Ignite Creation Date:
2025-12-25 @ 4:11 AM
Ignite Modification Date:
2026-03-05 @ 12:49 AM
Study NCT ID:
NCT03114020
Status:
COMPLETED
Last Update Posted:
2020-04-21
First Post:
2017-04-05
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Efficacy/Safety of HA Inhalation Solution for Hereditary Emphysema in Patients With Alpha-1 Antitrypsin Deficiency
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D004646', 'term': 'Emphysema'}, {'id': 'D019896', 'term': 'alpha 1-Antitrypsin Deficiency'}, {'id': 'D029424', 'term': 'Pulmonary Disease, Chronic Obstructive'}], 'ancestors': [{'id': 'D010335', 'term': 'Pathologic Processes'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}, {'id': 'D008107', 'term': 'Liver Diseases'}, {'id': 'D004066', 'term': 'Digestive System Diseases'}, {'id': 'D008171', 'term': 'Lung Diseases'}, {'id': 'D012140', 'term': 'Respiratory Tract Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D013352', 'term': 'Subcutaneous Emphysema'}, {'id': 'D008173', 'term': 'Lung Diseases, Obstructive'}, {'id': 'D002908', 'term': 'Chronic Disease'}, {'id': 'D020969', 'term': 'Disease Attributes'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'DOUBLE', 'whoMasked': ['PARTICIPANT', 'INVESTIGATOR'], 'maskingDescription': 'treatment has blinded labeling'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 27}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2017-03-22', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2020-04', 'completionDateStruct': {'date': '2019-11-12', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2020-04-17', 'studyFirstSubmitDate': '2017-04-05', 'studyFirstSubmitQcDate': '2017-04-10', 'lastUpdatePostDateStruct': {'date': '2020-04-21', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2017-04-14', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2019-11-12', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Measurement of sputum, plasma and urine concentrations of desmosine and isodesmosine', 'timeFrame': '28 days', 'description': 'measurement of biomarkers'}, {'measure': 'Assessment of pulmonary function tests', 'timeFrame': '28 days', 'description': 'measurement of pulmonary function'}], 'secondaryOutcomes': [{'measure': 'Assessment of St. George Respiratory Questionnaire', 'timeFrame': '28 days', 'description': 'measurement of patient abilities'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Emphysema', 'COPD', 'Alpha-1 Antitrypsin Deficiency'], 'conditions': ['Emphysema', 'Alpha 1-Antitrypsin Deficiency']}, 'descriptionModule': {'briefSummary': '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', 'detailedDescription': '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.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '80 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n1. Able and willing to provide written informed consent and comply with study requirements\n2. Men or women aged 18 through 80 years at the time of consent\n3. Diagnosis of emphysema at screening consistent with National Institutes of Health guidelines 19 GOLD COPD classification stages I, II or III\n4. Evidence of emphysema on radiographic imaging.\n5. A ratio of pre-bronchodilator FEV1 to forced vital capacity (FVC) of ≤ 80% at screening\n6. FEV1 ≥ 30% and ≤ 79% (post-bronchodilator) of predicted normal at screening\n7. Clinical laboratory tests (complete blood count, serum chemistry, and urinalysis) within normal limits or clinically acceptable to the PI and sponsor at screening\n8. 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.\n9. Evidence of alpha-1 antitrypsin deficiency (AATD) with any genotype except PiMZ deficiency. Individuals with PiMZ deficiency are not allowed in the study.\n10. Patients must have stopped using Intravenous alpha-1 antitrypsin protein (AAT) augmentation therapy at least 3 months before entering study.\n\nExclusion Criteria:\n\n1. Subjects with measured DLCO of ≤ 35%, or unable to perform a reproducible DLCO\n2. Subjects unable to perform 3 reproducible spirometry tests after 8 attempts\n3. Upper or lower respiratory tract infection within 2 weeks prior to screening and baseline (day1)\n4. Presence of clinically relevant abnormality on chest x-ray (other than evidence of emphysema) within the previous 12 months\n5. Use of supplemental oxygen therapy\n6. Requirement for ventilator support within the last year\n7. Exacerbation requiring treatment with systemic corticosteroids within the last 3 months\n8. History of lung transplant or liver transplant.\n9. Presence of clinically relevant abnormality on electrocardiogram (ECG)\n10. Any medical condition that, in the investigator's judgment, would compromise study participation or the evaluation of the study drug\n11. Women who are pregnant or breastfeeding\n12. Receipt of an investigational drug within 30 days prior to screening\n13. Patients who are current smokers or have smoked within the last 3 months -"}, 'identificationModule': {'nctId': 'NCT03114020', 'briefTitle': 'Efficacy/Safety of HA Inhalation Solution for Hereditary Emphysema in Patients With Alpha-1 Antitrypsin Deficiency', 'organization': {'class': 'INDIV', 'fullName': 'Turino, Gerard, M.D.'}, 'officialTitle': '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', 'orgStudyIdInfo': {'id': 'C100-008'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Hyaluronic Acid inhalation solution', 'description': '3mL of 0.03% Hyaluronic Acid inhalation solution BID for 28 days', 'interventionNames': ['Drug: Hyaluronic Acid Inhalation Solution']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo Inhalation Solution', 'description': '3mL matching placebo inhalation solution BID for 28 days', 'interventionNames': ['Drug: Placebo Inhalation Solution']}], 'interventions': [{'name': 'Hyaluronic Acid Inhalation Solution', 'type': 'DRUG', 'otherNames': ['active inhalation solution'], 'description': 'twice a day 3 mL of 0.03% Hyaluronic Acid Inhalation Solution', 'armGroupLabels': ['Hyaluronic Acid inhalation solution']}, {'name': 'Placebo Inhalation Solution', 'type': 'DRUG', 'description': 'Twice a day 3 ml of placebo inhalation solution', 'armGroupLabels': ['Placebo Inhalation Solution']}]}, 'contactsLocationsModule': {'locations': [{'zip': '33136', 'city': 'Miami', 'state': 'Florida', 'country': 'United States', 'facility': 'University of Miami Hospital', 'geoPoint': {'lat': 25.77427, 'lon': -80.19366}}, {'zip': '13066', 'city': 'Fayetteville', 'state': 'New York', 'country': 'United States', 'facility': 'Pulmonary Health Physicians, PC', 'geoPoint': {'lat': 43.02979, 'lon': -76.00436}}, {'zip': '29425', 'city': 'Charleston', 'state': 'South Carolina', 'country': 'United States', 'facility': 'Medical University of South Carolina', 'geoPoint': {'lat': 32.77632, 'lon': -79.93275}}, {'zip': '75708', 'city': 'Tyler', 'state': 'Texas', 'country': 'United States', 'facility': 'University of Texas Health Science Center', 'geoPoint': {'lat': 32.35126, 'lon': -95.30106}}, {'zip': '53226', 'city': 'Milwaukee', 'state': 'Wisconsin', 'country': 'United States', 'facility': 'Medical College of Wisconsin / Froedtert Hospital', 'geoPoint': {'lat': 43.0389, 'lon': -87.90647}}, {'zip': 'M5T 3A9', 'city': 'Toronto', 'state': 'Ontario', 'country': 'Canada', 'facility': 'Inspiration Research Limited', 'geoPoint': {'lat': 43.70643, 'lon': -79.39864}}], 'overallOfficials': [{'name': 'Gerard M Turino, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Mount Sinai St Lukes'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Gerard Turino', 'class': 'INDIV'}, 'responsibleParty': {'type': 'SPONSOR_INVESTIGATOR', 'investigatorTitle': 'Sponsor-Investigator', 'investigatorFullName': 'Gerard Turino', 'investigatorAffiliation': 'Turino, Gerard, M.D.'}}}}