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A Phase II Trial of Topical Halofuginone in Patients With HIV Related Kaposi's Sarcoma

Phase 2
16 Years
Not Enrolling
AIDS-related Kaposi Sarcoma, Recurrent Kaposi Sarcoma

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Trial Information

A Phase II Trial of Topical Halofuginone in Patients With HIV Related Kaposi's Sarcoma


I. To determine the tumor response rate of acquired immune deficiency syndrome
(AIDS)-related Kaposi's sarcoma to topical halofuginone (halofuginone hydrobromide) versus
vehicle control.

II. To evaluate the safety and tolerability of topical halofuginone and vehicle in patients
with AIDS-related Kaposi's sarcoma.


I. To determine the ability of topical halofuginone to inhibit expression of matrix
metallopeptidase 2 (MMP-2) and Collagen type I in AIDS-related Kaposi's sarcoma.

II. To explore the relationship between baseline cluster of differentiation (CD) 4 and CD8
counts, HIV viral load and human herpesvirus 8 (HHV-8) viral load and response to

III. To characterize the pharmacokinetics of halofuginone.

OUTLINE: Twelve treatable Kaposi's sarcoma lesions are selected on each patient, and these
12 lesions are randomized equally to 1 of 2 treatment arms (6 lesions receive study
treatment and 6 lesions receive placebo); each patient serves as his/her own control.

ARM I: Patients apply topical halofuginone hydrobromide ointment to each of 6 lesions twice
a day for 12 weeks.

ARM II: Patients apply topical placebo ointment to each of 6 lesions twice a day for 12

Patients with stable or responding disease in either or both groups of treated lesions
(halofuginone hydrobromide ointment or placebo ointment) may receive open-label treatment
with topical halofuginone hydrobromide ointment to all 12 lesions for an additional 12 weeks
as above in the absence of disease progression or unacceptable toxicity.

Patients are followed for at least 1 month.

Inclusion Criteria:

- Biopsy-proven Kaposi's sarcoma with at least 14 cutaneous lesions, 12 of which are
measurable in two dimensions and can serve as marker lesions; each of the 14 lesions
must measure a minimum of 0.5 cm in diameter, so that a 4 mm punch biopsy will be
entirely composed of Kaposi's sarcoma

- Serologic documentation of HIV infection by any of the Food and Drug Administration
(FDA) approved tests

- Karnofsky performance status >= 60%

- Hemoglobin >= 8 g/dl

- Absolute neutrophil count >= 750 cells/mm^3

- Platelet count >= 75,000/mm^3

- Creatinine < 1.5 times the upper limit of normal or creatinine clearance >= 60 mL/min

- Total bilirubin should be =< 1.5 x upper limit of normal (ULN); if, however, the
elevated bilirubin is felt to be secondary to indinavir therapy, patients will be
allowed to enroll on protocol if the total bilirubin is =< 3.5 mg/dl provided that
the direct bilirubin is normal

- Aspartate aminotransferase (AST) (serum glutamic oxaloacetic transaminase [SGOT]) and
alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase [SGPT]) =< 3 x
the upper limit of normal

- Life expectancy >= 3 months

- Ability and willingness to give informed consent; patients who are younger than 18
years of age will require the consent of a parent or guardian.

- All women of childbearing potential must have a negative serum b human chorionic
gonadotropin (HCG) within 72 hours prior to study entry and must practice adequate
birth control to prevent pregnancy while receiving treatment and for three months
after treatment is discontinued

- Patients must, in the opinion of the investigator, be capable of complying with the

- Patients receiving antiretroviral therapy must be on a stable regimen for at least 12
weeks prior to study entry without showing evidence of ongoing Kaposi's sarcoma (KS)
regression (ie, less than 25% decrease in the size, number or nodularity of KS
lesions in the opinion of the investigator); patients may receive any FDA approved
antiretroviral therapy or agents available through a treatment IND; concurrent
treatment with highly active antiretroviral therapy should be strongly encouraged, in
accordance with DHHS guidelines ( but
will not be required for participation

Exclusion Criteria:

- Concurrent, acute, active, untreated opportunistic infection other than oral thrush
or genital herpes within 14 days of enrollment

- Known active visceral Kaposi's sarcoma or symptomatic Kaposi's sarcoma-related edema
that interferes with function or requires cytotoxic therapy

- Concurrent neoplasia requiring cytotoxic therapy

- Acute treatment for an infection (other than oral thrush or genital herpes) or other
serious medical illness within 14 days of study entry

- Anti-neoplastic treatment for Kaposi's sarcoma (including chemotherapy, radiation
therapy, local therapy, biological therapy, or investigational therapy) within four
weeks of study entry

- Previous local therapy of any KS-indicator lesion within 60 days unless the lesion
has clearly progressed since treatment

- Corticosteroid treatment, other than replacement doses

- Use of investigational agents other than antiretroviral drugs available under
expanded access or compassionate use protocols

- Pregnant or breast feeding females are excluded from participation in this study
since the effects of halofuginone on an unborn or young child are unknown and may
potentially be toxic

Type of Study:


Study Design:

Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double-Blind, Primary Purpose: Treatment

Outcome Measure:

Response rate

Outcome Description:

McNemar's chi-square test will be used to compare vehicle control and halofuginone with respect to response rates.

Outcome Time Frame:

Up to 30 days

Safety Issue:


Principal Investigator

Susan Krown

Investigator Role:

Principal Investigator

Investigator Affiliation:

AIDS Associated Malignancies Clinical Trials Consortium


United States: Food and Drug Administration

Study ID:




Start Date:

May 2003

Completion Date:

Related Keywords:

  • AIDS-related Kaposi Sarcoma
  • Recurrent Kaposi Sarcoma
  • Sarcoma, Kaposi
  • AIDS-Related Opportunistic Infections
  • Sarcoma



AIDS - Associated Malignancies Clinical Trials Consortium Rockville, Maryland  20850