Expanded Cord Blood Cell Infusion Following Combination Chemotherapy in Younger Patients With Relapsed or Refractory Acute Myeloid Leukemia

NCT01701323 | Terminated Phase 1 DMC FDA Drug

• 4 other identifiers: 2584NCI-2012-017242584.00P30CA015704
• Study Type: interventional
• Target: 7
• Locations: 1 country
• Sites: 2

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.

Conditions

Acute Leukemia of Ambiguous Lineage; Acute Myeloid Leukemia

Outcome Measures

Primary Outcomes (5)

• Incidence of NCI CTCAE grade > 3 infusional toxicities

  Up to 2 years

• Occurrence of transfusion associated graft versus host disease

  Up to 2 years

• Incidence of platelet refractoriness in the presence of alloimmunization as a direct result of ex vivo expanded cord blood product infusion

  Up to 2 years

• Incidence of delayed marrow recovery

  Failure to achieve ANC \>= 500 cells/µl by day 42 post treatment with marrow cellularity \< 5% and marrow blast count \< 5%.

  Up to day 42

• Rate of treatment related mortality

  Up to 2 years

Secondary Outcomes (8)

• Time to neutrophil recovery

  Up to 2 years

• In vivo persistence of ex vivo expanded cellular therapy

  Up to 2 years

• Patient and infused expanded cord blood cells immune interaction

  Up to 2 years

• Incidence of NCI CTCAE grade 3 or 4 infections

  First 30 days following FLAG administration

• Incidence of NCI CTCAE grade > 3 chemotherapy-related toxicity in the first 30 days following fludarabine phosphate, cytarabine, and filgrastim (FLAG) therapy

  First 30 days following FLAG administration

Study Arms (1)

Treatment (Ex-vivo expanded cord blood progenitors)

EXPERIMENTAL

Patients receive filgrastim SC or IV on days 1-7, fludarabine phosphate IV QD over 30 minutes on days 2-6, cytarabine IV QD over 4 hours on days 2-6, and ex-vivo expanded cord blood progenitor cells IV over 30 minutes on day 8.

Biological: Ex-Vivo Expanded Cord Blood Progenitor Cell Infusion; Drug: Cytarabine; Drug: Filgrastim; Drug: Fludarabine Phosphate

Interventions

Ex-Vivo Expanded Cord Blood Progenitor Cell Infusion BIOLOGICAL

Given IV

Also known as: NLA101, Dilanubicel

Treatment (Ex-vivo expanded cord blood progenitors)

Cytarabine DRUG

Given IV

Treatment (Ex-vivo expanded cord blood progenitors)

Filgrastim DRUG

Given SC or IV

Treatment (Ex-vivo expanded cord blood progenitors)

Fludarabine Phosphate DRUG

Given IV

Treatment (Ex-vivo expanded cord blood progenitors)

Eligibility Criteria

• Age: 6 Months - 30 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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:
• weeks from local radiation therapy (XRT)
• weeks from prior craniospinal or if \> 50% of the pelvis has been irradiated
• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Nohla Therapeutics, Inc.: lead
• National Cancer Institute (NCI): collaborator
• Fred Hutchinson Cancer Center: collaborator

Study Sites (2)

Emory University/Winship Cancer Institute

Atlanta, Georgia, 30322, United States

Location

Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium

Seattle, Washington, 98109, United States

Location

MeSH Terms

Conditions

Leukemia, Biphenotypic, Acute; Leukemia, Myeloid, Acute

Interventions

Cytarabine; Filgrastim; fludarabine phosphate

Condition Hierarchy (Ancestors)

Leukemia, Lymphoid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases; Leukemia, Myeloid

Intervention Hierarchy (Ancestors)

Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Arabinonucleosides; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Granulocyte Colony-Stimulating Factor; Colony-Stimulating Factors; Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors

Study Officials

• Ann Dahlberg

  Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 3, 2012
• First Posted: October 5, 2012
• Study Start: December 10, 2012
• Primary Completion: May 10, 2018
• Study Completion: May 10, 2018
• Last Updated: March 1, 2019

Record last verified: 2019-02

Locations

US (2)