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Phase I Study To Evaluate The Toxicity And Feasibility Of Intra-Tumoral Injection Of Alpha-Gal Glycosphingolipids In Patients With Advanced Or Refractory Solid Tumors

Phase 1
18 Years
75 Years
Not Enrolling
Neoplasm Metastases

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Trial Information

Phase I Study To Evaluate The Toxicity And Feasibility Of Intra-Tumoral Injection Of Alpha-Gal Glycosphingolipids In Patients With Advanced Or Refractory Solid Tumors

Intratumoral injection of alpha gal glycolipid in experimental knockout mouse model systems
incorporates into tumor cell membranes and presents these xeno-transplantation epitopes to
antigen presenting cells with that particular tumor's tumor associated antigens (TAA). Thus
this maneuver converts any individual tumor into an in situ tumor vaccine without the need
to isolate, purify or supply TAA exogenously. The effects in these model systems
demonstrate both the upregulation of cytotoxic T cells which react against the particular
tumor's TAA, as well as resolution of injected primary tumor and eradication and prevention
of metastatic disease at distant sites. This current study was undertaken to investigate
the safety and feasibility of such an approach in humans. The major toxicity concerns are
acute allergic or complement activation reactions or development of autoimmunity. The
primary treatment is a single intratumoral injection of alpha gal glycolipid. The study
design is a standard dose escalation design and the primary endpoint is Dose limiting
toxicity at one month after injection (grade 3 or 4. Subjects are followed until death
utilizing standard clinical imaging and evaluation to judge overall tumor response.

Inclusion Criteria:

1. Patients with solid tumors who have failed standard therapies, or are not candidates
for standard therapies.

2. Patients must have at least one measurable lesion that is accessible and suitable for
injection of the GSL alpha-GAL.

3. Patients should not be undergoing any active treatment with chemotherapy,
radiotherapy, or steroids (either because the patient or the treating physician have
decided not to employ these therapies at this time, or because they had already been
tried and failed). If they have been treated with these modalities, the treatments
should have been completed at least two weeks prior to date of injection of GSL

4. Patients should be judged by the investigator to be able to undergo safely the
procedure needed to inject the tumor with GSL alpha-GAL.

5. Age equal or over 18 years old.

6. ECOG (Eastern Cooperative Oncology Group ) performance of less than 2. (International
Normalized Ratio) INR less than 1.5 and a (Partial Thromboplastin Time) PTT no
greater than normal limits within 1 week prior to intra-tumoral injection (For
patients who requires invasive procedure for intra-tumoral injection).

7. Laboratory Criteria (completed equal or less 2 weeks before enrollment) Hematologic:
(White Blood Cell Count) WBC equal or above 3500/millimeter-cubed or (Absolute
Neutrophil Count) ANC equal or above 1500/millimeter-cubed and platelet count equal
or above 100,000/ millimeter-cubed.

Hepatic: Total bilirubin equal or less 4.0 milligrams/deciliter. Renal: Creatinine
equal or less 2.2 milligrams/deciliter.

8. Patients must be negative for HIV (circulating antibody), Hepatitis B (circulating
antigen), and Hepatitis C (circulating antibody).

9. Patients should have an expected survival of more than 6 weeks and should not have
other systemic anti-tumor treatments planned during this time frame.

Exclusion Criteria:

1. Patients who are pregnant (as determined by a positive serum HCG (Human Chorionic
Gonadotropin) in patients of childbearing potential) or nursing.

2. Patients under the age of 18.

3. Patients with severe infections or septicemia.

4. Patients with a history of autoimmune disease.

5. Patients in, or about to be in, active treatment with chemotherapy or steroids.

6. Patients who refuse HIV/hepatitis testing and patients who do not sign an approved
consent form.

7. Patient has received other investigational drugs within 14 days before enrollment or
is expected to participate in an experimental drug study during this study treatment.

8. Serious medical or psychiatric illness likely to interfere with participation in this
clinical study.

Type of Study:


Study Design:

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

Outcome Measure:

Number of Subjects With Greater Than Grade 3 or 4 Toxicity

Outcome Description:

Grade 3/4 Toxicity occurring in a participant within a month of intratumoral injection

Outcome Time Frame:

1 month

Safety Issue:


Principal Investigator

Giles Whalen, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

University of Massachusetts, Worcester


United States: Food and Drug Administration

Study ID:




Start Date:

July 2007

Completion Date:

July 2011

Related Keywords:

  • Neoplasm Metastases
  • Cancerous solid tumors
  • Alpha-Gal Glycosphingolipids
  • Neoplasms
  • Neoplasm Metastasis



University of Massachusetts Medical SchoolWorcester, Massachusetts  01605