Clinical trial NCT01701323: Expanded Cord Blood Cell Infusion Following Combination Chemotherapy in Younger Patients With Relapsed or Refractory Acute Myeloid Leukemia
Official title: Pilot Study Evaluating the Use of Ex Vivo Expanded Cord Blood Progenitors as Supportive Care Following Chemotherapy (FLAG) in Patients With AML or Acute Leukemia of Ambiguous Lineage
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE1
Conditions: Acute Leukemia of Ambiguous Lineage
Acute Myeloid Leukemia
Lead sponsor: Nohla Therapeutics, Inc.
Sponsor class: INDUSTRY
Collaborators: name: National Cancer Institute (NCI)
name: Fred Hutchinson Cancer Center
First posted: 2012-10-05
Enrollment: 7
Sex: ALL
Minimum age (years): 6
Maximum age (years): 30
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This pilot clinical trial studies infusion of expanded cord blood hematopoietic progenitor cells following combination chemotherapy in treating younger patients with acute myeloid leukemia that has relapsed or has not responded to treatment. Chemotherapy drugs work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Chemotherapy also kills healthy infection-fighting cells, increasing the risk of infection. The infusion of expanded cord blood hematopoietic progenitor cells may be able to replace blood-forming cells that were destroyed by chemotherapy. This cellular therapy may decrease the risk of infection following chemotherapy.


Eligibility criteria: Inclusion Criteria:

* Patients must have a diagnosis of AML or acute leukemia of ambiguous lineage according to World Health Organization (WHO) classification with \>= 5% of disease in bone marrow (BM)
* Recipients of prior allogeneic hematopoietic stem cell transplantation for AML or acute leukemia of ambiguous lineage are eligible if they do not have graft-versus-host disease (GVHD) or they have quiescent GVHD whether or not they are receiving immunosuppressive therapy
* Must have a Lansky or Karnofsky performance status of \>= 50; use Karnofsky for patients \> 16 years of age and Lansky for patients =\< 16 years of age
* Patients must have recovered from the acute toxicity of all prior chemotherapy
* The following amounts of time must have elapsed prior to entry on study:

  * 2 weeks from local radiation therapy (XRT)
  * 8 weeks from prior craniospinal or if \> 50% of the pelvis has been irradiated
  * 6 weeks must have elapsed if other bone marrow radiation has occurred
* Adequate cardiac, renal, pulmonary, and hepatic function
* Patient must have a life expectancy of at least 2 months
* Females of childbearing potential must have a negative serum pregnancy test performed within 7 days prior to the start of treatment
* Females of childbearing potential and males should agree to use adequate contraception (barrier method of birth control) prior to study entry and for the duration of study participation

Exclusion Criteria:

* Recipients of prior allogeneic hematopoietic stem cell transplant (HSCT) with active acute or chronic GVHD
* Patients with history of Down's syndrome, Fanconi anemia or other known marrow failure condition
* Patients currently receiving other investigational drugs are not eligible
* Current concomitant chemotherapy, radiation therapy, or immunotherapy other than as specified in the protocol with the exception of intrathecal chemotherapy; this includes the tyrosine kinase inhibitor sorafenib which must not be initiated until patient demonstrates count recovery
* Patients with a systemic fungal, bacterial, viral, or other infection not controlled despite appropriate antibiotics or other treatment; uncontrolled systemic infections require infectious disease consultation for verification
* Patients who are platelet refractory prior to initiation of protocol therapy
* Pregnant or lactating patients
* Any significant concurrent disease, illness, or psychiatric disorder that would compromise patient safety or compliance, interfere with consent, study participation, follow up, or interpretation of study results

Primary outcomes:
measure: Incidence of NCI CTCAE grade > 3 infusional toxicities
timeFrame: Up to 2 years
measure: Occurrence of transfusion associated graft versus host disease
timeFrame: Up to 2 years
measure: Incidence of platelet refractoriness in the presence of alloimmunization as a direct result of ex vivo expanded cord blood product infusion
timeFrame: Up to 2 years
measure: Incidence of delayed marrow recovery
timeFrame: Up to day 42
measure: Rate of treatment related mortality
timeFrame: Up to 2 years
Secondary outcomes:
measure: Time to neutrophil recovery
timeFrame: Up to 2 years
measure: In vivo persistence of ex vivo expanded cellular therapy
timeFrame: Up to 2 years
measure: Patient and infused expanded cord blood cells immune interaction
timeFrame: Up to 2 years
measure: Incidence of NCI CTCAE grade 3 or 4 infections
timeFrame: First 30 days following FLAG administration
measure: Incidence of NCI CTCAE grade > 3 chemotherapy-related toxicity in the first 30 days following fludarabine phosphate, cytarabine, and filgrastim (FLAG) therapy
timeFrame: First 30 days following FLAG administration
measure: Rate of complete remission
timeFrame: Up to 2 years
measure: Leukemia-free survival
timeFrame: Up to 2 years
measure: Overall survival
timeFrame: Up to 2 years
Other outcomes:
Locations:
city: Atlanta
country: United States
facility: Emory University/Winship Cancer Institute
state: Georgia
city: Seattle
country: United States
facility: Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium
state: Washington
Study officials:
affiliation: Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium
name: Ann Dahlberg
role: PRINCIPAL_INVESTIGATOR
References: