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

NCT03114020 | Completed Phase 2 FDA Drug

• 1 other identifier: C100-008
• Study Type: interventional
• Target: 27
• Locations: 2 countries
• Sites: 6

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

Conditions

Emphysema; Alpha 1-Antitrypsin Deficiency

Keywords

Emphysema; COPD; Alpha-1 Antitrypsin Deficiency

Outcome Measures

Primary Outcomes (2)

• Measurement of sputum, plasma and urine concentrations of desmosine and isodesmosine

  measurement of biomarkers

  28 days

• Assessment of pulmonary function tests

  measurement of pulmonary function

  28 days

Secondary Outcomes (1)

• Assessment of St. George Respiratory Questionnaire

  28 days

Study Arms (2)

Hyaluronic Acid inhalation solution

EXPERIMENTAL

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

Drug: Hyaluronic Acid Inhalation Solution

Placebo Inhalation Solution

PLACEBO COMPARATOR

3mL matching placebo inhalation solution BID for 28 days

Drug: Placebo Inhalation Solution

Interventions

Hyaluronic Acid Inhalation Solution DRUG

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

Also known as: active inhalation solution

Hyaluronic Acid inhalation solution

Placebo Inhalation Solution DRUG

Twice a day 3 ml of placebo inhalation solution

Placebo Inhalation Solution

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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 -

Sponsors & Collaborators

• Gerard Turino: lead

Study Sites (6)

University of Miami Hospital

Miami, Florida, 33136, United States

Location

Pulmonary Health Physicians, PC

Fayetteville, New York, 13066, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

University of Texas Health Science Center

Tyler, Texas, 75708, United States

Location

Medical College of Wisconsin / Froedtert Hospital

Milwaukee, Wisconsin, 53226, United States

Location

Inspiration Research Limited

Toronto, Ontario, M5T 3A9, Canada

Location

MeSH Terms

Conditions

Emphysema; alpha 1-Antitrypsin Deficiency; Pulmonary Disease, Chronic Obstructive

Condition Hierarchy (Ancestors)

Pathologic Processes; Pathological Conditions, Signs and Symptoms; Liver Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Subcutaneous Emphysema; Lung Diseases, Obstructive; Chronic Disease; Disease Attributes

Study Officials

• Gerard M Turino, MD

  Mount Sinai St Lukes

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Masking Details: treatment has blinded labeling
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDIV
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Sponsor-Investigator

Study Record Dates

• First Submitted: April 5, 2017
• First Posted: April 14, 2017
• Study Start: March 22, 2017
• Primary Completion: November 12, 2019
• Study Completion: November 12, 2019
• Last Updated: April 21, 2020

Record last verified: 2020-04

Data Sharing

• IPD Sharing: Will not share

Locations

CA (1); US (5)