Genethon — Sponsor Intelligence | 5 EU Trials

Filter these 5 trials by condition, phase, country and more.

5 Registered trials

4 Recruiting

1 Completed trials

5 Countries active

17 Unique IMPs

100% Results reported

Phase II

Phase I (FiH)

Oversight events:

1 urgent safety measure 1 unexpected event across 1 trial

100%

1 of 1 completed trials have published summary results (EU CTR Article 37 — 12-month obligation).

Top indications

5 total

Efficacy and safety trial of GNT0003 following imlifidase pre-treatment in participants aged 16 years and older with severe Crigler-Najjar s… Recruiting

2023-510405-18-00 Phase II 1 country ADENO-ASSOCIATED VIRAL VECTOR SEROTYPE 2/8 CONTAINING THE HUMAN UGT1A1 GENE; IML…
A clinical study in 3 parts with a microdystrophin (called GNT0004), a new gene therapy in boys with Duchenne disease who can still walk. Th… Recruiting

2023-505187-11-00 Phase I (FiH) 3 countries ADENO-ASSOCIATED VIRAL VECTOR SEROTYPE 8 CONTAINING THE HUMAN MD1 GENE; ECULIZUM…
Gene Therapy for Crigler-Najjar Syndrome Recruiting

2023-507007-60-00 Phase I (FiH) 3 countries ADENO-ASSOCIATED VIRAL VECTOR SEROTYPE 2/8 CONTAINING THE HUMAN UGT1A1 GENE
Long-term follow-up of the WAS gene therapy study Recruiting

2024-512680-31-00 Phase II 1 country ETUVETIDIGENE AUTOTEMCEL
A phase I/II, non randomized, monocentric open-label study of autologous CD34+ cells transduced with the G1XCGD lentiviral vector in patient… Ended

2024-512790-27-00 Phase I (FiH) 1 country AUTOLOGOUS CD34+ CELLS TRANSDUCED WITH THE G1XCGD LENTIVIRAL VECTOR CONTAINING T…

Showing 1–5 of 5 trials