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New hope for rare lung disease: protein infusions may slow emphysema

NCT ID NCT01983241

Disease control Sponsor: Grifols Therapeutics LLC Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests whether weekly infusions of Alpha-1 MP (a protein replacement) can slow lung damage in 345 people with emphysema caused by Alpha-1 antitrypsin deficiency. Participants receive one of two doses or a placebo for three years, with lung CT scans tracking changes. The goal is to see if the treatment preserves lung function and reduces flare-ups.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Alpha-1 MP (Prolastin-C)
What this could lead to
If it works, this could provide a new treatment option to slow lung damage in people with Alpha-1 antitrypsin deficiency-related emphysema.
What could go wrong
This is a late-stage trial, but the treatment is not a cure—patients will need ongoing therapy. Results may not show significant benefit over placebo, and side effects are possible.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

345 people

The number who actually took part.

Started

Nov 2013

Expected to finish

Jan 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 70 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Have a documented total alpha1-PI serum level \< 11 µM. * Have a diagnosis of congenital AATD with an allelic combination of ZZ, SZ, Z(null), (null)(null), S(null), or "at-risk" alleles. * At the Screening (Week -3) Visit, have a post-bronchodilator forced expiratory volume in 1 second (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). * Have a carbon monoxide diffusing capacity (DLCO) ≤ 60% of predicted (corrected for HgB) within the past 2 years OR evidence of pulmonary emphysema on CT scan within the past 2 years per the Investigator's judgment. * Have clinical evidence of pulmonary emphysema per the Investigator's judgment. Exclusion Criteria: * Has received alpha1-PI augmentation therapy for more than 1 month within the six months prior to the Screening Visit. * Has received alpha1-PI augmentation therapy within one month of the Screening Visit. * Has had a chronic obstructive pulmonary disease (COPD) exacerbation within the 5 weeks prior to the Screening Visit or during the Screening Phase. * Unable to physically (e.g., unable to fit inside the CT scanner) or mentally (e.g., claustrophobic) undergo a CT scan. * History of lung or liver transplant. * Any lung surgery during the past 2 years (excluding lung biopsy). * On the waiting list for lung surgery, including lung transplant. * Smoking during the past 12 months or a positive urine cotinine test at screening that is due to smoking. Maybe on Nicotine replacement, including vapor cigarettes. * History of anaphylaxis or severe systemic response to any plasma-derived alpha1-PI preparation or other blood product(s). * Use of systemic steroids above a stable dose equivalent to 5 mg/day prednisone (i.e., 10 mg every 2 days) within the 5 weeks prior to the Screening Visit (inhaled steroids are not considered systemic steroids) or during the Screening Phase. * Use of systemic or aerosolized antibiotics for a COPD exacerbation within the 5 weeks prior to the Screening Visit or during the Screening Phase. * Known selective or severe Immunoglobulin A (IgA) deficiency.

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Conditions

The condition(s) this trial relates to.

alpha 1-antitrypsin deficiency pulmonary emphysema

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Pulmonary emphysema in alpha-1 pi deficiency

Contacts and locations

Locations

  • Grifols Investigative Site

    Phoenix, Arizona, 85013, United States

  • Grifols Investigative Site

    Gainesville, Florida, 32610, United States

  • Grifols Investigative Site

    Leesburg, Florida, 34748, United States

  • Grifols Investigative Site

    Miami, Florida, 33136, United States

  • Grifols Investigative Site

    St Louis, Missouri, 63110, United States

  • Grifols Investigative Site

    Wilmington, North Carolina, 28401, United States

  • Grifols Investigative Site

    Portland, Oregon, 97239, United States

  • Grifols Investigative Site

    Hershey, Pennsylvania, 17033, United States

  • Grifols Investigative Site

    Charleston, South Carolina, 29425, United States

  • Grifols Investigative Site

    Tyler, Texas, 75708, United States

  • Grifols Investigative Site

    Vicente López, Buenos Aires, B1602DOH, Argentina

  • Grifols Investigative Site

    Córdoba, Córdoba Province, 5000, Argentina

  • Grifols Investigative Site

    Sydney, New South Wales, 2010, Australia

  • Grifols Investigative Site

    Chermside, Queensland, 4032, Australia

  • Grifols Investigative Site

    Adelaide, South Australia, 5000, Australia

  • Grifols Investigative Site

    Fitzroy, Victoria, 3065, Australia

  • Grifols Investigative Site

    Nedlands, Western Australia, 6009, Australia

  • Grifols Investigative Site

    Santo André, São Paulo, 09060-650, Brazil

  • Grifols Investigative Site

    São Paulo, São Paulo, 04023-061, Brazil

  • Grifols Investigative Site

    São Paulo, São Paulo, 05403-000, Brazil

  • Grifols Investigative Site

    St. John's, Newfoundland and Labrador, A1B 3V6, Canada

  • Grifols Investigative Site

    Halifax, Nova Scotia, B3H3A8, Canada

  • Grifols Investigative Site

    Toronto, Ontario, M5T3A11, Canada

  • Grifols Investigative Site

    Arhus C, 8000, Denmark

  • Grifols Investigative Site

    Hellerup, 2900, Denmark

  • Grifols Investigative Site

    Tallinn, 13419, Estonia

  • Grifols Investigative Site

    Tartu, 51014, Estonia

  • Grifols Investigative Site

    Turku, 20520, Finland

  • Grifols Investigative Site

    Pessac, Gironde, 33604, France

  • Grifols Investigative Site

    Bron, Rhone, 69500, France

  • Grifols Investigative Site

    Heidelberg, Baden-Wurttemberg, 69126, Germany

  • Grifols Investigative Site

    Marburg, Hesse, 35043, Germany

  • Grifols Investigative Site

    Essen, North Rhine-Westphalia, 45239, Germany

  • Grifols Investigative Site

    Homburg Saar, 66421, Germany

  • Grifols Investigative Site

    Chisinau, 2025, Moldova

  • Grifols Investigative Site

    Auckland, 1051, New Zealand

  • Grifols Investigative Site

    Christchurch, 8013, New Zealand

  • Grifols Investigative Site

    Hamilton, 3240, New Zealand

  • Grifols Investigative Site

    Krakow, 31-066, Poland

  • Grifols Investigative Site

    Warsaw, 01-138, Poland

  • Grifols Investigative Site

    Oradea, 410176, Romania

  • Grifols Investigative Site

    Timișoara, 300312, Romania

  • Grifols Investigative Site

    Barnaul, 656038, Russia

  • Grifols Investigative Site

    Moscow, 105077, Russia

  • Grifols Investigative Site

    Yaroslavl, 150003, Russia

  • Grifols Investigative Site

    Oviedo, Principality of Asturias, 33011, Spain

  • Grifols Investigative Site

    Barakaldo, Vizcaya, 48903, Spain

  • Grifols Investigative Site

    Gothenburg, 41345, Sweden

  • Grifols Investigative Site

    Linköping, 587 58, Sweden

  • Grifols Investigative Site

    Malmö, 20502, Sweden

  • Grifols Investigative Site

    Stockholm, 11361, Sweden

  • Grifols Investigative Site

    Uppsala, 752 37, Sweden