Emphysema, Alpha 1-Antitrypsin Deficiency
Conditions
Keywords
Emphysema, COPD, Alpha-1 Antitrypsin Deficiency
Brief summary
The purpose of the study is to evaluate the safety and efficacy of administering repeated doses of Hyaluronic Acid Inhalation Solution to subjects with Emphysema that have Alpha-1-Antitrypsin deficiency
Detailed description
The study primarily aims to establish desmosine and isodesmosine concentrations in plasma, sputum and urine measured as markers of elastin degradation systemically in the lung and also markers of inflammation and fibrinogen. Assessment of vital signs, lab tests, carbon monoxide diffusing capacity, oxygen saturation, pulmonary function tests, ECGs, physical exams and adverse events.
Interventions
DRUGHyaluronic Acid Inhalation Solution
twice a day 3 mL of 0.03% Hyaluronic Acid Inhalation Solution
Twice a day 3 ml of placebo inhalation solution
Sponsors
Gerard Turino
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Masking description
treatment has blinded labeling
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
1. Able and willing to provide written informed consent and comply with study requirements 2. Men or women aged 18 through 80 years at the time of consent 3. Diagnosis of emphysema at screening consistent with National Institutes of Health guidelines 19 GOLD COPD classification stages I, II or III 4. Evidence of emphysema on radiographic imaging. 5. A ratio of pre-bronchodilator FEV1 to forced vital capacity (FVC) of ≤ 80% at screening 6. FEV1 ≥ 30% and ≤ 79% (post-bronchodilator) of predicted normal at screening 7. Clinical laboratory tests (complete blood count, serum chemistry, and urinalysis) within normal limits or clinically acceptable to the PI and sponsor at screening 8. Women of childbearing potential and men who are sexually active must agree to use an adequate method of contraception (oral contraceptives, depot progesterone, condom plus spermicidal, or IUD) during the study and for 1 month after the final dose of study drug. 9. Evidence of alpha-1 antitrypsin deficiency (AATD) with any genotype except PiMZ deficiency. Individuals with PiMZ deficiency are not allowed in the study. 10. Patients must have stopped using Intravenous alpha-1 antitrypsin protein (AAT) augmentation therapy at least 3 months before entering study.
Exclusion criteria
1. Subjects with measured DLCO of ≤ 35%, or unable to perform a reproducible DLCO 2. Subjects unable to perform 3 reproducible spirometry tests after 8 attempts 3. Upper or lower respiratory tract infection within 2 weeks prior to screening and baseline (day1) 4. Presence of clinically relevant abnormality on chest x-ray (other than evidence of emphysema) within the previous 12 months 5. Use of supplemental oxygen therapy 6. Requirement for ventilator support within the last year 7. Exacerbation requiring treatment with systemic corticosteroids within the last 3 months 8. History of lung transplant or liver transplant. 9. Presence of clinically relevant abnormality on electrocardiogram (ECG) 10. Any medical condition that, in the investigator's judgment, would compromise study participation or the evaluation of the study drug 11. Women who are pregnant or breastfeeding 12. Receipt of an investigational drug within 30 days prior to screening 13. Patients who are current smokers or have smoked within the last 3 months -
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Measurement of sputum, plasma and urine concentrations of desmosine and isodesmosine | 28 days | measurement of biomarkers |
| Assessment of pulmonary function tests | 28 days | measurement of pulmonary function |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Assessment of St. George Respiratory Questionnaire | 28 days | measurement of patient abilities |
Countries
Canada, United States
Outcome results
None listed