Clinical trial • Not applicable • Respiratory
BI 3720931 for Cystic fibrosis
Not applicable trial of BI 3720931 for Cystic fibrosis. open-label, none/not specified-controlled. 15 participants.
Overview
- Trial Therapeutic Area
- Respiratory
- Trial Disease
- Cystic fibrosis
- Trial Stage
- Not applicable
- Drug Modality
- Gene therapy
- Paediatric Trial
- Yes
Key dates
- Initial CTIS Submission Date
- 31-01-2024
- First CTIS Authorization Date
- 17-06-2024
Trial design
open-label, none/not specified-controlled Not applicable trial across 6 sites in France, Italy, Spain and others.
- Open Label
- Yes
- Comparator
- None/Not specified
- Target Sample Size
- 15
- Trial Duration For Participant
- 5475
Eligibility
Recruits 15 paediatric patients.
- Vulnerable Population
- No vulnerable populations selected. Signed and dated written informed consent is required in accordance with ICH-GCP and local legislation prior to admission to the trial. Assent for minors is not explicitly mentioned in the eligibility criteria; separate country-specific ICF documents are provided.
Inclusion criteria
- {"criterion_text":"- All trial participants who participated in a previous BI sponsored trial with BI 3720931 and have received at least one dose of BI 3720931, or matching placebo, are eligible for participation in extension trial 1504-0003 and should be enrolled."}
- {"criterion_text":"- Signed and dated written informed consent in accordance with International Council for Harmonisation (ICH)-Good Clinical Practice (GCP) and local legislation prior to admission to the trial."}
Exclusion criteria
- {"criterion_text":"- There are no exclusion criteria for enrolment into the trial."}
Endpoints
Primary endpoints
- {"endpoint_text":"- Occurrence of treatment-emergent delayed AEs defined as new malignancies, new serious neurologic disorders, new serious rheumatologic or autoimmune disorders, new serious hematologic disorders, or new serious potentially product-related infections, until 15 years from enrolment","definition_or_measurement_approach":"Treatment-emergent delayed adverse events defined as new malignancies, new serious neurologic disorders, new serious rheumatologic or autoimmune disorders, new serious hematologic disorders, or new serious potentially product-related infections, assessed up to 15 years from enrolment."}
Secondary endpoints
- {"endpoint_text":"- Time to loss of efficacy defined as the drop to below 5% (absolute) above baseline in forced expiratory volume in 1 second, percent of predicted value (FEV1pp)","definition_or_measurement_approach":"Defined as time until FEV1pp falls to less than 5% (absolute) above baseline."}
- {"endpoint_text":"- Time to first PEX from dosing","definition_or_measurement_approach":"Time from dosing to first pulmonary exacerbation (PEX)."}
- {"endpoint_text":"- Occurrence of AEs up to 2 years after enrolment","definition_or_measurement_approach":"Occurrence of adverse events recorded up to 2 years after enrolment."}
- {"endpoint_text":"- Occurrence of serious adverse events (SAEs) throughout the study (up to 15 years)","definition_or_measurement_approach":"Recording of serious adverse events throughout the study up to 15 years."}
- {"endpoint_text":"- Occurrence of replication-competent lentivirus (RCL) throughout the study (up to 15 years)","definition_or_measurement_approach":"Monitoring for replication-competent lentivirus (RCL) throughout the study up to 15 years."}
Recruitment
- Planned Sample Size
- 15
- Recruitment Window Months
- 278
- Consent Approach
- Signed and dated written informed consent in accordance with ICH-GCP and local legislation prior to admission to the trial. Country-specific subject information and informed consent form documents are provided (documents listed for Italy, France, Spain, Netherlands).
Geography
- Total Number Of Sites
- 6
- Total Number Of Participants
- 12
France
- Earliest CTIS Part Ii Submission Date
- 22-02-2024
- Latest Decision Or Authorization Date
- 24-03-2026
- Processing Time Days
- 761
- Number Of Sites
- 2
- Number Of Participants
- 4
Sites
- Site Name
- Hopital Necker Enfants Malades
- Department Name
- Centre d'Investigation Clinique
- Contact Person Name
- Isabelle Sermet-Gaudelus
- Contact Person Email
- isabelle.sermet@aphp.fr
- Site Name
- Centre Hospitalier Universitaire De Montpellier
- Department Name
- Centre d'Investigation Clinique
- Contact Person Name
- Raphaël Chiron
- Contact Person Email
- r-chiron@chu-montpellier.fr
Italy
- Earliest CTIS Part Ii Submission Date
- 26-04-2024
- Latest Decision Or Authorization Date
- 25-03-2026
- Processing Time Days
- 698
- Number Of Sites
- 2
- Number Of Participants
- 4
Sites
- Site Name
- Giannina Gaslini Institute For Scientific Hospitalization And Care
- Department Name
- Centro Fibrosi Cistica
- Contact Person Name
- Federico CRESTA
- Contact Person Email
- federicocresta@gaslini.org
- Site Name
- Bambino Gesu Childrens Hospital
- Department Name
- Pneumology and Cystic Fibrosis Unit
- Contact Person Name
- Federico ALGHISI
- Contact Person Email
- federico.alghisi@opbg.net
Spain
- Earliest CTIS Part Ii Submission Date
- 26-04-2024
- Latest Decision Or Authorization Date
- 25-03-2026
- Processing Time Days
- 698
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- Vall D'hebron Institut De Recerca
- Department Name
- Servicio Neumología
- Contact Person Name
- Antonio Álvarez Fernández
- Contact Person Email
- antonio.alvarez@vallhebron.cat
Netherlands
- Earliest CTIS Part Ii Submission Date
- 03-05-2024
- Latest Decision Or Authorization Date
- 24-03-2026
- Processing Time Days
- 690
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- Universitair Medisch Centrum Utrecht
- Department Name
- Department of Pulmonology and Tuberculosis
- Contact Person Name
- Kors van der Ent
- Contact Person Email
- k.vanderent@umcutrecht.nl
Sponsor
Primary sponsor
- Full Name
- Boehringer Ingelheim International GmbH
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- Germany
Third parties
- {"country":"Spain","full_name":"Boehringer Ingelheim Espana S.A.","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- BI 3720931
- Active Substance
- BI 3720931
- Modality
- Gene therapy
- Routes Of Administration
- Inhalation
- Route
- Inhalation
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