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New treatment option for Alpha 1-Antitrypsin Deficiency
Official title Gene Therapy for Alpha 1- Antitrypsin Deficiency
ClinicalTrials.gov ID: NCT06996756
What this study is testing
What is AAV8hAAT(AVL)?
AAV8hAAT(AVL) is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for alpha 1-antitrypsin deficiency.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This is a study of gene therapy to treat alpha 1-antitrypsin (AAT) deficiency. This study aims to treat AAT deficiency with a single administration of AAV8hAAT(AVL), a gene therapy that codes for an oxidation resistant form of the AAT protein, which if safe and if efficacious, will protect the lung on a persistent basis.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 70
You may be able to join if
- AAT genotype ZZ, or Z null heterozygotes, and if on augmentation therapy, pre-therapy AAT serum levels \<11 μM
- Emphysema as assessed by chest high resolution computational tomography (HRCT)
- Lung function parameters consistent with mild to moderate loss of lung function and the presence of emphysema.
- Troponin T within normal limits
- Normal liver ultrasound and serum alpha fetoprotein
You likely can't join if
- Individuals receiving systemic corticosteroids or other immunosuppressive medications for pre-existing conditions.
- Inability to tolerate immunosuppression with corticosteroids (e.g., uncontrolled diabetes)
- Individuals with an immunodeficiency disease, or evidence of active infection of any type, including human immunodeficiency virus
- Evidence of major central nervous system, major psychiatric, musculoskeletal or immune disorder
- Prior history of myocardial infarction or cancer within the past 5 years (other than basal cell carcinoma of the skin)
- Decompensated heart failure (NY4A class III-IV at time of baseline clinical assessment)
See the full eligibility criteria
- AAT genotype ZZ, or Z null heterozygotes, and if on augmentation therapy, pre-therapy AAT serum levels \<11 μM
- Emphysema as assessed by chest high resolution computational tomography (HRCT)
- Lung function parameters consistent with mild to moderate loss of lung function and the presence of emphysema.
- Troponin T within normal limits
- Normal liver ultrasound and serum alpha fetoprotein
- Normal kidney function
- No contraindications to receiving corticosteroid immunosuppression
- Individuals receiving systemic corticosteroids or other immunosuppressive medications for pre-existing conditions.
- Inability to tolerate immunosuppression with corticosteroids (e.g., uncontrolled diabetes)
- Individuals with an immunodeficiency disease, or evidence of active infection of any type, including human immunodeficiency virus
- Evidence of major central nervous system, major psychiatric, musculoskeletal or immune disorder
- Prior history of myocardial infarction or cancer within the past 5 years (other than basal cell carcinoma of the skin)
- Decompensated heart failure (NY4A class III-IV at time of baseline clinical assessment)
- Abnormal ECG at screening with findings consistent with cardiac disease
- Females who are currently pregnant or lactating
- Any history of allergies to drugs used for bronchoscopy, including xylocaine, lidocaine, versed, valium, atropine, pilocarpine, isoproterenol, terbutaline, aminophylline, or any local anesthetic
- Individuals receiving experimental medications or participating in another experimental protocol for at least 3 months prior to entry to the study
- Use of oxygen supplementation
- Risk for thromboembolic disease
- History of significant cardiovascular disease, hypertension, prior myocardial infarction and/or cerebrovascular event
- Individuals who are currently on beta-blockers, or other cardiac therapy related drugs
- Prior history of hypersensitivity or anaphylaxis associated with the administration of any AAT product
The study team makes the final eligibility decision.
Where it's taking place
- New York, New York, United States
Compensation & support
A stipend or compensation may be offered.
Compensation mentioned.
ClinicalTrials.gov doesn't provide a reliable structured field for payment or travel support - confirm details with the study team.
Questions & answers
Do participants get paid in this trial?
This study's listing includes signals that participants may be compensated or receive a stipend. Amounts vary and are set by the study team - confirm the details with them.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 70 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include New York, New York, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.