Ignite Creation Date:
2025-12-24 @ 3:17 PM
Ignite Modification Date:
2025-12-26 @ 12:54 PM
Study NCT ID:
NCT01661192
Status:
COMPLETED
Last Update Posted:
2017-01-11
First Post:
2012-08-07
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Long Term Treatment Effect of the Safety, Tolerability and Efficacy of AAT in Type 1 Diabetes
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D003922', 'term': 'Diabetes Mellitus, Type 1'}], 'ancestors': [{'id': 'D003920', 'term': 'Diabetes Mellitus'}, {'id': 'D044882', 'term': 'Glucose Metabolism Disorders'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D004700', 'term': 'Endocrine System Diseases'}, {'id': 'D001327', 'term': 'Autoimmune Diseases'}, {'id': 'D007154', 'term': 'Immune System Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C037741', 'term': 'alpha 1-antitrypsin-leukocyte elastase complex'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'PREVENTION', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 12}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2013-01'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2017-01', 'completionDateStruct': {'date': '2017-01', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2017-01-10', 'studyFirstSubmitDate': '2012-08-07', 'studyFirstSubmitQcDate': '2012-08-07', 'lastUpdatePostDateStruct': {'date': '2017-01-11', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2012-08-09', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2017-01', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Safety and tolerability of AAT in terms of adverse events and serious adverse events', 'timeFrame': 'At month 36', 'description': 'We will assess at each visit until final visit (month 36)the safety and tolerability of study drug in terms of adverse events and serious adverse events'}, {'measure': 'Safety and tolerability of the AAT in terms of laboratory values', 'timeFrame': 'At month 36', 'description': 'We will assess at each visit until final visit (month 36)the safety and tolerability of study drug in terms of laboratory values'}], 'secondaryOutcomes': [{'measure': 'Beta cell function-AUC (Area Under the Curve) of stimulated C-Peptide from stimulated MMTT (mixed meal tolerance test)', 'timeFrame': 'at month 36'}, {'measure': 'Percentage of patients that maintain stimulated peak C-peptide >=0.2 nmol/L', 'timeFrame': 'at month 36'}, {'measure': 'Percentage of patients that achieve glycemic target of HbA1c <=7.5%', 'timeFrame': 'At month 36'}, {'measure': 'Daily insulin dose adjusted to body weight', 'timeFrame': 'At month 36'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['Type 1 Diabetes', 'newly diagnosed', 'Beta cell preservation'], 'conditions': ['Type 1 Diabetes', 'Beta Cell Preservation']}, 'referencesModule': {'references': [{'pmid': '30236025', 'type': 'DERIVED', 'citation': 'Brener A, Lebenthal Y, Interator H, Horesh O, Leshem A, Weintrob N, Loewenthal N, Shalitin S, Rachmiel M. Long-term safety of alpha-1 antitrypsin therapy in children and adolescents with Type 1 diabetes. Immunotherapy. 2018 Sep;10(13):1137-1148. doi: 10.2217/imt-2018-0047.'}]}, 'descriptionModule': {'briefSummary': 'At a previous study the investigators have assessed the safety and efficacy of treatment with AAT(Alpha 1 Antitrypsin)in newly diagnosed type 1 diabetes subjects aiming at beta cells preservation .\n\nSince treatment with AAT is expected to be a chronic treatment; stopping treatment will probably result in eventual loss of the preserved beta-cell function. Indeed, other investigational drugs aiming at beta cells preservation have shown that patients who were initially treated and maintained their initial beta-cell function, required continuation of treatment or they lost the beta-cell function.\n\nTherefore, in this extension study, patients who were previously treated with AAT and maintained clinically significant beta-cell function are offered a continuation of treatment, since they are likely to benefit from use of the medication.\n\nThe proposed study is aimed to assess the long term effect of AAT in subjects with type 1 diabetes mellitus: safety and tolerability of treatment, and effect on beta-cell function.\n\nSubjects who have completed all visits of the 008 study will be offered to participate in the extension study.\n\nThe study will be consist off two main arms as following:\n\nArm 1: Subjects who maintained peak stimulated C-peptide secretion ≥ 0.2 nmol/L will continue treatment with AAT for up to 18 treatments according to the dosage group they were allocated to in the 008 study.\n\nArm 2:\n\nSubjects who have not maintained peak stimulated C-peptide secretion ≥ 0.2nmol/L and subjects with peak stimulated C -peptide secretion ≥ 0.2 nmol/L who are reluctant to receive additional study drug.\n\nClinical follow up for all subjects in both arms will be for 3 years'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '25 Years', 'minimumAge': '10 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Subject (or parent/guardian) willing and able to sign an informed consent\n* Ability to comply with all study requirements.\n* A patient that participated in Study 008 and received all doses of study medication, per protocol.\n* Evidence of clinically significant residual beta-cell function demonstrated by MMTT peak stimulated C-peptide concentrations ≥ 0.20 nmol/L (Arm 1 only).\n* Age 10-25 (inclusive) years\n* If a female is of childbearing potential, the subject is not pregnant or lactating, and will use oral hormonal contraception or other equally effective contraceptive methods throughout the study.\n\nExclusion Criteria:\n\n* IgA (immunoglobulin A ) deficient subjects.\n* Individuals with a history of severe immediate hypersensitivity reactions, including anaphylaxis, to plasma products.\n* History of life threatening allergy, anaphylactic reaction, or systemic response to human plasma derived products.\n* The subject is receiving immunosuppressive or immunomodulating agents or cytotoxic therapy or any medication that in the opinion of the Investigator might interfere with the study.\n* Clinically significant intercurrent illnesses, including (but not limited to): cardiac, hepatic, renal, neurological, hematological, neoplastic, immunological, skeletal or other) that in the opinion of the investigator, could interfere with the safety, compliance or other aspects of this study. Patients with well-controlled, chronic diseases could be possibly included after consultation with the treating physician.'}, 'identificationModule': {'nctId': 'NCT01661192', 'acronym': 'AAT Extension', 'briefTitle': 'Long Term Treatment Effect of the Safety, Tolerability and Efficacy of AAT in Type 1 Diabetes', 'organization': {'class': 'OTHER', 'fullName': 'Rabin Medical Center'}, 'officialTitle': 'Open Label Study (Extension 001)to Evaluate Long Term Treatment Effect of the Safety, Tolerability and Efficacy of Intervenous ALPHA-1 ANTITRYSIN (AAT)Glasia™ in Type 1 Diabetes Mellitus (Extension to KAMADA AAt 008, PHASE I/II Study)', 'orgStudyIdInfo': {'id': '006971ctil'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'AAT( Alpha 1 Antitrypsin)', 'description': 'Subjects who maintained peak stimulated C-peptide secretion ≥ 0.2nmol/L will continue treatment with AAT according to the dosage group they were allocated to at the previous study(40mg/kg or 60mg/kg or 80mg/kg), intravenously, once a week for 6 consecutive weeks, at 24-week intervals for a duration of \\~54 weeks.', 'interventionNames': ['Drug: AAT( Alpha 1 Antitrypsin)']}, {'type': 'NO_INTERVENTION', 'label': 'Follow up group', 'description': 'Subjects who have not maintained peak stimulated C-peptide secretion ≥ 0.2nmol/L or subjects with peak stimulated C -peptide secretion ≥ 0.2nmol/L who are reluctant to receive additional study drug, will be followed up only with no administration of investigational product'}], 'interventions': [{'name': 'AAT( Alpha 1 Antitrypsin)', 'type': 'DRUG', 'armGroupLabels': ['AAT( Alpha 1 Antitrypsin)']}]}, 'contactsLocationsModule': {'locations': [{'zip': '49202', 'city': 'Petah Tikva', 'country': 'Israel', 'facility': "Schneider Children's Medical Center", 'geoPoint': {'lat': 32.08707, 'lon': 34.88747}}, {'city': 'Ẕerifin', 'country': 'Israel', 'facility': 'Assaf Haroffeh Medical Center', 'geoPoint': {'lat': 31.95731, 'lon': 34.84852}}], 'overallOfficials': [{'name': 'Yael Lebenthal, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Rabin Medical Center'}, {'name': 'Mariana Rachmiel, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Assaf Haroffe Medical Center'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Rabin Medical Center', 'class': 'OTHER'}, 'collaborators': [{'name': 'Kamada, Ltd.', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'SPONSOR'}}}}