Ignite Creation Date:
2025-12-25 @ 2:47 AM
Ignite Modification Date:
2025-12-30 @ 1:11 PM
Study NCT ID:
NCT06405633
Status:
RECRUITING
Last Update Posted:
2025-10-07
First Post:
2024-05-03
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Phase 1b/2a, Open-label Single Ascending Doses and Multiple Ascending Doses Study in Participants With Pi*ZZ AATD
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'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SEQUENTIAL'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 24}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2024-07-19', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-10', 'completionDateStruct': {'date': '2026-09', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-10-01', 'studyFirstSubmitDate': '2024-05-03', 'studyFirstSubmitQcDate': '2024-05-03', 'lastUpdatePostDateStruct': {'date': '2025-10-07', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2024-05-08', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2026-09', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'The proportion of participants with adverse events', 'timeFrame': 'Up to 36 Weeks'}], 'secondaryOutcomes': [{'measure': 'Single Ascending Dose - Change from baseline in levels of serum M-AAT protein.', 'timeFrame': 'Up to 12 Weeks'}, {'measure': 'Multiple Ascending Dose - Change from baseline in levels of serum M-AAT protein', 'timeFrame': 'Up to 24 Weeks'}, {'measure': 'Single Ascending Dose - Area under the plasma concentration time curve for WVE-006 from time of dosing to the last measurable concentration (AUClast)', 'timeFrame': 'Up to 12 Weeks'}, {'measure': 'Single Ascending Dose - Maximum concentration of WVE-006 in plasma', 'timeFrame': 'Up to 12 Weeks'}, {'measure': 'Multiple Ascending Doses - Area under the plasma concentration time curve for WVE-006 from time of dosing to the last measurable concentration (AUClast)', 'timeFrame': 'Up to 24 Weeks'}, {'measure': 'Multiple Ascending Doses - Maximum concentration of WVE-006 in plasma (Cmax)', 'timeFrame': 'Up to 24 Weeks'}]}, 'oversightModule': {'isUsExport': True, 'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Alpha-1 Antitrypsin Deficiency']}, 'descriptionModule': {'briefSummary': 'The purpose of this open-label study is to assess the safety, tolerability, pharmacodynamics, and pharmacokinetics of WVE-006 in participants with alpha-1 antitrypsin deficiency (AATD) following Period 1 single ascending dose (SAD) and Period 2 multiple ascending doses (MAD), respectively.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '70 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Healthy as determined by the Investigator, based on a medical evaluation, or has mild to moderate AATD-induced lung disease (FEV1 ≥50%) and/or stable mild AATD-induced liver disease (≤F2 (≤10 kPa) on FibroScan.\n* Genetic testing confirming Pi\\*ZZ.\n* Participant has been a non-smoker for at least 1 year prior to screening.\n\nExclusion Criteria:\n\n* • Participant has a history of multiple drug allergies or of allergic reaction to an oligonucleotide or to N-acetylgalactosamine (GalNAc).\n\n * Participant has a history of intolerance or any medical condition that might interfere with subcutaneous injections.\n * Any ongoing or recent infections.\n * Any recent or planned vaccinations during the study.\n * Participant has a history of regular alcohol consumption exceeding 14 standard drinks/week.\n * Unwilling to abstain from alcohol for 48 hours prior to dosing at each of the dosing visits.\n * Any recent or planned major surgery during the study.\n * Participant has any medical condition or social circumstance that, in the opinion of the Investigator, would make the participant unsuitable for participation in the study or for dosing on Day 1, or could interfere with the assessments of safety, pharmacodynamics, or pharmacokinetics, or completion of the study.\n * Participant currently on AAT augmentation therapy, planned to be on augmentation therapy anytime during the study, or has been on augmentation therapy within 30 days prior to Screening Visit.\n * Donation of blood or blood products in excess of 500 mL within 12 weeks prior to Screening Visit and/or unwilling to refrain from blood donation for the duration of the study.\n * Participant has received an investigational agent within 3 months of the Screening Visit.'}, 'identificationModule': {'nctId': 'NCT06405633', 'briefTitle': 'A Phase 1b/2a, Open-label Single Ascending Doses and Multiple Ascending Doses Study in Participants With Pi*ZZ AATD', 'organization': {'class': 'INDUSTRY', 'fullName': 'Wave Life Sciences Ltd.'}, 'officialTitle': 'A Phase 1b/2a Open-label Single Ascending Doses (SAD) and Multiple Ascending Doses (MAD) Research Study to Evaluate Safety, Tolerability, Pharmacokinetics and Pharmacodynamics in Participants With AATD Pi*ZZ on WVE-006 (RestorAATion-2)', 'orgStudyIdInfo': {'id': 'WVE-006-002'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'WVE-006 (Dose 1)', 'interventionNames': ['Drug: WVE-006']}, {'type': 'EXPERIMENTAL', 'label': 'WVE-006 (Dose 2)', 'interventionNames': ['Drug: WVE-006']}, {'type': 'EXPERIMENTAL', 'label': 'WVE-006 (Dose 3)', 'interventionNames': ['Drug: WVE-006']}], 'interventions': [{'name': 'WVE-006', 'type': 'DRUG', 'description': 'RNA editing oligonucleotide', 'armGroupLabels': ['WVE-006 (Dose 1)', 'WVE-006 (Dose 2)', 'WVE-006 (Dose 3)']}]}, 'contactsLocationsModule': {'locations': [{'zip': '3065', 'city': 'Fitzroy', 'state': 'Victoria', 'status': 'RECRUITING', 'country': 'Australia', 'contacts': [{'name': 'Sue Brenton', 'role': 'CONTACT', 'email': 'Sue.brenton@svha.org.au'}], 'facility': "St. Vincent's Hospital, Melbourne", 'geoPoint': {'lat': -37.79839, 'lon': 144.97833}}, {'zip': 'B3H 1V7', 'city': 'Halifax', 'state': 'Nova Scotia', 'status': 'RECRUITING', 'country': 'Canada', 'contacts': [{'name': 'Scott Fulton', 'role': 'CONTACT', 'email': 'Scott.Fulton@nshealth.ca'}], 'facility': 'Dalhousie University - Queen Elizabeth II Health Sciences Centre', 'geoPoint': {'lat': 44.64269, 'lon': -63.57688}}, {'zip': 'M5T 3A9', 'city': 'Toronto', 'state': 'Ontario', 'status': 'RECRUITING', 'country': 'Canada', 'contacts': [{'name': 'Kayla Cardoso', 'role': 'CONTACT', 'email': 'kcardoso@inspirationresearch.ca'}, {'name': 'Dhwani Patel', 'role': 'CONTACT', 'email': 'dpatel@inspirationresearch.ca'}], 'facility': 'Inspiration Research Limited', 'geoPoint': {'lat': 43.70643, 'lon': -79.39864}}, {'zip': '20520', 'city': 'Turku', 'status': 'RECRUITING', 'country': 'Finland', 'contacts': [{'name': 'Terhi Yla-Outinen', 'role': 'CONTACT', 'email': 'Terhi.Yla-Outinen@varha.fi'}], 'facility': 'Turku University Hospital', 'geoPoint': {'lat': 60.45148, 'lon': 22.26869}}, {'zip': '52074', 'city': 'Aachen', 'status': 'RECRUITING', 'country': 'Germany', 'contacts': [{'name': 'Sarah Henke', 'role': 'CONTACT', 'email': 'Shenke@ukaachen.de'}], 'facility': 'Universitaetsklinikum Aachen, AoeR', 'geoPoint': {'lat': 50.77664, 'lon': 6.08342}}, {'zip': '3204', 'city': 'Hamilton', 'status': 'RECRUITING', 'country': 'New Zealand', 'contacts': [{'name': 'Chris Tuffery', 'role': 'CONTACT', 'email': 'Christine.Tuffery@waikatodhb.health.nz'}], 'facility': 'Waikato Hospital', 'geoPoint': {'lat': -37.78333, 'lon': 175.28333}}, {'zip': 'B15 2GW', 'city': 'Birmingham', 'status': 'RECRUITING', 'country': 'United Kingdom', 'contacts': [{'name': 'Emma Bruce', 'role': 'CONTACT', 'email': 'Emma.Bruce@uhb.nhs.uk'}], 'facility': 'Queen Elizabeth Hospital, University Hospital Birmingham', 'geoPoint': {'lat': 52.48142, 'lon': -1.89983}}, {'zip': 'CB2 0QQ', 'city': 'Cambridge', 'status': 'RECRUITING', 'country': 'United Kingdom', 'contacts': [{'name': 'Abi Ford', 'role': 'CONTACT', 'email': 'abigail.ford4@nhs.net'}], 'facility': "Cambridge University - Addenbrooke's Hospital", 'geoPoint': {'lat': 52.2, 'lon': 0.11667}}, {'zip': 'SW8 3RN', 'city': 'London', 'status': 'RECRUITING', 'country': 'United Kingdom', 'contacts': [{'name': 'Cristina Ma', 'role': 'CONTACT', 'email': 'macristina.levy@nhs.net'}], 'facility': 'Royal Free London NHS Foundation Trust', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}], 'centralContacts': [{'name': 'Clinical Operations', 'role': 'CONTACT', 'email': 'Clinicaltrials@wavelifesci.com', 'phone': '855-215-4687'}], 'overallOfficials': [{'name': 'Cynthia Caracta, MD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Wave Life Sciences'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Wave Life Sciences Ltd.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}