Ignite Creation Date:
2025-12-24 @ 2:14 PM
Ignite Modification Date:
2026-01-23 @ 8:51 AM
Study NCT ID:
NCT03385395
Status:
WITHDRAWN
Last Update Posted:
2018-04-10
First Post:
2017-12-01
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Study Comparing Weekly Intravenous Administration of OctaAlpha1 With a Marketed Preparation Glassia® in Subjects With Alpha-1-antitrypsin Deficiency
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D019896', 'term': 'alpha 1-Antitrypsin Deficiency'}], 'ancestors': [{'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': 'D004646', 'term': 'Emphysema'}, {'id': 'D010335', 'term': 'Pathologic Processes'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D000515', 'term': 'alpha 1-Antitrypsin'}], 'ancestors': [{'id': 'D006023', 'term': 'Glycoproteins'}, {'id': 'D006001', 'term': 'Glycoconjugates'}, {'id': 'D002241', 'term': 'Carbohydrates'}, {'id': 'D015843', 'term': 'Serpins'}, {'id': 'D010455', 'term': 'Peptides'}, {'id': 'D000602', 'term': 'Amino Acids, Peptides, and Proteins'}, {'id': 'D000209', 'term': 'Acute-Phase Proteins'}, {'id': 'D001798', 'term': 'Blood Proteins'}, {'id': 'D011506', 'term': 'Proteins'}, {'id': 'D000510', 'term': 'Alpha-Globulins'}, {'id': 'D012712', 'term': 'Serum Globulins'}, {'id': 'D005916', 'term': 'Globulins'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 0}}, 'statusModule': {'whyStopped': 'IND has been closed.', 'overallStatus': 'WITHDRAWN', 'startDateStruct': {'date': '2018-07', 'type': 'ESTIMATED'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2018-04', 'completionDateStruct': {'date': '2019-12', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2018-04-06', 'studyFirstSubmitDate': '2017-12-01', 'studyFirstSubmitQcDate': '2017-12-20', 'lastUpdatePostDateStruct': {'date': '2018-04-10', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2017-12-28', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2019-12', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Non-inferiority of OctaAlpha1 compared to Glassia® in terms of the serum trough levels at steady state', 'timeFrame': '26 weeks', 'description': 'Non-inferiority of OctaAlpha1 compared to Glassia® in terms of the serum trough levels at steady state'}], 'secondaryOutcomes': [{'measure': 'Compare PK parameters following a single dose between the two treatment groups following principles of bio-equivalence testing (AUC)', 'timeFrame': 'Time period including days 1 to 14 after first infusion in study', 'description': 'Compare PK parameters following a single dose between the two treatment groups calculating area under the plasma concentration-time curve (AUC)-ratio: 90% confidence interval (CI) should lie within 80%-125%.'}, {'measure': 'Compare PK parameters following a single dose between the two treatment groups following principles of bio-equivalence testing (Cmax)', 'timeFrame': 'Time period including days 1 to 14 after first infusion in study', 'description': 'Compare PK parameters following a single dose between the two treatment groups calculating maximum plasma concentration (Cmax)-ratio: 90% CI should lie within 80%-125%'}, {'measure': 'Compare PK parameters following a single dose between the two treatment groups following principles of bio-equivalence testing (tmax)', 'timeFrame': 'Time period including days 1 to 14 after first infusion in study', 'description': 'Compare PK parameters following a single dose between the two treatment groups calculating tmax (time to reach maximum serum concentration)'}, {'measure': 'Compare PK parameters following a single dose between the two treatment groups following principles of bio-equivalence testing (t1/2)', 'timeFrame': 'Time period including days 1 to 14 after first infusion in study', 'description': 'Compare PK parameters following a single dose between the two treatment groups calculating t1/2 (apparent terminal half-life)'}, {'measure': 'Compare PK parameters following a single dose between the two treatment groups following principles of bio-equivalence testing (λZ)', 'timeFrame': 'Time period including days 1 to 14 after first infusion in study', 'description': 'Compare PK parameters following a single dose between the two treatment groups calculating λZ (apparent terminal elimination rate constant determined by log-linear regression analysis)'}, {'measure': 'Evaluate descriptively the safety and tolerability of OctaAlpha1 based on occurrence of adverse events', 'timeFrame': '26 weeks', 'description': 'Evaluate descriptively the safety and tolerability of OctaAlpha1 based on occurrence of adverse events'}, {'measure': 'Evaluate descriptively the safety and tolerability of OctaAlpha1 based on blood pressure', 'timeFrame': '26 weeks', 'description': 'Evaluate descriptively the safety and tolerability of OctaAlpha1 based on blood pressure'}, {'measure': 'Evaluate descriptively the safety and tolerability of OctaAlpha1 based on pulse', 'timeFrame': '26 weeks', 'description': 'Evaluate descriptively the safety and tolerability of OctaAlpha1 based on pulse'}, {'measure': 'Evaluate descriptively the safety and tolerability of OctaAlpha1 based on body temperature', 'timeFrame': '26 weeks', 'description': 'Evaluate descriptively the safety and tolerability of OctaAlpha1 based on body temperature'}, {'measure': 'Evaluate descriptively the safety and tolerability of OctaAlpha1 based on respiratory rate', 'timeFrame': '26 weeks', 'description': 'Evaluate descriptively the safety and tolerability of OctaAlpha1 based on respiratory rate'}, {'measure': 'Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hematological parameter hematocrit', 'timeFrame': '26 weeks', 'description': 'Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hematological parameter hematocrit via lab test'}, {'measure': 'Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hematological parameter hemoglobin', 'timeFrame': '26 weeks', 'description': 'Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hematological parameter hemoglobin via lab test'}, {'measure': 'Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hematological parameter white blood cells', 'timeFrame': '26 weeks', 'description': 'Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hematological parameter white blood cells via lab test'}, {'measure': 'Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hematological parameter platelet count', 'timeFrame': '26 weeks', 'description': 'Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hematological parameter platelet count via lab test'}, {'measure': 'Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hepatic parameter alanine aminotransferase', 'timeFrame': '26 weeks', 'description': 'Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hepatic parameter alanine aminotransferase via lab test'}, {'measure': 'Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hepatic parameter aspartate aminotransferase', 'timeFrame': '26 weeks', 'description': 'Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hepatic parameter aspartate aminotransferase via lab test'}, {'measure': 'Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring renal parameter creatinine', 'timeFrame': '26 weeks', 'description': 'Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring renal parameter creatinine via lab test'}, {'measure': 'Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring renal parameter Blood Urea Nitrogen (BUN)', 'timeFrame': '26 weeks', 'description': 'Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring renal parameter Blood Urea Nitrogen (BUN) via lab test'}, {'measure': 'Evaluate descriptively the safety and tolerability of OctaAlpha1 based on viral safety (HAV, HBV, HCV, HIV-1/2, HN, and parvovirus B19)', 'timeFrame': '26 weeks', 'description': 'Evaluate descriptively the safety and tolerability of OctaAlpha1 based on viral safety (HAV, HBV, HCV, HIV-1/2, HN, and parvovirus B19)'}, {'measure': 'Trough Levels of A1PI', 'timeFrame': '26 weeks', 'description': 'Investigate descriptively the trough levels of A1PI and anti-NE capacity of OctaAlpha1 compared to Glassia®'}, {'measure': 'Pharmacodynamics of OctaAlpha1 measuring Pulmonary function tests', 'timeFrame': '26 weeks', 'description': 'Investigate the pharmacodynamics of OctaAlpha1 measuring Pulmonary function tests (done according to the American Thoracic Society/European Respiratory Society Taskforce Standardisation of Lung Function Testing guideline)'}, {'measure': 'Investigate the pharmacodynamics of OctaAlpha1 measuring Induced sputum test measuring the amount of functional and total A1PI in the airway lining fluid of the lung.', 'timeFrame': '26 weeks', 'description': 'Investigate the pharmacodynamics of OctaAlpha1 measuring Induced sputum test measuring the amount of functional and total A1PI in the airway lining fluid of the lung.'}, {'measure': 'Investigate the pharmacodynamics of OctaAlpha1 measuring Induced sputum test measuring the amount of functional and total LTB4 in the airway lining fluid of the lung.', 'timeFrame': '26 weeks', 'description': 'Investigate the pharmacodynamics of OctaAlpha1 measuring Induced sputum test measuring the amount of functional and total LTB4 in the airway lining fluid of the lung.'}, {'measure': 'Investigate the pharmacodynamics of OctaAlpha1 measuring antibodies to A1PI using normal ranges of the central laboratory', 'timeFrame': '26 weeks', 'description': 'Investigate the pharmacodynamics of OctaAlpha1 measuring antibodies to A1PI using normal ranges of the central laboratory'}, {'measure': 'Determine PK parameters of the A1PI serum concentration versus time curve following a single dose of OctaAlpha1', 'timeFrame': '26 weeks', 'description': 'Determine PK parameters of the A1PI serum concentration versus time curve following a single dose of OctaAlpha1'}]}, 'oversightModule': {'isUsExport': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Alpha 1-Antitrypsin Deficiency']}, 'descriptionModule': {'briefSummary': 'This randomized trial is being conducted to show non-inferiority of OctaAlpha1 compared to Glassia® in terms of the serum trough levels at steady state. This will be conducted in individuals with alpha-1-antitrypsin deficiency and clinical evidence of emphysema.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Any subject who needs chronic IV augmentation and maintenance therapy with A1PI because of congenital alpha-1-proteinase inhibitor (A1PI) deficiency and clinically diagnosed emphysema\n* ≥18 years of age\n* Individuals with A1PI serum concentration \\<11 µM at screening\n* Following bronchodilators:\n\n * Initial FEV1(pred) between 25% and 75% or\n * If the initial FEV1 was greater than 75% of predicted, a diffusing capacity of the lung for carbon monoxide (DLC O) less than 70% of predicted\n* Following bronchodilators: Initial forced expiratory volume/forced vital capacity (FEV1/FVC) ratio less than 70%\n* Non-smoking for at least 6 months before study treatment starts\n* Able to understand and provide written informed consent\n* Women of reproductive age: negative result of pregnancy test (human chorionic gonadotropin \\[HCG\\]-based assay) and agreement to use adequate contraception for the duration of the trial\n\nExclusion Criteria:\n\n* Any inflammatory condition or malignant tumor in the 7 days before treatment starts that according to investigator judgment might influence the metabolism of an enzyme inhibitor such as A1PI\n* More than one A1PI-deficiency related exacerbation and/or hospitalization during the 3 months before study treatment starts\n* Clinically significant liver or kidney disease in the preceding 6 months before study treatment starts\n* Severe gas exchange abnormality (i.e., PaCO2 ≥46 mmHg)\n* Known IgA deficiency with documented antibodies against IgA\n* History of hypersensitivity to blood or plasma derived products, or any component of the product\n* Known presence of antibodies against A1PI\n* Seropositivity for HBsAg or HCV, HIV-1/2 IgG antibodies\n* Administration of A1PI products in the 4 weeks before study treatment starts\n* Participating in another clinical study currently or during the 3 months before study treatment starts.\n* Live viral vaccination within the last month before study treatment starts\n* A current life-threatening malignancy\n* Emergency operation within 3 months before study treatment starts\n* History of, or suspected, alcohol or drug abuse within 1 year before study treatment starts or currently on drug abuse therapy\n* Pregnant and nursing women'}, 'identificationModule': {'nctId': 'NCT03385395', 'briefTitle': 'Study Comparing Weekly Intravenous Administration of OctaAlpha1 With a Marketed Preparation Glassia® in Subjects With Alpha-1-antitrypsin Deficiency', 'organization': {'class': 'INDUSTRY', 'fullName': 'Octapharma'}, 'officialTitle': 'A Randomized, Double-blind, Parallel-group, Multicenter, Pharmacokinetic Study Comparing Weekly Intravenous Administration of OctaAlpha1 (Octapharma) With a Marketed Preparation Glassia® (Kamada Ltd.) in Subjects With Alpha-1-antitrypsin Deficiency', 'orgStudyIdInfo': {'id': 'OctaAlpha1'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'OctaAlpha1', 'interventionNames': ['Drug: OctaAlpha1']}, {'type': 'ACTIVE_COMPARATOR', 'label': 'Glassia®', 'interventionNames': ['Drug: Glassia']}], 'interventions': [{'name': 'OctaAlpha1', 'type': 'DRUG', 'description': 'For OctaAlpha1 the standard weekly dose of 60mg/kg will be given for 24 consecutive infusions', 'armGroupLabels': ['OctaAlpha1']}, {'name': 'Glassia', 'type': 'DRUG', 'description': 'For Glassia the standard weekly dose of 60mg/kg will be given for 24 consecutive infusions', 'armGroupLabels': ['Glassia®']}]}, 'ipdSharingStatementModule': {'ipdSharing': 'UNDECIDED'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Octapharma', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}