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About
The main purpose of this study is to evaluate the efficacy of RYMPHYSIA [Alpha1-Proteinase Inhibitor (Human)] compared to another available alpha-1 proteinase Inhibitor (A1PI) in adults with A1PI deficiency and COPD-emphysema.
In Part A of the study, participants will be randomly assigned to receive either RYMPHYSIA or another available A1PI for 104 weeks. Participants who were randomized to another available A1PI will enter a 2-week follow-up period after the treatment phase is completed; participants who were randomized to RYMPHYSIA will enter Part B.
In Part B, participants will be randomly assigned to one of two groups and will receive either the same dose of RYMPHYSIA as in Part A or a different dose for an additional 104 weeks, followed by a 2-week follow-up period.
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Volunteers
Inclusion and exclusion criteria
Inclusion:
Adult 18 to 75 years at the time of screening.
Diagnosis of alpha1-proteinase inhibitor (A1PI) deficiency, defined as follows:
Participants eligible for enrollment include newly diagnosed, previously untreated, currently treated, and currently not on treatment but who have received treatment in the past. For those currently on augmentation therapy, a 2-week minimum washout is required until A1PI levels are <11 μM.
Clinically evident chronic obstructive pulmonary disease (COPD)-emphysema at the time of screening defined by post-bronchodilator FEV1 of greater than or equal to (≥)30 percent (%) and lesser than or equal to (≤)80% predicted and FEV1/forced vital capacity (FVC) <70%, corresponding to mild to severe COPD (according to Global Initiative for Chronic Obstructive Lung Disease [GOLD] Criteria, stage I-III) (Global Initiative for Chronic Obstructive Lung Disease 2021) and evidence of emphysema on chest imaging (confirmed by the baseline computer topography [CT] densitometry scan).
If treated with any respiratory medications including inhaled bronchodilators, inhaled corticosteroids, or systemic corticosteroids (e.g., prednisone ≤10 micrograms per day [mg/day] or its equivalent), the doses of medications should have remained stable for at least 28 days prior to screening.
No clinically significant abnormalities (other than emphysema, bronchitis, or bronchiectasis) detected via chest imaging at the time of screening.
Males and non-pregnant, non-lactating females whose screening pregnancy test is negative and willing and able to employ adequate contraceptive methods.
Willing and able to refrain from smoking (including e-cigarettes and vaping of any other substance) for the duration of study.
Willing and able to comply with the requirements of the protocol and able to voluntarily provide written, signed, and dated (personally or via a legally authorized representative) informed consent to participate in the study.
Exclusion:
Primary purpose
Allocation
Interventional model
Masking
0 participants in 4 patient groups
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Data sourced from clinicaltrials.gov
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