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Phase I Study To Evaluate The Toxicity And Feasibility Of Intratumoral Injection Of Alpha-Gal (Beta-galactosidase ) Glycosphingolipids In Patients With Advanced Melanoma

Phase 1
18 Years
75 Years
Open (Enrolling)
Metastatic Melanoma

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Trial Information

Phase I Study To Evaluate The Toxicity And Feasibility Of Intratumoral Injection Of Alpha-Gal (Beta-galactosidase ) Glycosphingolipids In Patients With Advanced Melanoma

A standard Phase I dose escalation model will be used to define the maximum tolerated dose
(MTD) of GSL alpha-GAL that can be administered directly into the tumor lesion on two
separate injections separated by 4-weeks. This trial will serve as the basis for future
Phase II trials utilizing multiple injections of GSL alpha-GAL in refractory solid tumors.

Additionally, in this study we will look for histologic evidence of an immune response
against the injected melanoma lesions which matches that seen in mice. Our hypothesis for
this study is that a second injection of GSL alpha-GAL into a melanoma lesion will not
precipitate an allergic or autoimmune reaction, but will cause a histologically evident
immune response to the tumor.

Inclusion Criteria:

1. Patients with recurrent melanoma who have failed standard therapies, or are not
candidates for standard therapies.

2. Patients must have at least one measurable cutaneous 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 has
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 >18 years old.

6. ECOG (Eastern Cooperative Oncology Group) performance of <2. International Normalized
Ratio (INR)<1.5 and a PTT (partial thromboplastin time ) no greater than normal
limits within 1 week prior to intra-tumoral injection (For patients who may be on
blood thinners)

7. Laboratory Criteria (completed <2 weeks before enrollment) Hematologic: (White Blood
Cell Count) WBC > 3500/mm3 or (absolute neutrophil count) ANC > 1500/mm3 and platelet
count > 100 000/ mm3 Hepatic: Total bilirubin < 4.0 mg/dl Renal: Creatinine < 2.2

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

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

Exclusion Criteria:

Patients meeting any of the following exclusion criteria are not eligible:

1. Patients who are pregnant or nursing (PRN serum pregnancy test to be done at week

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 experiment 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:

Grade 3/4 toxicity

Outcome Description:

Grade 3/4 toxicity or adverse event during injection protocol or up to a month after

Outcome Time Frame:

11-12 weeks

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:

March 2007

Completion Date:

March 2014

Related Keywords:

  • Metastatic Melanoma
  • metastatic cutaneous melanoma
  • alpha-gal glycolipids
  • Melanoma



Universiity of Wisconsin Madison, Wisconsin  53792