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

Related Clinical Trial
A Single Ascending and Repeated Dose Study of Oral ZF874 in Healthy Volunteers and PiMZ Subjects ARALAST NP Alpha-1 Lung Density Chronic Obstructive Pulmonary Disease-Emphysema (COPD-E) Study Early Access Program Using Alpha 1 Antitrypsin Infusion for Patients With Steroid Refractory Acute GvHD After Hematopoietic Stem Cell Transplantation (HSCT) Characterization of the Pathobiology of Early Lung Destruction in Alpha 1-Antitrypsin Deficient Individuals COPD Exacerbation Blood and Urine Biomarkers Study Alpha-1 Carrier Genomics Study Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZ Genotype Study of ARO-AAT in Normal Adult Volunteers Safety Study of Alfalastin (Human Alpha-1 Antitrypsin) Administered at Home AL1TER™: Alpha-1 Therapy, Evaluation, and Research Patient Registry Respreeza® Self-administration and Learning Program (AmAREtTI Study) Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin (AAT) Deficiency Lung Disease and Its Affect on the Work of White Blood Cells in the Lungs A 12-week Study Treating Participants Who Have alpha1-antitrypsin-related COPD With Alvelestat (MPH966) or Placebo. Environment Effect on Six-Minute Walk Test Performance Alpha-1 Foundation DNA and Tissue Bank Alpha1-antitrypsin Deficiency Registry Alpha-1 Research Registry Stage 1 Study of ARALAST NP and GLASSIA in A1PI Deficiency Alpha-1 Coded Testing(ACT) Study Long-Term Follow-up Study of ADVM-043 Safety Dose Finding Study of ADVM-043 Gene Therapy to Treat Alpha-1 Antitrypsin (A1AT) Deficiency 4-PBA: Will it Increase the Level of Alpha 1-Antitrypsin(AAT) in Persons With AAT Deficiency? Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin Deficiency Management of Patients With Alpha-1 Antitrypsin Deficiency Associated Emphysema Phase II, Safety and Efficacy Study of Kamada-alpha-1-antitrypsin (AAT) for Inhalation” EARCO REGISTRY. History Of Patients With Alpha-1 Antitrypsin GLASSIA Safety, Immunogenicity, and Bronchoalveolar Lavage Study Safety, Tolerability and Effect of ARC-AAT Injection on Circulating and Intrahepatic Alpha-1 Antitrypsin Levels Efficacy/Safety of HA Inhalation Solution for Hereditary Emphysema in Patients With Alpha-1 Antitrypsin Deficiency Safety and Pharmacokinetics of Alpha-1 MP in Patients With Alpha1-Antitrypsin Deficiency Phase II/III Study of an Alpha-1 Proteinase Inhibitor (Kamada-API) in Individuals With Alpha-1 Antitrypsin Deficiency Study of the Effect of Aerosolized, Recombinant Alpha 1-Antitrypsin on Epithelial Lining Fluid Analytes in Subjects With Alpha 1-Antitrypsin Deficiency Alvelestat (MPH966) for the Treatment of ALpha-1 ANTitrypsin Deficiency The Safety and Tolerability of Alpha-1 Modified Process (MP) In Subjects With Alpha-1-antitrypsin (AAT) Deficiency Lung Volume Reduction Coils for Emphysema in Alpha-1 Antitrypsin Deficiency Study of Genotype and Phenotype in Patients With Alpha 1-Antitrypsin Deficiency Alpha-1 Foundation Research Registry Targeting Pulmonary Perfusion in Alpha-1 Antitrypsin Deficiency Effects of Exercise Training in Chronic Obstructive Pulmonary Disease Versus Alpha-1-Antitrypsin-deficiency-patients Evaluate Efficacy and Safety of “Kamada-AAT for Inhalation” in Patients With AATD A Study of DCR-A1AT in Healthy Adult Volunteers and Patients With A1ATD-Associated Liver Disease Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency Effects of Different Exercise Training Modalities in Alpha-1 Antitrypsin Deficiency Patients Safety Study of an Aerosolized, Recombinant Alpha 1-Antitrypsin in Subjects With Alpha 1-Antitrypsin Deficiency Study Comparing Weekly Intravenous Administration of OctaAlpha1 With a Marketed Preparation Glassia® in Subjects With Alpha-1-antitrypsin Deficiency Long-term Safety of Alpha1-Proteinase Inhibitor (Human) in Japanese Subjects With Alpha1 Antitrypsin Deficiency (GTI1401-OLE) Efficacy and Safety Study of Augmentation Therapy With ARALAST Fraction IV-1 (Human Alpha 1 – Proteinase Inhibitor) Aralast alpha1-proteinase Inhibitor Surveillance Study Pharmacokinetic Study of ARALAST (Human Alpha1- PI) The Use of High Resolution Chest Computed Tomography in Alpha-1 Antitrypsin Deficiency Prevalence of Alpha-1 Antitrypsin Deficiency in Chronic Obstructive Pulmonary Disease (COPD) Comparison of Pharmacokinetic, Safety, Tolerability of Alpha-1 MP and Prolastin In Alpha1-antitrypsin Deficient Adults Phase 1 Study to Assess the Safety, PK and PD of INBRX-101 in Adults With Alpha-1 Antitrypsin Deficiency Safety and Pharmacokinetics of Alpha-1 Proteinase Inhibitor in Subjects With Alpha1-Antitrypsin Deficiency The Impact of Delayed Diagnosis of Alpha-1 Antitrypsin Deficiency Epigenetic Regulation of Immunity in Alpha-1 Anti-trypsin Deficiency Microbioma in Sputa From COPD With Alpha-1 Antitrypsin Deficiency A Study of ARC-AAT in Healthy Volunteer Subjects and Patients With Alpha-1 Antitrypsin Deficiency (AATD) A Study to Assess Safety and PK of Liquid Alpha₁-Proteinase Inhibitor (Human) in Treating Alpha₁-Antitrypsin Deficiency

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


Primary Outcome

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

Secondary Outcome

 Assessment of St. George Respiratory Questionnaire




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


* 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


Estimated Enrollment


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

          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

          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

          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

          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 -




18 Years - 80 Years

Accepts Healthy Volunteers



Gerard M Turino, MD, , 

Location Countries


Location Countries


Administrative Informations



Organization ID


Responsible Party


Study Sponsor

Gerard Turino

Study Sponsor

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

Verification Date

April 2020