Trial Outcomes & Findings for An Open-Label Single-Arm Clinical Trial to Evaluate The Efficacy of Abatacept in Moderate to Severe Patch Type Alopecia Areata (NCT NCT02018042)

NCT ID: NCT02018042

Last Updated: 2019-04-30

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

15 participants

Primary outcome timeframe

Week 24

Results posted on

2019-04-30

Participant Flow

Participant milestones

Participant milestones
Measure	Abatacept 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. Abatacept: 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.
Overall Study STARTED	15
Overall Study COMPLETED	14
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Abatacept 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. Abatacept: 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.
Overall Study Withdrawal by Subject	1

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Abatacept n=15 Participants 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. Abatacept: 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.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	15 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	39 years n=5 Participants
Sex: Female, Male Female	9 Participants n=5 Participants
Sex: Female, Male Male	6 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	14 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	3 Participants n=5 Participants
Race (NIH/OMB) White	12 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	15 Participants n=5 Participants

PRIMARY outcome

Timeframe: Week 24

Outcome measures

Outcome measures
Measure	Abatacept n=15 Participants 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. Abatacept: 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.
Total Number of Participants With At Least 50% Hair Regrowth	1 Participants

SECONDARY outcome

Timeframe: Week 24

Percent hair regrowth from baseline determined by global overall improvement in SALT measurements following 24 weeks of treatment.

Outcome measures

Outcome measures
Measure	Abatacept n=15 Participants 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. Abatacept: 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.
Percentage of Hair Regrowth	21.3 percentage Interval 2.9 to 91.0

SECONDARY outcome

Timeframe: Baseline, Week 12, Week 24

Quality of life measures were based on changes in the Dermatology Life Quality Index (DLQI). The DLQi is a 10-item questionnaire with each question scored from 0 (not at all) to 3 (very much). The DLQI is calculated by summing the score of each question resulting in a maximum of 30 and a minimum of 0 (better outcome). The higher the score, the more quality of life is impaired.

Outcome measures

Outcome measures
Measure	Abatacept n=15 Participants 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. Abatacept: 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.
Measurement of Quality of Life Baseline	4.2 score on a scale Standard Deviation 3.8
Measurement of Quality of Life Week 12	3.9 score on a scale Standard Deviation 3.8
Measurement of Quality of Life Week 24	4.6 score on a scale Standard Deviation 3.8

Adverse Events

Abatacept

Serious events: 0 serious events

Other events: 6 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Abatacept n=15 participants at risk 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. Abatacept: 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.
Respiratory, thoracic and mediastinal disorders Upper Respiratory Infection	40.0% 6/15 • 24 weeks plus an additional 6 months post-treatment. There were no serious adverse effects, no major clinical laboratory abnormalities and no patients required discontinuation of therapy. Most common adverse event was upper respiratory infection symptoms in 6 subjects. All occurrences resolved with supportive measures and/or antibiotics. One subject experienced recurrent yeast infection. Adverse effects related to site injection was reported in 3 subjects and included body aches, tightness at the injection site, and tingling of the hands/arms.
Product Issues Site Injection	20.0% 3/15 • 24 weeks plus an additional 6 months post-treatment. There were no serious adverse effects, no major clinical laboratory abnormalities and no patients required discontinuation of therapy. Most common adverse event was upper respiratory infection symptoms in 6 subjects. All occurrences resolved with supportive measures and/or antibiotics. One subject experienced recurrent yeast infection. Adverse effects related to site injection was reported in 3 subjects and included body aches, tightness at the injection site, and tingling of the hands/arms.
Reproductive system and breast disorders Candidiasis/Yeast Infection	6.7% 1/15 • 24 weeks plus an additional 6 months post-treatment. There were no serious adverse effects, no major clinical laboratory abnormalities and no patients required discontinuation of therapy. Most common adverse event was upper respiratory infection symptoms in 6 subjects. All occurrences resolved with supportive measures and/or antibiotics. One subject experienced recurrent yeast infection. Adverse effects related to site injection was reported in 3 subjects and included body aches, tightness at the injection site, and tingling of the hands/arms.

Additional Information

Grace Ulerio, CCRC

Columbia University Department of Dermatology Clinical Research Unit

Phone: 212-305-6593

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place