Granulomatous and Lymphocytic Interstitial Lung Disease Clinical Trial
Official title:
Clinical Trial to Assess the Efficacy of Rituximab and Azathioprine in the Treatment of Granulomatous and Lymphocytic Interstitial Lung Disease (GLILD) in Adult Patients With Common Variable Immunodeficiency (CVID)
This phase II study will assess the effect of a treatment combination of Rituximab and azathioprine in patients with Granulomatous and Lymphocytic Interstitial Lung Disease (GLILD) compared to placebo, based on change in lung function at 18 months compared to baseline. The researchers will also assess if the drugs improved quality of life.
BACKGROUND Common Variable Immunodeficiency (CVID) is one of the most clinically important
primary immunodeficiencies due to its frequency, serious complications, and long-term costs
of therapy. A form of lung disease known as granulomatous and lymphocytic interstitial lung
disease (GLILD) occurs in 10-15% of patients with CVID. The causes of GLILD are unknown; no
long-term study has defined the natural course of GLILD; and no clinical trials have been
done to define the best possible treatment for this condition. As a result, currently there
is no proven standard of care for the treatment of GLILD.
The best treatment for individuals with GLILD is not currently known. Some doctors believe
that GLILD does not always continue to get worse and patients should only be treated unless
this happens. Other doctors believe GLILD is always progressive and should be treated early
to prevent more problems later.
There is compelling evidence to support that treatment using rituximab (RTX) in conjunction
with azathioprine (AZA), may improve the lung function and abnormalities seen on high
resolution CT (HRCT) scans of the chest.
STUDY GROUPS Patients in this study will either receive a placebo or a combination of
Rituximab and azathioprine. These drugs are approved by the US Food and Drug Administration
for other conditions, but not yet for this disease.
Because no one knows which of the treatments is best, patients will be "randomized" into one
of the two study groups. Randomization means that you are put into a group by chance.
TREATMENT Eligible patients will be randomized to receive either 18 months of Rituximab and
Azathioprine (20 patients) or placebo (20 patients). Rituximab will be administered
intravenously (IV) weekly for four consecutive weeks at enrollment and months 6 and 12. IV
placebo will be administered on the same schedule as Rituximab. Azathioprine or oral placebo
will be administered by mouth daily for 18 months.
SUMMARY OF STUDY PROCEDURES
-Month 1, 6, 12
Patients will be required to travel to a study site weekly for four consecutive weeks at
enrollment and at 6 and 12 months to receive study infusions. At each of these visits,
patients will be given:
- Your study infusions
- Physical exams with vital signs
- Blood tests to check your organ function
Every six months (Enrollment, 6, 12 & 18 months) while receiving study treatment, patients
will be asked to complete the following study tests:
- Lung Function testing
- High resolution CT of the chest
- Quality of Life Questionnaire, 6-min walk distance test and Karnofsky performance scale.
- Blood for research - approximately 10 teaspoons of blood will be collected
Monthly Labs Following the first month of study treatment, patients will be required to visit
their local clinic/hospital for a blood draw to monitor their lab values twice monthly for
the second and third months of treatment, then monthly.
Final Study Visit
The final study visit will take place at Month 24 after start of study treatment. Patients
will also have the following tests done:
- Physical exams with vital signs
- Lung Function testing
- High resolution CT of the chest
- Quality of Life Questionnaire, 6-min walk distance test and Karnofsky performance scale.
- Blood for research - approximately 10 teaspoons of blood will be collected
;