A Two Stage Phase II Randomized Bilateral Comparison of Topical Targretin® Gel 1% in Alopecia Areata
Targretin® (bexarotene) Gel 1% a synthetic vitamin A compound that is approved for the
topical treatment and oral treatment of cutaneous T cell lymphoma (CTCL). Vitamin A
compounds can influence the growth of skin cells, and can cause the death of T-cells.
Researchers hope that the bexarotene may get rid of T-cells around the hair follicles in AA
(alopecia areata) lesions.
Before treatment starts, patients will have a complete medical history and a brief physical
exam. Researchers will ask about alopecia disease history, date of first diagnosis, and
earlier treatments and responses. Blood test (about 4 teaspoons) will be performed before
and after the end of treatment. Additional blood tests will be done if needed. Women able
to have children must have a negative blood pregnancy test within 7 days before the start of
treatment. Blood pregnancy test must be repeated once a month while receiving treatment.
Participants in this study will be randomly assigned (as in the toss of a coin) to treat
alopecia areata lesions on only one half of their head. Half of the participants will have
the left side treated while the other half will have the right side treated. Sealed
envelopes will be given to participants, telling them which side of the head to treat. The
other half of the head will remain untreated and serve as the control.
Patients will treat alopecia lesions on one-half of the head including facial hair as
designated by the investigator. The other half of the head will have control (untreated)
alopecia lesions. Up to 5 index lesions for treatment and 5 control lesions will be
designated at baseline to follow and measure during the study. Patients will be treated
with Targretin® gel 1% in a dose escalation regimen starting at once every day. At the
start of Week 3 patients will begin applications of twice a day applications if tolerated.
Patients will continue BID dosing, if tolerated, unless the investigator determines a
further increase in application frequency may benefit the patient and the patient agrees to
Patients will be seen at baseline and at weeks 2, 4, 8, 12, 16, 20, 24 and at a 4-week
follow-up. Telephone evaluations will be done at week 1 and as needed.
Study visits for evaluation of safety and efficacy will be made at Week 2 and 4 after the
start of treatment and then every 4 weeks up until Week 24. Telephone safety evaluations
will also be made at Week 1 and other times as needed.
Skin irritation or changes in health during the study may require participants to stop
treatment or withdraw from the study.
At withdrawal from the study, about 4 teaspoons of blood will be drawn. The total study
duration is 24 Weeks with a 4-week follow-up period. At the 4-week follow-up visit, a
physical exam, including measurement of vital signs and weight will be performed. An
evaluation of both treated and untreated sides of the head will be performed.
Patients who show a response at 6 months (24 weeks) will be given the option to continue
therapy for an additional 6 months and will be able to apply the medication to both sides of
the head. Patients will come every 2 months (3 additional visits) and will have the same
examination as they have had during the study with evaluation of irritation, hair loss, hair
thickness, measurement of lesions, and the physician's global assessment, which is the
evaluation of hair re-growth. The same dose adjustment and withdrawal from treatment will
be applied during this 6-month period. Every 2 months, blood (about 2 tablespoons) will be
drawn for pregnancy tests and to look at your triglycerides (fat) levels and complete blood
This is an investigational study. Targretin® Gel 1% is commercially available and approved
by the FDA for use in the treatment of cutaneous lesions in patients with CTCL. Initially up
to 42 participants will be enrolled in the study. All will be enrolled at M. D. Anderson.
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Response and toxicity of Targretin Gel 1% treatment for alopecia areata
"Response" defined as hair growth at PGA grade 3 or 4, and "toxicity" as vesiculation grade 3 or ulceration at grade 4 using design of Thall, Simon and Estey (1995, 1996). To monitor response and toxicity, four possible elementary outcomes are: 1 = [no toxicity, no response], 2 = [no toxicity, response], 3 = [toxicity, no response] , 4 = [toxicity, response].
Baseline and at weeks 2,4,8,12,16,20,24 and at a 4-week follow-up
Madeleine Duvic, MD
UT MD Anderson Cancer Center
United States: Food and Drug Administration
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