Phase I Study of GNKG168 in Acute Lymphoblastic Leukemia and Acute Myelogenous Leukemia



Status:Terminated
Conditions:Blood Cancer, Hematology
Therapuetic Areas:Hematology, Oncology
Healthy:No
Age Range:1 - 21
Updated:4/21/2016
Start Date:November 2012
End Date:July 2014

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A Phase I Study of GNKG168 in Pediatric Patients With Acute Lymphoblastic Leukemia or Acute Myeloid Leukemia (IND#113600)

This is a phase I trial of an investigational drug called GNKG168 in patients with relapsed
and refractory acute lymphoblastic leukemia (ALL) and acute myelogenous leukemia (AML) who
are in morphologic remission but are positive for Minimum Residual Disease (MRD). GNKG168 is
a Toll-like receptor (TLR) agonist. TLR agonists are a novel approach to stimulate an
effective anti-tumor immune response as they are able to stimulate both innate and adaptive
immune responses. There will be two strata i.e patients who have received hematopoietic stem
cell transplant (HSCT) and patients who have never undergone HSCT. GNKG168 will be
administered as a 60 min iv infusion. One 14-day cycle consists of 5-day treatment followed
by 9 day-rest. Patients will receive 2 cycles before evaluation. The primary objective is to
determine the maximum tolerated dose of GNKG168 in relapsed ALL and AML patients.


Inclusion Criteria:

- Patients must be ≥1 and ≤ 21 years of age when originally diagnosed with ALL or AML.

- Diagnosis

1. Patients must have previously histologically confirmed ALL or AML at original
diagnosis or previous relapse.

2. Patients must be in complete remission (CR) with less than 5% blasts in the bone
marrow.

- Post-HSCT patients should be in first or greater CR

- Patients who have never received HSCT should be in second or greater CR c. Patient
must have detectable MRD (≥0.01%) by flow cytometry as confirmed by Brent Woods' lab.
Results must be available at the time of enrollment.

- Karnofsky ≥ 50% for patients >16 years of age and Lansky ≥ 50% for patients ≤16 years
of age. (See Appendix I for Performance Scales)

- Patients must have fully recovered from the acute toxic effects of all prior
anti-cancer therapy.

- At least 14 days must have elapsed since any treatment with systemic chemotherapy
including high-dose steroid (prednisone>0.5 mg/kg or equivalent), radiotherapy,
biological therapy or any other investigational therapy. (Note: low-dose steroid;
prednisone ≤0.5 mg/kg/day or equivalent is allowed.)

- Patients who have never had a Hematopoietic Stem Cell Transplant (HSCT) must not be a
suitable candidate for HSCT.

- Previous Hematopoietic Stem Cell Transplant:

1. Patients having received HSCT are eligible.

2. Patients having received donor lymphocyte infusions (DLI) are eligible.

3. At least 60 days must have elapsed from the last DLI.

4. Must have ≥95% donor T-cell chimerism.

5. Patients must have been off all immune suppression drugs for 7 days before study
entry. (at least 2 weeks for high-dose steroid, i.e. prednisone>0.5 mg/kg or
equivalent; see section 3.3.4 b) (Note; low-dose steroid; prednisone ≤0.5
mg/kg/day or equivalent is allowed.)

- Patients must have a serum creatinine that is less than or equal to 1.5 x the
institutional upper limit of normal according to age.

- Patient's alanine aminotransferase (ALT) and aspartate aminotransferase (AST) must be
less than or equal to 3 x institutional upper limit of normal.

- Patient's total bilirubin must be less than or equal to 1.5 x institutional upper
limit of normal.

- Patient must have a shortening fraction > 27% or an ejection fraction > 45% by
echocardiogram (ECHO) or multigated radionuclide angiography (MUGA) .

- Female patients of childbearing potential must have a negative urine or serum
pregnancy test confirmed prior to enrollment.

- Female patients with infants must agree not to breastfeed their infants while on this
study.

- Male and female patients of child-bearing potential must agree to use an effective
method of contraception approved by the investigator during the study.

- Patients must have an absolute neutrophil count > 1000/dL, platelets > 100,000/dL AND
absolute lymphocyte count > 200 which is not decreasing. Patients with previous HSCT
may have a platelet count > 50,000/dL.

Exclusion Criteria:

- Active grade 2 or higher acute GVHD at the time of study entry.

- Active chronic GVHD (moderate or severe). See Appendix 2 for Chronic GVHD Grading.

- Plan for donor lymphocyte infusions during the study period.

- Need for immunosuppressive medications including high-dose corticosteroids
(prednisone >0.5 mg/kg or equivalent) (Note: low-dose steroid; prednisone ≤0.5
mg/kg/day or equivalent is allowed.)

- Patients with a systemic fungal, bacterial, viral, or other infection not controlled
(defined as exhibiting ongoing signs/symptoms related to the infection and without
improvement, despite appropriate antibiotics or other treatment).

- Patient will be excluded if they are currently receiving other investigational drugs.

- Patients will be excluded if there is a plan to administer non-protocol chemotherapy,
radiation therapy, or immunotherapy during the study period.

- Patients will be excluded if they have significant concurrent disease, illness,
psychiatric disorder or social issue that would compromise patient safety or
compliance with the prescribed protocol therapy, interfere with consent, study
participation, follow up, or interpretation of study results.

- Patients with central nervous system 3 disease are excluded.
We found this trial at
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Ann Arbor, MI 48109Bus: -
Ann Arbor, Michigan 48109
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3400 N Charles St
Baltimore, Maryland 21205
410-516-8000
Johns Hopkins University The Johns Hopkins University opened in 1876, with the inauguration of its...
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700 Childrens Drive
Columbus, Ohio 43205
(616) 722-2000
Nationwide Children's Hospital At Nationwide Children’s, we are creating the future of pediatric health care....
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4650 Sunset Blvd
Los Angeles, California 90027
 (323) 660-2450
Childrens Hospital Los Angeles Children's Hospital Los Angeles is a 501(c)(3) nonprofit hospital for pediatric...
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South 34th Street
Philadelphia, Pennsylvania 19104
 215-590-1000
Children's Hospital of Philadelphia Since its start in 1855 as the nation's first hospital devoted...
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3181 Southwest Sam Jackson Park Road
Portland, Oregon 97239
503 494-8311
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Boston, Massachusetts
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Chicago, Illinois 60614
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Dallas, Texas 75390
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Forth Worth, Texas 76104
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Kansas City, Missouri 64108
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2801 Atlantic Ave
Long Beach, California 90806
(562) 933-5437
Miller Children's Hospital Miller Children
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Memphis, Tennessee 38105
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Miami, Florida 33136
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Minneapolis, Minnesota 55404
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2200 Children's Way
Nashville, Tennessee 37232
(615) 936-1000
Vanderbilt Children's Hospital Monroe Carell Jr. Children's Hospital at Vanderbilt is one of the nation's...
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New York, New York 10065
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New York, New York 10021
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Oakland, California
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Palo Alto, California 94304
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1919 E Thomas Rd
Phoenix, Arizona 85006
(602) 933-1000
Phoenix Children's Hospital Phoenix Children's Hospital has provided hope, healing, and the best healthcare for...
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San Francisco, California 94143
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4800 Sand Point Way NE
Seattle, Washington 98105
(206) 987-2000
Seattle Children's Hospital Seattle Children’s Hospital specializes in meeting the unique physical, emotional and developmental...
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111 Michigan Ave NW
Washington, District of Columbia
(202) 476-5000
Childrens National Medical Center As the nation’s children’s hospital, the mission of Children’s National Medical...
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