Ignite Creation Date:
2025-12-24 @ 3:42 PM
Ignite Modification Date:
2026-01-04 @ 7:14 PM
Study NCT ID:
NCT06186492
Status:
COMPLETED
Last Update Posted:
2025-04-29
First Post:
2023-11-09
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Phase 1 Research Study to Evaluate Safety, Tolerability, and Pharmacokinetics of WVE-006 in Healthy Participants With Wild-type AAT Expression (RestorAATion-1)
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'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'DOUBLE', 'whoMasked': ['PARTICIPANT', 'INVESTIGATOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SEQUENTIAL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 47}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2023-11-14', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-04', 'completionDateStruct': {'date': '2025-02-13', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-04-24', 'studyFirstSubmitDate': '2023-11-09', 'studyFirstSubmitQcDate': '2023-12-15', 'lastUpdatePostDateStruct': {'date': '2025-04-29', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2024-01-02', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2025-02-13', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'The proportion of participants with adverse events', 'timeFrame': 'Day 1 through Day 85 after last dose.'}], 'secondaryOutcomes': [{'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': 'Day 0 through Day 84 postdose'}, {'measure': 'Single Ascending Dose - Maximum concentration of WVE-006 in plasma (Cmax)', 'timeFrame': 'Day 0 through Day 84 postdose'}, {'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': 'Day 0-14 after the first and Day 0-84 days after the last dose, respectively'}, {'measure': 'Multiple Ascending Doses - Maximum concentration of WVE-006 in plasma (Cmax)', 'timeFrame': 'Day 0-14 after the first and Day 0-84 days after the last dose, respectively'}]}, 'oversightModule': {'isUsExport': True, 'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Alpha-1 Antitrypsin Deficiency']}, 'descriptionModule': {'briefSummary': 'This study is the first study in the RestorAATion clinical program.\n\nThe purpose of this first-in human (FIH), double-blind, randomized, placebo-controlled, single ascending dose (SAD) and multiple-dose Phase 1 study is to assess the safety, tolerability, and PK of WVE-006 compared to placebo in healthy participants following a single dose (Period 1) and multiple doses (Period 2) of WVE-006.\n\nThis information will be used to determine doses and regimes that have the potential to be pharmacologically active in patients with Alpha-1 antitrypsin deficiency in the RestorAATion 2 study, and the maximum safe and tolerable dose that may be given to these patients.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '65 Years', 'minimumAge': '18 Years', 'healthyVolunteers': True, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Healthy as determined by the Investigator, based on a medical evaluation.\n* Genetic testing confirming PI\\*MM.\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* 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* Participant has a history of caffeine consumption exceeding 8 cups of coffee/day.\n* Use of prescription or non-prescription medications, including vitamin, dietary, and herbal supplements (including St John's Wort) within 7 days prior to the first dose of study treatment unless, in the opinion of the Investigator and Sponsor, the medication will not interfere with interpretation of study assessments. Contraception and hormone replacement therapy (HRT) are permitted. If needed, over-the-counter (OTC) medications such as paracetamol/acetaminophen may be used acutely.\n* Any recent or planned major surgery during the study.\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': 'NCT06186492', 'acronym': 'RestorAATion-1', 'briefTitle': 'A Phase 1 Research Study to Evaluate Safety, Tolerability, and Pharmacokinetics of WVE-006 in Healthy Participants With Wild-type AAT Expression (RestorAATion-1)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Wave Life Sciences Ltd.'}, 'officialTitle': 'A Phase 1, Randomized, Double-blind, Placebo-controlled, Safety, Tolerability, and Pharmacokinetic Study of Single Ascending Doses and Multiple Doses of WVE-006 in Healthy Participants', 'orgStudyIdInfo': {'id': 'WVE-006-001'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Experimental WVE-006 (Dose A) or placebo', 'interventionNames': ['Drug: WVE-006']}, {'type': 'EXPERIMENTAL', 'label': 'Experimental WVE-006 (Dose B) or placebo', 'interventionNames': ['Drug: WVE-006']}, {'type': 'EXPERIMENTAL', 'label': 'Experimental WVE-006 (Dose C) or placebo', 'interventionNames': ['Drug: WVE-006']}, {'type': 'EXPERIMENTAL', 'label': 'Experimental WVE-006 (Dose D) or placebo', 'interventionNames': ['Drug: WVE-006']}, {'type': 'EXPERIMENTAL', 'label': 'Experimental WVE-006 (Dose E) or placebo', 'interventionNames': ['Drug: WVE-006']}, {'type': 'EXPERIMENTAL', 'label': 'Experimental WVE-006 (Dose F) or placebo', 'interventionNames': ['Drug: WVE-006']}, {'type': 'EXPERIMENTAL', 'label': 'Experimental WVE-006 (Dose G) or placebo', 'interventionNames': ['Drug: WVE-006']}], 'interventions': [{'name': 'WVE-006', 'type': 'DRUG', 'description': 'RNA editing oligonucleotide', 'armGroupLabels': ['Experimental WVE-006 (Dose A) or placebo', 'Experimental WVE-006 (Dose B) or placebo', 'Experimental WVE-006 (Dose C) or placebo', 'Experimental WVE-006 (Dose D) or placebo', 'Experimental WVE-006 (Dose E) or placebo', 'Experimental WVE-006 (Dose F) or placebo', 'Experimental WVE-006 (Dose G) or placebo']}]}, 'contactsLocationsModule': {'locations': [{'zip': 'CF48 4DR', 'city': 'Merthyr Tydfil', 'state': 'Wales', 'country': 'United Kingdom', 'facility': 'Simbec-Orion Clinical Pharmacology,', 'geoPoint': {'lat': 51.74794, 'lon': -3.37779}}], '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'}}}}