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A Phase I Trial of in Vitro Expanded Autologous Invariant Natural Killer Cells in Cancer

Phase 1
18 Years
Open (Enrolling)
Malignant Melanoma

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Trial Information

A Phase I Trial of in Vitro Expanded Autologous Invariant Natural Killer Cells in Cancer

- The first step in the study will be to collect iNKT cells from the participants
peripheral blood. For this procedure an intravenous catheter will be used to remove
the blood. The white blood cells will be removed from the blood and the red blood
cells and plasma will be returned to the participant (leukopheresis).

- We will then purify the iNKT cells from these collected white blood cells, and will
grow them in culture plates in the lab under strictly controlled sterile conditions.
This can take 4-6 weeks.

- If we are successful in growing the iNKT cells to large enough numbers, they will be
divided into 3 equal doses. Participants will receive one dose of these cells by
intravenous infusion every 2 weeks or days 1, 15 and 29. A blood sample will be taken
immediately before each infusion, and at 1 and 4 hours after each infusion.
Participants will be asked to return for blood samples on day 2, 3, 4 and 8 after

- The initial group of 3-6 participants will not receive any other therapy with the iNKT
cell infusions. However, the subsequent group of 6 patients will in addition receive
GM-CSF, which can further stimulate the immune system and may increase the effects of
the iNKT cells. This medication is administered by subcutaneous injection and will be
given daily for 10 days beginning the day of the second and third infusion.

- Participants will be on this research study for about 14 weeks, which includes the time
for the cell purification and culture, treatment, and follow-up observation.

Inclusion Criteria:

- Stage IV melanoma

- ECOG Performance Status 0-1

- Estimated life expectancy of 6 months or greater

- 18 years of age or older

- Adequate renal, hepatic and hematological function as outlined in protocol

- Adequate pulmonary and cardiac function as outlined in protocol

- Prior therapies must be discontinued at least 4 weeks prior to the leukopheresis to
obtain iNKT cells. This does not include palliative surgery or radiation therapy,
which may be used prior to leukopheresis or during the interval between leukopheresis
and iNKT cell reinfusion

- Melanoma patients must not have brain metastases based on a negative MRI obtained
within 4 weeks prior to screening, and must not have a history of brain metastases

- No other significant medical, surgical or psychiatric condition that, in the judgment
of the PI, would interfere with compliance to the protocol regimen

Exclusion Criteria:

- Pregnant or nursing women

- Active systemic infection, positive HIV, HBV, or HCV serology, or immune deficiency

- Autoimmune disease that currently requires systemic therapy with immunosuppressive

- Known hypersensitivity to GM-CSF or DMSO

- Other active malignancy other than squamous cell or basal cell of the skin

Type of Study:


Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

To determine the feasibility of isolating and expanding in vitro autologous iNKT cells from cancer patients for therapeutic use.

Outcome Time Frame:

2 years

Safety Issue:


Principal Investigator

Steven Balk, MD, PhD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Beth Israel Deaconess Medical Center


United States: Food and Drug Administration

Study ID:




Start Date:

February 2008

Completion Date:

Related Keywords:

  • Malignant Melanoma
  • iNKT
  • in vitro autologous iNKT
  • Melanoma



Beth Israel Deaconess Medical Center Boston, Massachusetts  02215
Dana-Farber Cancer Institute Boston, Massachusetts  02115