Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
RECRUITING
NA
17 participants
INTERVENTIONAL
2018-03-19
2030-03-30
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
* a follow-up of approximately 12 months (48 weeks)
* a long term follow-up of approximately 48 months (4 years), in order to be in line with the latest EMEA Guideline on follow-up of patients administered with gene therapy medicinal products, released on 22 Oct.2009 by the Committee for medicinal products for human use.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
Gene Therapy for Crigler Najjar Syndrome Type I (AlphaCN)
NCT06641154
Efficacy and Safety of GNT0003 Following Imlifidase Pre-treatment in Severe Crigler-Najjar Syndrome
NCT06518005
Gene Transfer Clinical Study in Crigler-Najjar Syndrome
NCT03223194
Safety and Efficacy of a Single Subretinal Injection of JWK002 Gene Therapy in Subjects With X-linked Retinoschisis(XLRS)
NCT06345898
Evaluation of the Safety, Tolerability and Efficacy of Gene Therapy Drug for Late Onset Pompe Disease (LOPD)
NCT06178432
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
NA
SEQUENTIAL
OTHER
NONE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
GNT0003
2 doses of the IMP assessed in the dose escalation, open-label, phase 1/2 study
GNT0003
Intravenous infusion, single dose
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
GNT0003
Intravenous infusion, single dose
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* Male or female at least 9 years at the date of signature of informed consent
* Patient able to give informed assent and/or consent in writing
Exclusion Criteria
* Patients with chronic hepatitis B or C
* Patients infected with Human immunodeficiency virus (HIV)
* Patients with significant underlying liver disease
* Patients with significant encephalopathy
* Participation in any other investigational trial during this trial
* Patients unable or unwilling to comply with the protocol requirements
9 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Genethon
OTHER
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
LABRUNE Philippe, Prof
Role: PRINCIPAL_INVESTIGATOR
Hopital Antoine Beclere
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
Hopital Antoine BECLERE
Clamart, , France
ASST Papa Giovanni XXIII
Bergamo, , Italy
Azienda Ospedaliera Universitaria Federico II
Napoli, , Italy
AMC
Amsterdam, , Netherlands
Countries
Review the countries where the study has at least one active or historical site.
Central Contacts
Reach out to these primary contacts for questions about participation or study logistics.
Facility Contacts
Find local site contact details for specific facilities participating in the trial.
Role: primary
Role: primary
Role: primary
Role: primary
References
Explore related publications, articles, or registry entries linked to this study.
D'Antiga L, Beuers U, Ronzitti G, Brunetti-Pierri N, Baumann U, Di Giorgio A, Aronson S, Hubert A, Romano R, Junge N, Bosma P, Bortolussi G, Muro AF, Soumoudronga RF, Veron P, Collaud F, Knuchel-Legendre N, Labrune P, Mingozzi F. Gene Therapy in Patients with the Crigler-Najjar Syndrome. N Engl J Med. 2023 Aug 17;389(7):620-631. doi: 10.1056/NEJMoa2214084.
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
GNT-012-CRIG
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.