A Clinical Trial to Evaluate the Efficacy of Abatacept in Moderate to Severe Alopecia Areata
The purpose of this study is to determine if receiving sub-cutaneous injections of a medication called abatacept causes regrowth of hair in people with alopecia areata.
Among patients with alopecia areata, patients with worse disease are unlikely to have satisfactory outcomes with current therapies. Our hypothesis is that Abatacept will be effective therapy in moderate to severe alopecia areata by blocking re-activation of a special type of immunecell call a memory T-Cell (CD8+NKG2D+)thereby blocking the inflammatory response underlying alopecia areata.
|Study Design:||Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
|Official Title:||A Randomized Clinical Trial to Evaluate the Efficacy of Abatacept in Moderate to Severe Alopecia Areata|
- SALT Score (Severity of Alopecia Tool) [ Time Frame: 24 Weeks ] [ Designated as safety issue: No ]The primary efficacy endpoint of this intention-to-treat trial will be the proportion of responders in the treated compared to the control group after 6 months of treatment, defined as 50% or greater hair re-growth from baseline as assessed by SALT score at week 24. This patient group and relatively strict definition for defining responders were chosen to minimize responses in the "vehicle" arm, in which 8% are expected to achieve this magnitude of hair regrowth spontaneously. To assess the durability of responses, patients who achieve 50% regrowth from baseline during the first 6 months, will continue to be followed for an additional 6 months off treatment or until it is determined that relapse has occurred.
- Efficacy [ Time Frame: 24 Weeks of Treatment and an additional 6 months off treatment or until determined that relapse has ocurred ] [ Designated as safety issue: Yes ]Efficacy will be measured by changes in hair re-growth as a continuous variable as determined by physical exam and Canfield photography, as well as patient and physician global evaluation scores. To assess the durability of responses, patients who achieve 50% regrowth from baseline during the first 6 months, will continue to be followed for an additional 6 months off treatment or until it is determined that relapse has occurred.
- Safety [ Time Frame: 24 Weeks of Treatment and an additional 6 months off treatment or until determined that relapse has ocurred ] [ Designated as safety issue: Yes ]
Patient reported outcomes, safety measures, incidence and timing of relapse will be important secondary outcomes.
Safety will be evaluated as a secondary endpoint using descriptive statistics to summarize the cumulative incidence and types of Adverse Events.
|Study Start Date:||August 2013|
|Estimated Study Completion Date:||August 2016|
|Estimated Primary Completion Date:||August 2016 (Final data collection date for primary outcome measure)|
|Active Comparator: abatacept||
This will be a randomized double blind placebo controlled study. Patients will be randomly assigned to abatacept or placebo in a 1:1 ratio based on a computer generated randomization list. The list will be generated prior to study initiation. Based on the randomization list, patients will be assigned to treatment vs. vehicle (provided by BMS) in the order in which they are enrolled. The assignment will be made by study personnel who are not involved in making clinical assessments or evaluations. The patient and the assessing physicians will remain blinded to the treatment assignment for the duration of the study.
Patients will be treated with abatacept 125mg SC self-administered each week. Treatment will be continued for 6 months to provide adequate time to assess the short-term efficacy and safety of abatacept in patients with alopecia areata. Patients will then be followed for an additional 6 months to assess the timing and incidence of relapse.
Other Name: Brand Name: ORENCIA
|Placebo Comparator: abatacept Subcutaneous vehicle||Drug: placebo|
Alopecia areata (AA) is a common disease of the immune system, known as an "autoimmune" disease. In the disease, the immune system mistaken 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. Abatacept (made by Bristol-Myers Squibb) is a safe intervention known to effectively treat rheumatoid arthritis,another "autoimmune" disease, by fighting inflammation. There are some genetic and chemical similarities between those with active rheumatoid arthritis and AA, suggesting that treatment with the same drug is likely to be effective.
In mice specially designed for testing drugs for the treatment of human alopecia, this medication worked to prevent the disease AA from starting. To test Abatacept, we are going to treat 60 patients with moderate to severe AA for 6 months. To make the study results meaningful, there will be a control or "placebo" group that does not receive the study drug. Patients will be randomly assigned to either receive the real or the inactive medication, and neither the patient nor the doctor will know which it is. 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 6 months off of the drug to see if the effects of treatment last and if there is delayed response. Small scalp biopsies and peripheral blood will be taken at the beginning of the study before treatment and then after 4,12 and 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 even guide us to better treatments in the future.
|Contact: Julian Mackay-Wiggan, MD, MSfirstname.lastname@example.org|
|Contact: Grace Ulerioemail@example.com|
|United States, New York|
|Columbia University Medical Center, Dept of Dermatology|
|New York, New York, United States, 10032|
|Principal Investigator:||Julian Mackay-Wiggan, MD, MS||Columbia University|