Abatacept for the Treatment of Common Variable Immunodeficiency With Interstitial Lung Disease
Children's Hospital Medical Center, Cincinnati
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. Funding Source - FDA OOPD
Description
There is no standard of care therapy for patients with granulomatous-lymphocytic interstitial lung disease (GLILD) seen in common variable immunodeficiency (CVID). Abatacept is a recombinant, human fusion protein of cytotoxic T lymphocyte-associated protein 4 (CTLA-4) and human IgG1 that blocks T cell activation by binding to CD80 and CD86, thereby blocking CD28 engagement- the "second signal" needed for T cell activation. 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 clinic…
Eligibility
- Age range
- 4+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. Diagnosis of CVID according to the international consensus document (ICON) 1. Age 4 years or above 2. Serum IgG at least 2 standard deviations below the age adjusted normal 3. Decreased serum IgA and/or serum IgM 4. Abnormal specific antibody response to immunization 5. 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…
Interventions
- DrugAbatacept
Abatacept is a selective costimulation modulator, inhibiting T lymphocyte activation by binding to CD80 and CD86, thereby blocking interaction with CD28. Orencia solution supplied in a prefilled syringe should be refrigerated at 2C to 8C (36F to 46F). Orencia should not be used beyond the expiration date on the prefilled syringe. The product should be protected from light by storing in the original package until time of use. The prefilled syringe should not be frozen.
- OtherPlacebo
The composition of the placebo for Orencia is the same as the active study drug without the abatacept. The placebo will be packaged and labeled as described above for the Orencia prefilled syringes. To maintain the blind, injection volumes will be the same as the active treatment.
Locations (6)
- University of California, San FranciscoSan Francisco, California
- University of South FloridaTampa, Florida
- Lahey Hospital and Medical CenterBurlington, Massachusetts
- Mayo ClinicRochester, Minnesota
- Duke University Health SystemDurham, North Carolina
- Cincinnati Children's Hospital Medical CenterCincinnati, Ohio