A Phase I Clinical Trial to Evaluate the Safety of the Infusion of Autologous CD34+ Cells Transduced With a Lentiviral Vector Carrying the FANCA Gene in Pediatric Subjects With Fanconi Anemia Subtype A
Phase of Trial: Phase I
Latest Information Update: 31 Jan 2019
At a glance
- Drugs RP L102 (Primary)
- Indications Fanconi's anaemia
- Focus Adverse reactions
- Sponsors Rocket Pharmaceuticals
- 18 Jan 2019 Status changed from planning to recruiting.
- 25 Nov 2018 New trial record
- 07 Nov 2018 According to a Rocket Pharmaceuticals media release, the company looks forward to engaging with regulatory authorities on a final registration path in the second half of 2019 after initial patients have been treated in this trial.