan	Conditioning Regimen
DRUG	GVHD Prophylaxis	Can be FK506 (Tacrolimus/Prograf®), mycophenolate mofetil ((MMF)/Cellcept®), or methotrexate (MTX, amethopterin)
DRUG	Gemtuzumab Ozogamicin	Dose Escalation
DRUG	Anti-Thymocyte Globulin	Unrelated Donors only
DRUG	Isotretinoin	JMML patients only

Timeline

• Start date: 2002-09-11
• Primary completion: 2011-07-20
• Completion: 2020-12-01
• First posted: 2009-11-25
• Last updated: 2021-08-06

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study