A Study to Evaluate Safety, Tolerability and Pharmacokinetics of Two Different Doses of Alpha1-Pr… (NCT04722887) | Clinical Trial Compass
CompletedPhase 1/2
A Study to Evaluate Safety, Tolerability and Pharmacokinetics of Two Different Doses of Alpha1-Proteinase Inhibitor Subcutaneous (Human) 15% in Participants With Alpha1-Antitrypsin Deficiency
United States17 participantsStarted 2021-08-13
Plain-language summary
The purpose of this study is to evaluate the safety and tolerability of 72 milligrams per kilogram (mg/kg) and 180 mg/kg Alpha-1 15%, administered as a single-dose subcutaneous (SC) infusion and subsequently as weekly SC infusions over 8 weeks in participants with Alpha1-Antitrypsin Deficiency (AATD).
Who can participate
Age range18 Years – 80 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Have a diagnosis of congenital Alpha1-antitrypsin deficiency (AATD) with an allelic combination of ZZ, SZ, Z(null), (null)(null), S(null), or "at-risk" alleles (subjects with "at-risk" alleles must be individually evaluated for eligibility by the Medical Monitor).
* Have a documented pre-Alpha1-Proteinase Inhibitor (PI) augmentation therapy serum alpha-1 antitrypsin (AAT) level \<11 micrometer (μM) (80 milligrams per decilitre (mg/dL) if measured by radial immunodiffusion or 50 mg/dL if measured by nephelometry).
* Subjects may be naïve to Alpha1-PI augmentation therapy or may be currently receiving Alpha1-PI augmentation therapy or received Alpha1-PI augmentation therapy within the past. If the subject is currently receiving Alpha1-PI augmentation therapy of any kind, he/she must be willing to discontinue that treatment for at least 25 days prior to the Week 1 (Baseline) Visit and remain off any kind of Alpha1-PI treatment, other than the IPs for this study, while participating in the study.
* At the Screening Visit, have a post-bronchodilator forced expiratory volume (FEV1) ≥30% and \<80% of predicted and FEV1/forced vital capacity (FVC) \<70% (Global Initiative for Chronic Obstructive Lung Disease (GOLD) stage II or III).
Exclusion Criteria:
* Have had a moderate or severe Chronic obstructive pulmonary disease (COPD) exacerbation during the 4 weeks before the Week 1 (Baseline) Visit.
* Have history of lung or liver transplant.
* Have any lung surge…
What they're measuring
1
Number of Participants With Adverse Events (AEs)
Timeframe: Up to 668 days
2
Number of Participants With Suspected Adverse Drug Reactions (ADRs)
Timeframe: Up to 668 days
3
Number of Participants With Infusion Site Reactions
Timeframe: Up to 668 days
4
Number of Participants With Serious Adverse Events (SAEs)
Timeframe: Up to 668 days
5
Number of Participants With AEs and SAEs Leading to Discontinuation
Timeframe: Up to 668 days
6
Number of Participants With Chronic Obstructive Pulmonary Disease (COPD) Exacerbations
Timeframe: Up to 668 days
7
Number of Participants With Clinically Significant Abnormalities in Vital Signs (Heart Rate, Blood Pressure, Respiratory Rate, and Temperature)
Timeframe: Up to 668 days
8
Change from Baseline in Forced Expiratory Volume in 1 Second (FEV1)