|Age:||18 years or older|
|Keywords:||A1PI-Deficiency, A1PI-Deficient, Epithelial Lining Fluid, ELF, Alpha1-Proteinase Inhibitor, A1PI|
|Type:||Biological study, Phase 3|
If you decide to be in this study, you will complete a series of tests to check that you meet study requirements:
-Informed Consent -Eligibility Check -Medical and Medication History -Physical Exam -Height and Weight -Vital Signs -Chest X-ray/CT Chest Scan -Pulmonary Function Tests -Blood Draw -Urine Test -Pregnancy Test -Adverse Events
You may undergo a test called bronchoalveolar lavage (BAL) during the baseline visit. This is a medical test in which a bronchoscope is passed through your mouth or nose into the lungs. A bronchoscope is a thin flexible tube with a camera and light source at the end. Once the study doctor reaches the targeted airway, a small amount of fluid is released into the airway. It is then collected to test for the levels of A1PI in the lining of your lungs. The study doctor will use standard medications for this test. Your heart will be monitored as well as your blood pressure, breathing and pulse.
A blood test will be collected on the same day of your BAL visit. After the BAL test, you will be contacted by phone or asked to return to the site in person in approximately 2 weeks to check on your health status.
There will be a group of subjects that will complete the BAL test and a separate group that will not. The group that completes the BAL will be enrolled first. You may not have to undergo a BAL once the target number of BAL subjects has been reached. If you are not eligible to undergo the BAL test based on your medical history, the test may be waived. You may still be able to participate in the study with sponsor approval before the target number of subjects for the BAL test has been reached. The study team will let you know whether or not you will have to undergo the BAL procedure.
If you meet the study requirements and you agree to be in this study, you will be assigned to one of two groups:
-GLASSIA (high particles) or -GLASSIA (low particles)
Your group assignment will be decided by a process called randomization. This means that the drug choice will be determined by chance, like flipping a coin. There is a 50% chance that you will be assigned to one group over the other. This is a double-blind study which means that you and the study team will not know to which group you are assigned. However, in the event of an emergency, this information can be made available.
WEEKLY STUDY DRUG INFUSIONS
After you are randomized, the study drug will be given to you through one of your veins (intravenous infusions). At this dose and rate, each study drug infusion will take approximately 15 minutes. There will be a total of 25 infusions over approximately 6 months. The dose of study drug given to you in this study is the dose approved by the FDA. Study drug infusions may take place at the study site or at another suitable location, like your home. If the study doctor allows for home infusions and you choose to have the drug given in your home, a home care nurse will visit your home and perform the infusion. Please be aware that the study drug infusions at Weeks 1, 7, 13, 19, and 25 must be done at the study site.
FOLLOW-UP VISITS (WEEKS 1, 7, 13, 19, AND 25)
There will be at least five visits where you will be asked to return to the study site. At these visits, the following will be performed:
-Body Weight -Physical Exam -Vital Signs -Blood Draw -Urine Test (Weeks 1, 13, and 25 only) -Pregnancy Test -Adverse Events -Concomitant Medications and Non-Drug Therapies -Infusion
BAL VISIT (WEEKS 12-14)
You will be asked to come in for one BAL visit sometime in between Weeks 12-14. The visit will be similar to the BAL procedure that you had at the Baseline Visit. You may not have to undergo this visit if the study has approximately 15-18 patients whom have completed a BAL for the study or if your participation in the BAL portion of the study is waived by the sponsor. However, if you completed a BAL at your Baseline visit, you will likely have to undergo this test during Weeks 12-14 (even if the required number of patients has been reached). The study team will let you know whether or not you will have to undergo this test.
END OF STUDY (WEEK 26) or EARLY TERMINATION VISIT
Once you have finished the last dose of the study drug, you will be asked to return to the study site approximately one week later to have the following tests performed:
-Blood Draw -Urine Test -Pregnancy Test -Adverse Events -Concomitant Medications and Non-Drug Therapies
If you are discontinued from the study early and have had at least one dose of study drug, you will be asked to return to the site approximately 1 week after your last infusion to complete the above tests.
2+proteinuria on urine dipstick analysis
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