Actively Recruiting
Abatacept for Common Variable Immunodeficiency With Interstitial Lung Disease in Pediatric and Adult Patients
Led by Children's Hospital Medical Center, Cincinnati · Updated on 2025-11-06
38
Participants Needed
6
Research Sites
N/A
Total Duration
On this page
Sponsors
C
Children's Hospital Medical Center, Cincinnati
Lead Sponsor
B
Bristol-Myers Squibb
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating abatacept as a treatment for granulomatous-lymphocytic interstitial lung disease (GLILD) in patients with common variable immunodeficiency (CVID), a condition without a standard therapy. This multi-site, phase II randomized, blinded, placebo-controlled clinical trial includes both pediatric and adult participants to compare the effects of abatacept against placebo. The study aims to assess the efficacy of abatacept in this complex immune condition, funded by the FDA Office of Orphan Products Development. Participants are divided into two cohorts based on weight. Cohort 1 includes pediatric subjects weighing 50 kg or more and adults, randomized in a 1:2 ratio to receive either abatacept or placebo weekly for six months, followed by open-label abatacept for all. Cohort 2 includes pediatric subjects under 50 kg receiving open-label abatacept with dosing based on weight. After the initial 12 months, patients can opt to continue abatacept treatment for up to three years, with safety monitoring every three months. During the study, participants will undergo assessments including high-resolution chest CT scans, lung function tests such as forced vital capacity and forced expiratory volume, quality of life questionnaires, and monitoring for infections and adverse events at 6 and 12 months. Additional evaluations include pediatric growth measures and steroid use. Safety monitoring continues for those opting for extended treatment, ensuring a comprehensive overview of abatacept’s effects over time.
CONDITIONS
Brief Title
Abatacept for the Treatment of Common Variable Immunodeficiency With Interstitial Lung Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of common variable immunodeficiency (CVID) according to international consensus
- Age 4 years or older
- Serum IgG at least 2 standard deviations below age-adjusted normal
- Decreased serum IgA and/or IgM
- Abnormal specific antibody response to immunization
- On replacement immunoglobulin therapy for at least 6 months and willing to maintain this during the study
- Diagnosis of granulomatous-lymphocytic interstitial lung disease confirmed by lung biopsy
- Persistence or worsening of interstitial lung disease shown on CT scans at least 6 months apart
- Signed informed consent
- Willingness to allow storage of biological specimens for future research
- Female participants of childbearing potential must agree to effective birth control
- Fertile, non-vasectomized males with female partners of childbearing potential must use condoms during the study and for 3 months after last dose
You will not qualify if you...
- History of hypersensitivity to abatacept or its components
- Use of lymphocyte depleting agents within 6 months prior to study
- Use of abatacept, cyclophosphamide, tumor necrosis factor inhibitors, or high-dose steroids in past 3 months
- Recent start or increase of certain immune modulating drugs within 3 months prior to enrollment
- History of HIV infection
- Untreated chronic hepatitis B or C infection
- Active tuberculosis or untreated latent TB
- Persistent Epstein-Barr Virus load above threshold
- Other uncontrolled infections
- Live vaccine administered within 6 weeks before trial start
- Malignancy within past year
- Currently pregnant or breastfeeding
- Life expectancy less than 1 month
- Unwillingness or inability to self-administer or have caregiver administer subcutaneous injections
- Other conditions deemed contraindicated by investigators
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 screening and enrollment visit
Duration - 12 months
Participants receive weekly subcutaneous injections of abatacept or placebo for the first 6 months. After 6 months, all participants receive abatacept weekly.
Weekly visits for up to 6 months, then weekly treatment visits up to 12 months
Duration - Up to 3 years
Participants who choose to continue abatacept after the initial 12 months are monitored for safety, including infections.
Safety monitoring visits approximately every 3 months
Trial Site Locations
Total: 6 locations
1
University of California, San Francisco
San Francisco, California, United States, 94143
Actively Recruiting
2
University of South Florida
Tampa, Florida, United States, 33620
Actively Recruiting
3
Lahey Hospital and Medical Center
Burlington, Massachusetts, United States, 01805
Actively Recruiting
4
Mayo Clinic
Rochester, Minnesota, United States, 55902
Actively Recruiting
5
Duke University Health System
Durham, North Carolina, United States, 27710
Actively Recruiting
6
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Actively Recruiting
Research Team
M
Michael Jordan
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
Similar Trials
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here