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

Emphysema Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03114020
• Other study ID #: C100-008
• Status: Completed
• Phase: Phase 2
• Start date: March 22, 2017
• Est. completion date: November 12, 2019

Study information

• Verified date: April 2020
• Source: Turino, Gerard, M.D.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 27
• Est. completion date: November 12, 2019
• Est. primary completion date: November 12, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 80 Years

Eligibility

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 -

Related Conditions & MeSH terms

• Alpha 1-Antitrypsin Deficiency
• Emphysema
• Pulmonary Emphysema

Intervention

Drug:
• Hyaluronic Acid Inhalation Solution
twice a day 3 mL of 0.03% Hyaluronic Acid Inhalation Solution
• Placebo Inhalation Solution
Twice a day 3 ml of placebo inhalation solution

Locations

Country	Name	City	State
Canada	Inspiration Research Limited	Toronto	Ontario
United States	Medical University of South Carolina	Charleston	South Carolina
United States	Pulmonary Health Physicians, PC	Fayetteville	New York
United States	University of Miami Hospital	Miami	Florida
United States	Medical College of Wisconsin / Froedtert Hospital	Milwaukee	Wisconsin
United States	University of Texas Health Science Center	Tyler	Texas

Sponsors (1)

Lead Sponsor	Collaborator
Gerard Turino

Countries where clinical trial is conducted

United States,  Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Measurement of sputum, plasma and urine concentrations of desmosine and isodesmosine	measurement of biomarkers	28 days
Primary	Assessment of pulmonary function tests	measurement of pulmonary function	28 days
Secondary	Assessment of St. George Respiratory Questionnaire	measurement of patient abilities	28 days