Lung Disease Effects for Alpha-1 Antitrypsin Deficiency and Cystic Fibrosis

Overseen By Allison E. Faunce, B.A.

Age: 18+

Sex: Any

Trial Phase: Academic

Sponsor: University of Florida

Disqualifiers: Pregnancy, Anemia, Hypertension, Cancer, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 3

220 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to understand how two specific lung conditions, Alpha-1-antitrypsin (AAT) deficiency and Cystic Fibrosis (CF), affect the function of certain white blood cells in the lungs. Participants will use an albuterol inhaler (a medication that helps open airways) and undergo tests such as blood draws and lung function tests. The trial includes three groups: one with CF, one with AAT deficiency, and one without either condition. Suitable candidates for this trial include individuals diagnosed with CF (specifically with the Delta F508 mutation), those with AAT deficiency, or those without any lung disease, who have a negative nicotine test and do not smoke.

As an unphased trial, this study offers participants the chance to contribute to foundational research that could lead to a better understanding and treatment of lung conditions.

Do I have to stop taking my current medications for the trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial organizers or your doctor.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research shows that albuterol inhalers are generally safe for individuals with lung conditions like cystic fibrosis and AAT deficiency. Studies have found that albuterol is usually well-tolerated, with most users experiencing no serious side effects. Some individuals might feel jittery or notice a fast heartbeat, but these mild effects are typically not serious and often resolve on their own. The FDA has approved albuterol, and it has been used for a long time to help people breathe easier by relaxing airway muscles.

For those with AAT deficiency, albuterol has been shown to improve lung function, although individual responses can vary. This treatment is widely used, and its safety is well-understood. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Researchers are excited about this trial because it aims to shed light on how lung diseases like Cystic Fibrosis and Alpha-1 Antitrypsin Deficiency affect the function of white blood cells in the lungs. By using a combination of albuterol inhalers, blood draws, and pulmonary function testing, the study examines how these conditions impact lung function and immune response. This approach could potentially reveal new insights into disease mechanisms, leading to better-targeted treatments in the future. Unlike standard treatments that focus on symptom management, this trial seeks to understand underlying biological changes, offering hope for more effective interventions down the line.

What evidence suggests that this trial's treatments could be effective for AAT deficiency and CF?

This trial will include participants with Cystic Fibrosis (CF), Alpha-1 Antitrypsin (AAT) Deficiency, and those without lung disease. Studies have shown that albuterol inhalers, which participants in this trial will use, can improve lung function in people with CF. One study found that patients using albuterol breathed better than those using a placebo. For individuals with AAT deficiency, research suggests that inhaled treatments can reduce lung inflammation, though albuterol is not specifically noted as effective for AAT deficiency as it is for CF. Overall, albuterol is known to open up the airways, making breathing easier, especially for CF patients. ^{2 6 7 8 9}

Who Is on the Research Team?

Karina Serban, MD

Principal Investigator

University of Florida, College of Medicine, Division of Pulmonary, Critical Care, and Sleep Medicine

Are You a Good Fit for This Trial?

Inclusion Criteria

Signed informed consent

Negative pregnancy test for women of childbearing potential

Hemoglobin >12.5 g/dl measured on the day of participation

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Study Visits

Participants undergo history and physical exam, blood draw, and pulmonary function testing with albuterol inhaler use at every study visit

4 weeks

3 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after study visits

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Albuterol inhaler
• Blood draw
• History and physical exam
• Pulmonary function testing

How Is the Trial Designed?

Group I: Without Lung Disease Diagnosis Experimental Treatment 4 Interventions

Those without the diagnosis of AAT Deficiency or CF. At every study visit, a history and physical exam (H\&P), blood draw, and pulmonary function testing (PFTs) with the use of an albuterol inhaler will be done.

Group II: Cystic Fibrosis Experimental Treatment 4 Interventions

Those diagnosed with Cystic Fibrosis (CF) with mutation Delta F508. At every study visit, a history and physical exam (H\&P), blood draw, and pulmonary function testing (PFTs) with the use of an albuterol inhaler will be done.

Group III: AAT Deficiency Experimental Treatment 4 Interventions

Those diagnosed with Alpha-1 Antitrypsin (AAT) Deficiency. At every study visit, a history and physical exam (H\&P), blood draw, and pulmonary function testing (PFTs) with the use of an albuterol inhaler will be done.

Who Is Running the Clinical Trial?

University of Florida

Lead Sponsor

Trials

1,428

Recruited

987,000+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC7857706/

Feasibility of Aerosolized Alpha-1 Antitrypsin as a ... Patients with alpha-1 antitrypsin deficiency (AATD) are at an increased risk of developing COPD and pulmonary emphysema and it is the emphysematous lung ...

2. alpha1.org alpha1.org/inhaled-alpha-1-antitrypsin-randomized-phase-2-study/

Inhaled AAT Reduces Inflammation in Study These results suggest that aerosol AAT replacement is highly effective in relieving the elastolytic burden in the lower airways of AATD patients ...

3. fda.gov fda.gov/media/136516/download

Patient-Focused Drug Development for Alpha-1 Antitrypsin ... Progression of emphysema in. AATD may lead to respiratory failure, a need for lung transplantation, and eventually death. The only specific ...

4. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/31467115/

Efficacy and safety of inhaled α1-antitrypsin in patients with ... We conclude that in AATD patients with severe COPD and frequent exacerbations, AAT inhalation for 50 weeks showed no effect on time to first exacerbation.

5. link.springer.com link.springer.com/article/10.1007/s13346-023-01346-3

Nebulizers effectiveness on pulmonary delivery of alpha-1 ... All results agreed on the safety of this approach and provided evidence of its efficacy in reducing lung inflammation. On the other hand, when ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC3974452/

Mysteries of α1-antitrypsin deficiency The classical form of α1-antitrypsin deficiency (ATD) is an autosomal co-dominant disorder that affects ~1 in 3000 live births and is an important genetic cause ...

7. clinicaltrials.gov clinicaltrials.gov/study/NCT01851642

Lung Disease and Its Affect on the Work of White Blood ... The purpose of this study is to look at how Alpha-1-antitrypsin (AAT) deficiency and Cystic Fibrosis (CF) affect white blood cells in the lungs, called ...

8. journal.copdfoundation.org journal.copdfoundation.org/jcopdf/id/1288/Feasibility-of-Aerosolized-Alpha-1-Antitrypsin-as-a-Therapeutic-Option

Aerosolized Alpha-1 Antitrypsin Patients with alpha-1 antitrypsin deficiency (AATD) are at an increased risk of developing COPD and pulmonary emphysema and it is the emphysematous lung ...

9. jaci-inpractice.org jaci-inpractice.org/article/S2213-2198(23)00468-3/fulltext

Improving Detection of Alpha-1 Antitrypsin Deficiency: Role ... With an albuterol inhaler, his FEV1 improved by 16% but he was still very symptomatic. The patient was started on dupilumab, which resulted ...