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A Phase I/II Trial of SGN-00101 in the Treatment of High-Grade Anal Intraepithelial Neoplasia (AIN) in HIV-Positive Individuals

Phase 1/Phase 2
18 Years
Not Enrolling
Anal Cancer

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Trial Information

A Phase I/II Trial of SGN-00101 in the Treatment of High-Grade Anal Intraepithelial Neoplasia (AIN) in HIV-Positive Individuals


I. Determine the safety and maximum tolerated dose of SGN-00101 in HIV-positive patients
with high-grade anal squamous intraepithelial lesions.

II. Determine clinical response and histologic/cytologic regression in patients treated with
this drug.

III. Determine immune response in patients treated with this drug. IV. Determine the effect
of this drug on HIV viral load and CD4 level in these patients.

OUTLINE: This is a dose-escalation study.

Patients receive SGN-00101 subcutaneously once on weeks 0, 4, and 8. Treatment continues in
the absence of disease progression or unacceptable toxicity.

Cohorts of 5-6 patients receive escalating doses of SGN-00101 until the maximum tolerated
dose (MTD) is determined. The MTD is defined as the dose at which no more than 1 of 6
patients experience dose-limiting toxicity.

Patients are followed at 1, 4, and 10 months.

Inclusion Criteria


- Histologically confirmed high-grade anal squamous intraepithelial lesions (HSIL) with
residual HSIL of the anal canal or margin by high-resolution anoscopy

- Declined routine surgery or not a candidate for surgical excision of HSIL

- Documented evidence of HIV infection by one of the following methods:

- Serologic (ELISA or western blot)

- Culture

- Quantitative polymerase chain reaction or bDNA assays

- HIV RNA no greater than 500 copies/mL

- CD4 at least 200 x 10^6/L

- Must have received stable highly active antiviral therapy (HAART) for at least 4
weeks before study

- HAART defined as 3 or more agents, including a protease inhibitor or
nonnucleoside reverse transcriptase inhibitor that is approved or available
through expanded access combination antiviral therapy

- No prior history of invasive anal or cervical cancer

- No concurrent untreated cervical HSIL



- 18 and over

Life expectancy

- At least 12 months


- Hemoglobin at least 10 g/dL

- Platelet count at least 75,000/mm^3

- Absolute neutrophil count at least 1,000/mm^3


- AST and ALT no greater than 3 times upper limit of normal (ULN)


- Creatinine no greater than 1.5 times ULN


- No prior severe allergic reactions (i.e., anaphylactic response) to drugs or any
other allergen

- No history of collagen-vascular or autoimmune disorder requiring treatment within the
past 5 years

- No other concurrent illness that compromises the immune system

- No active serious opportunistic infection


- Not pregnant or nursing

- Negative pregnancy test

- Fertile patients must use 2 forms of effective contraception during and for 3 months
after study

- No concurrent participation in a conception process (e.g., active attempt to become
pregnant or impregnate, sperm donation, or in vitro fertilization)

- No other concurrent medical or psychiatric illness that would preclude study


Biologic therapy

- No concurrent immunostimulants (including interferon or interleukin-12)


- More than 1 year since prior chemotherapy for cancer

Endocrine therapy

- No concurrent steroids that compromise immune function

- Concurrent topical corticosteroids allowed if dose determined not to suppress
immune function


- More than 1 year since prior radiotherapy for cancer


- More than 30 days since other prior investigational agents

- No concurrent medications that suppress immune function

Type of Study:


Study Design:

Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Prevention

Principal Investigator

Joel Palefsky, MD

Investigator Role:

Study Chair

Investigator Affiliation:

University of California, San Francisco


United States: Food and Drug Administration

Study ID:




Start Date:

December 2002

Completion Date:

Related Keywords:

  • Anal Cancer
  • anal cancer
  • Anus Neoplasms
  • Neoplasms



UCSF Comprehensive Cancer Center San Francisco, California  94115