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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03114020
Recruitment Status : Recruiting
First Posted : April 14, 2017
Last Update Posted : August 29, 2019
Information provided by (Responsible Party):
Gerard Turino, Turino, Gerard, M.D.

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

Condition or disease Intervention/treatment Phase
Emphysema Alpha 1-Antitrypsin Deficiency Drug: Hyaluronic Acid Inhalation Solution Drug: Placebo Inhalation Solution Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 40 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Masking Description: treatment has blinded labeling
Primary Purpose: Treatment
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
Actual Study Start Date : March 22, 2017
Estimated Primary Completion Date : March 2020
Estimated Study Completion Date : March 2020

Arm Intervention/treatment
Experimental: Hyaluronic Acid inhalation solution
3mL of 0.03% Hyaluronic Acid inhalation solution BID for 28 days
Drug: Hyaluronic Acid Inhalation Solution
twice a day 3 mL of 0.03% Hyaluronic Acid Inhalation Solution
Other Name: active inhalation solution

Placebo Comparator: Placebo Inhalation Solution
3mL matching placebo inhalation solution BID for 28 days
Drug: Placebo Inhalation Solution
Twice a day 3 ml of placebo inhalation solution

Primary Outcome Measures :
  1. Measurement of sputum, plasma and urine concentrations of desmosine and isodesmosine [ Time Frame: 28 days ]
    measurement of biomarkers

  2. Assessment of pulmonary function tests [ Time Frame: 28 days ]
    measurement of pulmonary function

Secondary Outcome Measures :
  1. Assessment of St. George Respiratory Questionnaire [ Time Frame: 28 days ]
    measurement of patient abilities

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts 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 -

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03114020

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Contact: Gerard M Turino, MD 2125235919

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United States, Florida
University of Miami Hospital Recruiting
Miami, Florida, United States, 33136
Contact: Eliana Mendes, MD    305-243-2568   
Contact: Patricia Rebolledo   
United States, New York
Pulmonary Health Physicians, PC Recruiting
Fayetteville, New York, United States, 13066
Contact: Jeannine Moore    315-234-0823   
United States, South Carolina
Medical University of South Carolina Recruiting
Charleston, South Carolina, United States, 29425
Contact: Gwen Blanton    843-792-8438   
United States, Texas
University of Texas Health Science Center Recruiting
Tyler, Texas, United States, 75708
Contact: Rebekah Hibbard    903-877-8246   
United States, Wisconsin
Medical College of Wisconsin / Froedtert Hospital Recruiting
Milwaukee, Wisconsin, United States, 53226
Contact: Amy Blair    414-955-7007   
Sponsors and Collaborators
Gerard Turino
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Principal Investigator: Gerard M Turino, MD Mount Sinai St Lukes

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Responsible Party: Gerard Turino, Sponsor-Investigator, Turino, Gerard, M.D. Identifier: NCT03114020     History of Changes
Other Study ID Numbers: C100-008
First Posted: April 14, 2017    Key Record Dates
Last Update Posted: August 29, 2019
Last Verified: August 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Gerard Turino, Turino, Gerard, M.D.:
Alpha-1 Antitrypsin Deficiency
Additional relevant MeSH terms:
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Alpha 1-Antitrypsin Deficiency
Pulmonary Emphysema
Respiratory Tract Diseases
Pathologic Processes
Pulmonary Disease, Chronic Obstructive
Lung Diseases, Obstructive
Lung Diseases
Liver Diseases
Digestive System Diseases
Genetic Diseases, Inborn
Subcutaneous Emphysema
Pharmaceutical Solutions
Hyaluronic Acid
Alpha 1-Antitrypsin
Adjuvants, Immunologic
Immunologic Factors
Physiological Effects of Drugs
Protective Agents
Trypsin Inhibitors
Serine Proteinase Inhibitors
Protease Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action