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

  • STATUS
    Recruiting
  • End date
    Jul 27, 2025
  • participants needed
    60
  • sponsor
    Children's Hospital Medical Center, Cincinnati
Updated on 27 May 2022
immunodeficiency
immune globulin
serum igg
interstitial lung disease
lung biopsy

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 clinical trial in adult and pediatric subjects ≥50 kg (cohort 1), with an additional cohort (#2) of pediatric subjects <50 kg tested as a single arm, receiving open-label abatacept. Cohort 1 utilizes a 'delayed-start' design to obtain maximum statistical power from this cohort. Cohort 2 will be open label due to the lack of a suitable placebo for pediatric dose abatacept syringes. A total of 45 evaluable subjects will be treated in cohort 1 and 15 evaluable subjects in cohort 2.

Details
Condition Interstitial Lung Disease, Common Variable Immunodeficiency
Treatment Placebo, Abatacept
Clinical Study IdentifierNCT04925375
SponsorChildren's Hospital Medical Center, Cincinnati
Last Modified on27 May 2022

Eligibility

Yes No Not Sure

Inclusion Criteria

Diagnosis of CVID according to the international consensus document (ICON)
a 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 2 months of study entry
Signed written informed consent
Willing to allow storage of biological specimens for future use in medical research
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

Persistent Epstein-Barr Virus (EBV) load ≥ 1,000 units/mL blood checked twice at least 1 month apart
History of hypersensitivity to abatacept or any of its components
Has received any lymphocyte depleting agents including anti-CD20 monoclonal antibodies, alemtuzumab, ATG in the preceding 6 months
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
History of HIV infection (positive PCR)
Chronic untreated hepatitis B or C (positive PCR)
Active tuberculosis (TB) by positive QuantiFERON gold. If history of latent TB, then must supply evidence of completing treatment
Other uncontrolled infections
Live vaccine given within 6 weeks of the start of the trial
Malignancy or treated for malignancy within the past year
Currently pregnant or breast feeding
Life expectancy less than 1 month
Subjects unwilling to self-administer or have a parent/caregiver self-administer subcutaneous injections at home
Other conditions that the investigators feel contraindicate participation in the study
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