Abatacept for the Treatment of Common Variable Immunodeficiency With Interstitial Lung Disease

• Status: Recruiting
• Trial end date: 2025-07-01
• Target enrollment: 0
• Participant gender: All

Summary

There is no standard of care therapy for patients with granulomatous-lymphocytic interstitial lung disease (GLILD) seen in common variable immunodeficiency (CVID). Abatacept has recently looked promising for the treatment of patients with complex CVID. This study is a multi-site, phase II, randomized, blinded/placebo-controlled clinical trial in pediatric and adult subjects to determine the efficacy of abatacept compared to placebo for treatment of subjects with GLILD in the context of CVID.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Children's Hospital Medical Center, Cincinnati
• Collaborator: Bristol-Myers Squibb
• Treatments: Abatacept

Criteria

Inclusion Criteria:

1. Diagnosis of CVID according to the international consensus document (ICON)

a Age 4 years or above

b. Serum IgG at least 2 standard deviations below the age adjusted normal

c. Decreased serum IgA and/or serum IgM

d. Abnormal specific antibody response to immunization

e. Exclusion of secondary immunodeficiency

2. On replacement immunoglobulin for at least 6 months and willing to maintain throughout study

3. Granulomatous-lymphocytic interstitial lung disease with a lymphocytic component diagnosed by lung biopsy prior to study entry, wedge biopsy preferred.

4. Persistence or worsening of interstitial lung disease measured on serial CT imaging of the lung at least 6 months apart, with the latest assessment within 2 months of study entry.

5. Signed written informed consent

6. Willing to allow storage of biological specimens for future use in medical research.

7. Females of childbearing potential must use a highly effective form of birth control such as hormone-based contraceptive, intrauterine device, or double barrier method.

Exclusion Criteria:

1. History of hypersensitivity to abatacept or any of its components

2. Has received any lymphocyte depleting agents including anti-CD20 monoclonal antibodies, alemtuzumab, ATG in the preceding 6 months

3. Has received abatacept, cyclophosphamide, tumor necrosis factor inhibitors, or pulse steroids (defined as >15mg/kg/day of methylprednisone or corticosteroid equivalent) within the past 3 months

4. History of HIV infection (positive PCR)

5. Chronic untreated hepatitis B or C (positive PCR)

6. Active tuberculosis (TB) by positive QuantiFERON gold. If history of latent TB, then must supply evidence of completing treatment.

7. Persistent Epstein-Barr Virus (EBV) load ≥ 1,000 units/mL blood checked twice at least 1 month apart

8. Other uncontrolled infections

9. Live vaccine given within 6 weeks of the start of the trial

10. Malignancy or treated for malignancy within the past year

11. Currently pregnant or breast feeding

12. Life expectancy less than 1 month

13. Subjects unwilling to self-administer or have a parent/caregiver self-administer subcutaneous injections at home

14. Other conditions that the investigators feel contraindicate participation in the study