Pilot Study to Evaluate the Efficacy of Ruxolitinib in Alopecia Areata
| Status: | Completed |
|---|---|
| Conditions: | Dermatology, Dermatology, Hair Loss |
| Therapuetic Areas: | Dermatology / Plastic Surgery |
| Healthy: | No |
| Age Range: | 18 - 75 |
| Updated: | 10/14/2017 |
| Start Date: | August 2013 |
| End Date: | April 2016 |
An Open-Label Pilot Study to Evaluate the Efficacy of Ruxolitinib in Moderate to Severe Alopecia Areata
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.
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.
Alopecia areata (AA) is a common autoimmune disease resulting from immune destruction of the
hair follicle and subsequent hair loss. Despite its high prevalence, research into the
pathogenesis and the development of innovative therapies in AA has lagged far behind other
autoimmune diseases. Currently, there are no FDA approved drugs for AA. Ruxolitinib (Incyte)
is an intervention known to effectively treat myelofibrosis and also rheumatoid arthritis by
modulating the inflammatory response of the interferon response pathway by inhibition of
Jak1/Jak2. Rheumatoid arthritis shares several susceptibility genes in common with AA. All
three diseases share the central role of the interferon-gamma response pathway, which is the
rationale for selecting Ruxolitinib for evaluation in this clinical trial. Both systemic and
topical Ruxolitinib have been shown to prevent the onset of AA in the C3H-HeJ animal model of
AA, demonstrating preclinical proof-of-concept data in AA. To test the safety and efficacy of
Ruxolitinib in patients with moderate to severe AA, we propose this open-label, single arm
pilot study with a total of 12 patients, treated for a minimum of 3 months up to 6 months.
Efficacy will be measured by changes in hair re-growth as determined by physical exam and
photography, as well as by patient and physician global evaluation scores. Patients will be
followed for another 3 months to evaluate durability of response following the treatment
phase. Punch biopsies and peripheral blood will be obtained at baseline prior to treatment
and then after 12 and possibly 24 weeks for immune monitoring and for molecular studies.
hair follicle and subsequent hair loss. Despite its high prevalence, research into the
pathogenesis and the development of innovative therapies in AA has lagged far behind other
autoimmune diseases. Currently, there are no FDA approved drugs for AA. Ruxolitinib (Incyte)
is an intervention known to effectively treat myelofibrosis and also rheumatoid arthritis by
modulating the inflammatory response of the interferon response pathway by inhibition of
Jak1/Jak2. Rheumatoid arthritis shares several susceptibility genes in common with AA. All
three diseases share the central role of the interferon-gamma response pathway, which is the
rationale for selecting Ruxolitinib for evaluation in this clinical trial. Both systemic and
topical Ruxolitinib have been shown to prevent the onset of AA in the C3H-HeJ animal model of
AA, demonstrating preclinical proof-of-concept data in AA. To test the safety and efficacy of
Ruxolitinib in patients with moderate to severe AA, we propose this open-label, single arm
pilot study with a total of 12 patients, treated for a minimum of 3 months up to 6 months.
Efficacy will be measured by changes in hair re-growth as determined by physical exam and
photography, as well as by patient and physician global evaluation scores. Patients will be
followed for another 3 months to evaluate durability of response following the treatment
phase. Punch biopsies and peripheral blood will be obtained at baseline prior to treatment
and then after 12 and possibly 24 weeks for immune monitoring and for molecular studies.
Inclusion Criteria:
- Patients between 18 to 75 years of age.
- Patients with a diagnosis of patch type alopecia areata.
- Patients will have >30% and <95% total scalp hair loss at baseline as measured using
the SALT score. Two patients with current episodes of alopecia totalis/universalis may
be included in this study.
- Duration of hair loss greater than 3 months.
- No evidence of regrowth present at baseline.
- Patients may be naïve to treatment or unresponsive to intralesional (IL) steroids or
other treatments for alopecia areata.
Exclusion Criteria:
- Patients with a history of or active skin disease on the scalp such as psoriasis or
seborrheic dermatitis.
- Patients in whom the diagnosis of alopecia areata is in question.
- Patients with active medical conditions or malignancies (except adequately treated
basal or squamous cell carcinoma) that in the opinion of the investigator would
increase the risks associated with study participation, including patients with a
history of recurrent infections.
- Women of childbearing potential who are unable or unwilling to use two forms of birth
control for the study duration.
- Women who are pregnant or nursing.
- Patients known to be HIV or hepatitis B or C positive.
- Patients with history or evidence of hematopoietic abnormality.
- Patients with <200K platelet count at baseline.
- Patients with history or evidence of renal or hepatic impairment.
- Patients with history of immunosuppression or history of recurrent serious infections.
- Patients unwilling or unable to discontinue treatments known to affect hair regrowth
in AA.
- Patients taking any medication considered a strong CYP3A4 inhibitor who is unable or
unwilling to stop this medication for the duration of the study.
- Patients receiving treatment deemed to affect alopecia areata within 2 weeks to one
month of baseline visit depending on the specific treatment.
We found this trial at
1
site
Click here to add this to my saved trials
