An Open-Label Pilot Study to Evaluate the Efficacy of Ruxolitinib in Moderate to Severe Alopecia Areata
Overview[ - collapse ][ - ]
Purpose | 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 10 patients with moderate to severe AA for 3 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. 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. |
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Condition | Alopecia Areata |
Intervention | Drug: Ruxolitinib |
Phase | Phase 2 |
Sponsor | Columbia University |
Responsible Party | Columbia University |
ClinicalTrials.gov Identifier | NCT01950780 |
First Received | September 23, 2013 |
Last Updated | November 11, 2013 |
Last verified | November 2013 |
Tracking Information[ + expand ][ + ]
First Received Date | September 23, 2013 |
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Last Updated Date | November 11, 2013 |
Start Date | August 2013 |
Estimated Primary Completion Date | August 2015 |
Current Primary Outcome Measures | Change in SALT Score [Time Frame: Baseline to week 12.] [Designated as safety issue: No]The study's primary efficacy endpoint will be the proportion of responders after 3 months of treatment, with response defined as 50% or greater hair re-growth from baseline as assessed by SALT score at week 12. |
Current Secondary Outcome Measures |
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Descriptive Information[ + expand ][ + ]
Brief Title | An Open-Label Pilot Study to Evaluate the Efficacy of Ruxolitinib in Moderate to Severe Alopecia Areata |
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Official Title | An Open-Label Pilot Study to Evaluate the Efficacy of Ruxolitinib in Moderate to Severe Alopecia Areata |
Brief 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 10 patients with moderate to severe AA for 3 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. 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. |
Detailed Description | 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 10 patients, treated for 3 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. |
Study Type | Interventional |
Study Phase | Phase 2 |
Study Design | Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment |
Condition | Alopecia Areata |
Intervention | Drug: Ruxolitinib A fixed dose of ruxolitinib (20mg) will be self-administered orally twice daily for 12 weeks. Dosing may be decreased or held if needed due to adverse effects. |
Study Arm (s) | Experimental: Ruxolitinib A fixed dose of ruxolitinib (20mg) will be self-administered orally twice daily for 12 weeks. Dosing may be decreased or held if needed due to adverse effects. |
Recruitment Information[ + expand ][ + ]
Recruitment Status | Recruiting |
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Estimated Enrollment | 10 |
Estimated Completion Date | August 2015 |
Estimated Primary Completion Date | August 2015 |
Eligibility Criteria | 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. - 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 alopecia totalis/universalis. - 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. |
Gender | Both |
Ages | 18 Years |
Accepts Healthy Volunteers | No |
Contacts | Contact: Julian Mackay-Wiggan, MD, MS 212-305-6953 jc299@columbia.edu |
Location Countries | United States |
Administrative Information[ + expand ][ + ]
NCT Number | NCT01950780 |
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Other Study ID Numbers | AAAL7102 |
Has Data Monitoring Committee | No |
Information Provided By | Columbia University |
Study Sponsor | Columbia University |
Collaborators | Alopecia Areata Initiative |
Investigators | Principal Investigator: Julian Mackay-Wiggan, MD, MS Columbia University |
Verification Date | November 2013 |
Locations[ + expand ][ + ]
Columbia University Medical Center, Department of Dermatology | New York, New York, United States, 10032 Contact: Julian Mackay-Wiggan, MD, MS | 212-305-6953 | jc299@columbia.eduPrincipal Investigator: Julian Mackay-Wiggan, MD, MS Recruiting |
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