Could a simple shot replace IV drips for Alpha-1 patients?
NCT ID NCT07555483
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
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Phase 3
Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.
- Participants
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About 40 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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May 2026
- Expected to finish
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Sep 2027
An estimate. End dates often move.
- Lead sponsor
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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
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18 to 80 years
- Sex
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Anyone
- Healthy volunteers
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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 Hide 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.
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The places running it
22 sites in 9 countries. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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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
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GC2501 Study Site 100
NOT_YET_RECRUITINGLos Angelas, California, 90095, United States
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GC2501 Study Site 101
NOT_YET_RECRUITINGCharleston, South Carolina, 29425, United States
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GC2501 Study Site 102
NOT_YET_RECRUITINGPhoenix, Arizona, 85032, United States
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GC2501 Study Site 103
NOT_YET_RECRUITINGGainesville, Florida, 32610, United States
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GC2501 Study Site 104
NOT_YET_RECRUITINGCleveland, Ohio, 44195, United States
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GC2501 Study Site 105
NOT_YET_RECRUITINGMiami, Florida, 33136, United States
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GC2501 Study Site 106
NOT_YET_RECRUITINGBirmingham, Alabama, 35233, United States
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GC2501 Study Site 107
NOT_YET_RECRUITINGScottsdale, Arizona, 85259, United States
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GC2501 Study Site 108
NOT_YET_RECRUITINGRochester, Minnesota, 55905, United States
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GC2501 Study Site 109
NOT_YET_RECRUITINGDallas, Texas, 75390, United States
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GC2501 Study Site 150
RECRUITINGVejle, Denmark
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GC2501 Study Site 250
NOT_YET_RECRUITINGFreiburg im Breisgau, Germany
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GC2501 Study Site 300
NOT_YET_RECRUITINGDublin, D09YD60, Ireland
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GC2501 Study Site 350
RECRUITINGLeiden, Netherlands
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GC2501 Study Site 400
NOT_YET_RECRUITINGWarsaw, Poland
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GC2501 Study Site 450
RECRUITINGGuimarães, Portugal
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GC2501 Study Site 451
RECRUITINGLoures, Portugal
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GC2501 Study Site 500
RECRUITINGVigo, Spain
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GC2501 Study Site 501
NOT_YET_RECRUITINGMadrid, Spain
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GC2501 Study Site 502
NOT_YET_RECRUITINGSan Cristóbal de La Laguna, Santa Cruz de Tenerife, Spain
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GC2501 Study Site 503
RECRUITINGSeville, Spain
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GC2501 Study Site 550
RECRUITINGMalmö, Sweden
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