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

Description

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

Conditions

Interstitial Lung Disease, Common Variable Immunodeficiency

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Study Overview

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Study Details

Study overview

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

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

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

Condition
Interstitial Lung Disease
Intervention / Treatment

-

Contacts and Locations

San Francisco

University of California, San Francisco, San Francisco, California, United States, 94143

Tampa

University of South Florida, Tampa, Florida, United States, 33620

Burlington

Lahey Hospital and Medical Center, Burlington, Massachusetts, United States, 01805

Rochester

Mayo Clinic, Rochester, Minnesota, United States, 55902

Durham

Duke University Health System, Durham, North Carolina, United States, 27710

Cincinnati

Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, United States, 45229

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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.
  • 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 3 months of study entry.
  • 5. Signed written informed consent
  • 6. Willing to allow storage of biological specimens for future use in medical research.
  • 7. 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.
  • 8. 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
  • 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. Have started or increased any of the following immune modulating drugs within 3 months of enrolling and 3 months from initial CT chest: azathioprine, cyclosporine, tacrolimus, mercaptopurine, methotrexate, mycophenolate mofetil, or sirolimus
  • 5. History of HIV infection (positive PCR)
  • 6. Chronic untreated hepatitis B or C (positive PCR)
  • 7. Active tuberculosis (TB) by positive QuantiFERON gold. If history of latent TB, then must supply evidence of completing treatment.
  • 8. Persistent Epstein-Barr Virus (EBV) load ≥ 1,000 units/mL blood checked twice at least 1 month apart
  • 9. Other uncontrolled infections
  • 10. Live vaccine given within 6 weeks of the start of the trial
  • 11. Malignancy or treated for malignancy within the past year
  • 12. Currently pregnant or breast feeding
  • 13. Life expectancy less than 1 month
  • 14. Subjects unwilling to self-administer or have a parent/caregiver self-administer subcutaneous injections at home
  • 15. Other conditions that the investigators feel contraindicate participation in the study

Ages Eligible for Study

4 Years to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Children's Hospital Medical Center, Cincinnati,

Study Record Dates

2025-07