Abatacept for the Treatment of Common Variable Immunodeficiency With Interstitial Lung Disease
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. Funding Source - FDA OOPD
⁃ Diagnosis of CVID according to the international consensus document (ICON)
∙ Age 4 years or above
‣ Serum IgG at least 2 standard deviations below the age adjusted normal
‣ Decreased serum IgA and/or serum IgM
‣ Abnormal specific antibody response to immunization
‣ Exclusion of secondary immunodeficiency
⁃ On replacement immunoglobulin for at least 6 months and willing to maintain throughout study
⁃ Granulomatous-lymphocytic interstitial lung disease with a lymphocytic component diagnosed by lung biopsy prior to study entry, wedge biopsy preferred.
⁃ 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 3 months of study entry.
⁃ Signed written informed consent
⁃ Willing to allow storage of biological specimens for future use in medical research.
⁃ Female subjects of childbearing potential must agree to an effective form of birth control such as hormone based contraceptive, intrauterine device, condoms/barrier, surgically sterile partner, or abstinence.
⁃ Fertile, non-vasectomized males with a female partner of childbearing potential should use condoms throughout the study and for 3 months after the last dose
• Patients must have completed the abatacept for the treatment of Interstitial Lung Disease in Common Variable Immunodeficiency (ABCVILD) trial
• Patients must have demonstrated positive response to abatacept.
• Patients must provide informed consent to participate in the Extended Treatment Plan.