Trial | NCT03114020  Report issue

Title

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

  • Phase

    Phase 2

  • Study Type

    Interventional

  • Status

    Completed No Results Posted

  • Intervention/Treatment

    hyaluronic acid ...

  • Study Participants

    27
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.
Study Started
Mar 22
2017
Primary Completion
Nov 12
2019
Study Completion
Nov 12
2019
Last Update
Apr 21
2020

Drug Hyaluronic Acid Inhalation Solution

twice a day 3 mL of 0.03% Hyaluronic Acid Inhalation Solution

  • Other names: active inhalation solution

Drug Placebo Inhalation Solution

Twice a day 3 ml of placebo inhalation solution

Hyaluronic Acid inhalation solution Experimental

3mL of 0.03% Hyaluronic Acid inhalation solution BID for 28 days

Placebo Inhalation Solution Placebo Comparator

3mL matching placebo inhalation solution BID for 28 days

Criteria 

Inclusion Criteria:

Able and willing to provide written informed consent and comply with study requirements
Men or women aged 18 through 80 years at the time of consent
Diagnosis of emphysema at screening consistent with National Institutes of Health guidelines 19 GOLD COPD classification stages I, II or III
Evidence of emphysema on radiographic imaging.
A ratio of pre-bronchodilator FEV1 to forced vital capacity (FVC) of ≤ 80% at screening
FEV1 ≥ 30% and ≤ 79% (post-bronchodilator) of predicted normal at screening
Clinical laboratory tests (complete blood count, serum chemistry, and urinalysis) within normal limits or clinically acceptable to the PI and sponsor at screening
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.
Evidence of alpha-1 antitrypsin deficiency (AATD) with any genotype except PiMZ deficiency. Individuals with PiMZ deficiency are not allowed in the study.
Patients must have stopped using Intravenous alpha-1 antitrypsin protein (AAT) augmentation therapy at least 3 months before entering study.

Exclusion Criteria:

Subjects with measured DLCO of ≤ 35%, or unable to perform a reproducible DLCO
Subjects unable to perform 3 reproducible spirometry tests after 8 attempts
Upper or lower respiratory tract infection within 2 weeks prior to screening and baseline (day1)
Presence of clinically relevant abnormality on chest x-ray (other than evidence of emphysema) within the previous 12 months
Use of supplemental oxygen therapy
Requirement for ventilator support within the last year
Exacerbation requiring treatment with systemic corticosteroids within the last 3 months
History of lung transplant or liver transplant.
Presence of clinically relevant abnormality on electrocardiogram (ECG)
Any medical condition that, in the investigator's judgment, would compromise study participation or the evaluation of the study drug
Women who are pregnant or breastfeeding
Receipt of an investigational drug within 30 days prior to screening
Patients who are current smokers or have smoked within the last 3 months -
No Results Posted