CompletedPhase 2

Safety and Pharmacokinetics of Alpha-1 Proteinase Inhibitor in Subjects With Alpha1-Antitrypsin Deficiency

United States30 participantsStarted 2010-11

Plain-language summary

This is a study to assess the safety and pharmacokinetics of weekly infusions of 120 mg/kg of Prolastin-C (alpha1-proteinase inhibitor \[alpha1-PI\] \[Human\]), compared to weekly infusions of 60 mg/kg of Prolastin-C in patients with alpha 1-antitrypsin deficiency (AATD).

Who can participate

Age range

18 Years – 70 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Be between 18 and 70 years of age * Have a documented diagnosis of congenital AATD * Have a post-bronchodilator Forced Expired Volume in 1 second (FEV1) of ≥30% and \<80% and FEV1/forced vital capacity (FVC) \<70% * If receiving alpha-1 PI augmentation therapy, be willing to discontinue the treatment for the duration of the study Exclusion Criteria: * Had a moderate or severe pulmonary exacerbation during the 4 weeks before the study * History of lung or liver transplant * Any lung surgery during the past 2 years * Confirmed liver cirrhosis * Elevated liver enzymes * Severe concurrent disease * Females who are pregnant or breast-feeding or unwilling to practice effective contraception during the study * Infection with hepatitis A, B, or C, human immunodeficiency or parvovirus B19 * Smoking during the past 6 months * Use of systemic steroids within 4 weeks of the study * Use of antibiotics for an exacerbation within 4 weeks of the study

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Subjects With Treatment-Emergent Adverse Events (TEAEs)

Timeframe: 22 weeks

Subjects With Drug-Related TEAE(s)

Timeframe: 22 weeks

Subjects With Treatment-Emergent Serious Adverse Events (SAEs)

Timeframe: 22 weeks

Subjects Withdrawn Due to an AE(s)

Timeframe: 22 weeks

Subjects With Treatment-Emergent Pulmonary Exacerbation(s)

Timeframe: 22 weeks

Subjects With Severe TEAE(s) or Pulmonary Exacerbation(s)

Timeframe: 22 weeks

Number of TEAEs

Timeframe: 22 Weeks

Number of Drug-related TEAEs

Trial details

NCT IDNCT01213043

SponsorGrifols Therapeutics LLC

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2012-01

Results submitted2013-02-26

View on ClinicalTrials.gov More Emphysema trials

Who can participate

Age range

18 Years – 70 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Subjects With Treatment-Emergent Adverse Events (TEAEs)

Timeframe: 22 weeks

Subjects With Drug-Related TEAE(s)

Timeframe: 22 weeks

Subjects With Treatment-Emergent Serious Adverse Events (SAEs)

Timeframe: 22 weeks

Subjects Withdrawn Due to an AE(s)

Timeframe: 22 weeks

Subjects With Treatment-Emergent Pulmonary Exacerbation(s)

Timeframe: 22 weeks

Subjects With Severe TEAE(s) or Pulmonary Exacerbation(s)

Timeframe: 22 weeks

Number of TEAEs

Timeframe: 22 Weeks

Number of Drug-related TEAEs