Alpha1-Antitrypsin DeficiencyPhase 1/2NCT04722887
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
A Phase 1/2 study testing Alpha-1 15% and Liquid Alpha1-Proteinase Inhibitor (Human) in people with Alpha1-Antitrypsin Deficiency, run by Grifols Therapeutics LLC at 5 sites in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Alpha1-Antitrypsin Deficiency
- What is being tested?
- Alpha-1 15% and Liquid Alpha1-Proteinase Inhibitor (Human)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 17 participants
- Where
- 5 sitesUnited States
- Study dates
- 16 Dec 2021to 1 Aug 2025
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Main 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 micromolar (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).
Main 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 surgery during the past 2 years (excluding lung biopsy).
- Have severe concomitant disease (example, 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.
- Have smoked during the past 6 months or a positive urine cotinine test at the Screening Visit that is due to smoking.
- Participate in another Investigational product (IP) study within one month prior to the Week 1 (Baseline) Visit.
- 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. It is recommended to maintain the same dose throughout the study.
- Use systemic or aerosolized antibiotics for a chronic COPD exacerbation within the 4 weeks prior to the Week 1 (Baseline) Visit.
- Have known selective or severe Immunoglobulin A (IgA) deficiency.
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
GC2008 Study Site 101
Charleston, South Carolina, 29425
GC2008 Study Site 103
Cleveland, Ohio, 44195
GC2008 Study Site 108
Gainesville, Florida, 32610
GC2008 Study Site 107
Los Angeles, California, 90025
GC2008 Study Site 102
Miami, Florida, 33136
About this study
Official title: A Multi-Center, Single-Dose and Repeat-Dose Over Eight Weeks, Sequential Cohort Study to Evaluate Safety and Tolerability as Well as Pharmacokinetics of Two Different Doses of Alpha1-Proteinase Inhibitor Subcutaneous (Human) 15% Administered Subcutaneously in Subjects With Alpha1-Antitrypsin Deficiency
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).
- Conditions
- Alpha1-Antitrypsin Deficiency
- Being tested
- Alpha-1 15%, Liquid Alpha1-Proteinase Inhibitor (Human)
- Keywords
- Pulmonary Emphysema, Alpha-1 Antitrypsin Deficiency, AATD, Alpha-1 PI Deficiency, Alpha-1 Proteinase Inhibitor, Emphysema, Pathologic Processes, Pulmonary Disease, Chronic Obstructive, Lung Diseases, Obstructive, Lung Diseases, Respiratory Tract Diseases, Liver Diseases, Digestive System Diseases, Genetic Diseases, Inborn, Subcutaneous Emphysema, Alpha1-Antitrypsin, Trypsin Inhibitors, Serine Proteinase Inhibitors, Protease Inhibitors, Enzyme Inhibition
- Medical subject headings
- alpha 1-Antitrypsin Deficiency, Pulmonary Emphysema, Emphysema, Pathologic Processes, Pulmonary Disease, Chronic Obstructive, Lung Diseases, Obstructive, Lung Diseases, Respiratory Tract Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Alpha-1 15%Biological
Alpha1-Proteinase Inhibitor (Human), 15%, Subcutaneous infusion
Liquid Alpha1-Proteinase Inhibitor (Human)Biological
Also called Prolastin®-C Liquid
Intravenous infusion
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1: Treatment Period 1 (Alpha-1 15%, 72 mg/kg) | Experimental | Participants will receive Alpha-1 15% 72 mg/kg, single subcutaneous (SC) infusion in treatment-period 1 (Single-Dose) at Week 1. Receives: Biological: Alpha-1 15% |
| Cohort 1: Single-Dose Data Evaluation Period (Liquid Alpha 1-Proteinase Inhibitor 60 mg/kg) | Experimental | Following the week-long treatment period 1, participants in Cohort 1 will enter 21 days of washout/serial pharmacokinetic (PK) phase and then the single-dose data evaluation period. During the single-dose data evaluation period, Liquid Alpha1- Proteinase Inhibitor (PI) 60 mg/kg, weekly intravenous (IV) Infusions will be administered from intravenous-dose Week 1 (single-dose Week 5) for up to Week 78, with the last IV dose given 1 week prior to the first repeat Alpha-1 15% SC dose. Receives: Biological: Liquid Alpha1-Proteinase Inhibitor (Human) |
| Cohort 1: Treatment Period 2 (Alpha-1 15%, 72 mg/kg) | Experimental | Following treatment period 1 and single-dose data evaluation period, participants in Cohort 1 will enter treatment period 2 (Repeat-Dose) and will receive Alpha-1 15% 72 mg/kg, for 8 weekly SC infusions. Receives: Biological: Alpha-1 15% |
| Cohort 2: Treatment Period 1 (Alpha-1 15%, 180 mg/kg) | Experimental | Participants will receive Alpha-1 15% 180 mg/kg, single SC infusion in treatment-period 1 (Single-Dose) at Week 1. Receives: Biological: Alpha-1 15% |
| Cohort 2: Single-Dose Data Evaluation Period (Liquid Alpha1-Proteinase Inhibitor 120 mg/kg) | Experimental | Following the week-long treatment period 1, participants in Cohort 2 will enter 21 days of washout/serial pharmacokinetic (PK) phase and then the single-dose data evaluation period. During the single-dose data evaluation period, Liquid Alpha1-PI 120 mg/kg, weekly IV Infusions will be administered from intravenous-dose Week 1 (single-dose Week 5) for up to Week 78, with the last IV dose given 1 week prior to the first repeat Alpha-1 15% SC dose. Receives: Biological: Liquid Alpha1-Proteinase Inhibitor (Human) |
| Cohort 2: Treatment Period 2 (Alpha-1 15%, 180 mg/kg) | Experimental | Following treatment period 1 and single-dose data evaluation period, participants in Cohort 2 will enter treatment period 2 (Repeat-Dose) and will receive Alpha-1 15% 180 mg/kg, for 8 weekly SC infusions. Receives: Biological: Alpha-1 15% |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
AE that occurred at any time from the beginning of IP administration until the final visit of the clinical trial was considered TEAE.
Measured: Up to 668 days
Number of Participants With Suspected Adverse Drug Reactions (ADRs)
All noxious and unintended responses to a medicinal product or study treatment related to any dose was considered suspected ADRs. The phrase "responses to a medicinal product" means that a causal relationship between a medicinal product or study treatment and an AE is at least a reasonable possibility.
Measured: Up to 668 days
Number of Participants With Infusion Site Reactions (ISRs)
Treatment-emergent ISRs have onset on or after date of the first administration of study treatment up to on or before 28 days after last administration of study treatment. An ISR was counted in the period during which it started.
Measured: Up to 668 days
Number of Participants With Serious Adverse Events (SAEs)
An adverse event is considered serious if, in the judgment of the investigator or sponsor, it results in death, is life-threatening, requires hospitalization or prolongs an existing hospitalization (admission for 24 hours or more, including emergency room stays), causes significant or persistent disability, results in a congenital anomaly or birth defect, or is an important medical event that may jeopardize the subject or require intervention to prevent these outcomes.
Measured: Up to 668 days
Number of Participants With Any TEAE Leading to Study Withdrawal
TEAE Leading to Study Withdrawal refers to a TEAE that results in a participant permanently discontinuing or withdrawing from a clinical study because of the adverse event.
Measured: Up to 668 days
Number of Participants With Chronic Obstructive Pulmonary Disease (COPD) Exacerbations
The definition of an exacerbation of COPD is an increase in respiratory symptoms (dyspnea, increased cough, and/or production of sputum) over baseline that usually requires medical intervention.
Measured: Up to 668 days
Number of Participants With Any Adverse Events Associated With Abnormalities in Vital Signs (Heart Rate, Blood Pressure, Respiratory Rate, and Temperature)
Clinically significant vital sign abnormalities were presented as adverse events, with clinical relevance based on the Investigator's judgment.
Measured: Up to 668 days
Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1)
FEV1 was performed according to ATS/ERS guidelines. At each visit, testing was performed both pre- and post-bronchodilator (BD) administration.
Measured: Single-Dose Week 5/Intravenous Week 1, and Repeat-Dose Weeks 1, 4, and 9
Change From Baseline in Forced Vital Capacity (FVC)
FVC was performed according to ATS/ERS guidelines. At each visit, testing was performed both pre- and post-bronchodilator (BD) administration.
Measured: Single-Dose Week 5/Intravenous Week 1, and Repeat-Dose Weeks 1, 4, and 9
Number of Participants With Any Adverse Events Associated With Abnormalities in Laboratory Parameters (Chemistry, Hematology, Urinalysis)
Out of the normal range laboratory results judged by the Investigator as clinically significant in the context of the participant's medical history were considered adverse events.
Measured: Up to 668 days
Immunogenicity: Number of Participants With Alpha1-PI Antibodies
Immunogenicity testing was performed using validated assays in a multi-tiered approach.
Measured: Treatment Period 1- Single-Dose Week 1; Treatment Period 2- Repeat-Dose Weeks 1 and 9
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Jan 2021
- Study start16 Dec 2021
- Primary completion1 Aug 2025
- Study completion1 Aug 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Alpha1-Antitrypsin DeficiencyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 17 participants (actual)
Sponsor and investigators
- Lead sponsor
- Grifols Therapeutics LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04722887
- Sponsor's protocol ID
- GC2008
- Status last verified
- Sep 2026
- First submitted
- 20 Jan 2021
- Last updated
- 8 Oct 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-Center, Single-Dose and Repeat-Dose Over Eight Weeks, Sequential Cohort Study to Evaluate Safety and Tolerability as Well as Pharmacokinetics of Two Different Doses of Alpha1-Proteinase Inhibitor Subcutaneous (Human) 15% Administered Subcutaneously in Subjects With Alpha1-Antitrypsin Deficiency
- ClinicalTrials.gov ID
- NCT04722887
- Acronym / sponsor ID
- GC2008
- Conditions
- Alpha1-Antitrypsin Deficiency
- Keywords
- Pulmonary Emphysema, Alpha-1 Antitrypsin Deficiency, AATD, Alpha-1 PI Deficiency, Alpha-1 Proteinase Inhibitor, Emphysema, Pathologic Processes, Pulmonary Disease, Chronic Obstructive
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 17 participants (actual)
- Lead sponsor
- Grifols Therapeutics LLCIndustry (pharma, biotech, devices)
- Study start
- 16 Dec 2021 (actual)
- Primary completion
- 1 Aug 2025 (actual)
- Study completion
- 1 Aug 2025 (actual)
- First posted
- 25 Jan 2021 (actual)
- Results
- Posted 8 Oct 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 6
| Arm | Type | Receives |
|---|---|---|
| Cohort 1: Treatment Period 1 (Alpha-1 15%, 72 mg/kg) | Experimental | Biological: Alpha-1 15%Participants will receive Alpha-1 15% 72 mg/kg, single subcutaneous (SC) infusion in treatment-period 1 (Single-Dose) at Week 1. |
| Cohort 1: Single-Dose Data Evaluation Period (Liquid Alpha 1-Proteinase Inhibitor 60 mg/kg) | Experimental | Biological: Liquid Alpha1-Proteinase Inhibitor (Human)Following the week-long treatment period 1, participants in Cohort 1 will enter 21 days of washout/serial pharmacokinetic (PK) phase and then the single-dose data evaluation period. During the single-dose data evaluation period, Liquid Alpha1- Proteinase Inhibitor (PI) 60 mg/kg, weekly intravenous (IV) Infusions will be administered from intravenous-dose Week 1 (single-dose Week 5) for up to Week 78, with the last IV dose given 1 week prior to the first repeat Alpha-1 15% SC dose. |
| Cohort 1: Treatment Period 2 (Alpha-1 15%, 72 mg/kg) | Experimental | Biological: Alpha-1 15%Following treatment period 1 and single-dose data evaluation period, participants in Cohort 1 will enter treatment period 2 (Repeat-Dose) and will receive Alpha-1 15% 72 mg/kg, for 8 weekly SC infusions. |
| Cohort 2: Treatment Period 1 (Alpha-1 15%, 180 mg/kg) | Experimental | Biological: Alpha-1 15%Participants will receive Alpha-1 15% 180 mg/kg, single SC infusion in treatment-period 1 (Single-Dose) at Week 1. |
| Cohort 2: Single-Dose Data Evaluation Period (Liquid Alpha1-Proteinase Inhibitor 120 mg/kg) | Experimental | Biological: Liquid Alpha1-Proteinase Inhibitor (Human)Following the week-long treatment period 1, participants in Cohort 2 will enter 21 days of washout/serial pharmacokinetic (PK) phase and then the single-dose data evaluation period. During the single-dose data evaluation period, Liquid Alpha1-PI 120 mg/kg, weekly IV Infusions will be administered from intravenous-dose Week 1 (single-dose Week 5) for up to Week 78, with the last IV dose given 1 week prior to the first repeat Alpha-1 15% SC dose. |
| Cohort 2: Treatment Period 2 (Alpha-1 15%, 180 mg/kg) | Experimental | Biological: Alpha-1 15%Following treatment period 1 and single-dose data evaluation period, participants in Cohort 2 will enter treatment period 2 (Repeat-Dose) and will receive Alpha-1 15% 180 mg/kg, for 8 weekly SC infusions. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Alpha-1 15%Alpha1-Proteinase Inhibitor (Human), 15%, Subcutaneous infusion | Biological | Cohort 1: Treatment Period 1 (Alpha-1 15%, 72 mg/kg), Cohort 1: Treatment Period 2 (Alpha-1 15%, 72 mg/kg), Cohort 2: Treatment Period 1 (Alpha-1 15%, 180 mg/kg), Cohort 2: Treatment Period 2 (Alpha-1 15%, 180 mg/kg) |
| Liquid Alpha1-Proteinase Inhibitor (Human)Also: Prolastin®-C LiquidIntravenous infusion | Biological | Cohort 1: Single-Dose Data Evaluation Period (Liquid Alpha 1-Proteinase Inhibitor 60 mg/kg), Cohort 2: Single-Dose Data Evaluation Period (Liquid Alpha1-Proteinase Inhibitor 120 mg/kg) |
Primary outcome
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
Time frame: Up to 668 days
AE that occurred at any time from the beginning of IP administration until the final visit of the clinical trial was considered TEAE.
Number of Participants With Suspected Adverse Drug Reactions (ADRs)
Time frame: Up to 668 days
All noxious and unintended responses to a medicinal product or study treatment related to any dose was considered suspected ADRs. The phrase "responses to a medicinal product" means that a causal relationship between a medicinal product or study treatment and an AE is at least a reasonable possibility.
Number of Participants With Infusion Site Reactions (ISRs)
Time frame: Up to 668 days
Treatment-emergent ISRs have onset on or after date of the first administration of study treatment up to on or before 28 days after last administration of study treatment. An ISR was counted in the period during which it started.
Number of Participants With Serious Adverse Events (SAEs)
Time frame: Up to 668 days
An adverse event is considered serious if, in the judgment of the investigator or sponsor, it results in death, is life-threatening, requires hospitalization or prolongs an existing hospitalization (admission for 24 hours or more, including emergency room stays), causes significant or persistent disability, results in a congenital anomaly or birth defect, or is an important medical event that may jeopardize the subject or require intervention to prevent these outcomes.
Number of Participants With Any TEAE Leading to Study Withdrawal
Time frame: Up to 668 days
TEAE Leading to Study Withdrawal refers to a TEAE that results in a participant permanently discontinuing or withdrawing from a clinical study because of the adverse event.
Number of Participants With Chronic Obstructive Pulmonary Disease (COPD) Exacerbations
Time frame: Up to 668 days
The definition of an exacerbation of COPD is an increase in respiratory symptoms (dyspnea, increased cough, and/or production of sputum) over baseline that usually requires medical intervention.
Number of Participants With Any Adverse Events Associated With Abnormalities in Vital Signs (Heart Rate, Blood Pressure, Respiratory Rate, and Temperature)
Time frame: Up to 668 days
Clinically significant vital sign abnormalities were presented as adverse events, with clinical relevance based on the Investigator's judgment.
Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1)
Time frame: Single-Dose Week 5/Intravenous Week 1, and Repeat-Dose Weeks 1, 4, and 9
FEV1 was performed according to ATS/ERS guidelines. At each visit, testing was performed both pre- and post-bronchodilator (BD) administration.
Change From Baseline in Forced Vital Capacity (FVC)
Time frame: Single-Dose Week 5/Intravenous Week 1, and Repeat-Dose Weeks 1, 4, and 9
FVC was performed according to ATS/ERS guidelines. At each visit, testing was performed both pre- and post-bronchodilator (BD) administration.
Number of Participants With Any Adverse Events Associated With Abnormalities in Laboratory Parameters (Chemistry, Hematology, Urinalysis)
Time frame: Up to 668 days
Out of the normal range laboratory results judged by the Investigator as clinically significant in the context of the participant's medical history were considered adverse events.
Immunogenicity: Number of Participants With Alpha1-PI Antibodies
Time frame: Treatment Period 1- Single-Dose Week 1; Treatment Period 2- Repeat-Dose Weeks 1 and 9
Immunogenicity testing was performed using validated assays in a multi-tiered approach.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Oct 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Alpha-1 15% and Liquid Alpha1-Proteinase Inhibitor (Human) in people with Alpha1-Antitrypsin Deficiency, run by Grifols Therapeutics LLC at 5 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 8 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 8 Oct 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Treatment Emergent Adverse Events (TEAEs) (time frame: Up to 668 days). The study lists 11 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Oct 2026.