An Open-Label Single-Arm Clinical Trial to Evaluate The Efficacy of Abatacept in Moderate to Severe Patch Type Alopecia Areata

NCT02018042 | Completed Phase 2 Results DMC

• 1 other identifier: AAAK2167
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief Summary

Alopecia areata (AA) is a common disease of the immune system, known as an "autoimmune" disease. In this 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 Alopecia Areata has lagged far behind other similar diseases of the immune system. Currently, there are no Federal Drug Administration approved drugs for Alopecia Areata. 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 Alopecia Areata, 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 Alopecia Areata from starting. To test Abatacept, we are going to treat 15 patients with moderate to severe Alopecia Areata for 6 months. Each person enrolling into this study will receive the active study drug. 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. We have recently changed the study to allow testing of abatacept in a few patients with alopecia totalis and universalis. 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 perhaps even guide us to better treatments in the future.

Conditions

Alopecia Areata

Keywords

Alopecia Areata; AA

Outcome Measures

Primary Outcomes (1)

• Total Number of Participants With At Least 50% Hair Regrowth

  The study's primary efficacy endpoint will be the proportion of responders after 6 months of treatment, with response 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 the potential for spontaneous remission, in which fewer than 10% are expected to achieve this magnitude of hair regrowth spontaneously.

  Week 24

Secondary Outcomes (2)

• Percentage of Hair Regrowth

  Week 24

• Measurement of Quality of Life

  Baseline, Week 12, Week 24

Study Arms (1)

Abatacept

EXPERIMENTAL

Patients will be treated with abatacept 125mg subcutaneous (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.

Drug: Abatacept

Interventions

Abatacept DRUG

After the screening period, subjects will begin weekly self-administered subcutaneous abatacept and will continue treatment for 6 months. Patients will be instructed in self-administration of study medication at baseline (week zero) and will be observed self-administering medication at each visit. Instructions regarding study drug administration will be reinforced as needed. The 6-month treatment period is expected to provide adequate time to assess the short-term efficacy and safety of abatacept in patients with moderate to severe AAP. Responders will then be followed for 6 months off drug.

Also known as: Orencia

Abatacept

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Signed Written Informed Consent Before any study procedures are performed, subjects will have the details of the study described to them, and they will be given a written informed consent document to read. Then, if subjects consent to participate in the study, they will indicate that consent by signing and dating the informed consent document in the presence of study personnel.
• 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 30% and equal to or less than 95% total scalp hair loss at baseline as measured using the SALT score. In addition, a few patients with 100% scalp hair loss will be enrolled.
• Duration of hair loss must be at least 3 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 (WOCBP) 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\]. WOCBP must follow instructions for birth control for the entire duration of the study including a minimum of 90 days after dosing has been completed.
• (Acceptable methods of highly effective birth control include: Condom with spermicide, Diaphragm and spermicide, Cervical cap and spermicide)
• The use of intrauterine devices, (IUDs) shall be at the discretion of the investigator.
• 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.
• Women must not be breastfeeding
• Sexually active fertile men must use highly effective birth control if their partners are WOCBP. Men that are sexually active with WOCBP must follow instructions for birth control for the entire duration of the study and a minimum of 90 days after dosing has been completed.

You may not qualify if:

• Sex and Reproductive Status
• WOCBP 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.
• WOCBP 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 WOCBP.
• 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
• Patients known to be HIV or hepatitis B or C positive.
• Patients with history or evidence of hematopoietic abnormality.
• 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
• Coexisting disease or concurrent medications

Sponsors & Collaborators

• Columbia University: lead
• Bristol-Myers Squibb: collaborator

Study Sites (1)

Columbia University Medical Center - Department of Dermatology

New York, New York, 10032, United States

Location

Related Publications (1)

• Biran R, Zlotogorski A, Ramot Y. The genetics of alopecia areata: new approaches, new findings, new treatments. J Dermatol Sci. 2015 Apr;78(1):11-20. doi: 10.1016/j.jdermsci.2015.01.004. Epub 2015 Jan 28.

  PMID: 25676427 DERIVED

MeSH Terms

Conditions

Alopecia Areata

Interventions

Abatacept

Condition Hierarchy (Ancestors)

Alopecia; Hypotrichosis; Hair Diseases; Skin Diseases; Skin and Connective Tissue Diseases

Intervention Hierarchy (Ancestors)

Immunoconjugates; Antibodies; Immunoglobulins; Serum Globulins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Globulins

Limitations and Caveats

Small study size and potential for spontaneous remission that might discount the validity of clinical response. A greater observational post treatment period would strengthen conclusions. Last, inherent observer bias, by SALT scoring \& photography.

Results Point of Contact

• Title: Grace Ulerio, CCRC
• Organization: Columbia University Department of Dermatology Clinical Research Unit

gu2102@cumc.columbia.edu

212-305-6593

Study Officials

• Julian Mackay-Wiggan, MD, MS

  Columbia University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 17, 2013
• First Posted: December 23, 2013
• Study Start: September 1, 2013
• Primary Completion: July 31, 2017
• Study Completion: July 31, 2017
• Last Updated: April 30, 2019
• Results First Posted: April 17, 2019

Record last verified: 2019-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)