Efficacy/Safety of HA Inhalation Solution for Hereditary Emphysema in Patients With Alpha-1 Antitrypsin Deficiency

Conditions:Chronic Obstructive Pulmonary Disease, Pulmonary
Therapuetic Areas:Pulmonary / Respiratory Diseases
Age Range:18 - 80
Start Date:March 22, 2017
End Date:July 2019
Contact:Gerard M Turino, MD

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Phase 2 Randomized Parallel-Group Double-Blind Placebo-Controlled Multiple-Dose Proof-of-Concept Study to Evaluate the Efficacy/Safety of Hyaluronic Acid Inhalation Solution for Treatment of Emphysema

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

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.

Inclusion Criteria:

1. Able and willing to provide written informed consent and comply with study

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

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

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

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 -
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