This study will test the effectiveness of topical thalidomide in healing mouth sores in HIV
infected patients. Oral (PO) thalidomide heals these sores at a dose of 200 mg per day.
However, PO thalidomide can cause drowsiness, skin rashes, allergic reactions, increased
viral load, and even nerve damage that may not be reversible. This study will evaluate the
efficacy of a topical formulation of thalidomide (placed directly on the surface of the
sore) for the healing of these sores.
Persons with HIV infection of acquired immunodeficiency of at least 18 years of age with one
or more chronic, painful intraoral lesions may be eligible for this study. Subjects must be
referred by a primary care physician who is managing their care, and must have HIV/AIDS
status confirmed. Patients' HIV treatment regimen will not be altered and those receiving
highly active therapy will not be excluded.
Patients will be excluded if they are concurrently being treated for mucosal lesions
(including topical or systemic steroids, viscous lidocaine, topical or systemic
anti-fungals, or mouthwashes), or concurrent thalidomide therapy; receving chemotherapy or
radiation therapy for neoplasms; using concurrent acute therapy for opportunistic
infections; concurrent use of sedatives (such as CNS depressants or alcohol use); history of
allergy to thalidomide; pre-existing peripheral neuropathy of grade II or higher; pregnant
or lactating females or those not practicing contraception according to FDA guidelines for
The proposed clinical trial will evaluate the efficacy of topically-applied thalidomide as a
treatment for painful oral lesions in HIV-infected patients. Limited data suggest that this
drug may be effective when given systemically, but is accompanied by a high incidence of
side effects. Administration of the drug topically onto the lesion should result in high
local concentrations within the lesion thereby suppressing tumor necrosis factor which is
thought to be related to the size and severity of the lesion. Subjects will be randomly
allocated to one of three possible groups: systemic administration of thalidomide, topical
administration of thalidomide, and placebo. The dose of thalidomide will be determined in a
dose escalation pilot study prior to the main study. Healing, pain, and the incidence of
side effects will be assessed at baseline and weekly for up to eight weeks. Successful
demonstration of an enhanced therapeutic effect or reduced toxicity may provide a basis for
the development of novel routes of administration and drugs for the treatment of painful
oral lesions associated with HIV infection and other diseases.
INCLUSION CRITERIA FOR PATIENTS:
Persons with HIV infection or acquired immunodeficiency of at least 18 years of age with
one or more chronic, painful intraoral lesions will constitute the study sample for both
Subjects must be referred by a primary care physician who is managing their care, and must
have HIV/AIDS status confirmed and current CD4 status provided by the primary physician.
Patients' HIV treatment regimen will not be altered and those receiving highly active
antiretroviral therapy will not be excluded.
EXCLUSION CRITERIA FOR PATIENTS:
Patients will be excluded from participation if taking any concurrent treatment for
mucosal lesions (including topical or systemic steroids, viscous lidocaine, topical or
systemic anti-fungals, or mouthwashes), prior to concurrent thalidomide therapy,
chemotherapy or radiation therapy for neoplasms, concurrent acute therapy for
opportunistic infection, concurrent use of sedatives (such as CNS depressants or alcohol
use), history of allergy to thalidomide, pre-existing peripheral neuropathy of grade II or
higher, and females of childbearing potential.
Pregnant or lactating females will be excluded.
INCLUSION CRITERIA FOR HEALTHY VOLUNTEERS:
Patients must be between ages 40 to 60 years.
EXCLUSION CRITERIA FOR HEALTHY VOLUNTEERS:
Cannot be using prescription or non-prescription medications except birth control.
Cannot have acute or chronic current infections or illness.
Cannot have autoimmune conditions, such as diabetes, lupus, or HIV (must be healthy).
Cannot use within 24 hours: anti-inflammatory drugs or other analgesics.
Cannot use within 24 hours: anti-histamines or allergy medications.
Cannot use within 3 weeks: antidepressants or steroids.