Clinical trial NCT02221310: Immunochemotherapy and AlloSCT in Patients With High Risk CD33+ AML/MDS
Official title: A Pilot Study of Gemtuzumab Ozogamicin in Combination With Busulfan and Cyclophosphamide (Immunochemotherapy) and Allogeneic Stem Cell Transplantation in Patients With High Risk Acute Myelogenous Leukemia and Myelodysplastic Syndrome
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Acute Myelogenous Leukemia
Myelodysplastic Syndrome
Keywords: targeted therapy
leukemia
MDS
immune therapy
Lead sponsor: New York Medical College
Sponsor class: OTHER
First posted: 2014-08-20
Enrollment: 25
Sex: ALL
Maximum age (years): 25
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: Targeted immune therapy with gemtuzumab ozogamicin (Mylotarg) in combination with chemotherapy followed by allogeneic stem cell transplantation will be given to patients with high risk acute myelogenous leukemia (AML) or myelodysplastic syndrome (MDS).

Detailed description: Gemtuzumab ozogamicin in combination with busulfan and cyclophosphamide (immunochemotherapy) conditioning followed by allogeneic stem cell transplantation + anti-thymocyte globulin (unrelated donors only) in patients with high risk CD33+AML or MDS meeting eligibility criteria.

Eligibility criteria: Inclusion Criteria:

* Disease Status:

  * History of AML Induction/Reinduction Failure
  * AML 1st Complete Remission (CR) with poor cytogenetics
  * AML 2nd CR with minimal residual disease (MRD)
  * AML 3rd CR
  * AML in refractory relapse but ≤25% bone marrow leukemia blasts
  * MDS with \>6% bone marrow blasts at diagnosis
  * Secondary MDS with ≤5% bone marrow myeloblasts at diagnosis

Disease Immunophenotype:

• Disease must express a minimum of \>/= 10% CD33+ for patients with AML

Organ Function:

• Adequate renal function, adequate liver function, adequate cardiac function, adequate pulmonary function Age: ≤25 years of age Donor: matched family donor, unrelated cord blood donor, unrelated adult donor

Exclusion Criteria:

* Patients with active central nervous system (CNS) AML/MDS disease at time of conditioning therapy
* Female patients who are pregnant
* Karnofsky \<50% or Lansky \<50% if 10 years or less
* Age \>25 years
* Has received gemtuzumab in the previous 30 days or has not recovered from prior gemtuzumab therapy.

Primary outcomes:
measure: Response rate
timeFrame: 1 year
Secondary outcomes:
Other outcomes:
Locations:
city: Valhalla
country: United States
facility: New York Medical College
state: New York
Study officials:
affiliation: New York Medical College
name: Mitchell S Cairo, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Satwani P, Bhatia M, Garvin JH Jr, George D, Dela Cruz F, Le Gall J, Jin Z, Schwartz J, Duffy D, van de Ven C, Foley S, Hawks R, Morris E, Baxter-Lowe LA, Cairo MS. A Phase I study of gemtuzumab ozogamicin (GO) in combination with busulfan and cyclophosphamide (Bu/Cy) and allogeneic stem cell transplantation in children with poor-risk CD33+ AML: a new targeted immunochemotherapy myeloablative conditioning (MAC) regimen. Biol Blood Marrow Transplant. 2012 Feb;18(2):324-9. doi: 10.1016/j.bbmt.2011.11.007. Epub 2011 Nov 9.