RCT2100 for Cystic fibrosis

Inclusion criteria

• {"criterion_text":"- Males or females aged 18 to 60 years, inclusive, on the date of informed consent\n- Confirmed diagnosis of CF\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 b) Eligible for CFTR modulators (based on local prescribing information) but not using CFTR modulators due to intolerance or contraindications\n- Forced expiratory volume in 1 second ≥50% and ≤100% of predicted mean value for age, sex, and height (equations of the Global Lung Function Initiative [GLI]) at the Screening Visit. Spirometry measurements must meet American Thoracic Society/European Respiratory Society criteria for acceptability and repeatability\n- Please refer to the protocol for details"}

Exclusion criteria

• {"criterion_text":"- 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 (Day 1)\n- Lung infection with organisms associated with a more rapid decline in pulmonary status (including, but not limited to, Burkholderia cenocepacia, Burkholderia dolosa, and Mycobacterium abscessus). For participants who have had a history of a positive culture, the Investigator will apply the following criteria to establish whether the participant is free of infection from such organisms: • The participant has not had a respiratory tract culture positive for these organisms within the 12 months before the date of informed consent • The participant has had at least two respiratory tract cultures negative for such organisms within the 12 months before the date of informed consent, with the first and last of these separated by at least 3 months, and the most recent one within the 6 months before the date of informed consent\n- Arterial oxygen saturation on room air less than 94% at screening\n- Estimated glomerular filtration rate < 30 mL/minute/1.73 m2 calculated with the Chronic Kidney Disease Epidemiology Collaboration formula\n- Treatment with a CFTR modulator (Eg. Kalydeco, Trikafta, Symdeko, Orkambi, Alyftrek) within 12 weeks of Screening\n- History of poorly controlled diabetes\n- Please refer to the protocol for details."}

Primary endpoints

• {"endpoint_text":"- Safety and tolerability as assessed by number of participants with AEs and SAEs","definition_or_measurement_approach":"Assessed by the number of participants reporting adverse events (AEs) and serious adverse events (SAEs)."}

Secondary endpoints

• {"endpoint_text":"- Biodistribution parameters may be derived from concentrations of RCT2100 components in blood (where feasible). Parameters may include but are not limited to the following: AUC, Cmax, Tmax, and t1/2","definition_or_measurement_approach":"Biodistribution parameters derived from blood concentrations of RCT2100 components; parameters may include AUC, Cmax, Tmax, t1/2."}
• {"endpoint_text":"- Absolute change in percent predicted FEV1 (ppFEV1) from baseline to Week 4 (escalation cohorts 1 to 3) or Week 12 (expansion cohort)","definition_or_measurement_approach":"Change in percent predicted FEV1 from baseline to Week 4 (escalation cohorts) or Week 12 (expansion cohort), measured by spirometry per GLI equations and ATS/ERS criteria."}
• {"endpoint_text":"- Change from baseline in CF questionnaire-revised (CFQ-R) respiratory domain score through Week 4 (escalation cohorts 1 to 3) or Week 12 (expansion cohort)","definition_or_measurement_approach":"Change from baseline in CFQ-R respiratory domain score through Week 4 (escalation cohorts) or Week 12 (expansion cohort)."}
• {"endpoint_text":"- Incidence and titer of anti-CFTR binding antibodies","definition_or_measurement_approach":"Measurement of incidence and titers of anti-CFTR binding antibodies (immunogenicity assays)."}
• {"endpoint_text":"- Based on safety and tolerability at projected efficacious doses","definition_or_measurement_approach":"Dose selection or evaluations based on safety and tolerability observed at doses projected to be efficacious."}
• {"endpoint_text":"- Please refer to the protocol for details.","definition_or_measurement_approach":"Refer to protocol for additional secondary endpoint details."}

France

Earliest CTIS Part Ii Submission Date

24-06-2024

Latest Decision Or Authorization Date

08-10-2025

Processing Time Days

471

Number Of Sites

3

Number Of Participants

9

Netherlands

Earliest CTIS Part Ii Submission Date

19-06-2024

Latest Decision Or Authorization Date

24-09-2025

Processing Time Days

462

Number Of Sites

1

Number Of Participants

8

Primary sponsor

Full Name

Recode Therapeutics Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

Novotech (Australia) Pty Limited

Responsibilities

Full Service CRO

Name

Pharmaceutical Product Development LLC

Responsibilities

code: 8

Name

Icon Public Limited Company

Responsibilities

code: 4

Third parties

• {"country":"United Kingdom","full_name":"Acm Global Central Laboratory Limited","duties_or_roles":"code: 4","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"Germany","full_name":"eResearchTechnology GmbH","duties_or_roles":"Respiratory Services","organisation_type":"Pharmaceutical company"}
• {"country":"Germany","full_name":"MARKEN Germany GmbH","duties_or_roles":"Kits for BioAnalytics","organisation_type":"Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"Illingworth Research Group Limited","duties_or_roles":"Home Visits, Patient Reimbursement","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"United States","full_name":"Pharmaceutical Product Development LLC","duties_or_roles":"code: 8","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Iqvia Biotech LLC","duties_or_roles":"CFQR Licensing","organisation_type":"Pharmaceutical company"}
• {"country":"Australia","full_name":"Novotech (Australia) Pty Limited","duties_or_roles":"Full Service CRO","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Korio Inc.","duties_or_roles":"code: 3","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Bioagilytix Labs LLC","duties_or_roles":"code: 4","organisation_type":"Pharmaceutical company"}
• {"country":"Ireland","full_name":"Icon Public Limited Company","duties_or_roles":"code: 4","organisation_type":"Pharmaceutical company"}
• {"country":"France","full_name":"ICTA Project Management En Abrege ICTA P.M.","duties_or_roles":"codes: 1, 12","organisation_type":"Pharmaceutical company"}
• {"country":"Belgium","full_name":"Harmony Clinical Research","duties_or_roles":"codes: 1, 12","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Avance Biosciences Inc.","duties_or_roles":"code: 4","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"Cardiac Safety Services","organisation_type":"Pharmaceutical company"}