Pilot Study to Evaluate the Efficacy of Ruxolitinib in Alopecia Areata
Status:
Completed
Trial end date:
2016-04-01
Target enrollment:
Participant gender:
Summary
Alopecia areata (AA) is a common disease of the immune system, known as an "autoimmune"
disease. In the disease, the immune system mistakenly destroys the hair follicle, causing
hair to fall out. Despite many people having this disease, research into its cause and into
new, better ways to treat AA has lagged far behind other similar diseases of the immune
system. Currently, there are no Federal Drug Administration approved drugs for AA.
Ruxolitinib (made by Incyte) is an intervention known to effectively treat a disease of the
bone marrow, known as myelofibrosis. It is also being studied in the treatment of rheumatoid
arthritis, another "autoimmune" disease, by fighting inflammation. There are some genetic and
chemical similarities between those with myelofibrosis, active rheumatoid arthritis and AA,
suggesting that treatment with ruxolitinib may be effective in AA. In mice specially designed
for testing drugs for the treatment of human alopecia areata, this medication worked to
prevent the disease AA from starting in mice that would have otherwise developed the disease.
To test Ruxolitinib, we are going to treat 12 patients with moderate to severe AA for a
minimum of 3 months up to 6 months. This is an "open-label" study, meaning that there will
not be a placebo group; all patients enrolled in the study will receive the active
medication. The effectiveness of the medication will be measured by changes in hair re-growth
as determined by physical exam and photography, as well as by patient and physician scoring.
Patients will be followed for another 3 months off of the drug to see if the effects of
treatment last and if there is delayed response. The safety of the medication, ruxolitinib,
in patients with alopecia areata will also be evaluated.
Blood work will be collected before medication is started, during the treatment period, and
after ruxolitinib is stopped, in order to monitor for adverse effects of the medication.
Small scalp biopsies and peripheral blood will be taken at the beginning of the study before
treatment and also after 12 and possibly 24 weeks. Optional biopsies may also be taken at
additional time points based on clinical considerations. The chemical analysis of these skin
samples and blood will help us to understand how the disease happens, how the treatment
works, and may even guide us to better treatments in the future.
Phase:
Phase 2
Details
Lead Sponsor:
Columbia University
Collaborators:
Alopecia Areata Initiative - Gates Foundation Locks of Love