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

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Brief Title

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

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

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
    

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.
    

Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

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

Secondary Outcome

 Assessment of St. George Respiratory Questionnaire

Condition

Emphysema

Intervention

Hyaluronic Acid Inhalation Solution

Study Arms / Comparison Groups

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

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

27

Start Date

March 22, 2017

Completion Date

November 12, 2019

Primary Completion Date

November 12, 2019

Eligibility Criteria

        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 -
      

Gender

All

Ages

18 Years - 80 Years

Accepts Healthy Volunteers

No

Contacts

Gerard M Turino, MD, , 

Location Countries

Canada

Location Countries

Canada

Administrative Informations


NCT ID

NCT03114020

Organization ID

C100-008


Responsible Party

Sponsor-Investigator

Study Sponsor

Gerard Turino


Study Sponsor

Gerard M Turino, MD, Principal Investigator, Mount Sinai St Lukes


Verification Date

April 2020