A Randomized Clinical Trial to Evaluate the Efficacy of Abatacept in Moderate to Severe Alopecia Areata
Overview
- Phase
- Phase 2
- Intervention
- Abatacept
- Conditions
- Alopecia Areata
- Sponsor
- Columbia University
- Locations
- 1
- Primary Endpoint
- SALT Score (Severity of Alopecia Tool)
- Status
- Withdrawn
- Last Updated
- 11 years ago
Overview
Brief Summary
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.
Detailed Description
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.
Investigators
Julian M. Mackay-Wiggan
Principal Investigator
Columbia University
Eligibility Criteria
Inclusion Criteria
- •Signed Written Informed Consent
- •Must be between 18 and 75 years of age.
- •Must have a diagnosis of moderate to severe AA - defined as the presence of equal to or more than 40% and equal to or less than 95% total scalp hair loss at baseline as measured using the SALT score.
- •Duration of hair loss must be between 3 to 12 months.
- •There may be no evidence of regrowth present at baseline.
- •Subjects may be naïve to treatment or unresponsive to intralesional (IL) steroids or other treatments for AA.
- •Must be willing to avoid live vaccines while on the study medication, and within 3 months of its discontinuation.
- •Women of childbearing potential must use highly effective methods of birth control \[for up to 12 weeks after the last dose of investigational product\] to minimize the risk of pregnancy\].
- •Women of childbearing potential must follow instructions for birth control for the entire duration of the study including a minimum of 90 days after dosing has been completed.
- •Women must have a negative serum or urine pregnancy test (minimum sensitivity 25 IU/L or equivalent units of HCG) within 24 hours prior to the start of investigational product.
Exclusion Criteria
- •Women of childbearing potential who are unwilling or unable to use an acceptable method to avoid pregnancy for the entire study period and for up to 10 weeks after the last dose of study drug.
- •Women of childbearing potential using a prohibited contraceptive method.
- •Women who are pregnant or breastfeeding.
- •Women with a positive pregnancy test on enrollment or before administration of abatacept.
- •Sexually active fertile men not using effective birth control if their partners are women of childbearing potential.
- •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.
- •Patients with COPD
Arms & Interventions
abatacept
Intervention: Abatacept
abatacept Subcutaneous vehicle
Intervention: placebo
Outcomes
Primary Outcomes
SALT Score (Severity of Alopecia Tool)
Time Frame: 24 Weeks
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.
Secondary Outcomes
- Safety(24 Weeks of Treatment and an additional 6 months off treatment or until determined that relapse has ocurred)
- Efficacy(24 Weeks of Treatment and an additional 6 months off treatment or until determined that relapse has ocurred)