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

Launched by CHILDREN'S HOSPITAL MEDICAL CENTER, CINCINNATI · Jun 7, 2021

Keywords

ClinConnect Summary

This clinical trial is studying the use of a medication called abatacept for treating a specific type of lung disease (called granulomatous-lymphocytic interstitial lung disease or GLILD) that occurs in people with common variable immunodeficiency (CVID). There isn’t a standard treatment for GLILD, so researchers are exploring whether abatacept can help improve lung function in both children and adults diagnosed with this condition. The study is taking place at multiple locations and involves comparing the effects of abatacept to a placebo (a treatment with no active medication) to see if it can provide better outcomes for patients.

To be eligible for this trial, participants need to be at least 4 years old, diagnosed with CVID, and have been on immunoglobulin therapy for at least six months. They must also have a confirmed diagnosis of GLILD through a lung biopsy. Participants will be required to agree to regular check-ups and may need to self-administer the treatment at home. It’s important to know that certain health conditions and recent treatments may exclude individuals from participating. The trial is currently recruiting participants, and those who are involved will help researchers learn more about this potential treatment for a challenging condition.

Gender

ALL

Eligibility criteria

• 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.
• • 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
• 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. 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
• Inclusion criteria for Extended Treatment Plan:
• • 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.
• Exclusion criteria for Extended Treatment Plan:
• • • Patients who experienced SAEs during the original trial, and such SAEs were determined as related to treatment, or patients who in the opinion of the investigator would not benefit from the extended treatment option.