Ignite Creation Date:
2025-12-24 @ 1:06 PM
Ignite Modification Date:
2026-01-09 @ 9:54 PM
Study NCT ID:
NCT00295061
Status:
COMPLETED
Last Update Posted:
2014-09-09
First Post:
2006-02-20
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Comparison of Pharmacokinetic, Safety, Tolerability of Alpha-1 MP and Prolastin In Alpha1-antitrypsin Deficient Adults
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D019896', 'term': 'alpha 1-Antitrypsin Deficiency'}, {'id': 'D011656', 'term': 'Pulmonary Emphysema'}], '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'}, {'id': 'D029424', 'term': 'Pulmonary Disease, Chronic Obstructive'}, {'id': 'D008173', 'term': 'Lung Diseases, Obstructive'}, {'id': 'D002908', 'term': 'Chronic Disease'}, {'id': 'D020969', 'term': 'Disease Attributes'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C114276', 'term': 'Mp alpha1 receptor'}, {'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'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'henry.li@grifols.com', 'phone': '1-800-520-2807', 'title': 'Henry Li', 'organization': 'Grifols Therapeutics'}, 'certainAgreement': {'otherDetails': 'The investigator must send a draft manuscript of the publication or abstract to the sponsor thirty days in advance of submission in order to obtain approval prior to submission of the final version for publication. This will be reviewed promptly and approval will not be withheld unreasonably. In case of a difference of opinion between the sponsor and the investigator(s), the contents of the publication will be discussed in order to find a solution which satisfies both parties.', 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'eventGroups': [{'id': 'EG000', 'title': 'Alpha-1 MP', 'description': 'Sequential, blinded treatment periods of Alpha-1 MP (experimental), then crossed-over to Prolastin (active comparator), followed by open-label Alpha-1 MP', 'otherNumAtRisk': 24, 'otherNumAffected': 3, 'seriousNumAtRisk': 24, 'seriousNumAffected': 0}, {'id': 'EG001', 'title': 'Prolastin', 'description': 'Sequential, blinded treatment periods of Prolastin (active comparator), then crossed-over to Alpha-1 MP (experimental), followed by open-label Alpha-1 MP', 'otherNumAtRisk': 24, 'otherNumAffected': 5, 'seriousNumAtRisk': 24, 'seriousNumAffected': 1}], 'otherEvents': [{'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 24, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations'}, {'term': 'Arthralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 24, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 24, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Nervous system disorders'}], 'seriousEvents': [{'term': 'Cervical spinal stenosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 24, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders'}, {'term': 'Spinal osteoarthritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 24, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Alpha-1 MP vs. Prolastin® of Area Under the Curve (AUC) From Day 0 to Day 7', 'denoms': [{'units': 'Participants', 'counts': [{'value': '24', 'groupId': 'OG000'}, {'value': '24', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Alpha-1 MP', 'description': 'Sequential, blinded treatment periods of Alpha-1 MP (experimental), then crossed-over to Prolastin (active comparator), followed by open-label Alpha-1 MP'}, {'id': 'OG001', 'title': 'Prolastin', 'description': 'Sequential, blinded treatment periods of Prolastin (active comparator), then crossed-over to Alpha-1 MP (experimental), followed by open-label Alpha-1 MP'}], 'classes': [{'categories': [{'measurements': [{'value': '155.9', 'groupId': 'OG000', 'lowerLimit': '0.97', 'upperLimit': '1.09'}, {'value': '152.4', 'groupId': 'OG001', 'lowerLimit': '0.97', 'upperLimit': '1.09'}]}]}], 'analyses': [{'groupIds': ['OG000', 'OG001'], 'paramType': 'Geometric least square means ratio', 'ciPctValue': '90', 'paramValue': '1.03', 'ciLowerLimit': '0.97', 'ciUpperLimit': '1.09', 'groupDescription': 'To compare AUC 0-7 days between the two treatments (Alpha-1 MP vs. Prolastin),natural log-transformed AUC 0-7 days values were analyzed by analysis of variance (ANOVA).', 'nonInferiorityType': 'NON_INFERIORITY_OR_EQUIVALENCE', 'testedNonInferiority': True, 'nonInferiorityComment': 'A total sample size of 20 subjects could demonstrate comparability of an AUC(0-7days) with 90% power up to a standard deviation of 0.288 of the difference in the log scale, expected mean treatment difference of 0 (= ratio of 1), a lower equivalence limit of -0.223 (= log 0.8), upper limit of 0.223 (= log 1.25) and a one-sided alpha of 0.05 (90% CI).'}], 'paramType': 'NUMBER', 'timeFrame': 'Day 0 to Day 7', 'description': 'The primary objective of this study was to demonstrate the pharmacokinetic comparability (geometric least square mean ratio of AUC between the Alpha-1 MP vs. Prolastin®, 90% confidence interval falls within 0.80-1.25, FDA Guidance as being "bioequivalent" between two treatments) of Alpha-1 MP to Prolastin® in subjects with alpha-1-anti-trypsin (AAT) deficiency by comparing AUC from Day 0 to Day 7 of plasma Alpha1-PI measured by the functional activity (potency) assay. AUC from Day 0 to Day 7 was calculated at steady state at the end of the first and second 8-week treatment periods during the 16-week double-blind, crossover phase.', 'unitOfMeasure': 'mg*h/mL', 'reportingStatus': 'POSTED', 'anticipatedPostingDate': '2009-09'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Alpha-1 MP / Prolastin', 'description': 'Sequential, blinded treatment periods of Alpha-1 modified process (MP) (experimental), then crossed-over to Prolastin (active comparator), followed by open-label Alpha-1 MP'}, {'id': 'FG001', 'title': 'Prolastin / Alpha-1 MP', 'description': 'Sequential, blinded treatment periods of Prolastin (active comparator), then crossed-over to Alpha-1 MP (experimental), followed by open-label Alpha-1 MP'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '12'}, {'groupId': 'FG001', 'numSubjects': '12'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '12'}, {'groupId': 'FG001', 'numSubjects': '12'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}]}]}], 'recruitmentDetails': 'First-subject-first-dose was on 22 May 2006, Last-subject-last-visit was on 28 Feb 2007. The trial was performed at 6 clinical sites in the United States.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'BG000'}, {'value': '12', 'groupId': 'BG001'}, {'value': '24', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'Alpha-1 MP / Prolastin', 'description': 'Sequential, blinded treatment periods of Alpha-1 MP (experimental), then crossed-over to Prolastin (active comparator), followed by open-label Alpha-1 MP'}, {'id': 'BG001', 'title': 'Prolastin / Alpha-1 MP', 'description': 'Sequential, blinded treatment periods of Prolastin (active comparator), then crossed-over to Alpha-1 MP (experimental), followed by open-label Alpha-1 MP'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '10', 'groupId': 'BG000'}, {'value': '10', 'groupId': 'BG001'}, {'value': '20', 'groupId': 'BG002'}]}, {'title': '>=65 years', 'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '4', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '58.4', 'spread': '6.86', 'groupId': 'BG000'}, {'value': '57.0', 'spread': '9.33', 'groupId': 'BG001'}, {'value': '57.7', 'spread': '8.04', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '6', 'groupId': 'BG000'}, {'value': '8', 'groupId': 'BG001'}, {'value': '14', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '6', 'groupId': 'BG000'}, {'value': '4', 'groupId': 'BG001'}, {'value': '10', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '12', 'groupId': 'BG000'}, {'value': '12', 'groupId': 'BG001'}, {'value': '24', 'groupId': 'BG002'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'CROSSOVER'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 24}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2006-05'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2014-08', 'completionDateStruct': {'date': '2007-02', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2014-08-28', 'studyFirstSubmitDate': '2006-02-20', 'resultsFirstSubmitDate': '2009-08-28', 'studyFirstSubmitQcDate': '2006-02-20', 'lastUpdatePostDateStruct': {'date': '2014-09-09', 'type': 'ESTIMATED'}, 'resultsFirstSubmitQcDate': '2014-08-28', 'studyFirstPostDateStruct': {'date': '2006-02-22', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2014-09-09', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2007-02', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Alpha-1 MP vs. Prolastin® of Area Under the Curve (AUC) From Day 0 to Day 7', 'timeFrame': 'Day 0 to Day 7', 'description': 'The primary objective of this study was to demonstrate the pharmacokinetic comparability (geometric least square mean ratio of AUC between the Alpha-1 MP vs. Prolastin®, 90% confidence interval falls within 0.80-1.25, FDA Guidance as being "bioequivalent" between two treatments) of Alpha-1 MP to Prolastin® in subjects with alpha-1-anti-trypsin (AAT) deficiency by comparing AUC from Day 0 to Day 7 of plasma Alpha1-PI measured by the functional activity (potency) assay. AUC from Day 0 to Day 7 was calculated at steady state at the end of the first and second 8-week treatment periods during the 16-week double-blind, crossover phase.'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['alpha 1-Antitrypsin Deficiency', 'alpha 1-Antitrypsin', 'pulmonary emphysema'], 'conditions': ['Alpha 1-Antitrypsin Deficiency']}, 'referencesModule': {'references': [{'pmid': '20920295', 'type': 'DERIVED', 'citation': 'Stocks JM, Brantly ML, Wang-Smith L, Campos MA, Chapman KR, Kueppers F, Sandhaus RA, Strange C, Turino G. Pharmacokinetic comparability of Prolastin(R)-C to Prolastin(R) in alpha(1)-antitrypsin deficiency: a randomized study. BMC Clin Pharmacol. 2010 Sep 30;10:13. doi: 10.1186/1472-6904-10-13.'}], 'seeAlsoLinks': [{'url': 'http://www.alphaone.org', 'label': 'The "Alpha-1 Foundation", dedicated to providing the leadership and resources that will result in increased research, improved health, worldwide detection and a cure for Alpha-1 Antitrypsin Deficiency'}, {'url': 'http://www.alphanet.org', 'label': 'AlphaNet, Inc devoted to improving the lives of individuals with Alpha-1 antitrypsin deficiency through comprehensive disease management services, clinical research administration, and consultative services'}]}, 'descriptionModule': {'briefSummary': 'The purpose of this clinical study (ChAMP - Comparability pharmacokinetics of Alpha-1 Modified Process) is to compare the pharmacokinetic, safety and tolerability of Alpha-1 Proteinase Inhibitor (Human), modified process (Alpha-1 MP) and Prolastin in adult Alpha1-antitrypsin deficient patients. Patients will be infused intravenously with study drug on a weekly schedule for 24 weeks.', 'detailedDescription': 'The objective of this study is to demonstrate the pharmacokinetic comparability of Alpha-1 MP to Prolastin® in subjects with Alpha1-antitrypsin deficiency.\n\nThis study is divided into three 8-week treatment sequences including an initial 8-week double-blind treatment period (with one of the 2 study drugs), a second 8-week double-blind treatment period (with the other study drug), and a third 8-week open-label treatment period (with Alpha-1 MP).'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Documented diagnosis of congenital Alpha1-antitrypsin deficiency\n* Must be receiving augmentation therapy with plasma-derived (human) Alpha1-Proteinase Inhibitor (Prolastin®) for at least one month prior to study entry.\n* Signed written informed consent prior to initiation of any study related procedures\n\nExclusion Criteria:\n\n* Females who are pregnant, breast feeding, or if of child-bearing potential, unwilling to practice adequate contraception throughout the study\n* Use of systemic steroids within the 2 weeks prior to receiving study treatment (this does not include the use of inhaled steroids used on a routine or as needed basis).\n* Subjects who have had exacerbations of their disease within one month of trial entry.'}, 'identificationModule': {'nctId': 'NCT00295061', 'acronym': 'ChAMP', 'briefTitle': 'Comparison of Pharmacokinetic, Safety, Tolerability of Alpha-1 MP and Prolastin In Alpha1-antitrypsin Deficient Adults', 'organization': {'class': 'INDUSTRY', 'fullName': 'Grifols Therapeutics LLC'}, 'officialTitle': 'Multi-center, Randomized, Double-blind, Crossover Trial to Evaluate the Pharmacokinetic Comparability of Alpha-1 MP to Prolastin in Subjects With Alpha1-antitrypsin Deficiency.', 'orgStudyIdInfo': {'id': '11816'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': '1 Alpha-1 MP', 'description': 'Sequential, blinded treatment periods of Alpha-1 MP (experimental), then crossed-over to Prolastin (active comparator), followed by open-label Alpha-1 MP', 'interventionNames': ['Drug: Alpha-1 MP']}, {'type': 'ACTIVE_COMPARATOR', 'label': '2 Prolastin', 'description': 'Sequential, blinded treatment periods of Prolastin (active comparator), then crossed-over to Alpha-1 MP (experimental), followed by open-label Alpha-1 MP', 'interventionNames': ['Drug: alpha-1 proteinase inhibitor (human)']}], 'interventions': [{'name': 'Alpha-1 MP', 'type': 'DRUG', 'otherNames': ['Alpha-1 antitrypsin (AAT)', 'TAL6004'], 'description': 'alpha-1 proteinase inhibitor (human), 60 mg/kg body weight', 'armGroupLabels': ['1 Alpha-1 MP']}, {'name': 'alpha-1 proteinase inhibitor (human)', 'type': 'DRUG', 'otherNames': ['Alpha-1 antitrypsin (AAT)', 'BAY x 5747', 'BAY 10-5233', 'TAL-05-00007'], 'description': 'Prolastin', 'armGroupLabels': ['2 Prolastin']}]}, 'contactsLocationsModule': {'locations': [{'zip': '80206', 'city': 'Denver', 'state': 'Colorado', 'country': 'United States', 'facility': 'National Jewish Medical and Research Center', 'geoPoint': {'lat': 39.73915, 'lon': -104.9847}}, {'zip': '32610-0225', 'city': 'Gainesville', 'state': 'Florida', 'country': 'United States', 'facility': 'University of Florida College of Medicine', 'geoPoint': {'lat': 29.65163, 'lon': -82.32483}}, {'zip': '33101', 'city': 'Miami', 'state': 'Florida', 'country': 'United States', 'facility': 'University of Miami School of Medicine', 'geoPoint': {'lat': 25.77427, 'lon': -80.19366}}, {'zip': '10019', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'St Lukes-Roosevelt Hospital Center, New York', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '44122', 'city': 'Cleveland', 'state': 'Ohio', 'country': 'United States', 'facility': 'Cleveland Clinic Foundation', 'geoPoint': {'lat': 41.4995, 'lon': -81.69541}}, {'zip': '19140', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'Temple University Hospital', 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '29425', 'city': 'Charleston', 'state': 'South Carolina', 'country': 'United States', 'facility': 'Medical University of South Carolina', 'geoPoint': {'lat': 32.77632, 'lon': -79.93275}}, {'zip': '75708-3154', 'city': 'Tyler', 'state': 'Texas', 'country': 'United States', 'facility': 'University of Texas Health Center at Tyler', 'geoPoint': {'lat': 32.35126, 'lon': -95.30106}}], 'overallOfficials': [{'name': 'Kim Hanna, MSc', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Grifols Therapeutics LLC'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Grifols Therapeutics LLC', 'class': 'INDUSTRY'}, 'responsibleParty': {'oldNameTitle': 'Gerald Klein, MD, Chief Medical Officer, Vice President of Medical and Clinical Affairs', 'oldOrganization': 'Talecris Biotherapeutics, Inc.'}}}}