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Could a simple shot replace IV drips for Alpha-1 patients?

NCT ID NCT07555483

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 26, 2026 · Last updated Aug 12, 2026 · Updated 5 times

Summary

This study tests a new way to give Alpha1-Proteinase Inhibitor—a medicine that helps protect the lungs in people with Alpha-1 Antitrypsin Deficiency. Instead of getting it through a vein at a clinic, participants will receive it as a weekly injection under the skin. The trial will compare two different doses of the new shot to the standard IV doses, focusing on how the body processes the drug and whether it is safe. About 40 adults are taking part.

What this could mean

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

Active substance
Alpha1-Proteinase Inhibitor (human) given under the skin
What this could lead to
If it works, this could offer a more convenient way to take the same medication at home instead of at a clinic.
What could go wrong
This is a small, early-stage study focused on safety and drug levels, not yet on long-term benefits. The new injection method may cause skin reactions or not work as well as the IV version.

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

About 40 people

The number the study aims to enrol. It can still change while the study runs.

Started

May 2026

Expected to finish

Sep 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 80 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 diagnosis of congenital AATD with an allelic combination of ZZ, SZ, Z(null), (null)(null), S(null), or "at-risk" alleles (patients with "at-risk" alleles must be individually evaluated for eligibility by the Medical Monitor). If the genotype has yet to be documented, a blood draw for genotyping (i.e., allelic discrimination) and phenotyping will be obtained at the Screening Visit. * Participants may be naïve to alpha1-PI augmentation therapy or may be currently receiving alpha1-PI augmentation therapy or received alpha1-PI augmentation therapy in the past. If the total alpha1-PI serum (alpha-1 antitrypsin \[AAT\]) level has yet to be documented as in a treatment-naïve patient, a blood draw for total alpha1-PI serum level will be obtained at the Screening Visit. For participants currently receiving alpha1-PI augmentation, a pre-alpha1-PI augmentation AAT level must be documented in the participant's medical history/records. * All participants must have a documented total alpha1-PI serum level \<11 μM (80mg/dL if measured by radial immunodiffusion or 50 mg/dL if measured by nephelometry) which is documented pre-alpha1-PI augmentation for participants receiving AAT augmentation. * At the Screening Visit, have post-bronchodilator Forced Expiratory Volume in 1 second (FEV₁) ≥25% and \<80% predicted of predicted FEV₁/Forced Vital Capacity (FVC) \<70% (Global Initiative for Chronic Obstructive Lung Disease \[GOLD\] stage II-III, and some individuals are GOLD stage IV). * If the participant has received alpha1-PI augmentation therapy of any kind, he/she must be willing to discontinue that treatment at the Week 1 (Baseline) Visit and remain off any kind of alpha1-PI treatment, other than the IPs of this study, while participating in the study. * Willing and able to provide written informed consent indicating that they understand the purpose of, and procedures required for the study and are willing to participate in it. 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 or on transplantation waiting list. * Have any lung surgery during the past 1 year (excluding lung biopsy). * At screening, have elevated liver enzymes (aspartate aminotransferase \[AST\], alanine aminotransferase \[ALT\], and alkaline phosphatase \[ALP\]) ≥ 2.5 times the upper limit of normal (ULN). * Have severe concomitant disease (e.g., congestive heart failure, clinically significant pulmonary fibrosis, malignant disease \[except for skin cancers other than melanoma\], history of acute hypersensitivity pneumonitis reaction, or current chronic hypersensitivity pneumonitis). * Females who are pregnant, breastfeeding or, if of child-bearing potential†, unwilling to practice a highly effective method of contraception (oral, injectable, or implanted hormonal methods of contraception, placement of an intrauterine device (IUD) or intrauterine system (IUS), condom or occlusive cap with spermicidal foam/gel/film/cream/suppository, male sterilization, or true abstinence\*) throughout the study. †Women of childbearing potential are defined as premenopausal and not surgically sterile, post tubal ligation, nor documented as infertile due to a concurrent medical condition. \*True abstinence: When this is in line with the preferred and usual lifestyle of the participant. (Periodic abstinence \[e.g., calendar, ovulation, symptothermal, post-ovulation methods\], declaration of abstinence for the duration of a study, and withdrawal are not acceptable methods of contraception.) * Have known previous infection with or clinical signs and symptoms consistent with current Hepatitis B Virus (HBV), Hepatitis C Virus (HCV), or Human Immunodeficiency Virus (HIV) infection. * Have smoked during the past 6 months (this includes electronic/vapor cigarettes) or a positive urine cotinine test at the Screening Visit that is due to smoking. * Received IP in another study within 30 days prior to the Week 1 (Baseline) Visit or received any recombinant human AAT-Fc fusion protein (e.g., INBRX-101) or other extended half-life AAT products within 5 half-lives of the product relative to the Screening Visit date. * Have history of anaphylaxis or severe systemic response to any plasma-derived alpha1- PI preparation or other blood product(s). * Use systemic steroids above a stable dose equivalent to 5 mg/day prednisone (i.e., 10 mg every 2 days) within the 4 weeks prior to the Week 1 (Baseline) Visit (Note: inhaled steroids are not considered systemic steroids). It is recommended to maintain the same dose throughout the study. * Use systemic or aerosolized antibiotics for a COPD exacerbation within the 4 weeks prior to the Week 1 (Baseline) Visit. * Have known selective or severe Immunoglobulin A (IgA) deficiency based on prior medical records. * In the opinion of the Investigator, the participant may have compliance problems or any medical condition that may place them at safety risk with the protocol and the procedures of the protocol, or because of unstable health be unable to come to the study site for in-person clinic visits required by the protocol.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

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

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    22 sites in 9 countries. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • GC2501 Study Site 100

    NOT_YET_RECRUITING

    Los Angelas, California, 90095, United States

  • GC2501 Study Site 101

    NOT_YET_RECRUITING

    Charleston, South Carolina, 29425, United States

  • GC2501 Study Site 102

    NOT_YET_RECRUITING

    Phoenix, Arizona, 85032, United States

  • GC2501 Study Site 103

    NOT_YET_RECRUITING

    Gainesville, Florida, 32610, United States

  • GC2501 Study Site 104

    NOT_YET_RECRUITING

    Cleveland, Ohio, 44195, United States

  • GC2501 Study Site 105

    NOT_YET_RECRUITING

    Miami, Florida, 33136, United States

  • GC2501 Study Site 106

    NOT_YET_RECRUITING

    Birmingham, Alabama, 35233, United States

  • GC2501 Study Site 107

    NOT_YET_RECRUITING

    Scottsdale, Arizona, 85259, United States

  • GC2501 Study Site 108

    NOT_YET_RECRUITING

    Rochester, Minnesota, 55905, United States

  • GC2501 Study Site 109

    NOT_YET_RECRUITING

    Dallas, Texas, 75390, United States

  • GC2501 Study Site 150

    RECRUITING

    Vejle, Denmark

  • GC2501 Study Site 250

    NOT_YET_RECRUITING

    Freiburg im Breisgau, Germany

  • GC2501 Study Site 300

    NOT_YET_RECRUITING

    Dublin, D09YD60, Ireland

  • GC2501 Study Site 350

    RECRUITING

    Leiden, Netherlands

  • GC2501 Study Site 400

    NOT_YET_RECRUITING

    Warsaw, Poland

  • GC2501 Study Site 450

    RECRUITING

    Guimarães, Portugal

  • GC2501 Study Site 451

    RECRUITING

    Loures, Portugal

  • GC2501 Study Site 500

    RECRUITING

    Vigo, Spain

  • GC2501 Study Site 501

    NOT_YET_RECRUITING

    Madrid, Spain

  • GC2501 Study Site 502

    NOT_YET_RECRUITING

    San Cristóbal de La Laguna, Santa Cruz de Tenerife, Spain

  • GC2501 Study Site 503

    RECRUITING

    Seville, Spain

  • GC2501 Study Site 550

    RECRUITING

    Malmö, Sweden

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