Ignite Creation Date:
2025-12-24 @ 10:02 PM
Ignite Modification Date:
2025-12-25 @ 7:39 PM
Study NCT ID:
NCT06237335
Status:
RECRUITING
Last Update Posted:
2025-11-17
First Post:
2024-01-23
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
A Phase 2 Study Evaluating Safety and Tolerability of RCT2100 (CFTR mRNA) in Healthy Participants and in Participants With CF
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D003550', 'term': 'Cystic Fibrosis'}], 'ancestors': [{'id': 'D010182', 'term': 'Pancreatic 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': 'D007232', 'term': 'Infant, Newborn, Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C545203', 'term': 'ivacaftor'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'DOUBLE', 'whoMasked': ['PARTICIPANT', 'INVESTIGATOR'], 'maskingDescription': 'Participant and investigator masking only applies to Part 1 which is randomized. For Part 2, there is no masking, and this part is Open Label.'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 192}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2024-02-01', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-11', 'completionDateStruct': {'date': '2026-12-31', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-11-13', 'studyFirstSubmitDate': '2024-01-23', 'studyFirstSubmitQcDate': '2024-01-23', 'lastUpdatePostDateStruct': {'date': '2025-11-17', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2024-02-01', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2026-08-30', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Part 1: The number of participants with Adverse Events (AEs) and Serious Adverse Events (SAEs).', 'timeFrame': 'From Baseline Through Day 29', 'description': 'Safety and tolerability as assessed by number of participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)'}, {'measure': 'Part 2: The number of participants with CF with AEs and SAEs.', 'timeFrame': 'From Day 1 through Safety Follow-up, Week 24', 'description': 'Safety and tolerability of multiple-ascending doses of inhaled RCT2100 administered to participants with CF'}, {'measure': 'Part 3: The number of participants with CF with AEs and SAEs.', 'timeFrame': 'From Day 1 through Safety Follow-up, Week 24', 'description': 'To assess the safety and tolerability of RCT2100 co-administered with ivacaftor in participants with CF.'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Cystic Fibrosis', 'CF', 'mRNA'], 'conditions': ['Cystic Fibrosis']}, 'descriptionModule': {'briefSummary': 'This is the first-in-human study with RCT2100 and is designed to provide safety and tolerability data for future clinical studies.', 'detailedDescription': 'This is a multi-part study to assess the safety, tolerability, and biodistribution of a single ascending dose of inhaled RCT2100 administered via nebulizer to healthy participants (Part 1), the safety and tolerability of multiple-ascending doses of inhaled RCT2100 administered to participants with CF (Part 2), and the safety and tolerability of RCT2100 co-administered with ivacaftor in participants with CF (Part 3).'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT'], 'maximumAge': '60 Years', 'minimumAge': '18 Years', 'healthyVolunteers': True, 'eligibilityCriteria': 'Part 1 Major Inclusion Criteria:\n\n* Healthy, adult, male or female, 18-55 years of age, inclusive, at screening.\n* Body weight greater than or equal to 50 kg and body mass index (BMI) between 16-32 kg/m2, inclusive\n* The participant has a forced expiratory volume in one second (FEV1) of at least 80% predicted\n* The participant is considered by the investigator to be in good general health as determined by medical history, clinical laboratory test results, vital sign measurements, 12-lead ECG results, and physical examination findings at screening.\n* Understands the study procedures in the informed consent form (ICF), and is willing and able to comply with the protocol.\n\nPart 1 Major Exclusion Criteria:\n\n* History or presence of clinically significant medical, surgical, clinical laboratory, or psychiatric condition or disease.\n* The participant has supine blood pressure (BP) \\>150 mm Hg (systolic) or \\>90 mm Hg (diastolic), following at least 5 minutes of supine rest.\n* The participant has abnormal clinical laboratory tests at screening, as assessed by the study-specific laboratory.\n* The participant is a smoker or has used nicotine or nicotine-containing products 6 weeks before the first dose of study drug. Former smokers with greater than 10 pack years of smoking history are excluded.\n\nPart 2 Major Inclusion Criteria:\n\n* Confirmed diagnosis of CF\n* Forced expiratory volume in 1 second ≥50% and ≤100% of predicted mean value for age, sex, and height\n* a) Not eligible for CFTR modulators based on having mutations of CFTR gene on both alleles that are not responsive to CFTR modulator therapy OR\n* b) Eligible for CFTR modulators (based on local prescribing information) but not using CFTR modulators due to intolerance or contraindications\n\nPart 2 Major Exclusion Criteria:\n\n* Hepatic cirrhosis with portal hypertension, moderate hepatic impairment (Child Pugh Score 7 to 9), or severe hepatic impairment (Child Pugh Score 10 to 15)\n* An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for sinopulmonary disease within 4 weeks before the first dose of study drug\n* Lung infection with organisms associated with a more rapid decline in pulmonary status\n* Arterial oxygen saturation on room air less than 94% at screening\n* Treatment with a CFTR modulator (Kalydeco, Trikafta, Symdeko, Orkambi, or Alyftrek) within 12 weeks of Screening\n\nOther protocol defined Inclusion/Exclusion criteria may apply.\n\nPart 3 Major Exclusion Criteria:\n\nConfirmed diagnosis of CF\n\n* Forced expiratory volume in 1 second ≥50% and ≤100% of predicted mean value for age, sex, and height\n* a) Not eligible for CFTR modulators based on having mutations of CFTR gene on both alleles that are not responsive to CFTR modulator therapy OR\n* b) Eligible for dual or triple CFTR modulators (based on local prescribing information) but not using CFTR modulators due to intolerance or contraindications\n\nPart 3 Major Exclusion Criteria:\n\n* Hepatic cirrhosis with portal hypertension, moderate hepatic impairment (Child Pugh Score 7 to 9), or severe hepatic impairment (Child Pugh Score 10 to 15)\n* An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for sinopulmonary disease within 4 weeks before the first dose of study drug\n* Lung infection with organisms associated with a more rapid decline in pulmonary status\n* Arterial oxygen saturation on room air less than 94% at screening\n* Treatment with a CFTR modulator (Kalydeco, Trikafta, Symdeko, Orkambi, or Alyftrek) within 12 weeks of Screening\n\nOther protocol defined Inclusion/Exclusion criteria may apply.'}, 'identificationModule': {'nctId': 'NCT06237335', 'briefTitle': 'A Phase 2 Study Evaluating Safety and Tolerability of RCT2100 (CFTR mRNA) in Healthy Participants and in Participants With CF', 'organization': {'class': 'INDUSTRY', 'fullName': 'ReCode Therapeutics'}, 'officialTitle': 'A Phase 1/2, Multicenter Study Evaluating the Safety, Tolerability, and Biodistribution of RCT2100 With Single-Ascending Doses in Healthy Participants and Multiple-Ascending Doses and Proof-of-Concept in Participants With Cystic Fibrosis', 'orgStudyIdInfo': {'id': 'RCT2100-101'}, 'secondaryIdInfos': [{'id': '2024-512169-15', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'RCT2100 (Part 1)', 'description': 'RCT2100 single dose', 'interventionNames': ['Drug: RCT2100']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo (Part 1)', 'description': 'Placebo single dose', 'interventionNames': ['Other: Placebo']}, {'type': 'EXPERIMENTAL', 'label': 'RCT2100 (Part 2) 4 week', 'description': 'RCT2100 multiple dose', 'interventionNames': ['Drug: RCT2100']}, {'type': 'EXPERIMENTAL', 'label': 'RCT2100 (Part 2) 12 week', 'description': 'RCT2100 multiple dose', 'interventionNames': ['Drug: RCT2100']}, {'type': 'EXPERIMENTAL', 'label': 'Experimental: RCT2100 (Part 3) 6 week', 'description': 'RCT2100 multiple dose', 'interventionNames': ['Drug: Ivacaftor', 'Drug: RCT2100']}], 'interventions': [{'name': 'RCT2100', 'type': 'DRUG', 'description': 'RCT2100 supplied as varying dose strengths administered via oral inhalation using nebulizer', 'armGroupLabels': ['RCT2100 (Part 1)']}, {'name': 'Placebo', 'type': 'OTHER', 'description': 'Placebo of similar volumes to experimental dose strengths administered via oral inhalation using nebulizer', 'armGroupLabels': ['Placebo (Part 1)']}, {'name': 'RCT2100', 'type': 'DRUG', 'description': 'RCT2100 supplied as varying dose strengths administered via oral inhalation using nebulizer for 4 weeks', 'armGroupLabels': ['RCT2100 (Part 2) 4 week']}, {'name': 'RCT2100', 'type': 'DRUG', 'description': 'RCT2100 supplied at a single dose strength administered via oral inhalation using nebulizer for 12 weeks', 'armGroupLabels': ['RCT2100 (Part 2) 12 week']}, {'name': 'Ivacaftor', 'type': 'DRUG', 'description': 'ivacaftor administered orally for 6 weeks', 'armGroupLabels': ['Experimental: RCT2100 (Part 3) 6 week']}, {'name': 'RCT2100', 'type': 'DRUG', 'description': 'RCT2100 supplied at varying dose strengths. Co- administered via oral inhalation using nebulizer for 4 weeks with ivacaftor after initial 2 weeks of ivacaftor dosing run in period', 'armGroupLabels': ['Experimental: RCT2100 (Part 3) 6 week']}]}, 'contactsLocationsModule': {'locations': [{'zip': '35294', 'city': 'Birmingham', 'state': 'Alabama', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'The University of Alabama at Birmingham', 'geoPoint': {'lat': 33.52066, 'lon': -86.80249}}, {'zip': '85719', 'city': 'Tucson', 'state': 'Arizona', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'University of Arizona', 'geoPoint': {'lat': 32.22174, 'lon': -110.92648}}, {'zip': '94304', 'city': 'Palo Alto', 'state': 'California', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'Stanford University', 'geoPoint': {'lat': 37.44188, 'lon': -122.14302}}, {'zip': '92037', 'city': 'San Diego', 'state': 'California', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'UCSD', 'geoPoint': {'lat': 32.71571, 'lon': -117.16472}}, {'zip': '80206', 'city': 'Denver', 'state': 'Colorado', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'National Jewish Health', 'geoPoint': {'lat': 39.73915, 'lon': -104.9847}}, {'zip': '30322', 'city': 'Atlanta', 'state': 'Georgia', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'Emory University', 'geoPoint': {'lat': 33.749, 'lon': -84.38798}}, {'zip': '02215', 'city': 'Boston', 'state': 'Massachusetts', 'status': 'RECRUITING', 'country': 'United States', 'facility': "Boston Children's Hospital", 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '10595', 'city': 'Valhalla', 'state': 'New York', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'New York Medical College', 'geoPoint': {'lat': 41.07482, 'lon': -73.77513}}, {'zip': '27599', 'city': 'Chapel Hill', 'state': 'North Carolina', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'The University of North Carolina at Chapel Hill', 'geoPoint': {'lat': 35.9132, 'lon': -79.05584}}, {'zip': '97239', 'city': 'Portland', 'state': 'Oregon', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'Oregon Health & Science University', 'geoPoint': {'lat': 45.52345, 'lon': -122.67621}}, {'zip': '15213', 'city': 'Pittsburgh', 'state': 'Pennsylvania', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'University of Pittsburgh', 'geoPoint': {'lat': 40.44062, 'lon': -79.99589}}, {'zip': '75390', 'city': 'Dallas', 'state': 'Texas', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'UT Southwestern Medical Center', 'geoPoint': {'lat': 32.78306, 'lon': -96.80667}}, {'zip': '98195', 'city': 'Seattle', 'state': 'Washington', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'University of Washington', 'geoPoint': {'lat': 47.60621, 'lon': -122.33207}}, {'city': 'Montpellier', 'status': 'RECRUITING', 'country': 'France', 'facility': 'Centre Hospitalier Régional Universitaire de Montpellier - Hôpital Arnaud de Villeneuve', 'geoPoint': {'lat': 43.61093, 'lon': 3.87635}}, {'city': 'Paris', 'status': 'RECRUITING', 'country': 'France', 'facility': 'Hôpital Necker Enfants Malades', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'city': 'Utrecht', 'status': 'RECRUITING', 'country': 'Netherlands', 'facility': 'UMC Utrecht', 'geoPoint': {'lat': 52.09083, 'lon': 5.12222}}, {'city': 'Auckland', 'status': 'COMPLETED', 'country': 'New Zealand', 'facility': 'New Zealand Clinical Research (Part 1 Only)', 'geoPoint': {'lat': -36.84853, 'lon': 174.76349}}, {'city': 'Birmingham', 'status': 'RECRUITING', 'country': 'United Kingdom', 'facility': 'University Hospitals Birmingham', 'geoPoint': {'lat': 52.48142, 'lon': -1.89983}}, {'city': 'Cambridge', 'status': 'RECRUITING', 'country': 'United Kingdom', 'facility': 'Royal Papworth Hospital', 'geoPoint': {'lat': 52.2, 'lon': 0.11667}}, {'city': 'Leeds', 'status': 'RECRUITING', 'country': 'United Kingdom', 'facility': 'Leeds Teaching Hospitals', 'geoPoint': {'lat': 53.79648, 'lon': -1.54785}}, {'city': 'London', 'status': 'RECRUITING', 'country': 'United Kingdom', 'facility': "King's College Hospital", 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'city': 'Nottingham', 'status': 'RECRUITING', 'country': 'United Kingdom', 'facility': 'Nottingham University Hospitals', 'geoPoint': {'lat': 52.9536, 'lon': -1.15047}}, {'city': 'Southampton', 'status': 'RECRUITING', 'country': 'United Kingdom', 'facility': 'University Hospital Southampton', 'geoPoint': {'lat': 50.90395, 'lon': -1.40428}}], 'centralContacts': [{'name': 'Priya Ryali, MBA', 'role': 'CONTACT', 'email': 'clinicaltrials@recodetx.com', 'phone': '650-629-7900'}], 'overallOfficials': [{'name': 'John Matthews, MBBS, MCRP, PhD', 'role': 'STUDY_CHAIR', 'affiliation': 'ReCode Therapeutics, Inc.'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'ReCode Therapeutics', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}