Condition: Crigler-Najjar Syndrome · Sponsor: Genethon
This is a Phase 1/2, multinational, open-label, study to evaluate the safety and efficacy of an intravenous infusion of GNT0003 in patients with Crigler-Najjar aged ≥10 years and requiring phototherapy. Patients will received a single administration of GNT0003 and will be followed for safety and efficacy of approximately 60 months (5 years): * 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.
This description comes directly from the study's public registry record.
Genethon Clinical Development Department · 00 33 (0)1 69 47 10 32 · clinical_development@genethon.fr
Always discuss trial participation with your own doctor first.
| Hopital Antoine BECLERE | Clamart, France | Recruiting |
| ASST Papa Giovanni XXIII | Bergamo, Italy | Recruiting |
| Azienda Ospedaliera Universitaria Federico II | Naples, Italy | Recruiting |
| AMC | Amsterdam, Netherlands | Recruiting |
Get one email when the public record changes — results posted, or the study's status changes. Nothing else, ever.
We email about this public record only. Unsubscribe anytime with one click. Never medical advice.
This page is independently generated by Eichor from the public ClinicalTrials.gov record and re-synced daily. It is not the sponsor's official website unless claimed. Nothing here is medical advice; eligibility is always determined by the study team — talk to your own doctor first.
Source record: clinicaltrials.gov/study/NCT03466463