Will Abatacept reduce priming of the hair follicle specific T cells and thereby reduce hair follicle associated infiltration and improve hair growth. This is a double blind placebo controlled study to test the safety and efficacy of Abatacept in the treatment of 64 subjects diagnosed with alopecia totalis or alopecia universalis. Subjects will be randomized 1:1 to the placebo or treatment arm and will receive 6 months of treatment with the study medication or placebo, followed by a 6 month observational period.
Alopecia Areata is a common autoimmune disease, affecting 1% of the general population resulting from autoimmune attack on the hair follicles and usually presents with patchy hair loss. One third of these patients will experience spontaneous remissions within the first year. However many patients will develop waxing and waning disease with some progressing to alopecia totalis (total scalp hair loss) or alopecia universalis (loss of all body hair). This population that suffers from a disfiguring disease represents a significant unmet medical need. Alopecia totalis/universalis seldom, if ever, remits spontaneously or with current treatment and is classified by the FDA as an Orphan Indication. There is no FDA approved drug for alopecia areata. A recent Cochrane report concluded that there was no evidence based support for any intervention in this disease. Standard of care remains observation for mild disease and lesional/oral steroids for more advanced cases. Abatacept is a soluble human fusion protein that selectively modulates the costimulatory signal required for full T-cell activation. It is approved for the treatment of moderately to severely active rheumatoid arthritis. It is also approved for the treatment of moderately to severely active polyarticular juvenile idiopathic arthritis in children 6 years of age or older. Abatacept is a lyophilized powder administered as a 30 minute intravenous infusion. Dosage, as in rheumatoid arthritis, is weight based and is fixed throughout the course of treatment. Abatacept or placebo will be administered as a 30 minute intravenous infusion at baseline, weeks 2, 4, and every 4 weeks for 5 cycles (weeks 8, 12, 16, 20) for a total treatment period of 6 months. There will be a 6 month observational period following the treatment period.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Abatacept will be administrated as a 30 minute intravenous infusion.
Placebo will be administered as a 30 minute intravenous infusion.
The study's primary endpoint will be the proportion of responders after 6 months of treatment, defined as 50% or greater hair re-growth from baseline as assessed by Severity of Alopecia Tool (SALT) score at week 24.
The study's primary endpoint of this intent 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 Severity of Alopecia Tool (SALT) score at week 24. This is a relatively strict definition for defining responders and non-responders and was chosen to minimize responses in the 'vehicle' arm, in which fewer than 10% are expected to achieve this magnitude of hair re-growth spontaneously.
Time frame: 24 weeks
Percent hair regrowth from baseline determined by SALT at weeks 4, 8, 12, 20, and 24 during treatment phase and at weeks 30, 36, 42 and 48 during the observational phase.
As secondary endpoints, efficacy will be measured by changes in hair re-growth as a continuous variable as determined by physical exam and photography, as well as subject and physician global evaluation scores. To assess the durability of responses, subjects will continue to be followed for an additional 6 months post treatment. All subjects will be assessed for safety at each study visit for incidence and severity of adverse effects (AE) and incidence of treatment emergent laboratory abnormalities.
Time frame: 24 weeks of treatment and 18 weeks observational phase
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