Trial Profile
Phase I/II safety, tolerability and initial efficacy study of adeno-associated viral vector serotype 9 containing human sulfamidase gene after intracerebroventricular administration to patients with MPSIIIA. Estudio de fase I/II para evaluar la seguridad, tolerabilidad y eficacia inicial del vector viral adenoasociado de serotipo 9 conteniendo el gen de la sulfamidasa humana tras su administración por vía intracerebroventricular a pacientes con MPSIIIA.
Status:
Recruiting
Phase of Trial:
Phase I/II
Latest Information Update: 14 Jun 2016
Price :
$35
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At a glance
- Drugs EGT 101 (Primary)
- Indications Mucopolysaccharidosis III
- Focus Adverse reactions; First in man; Pharmacodynamics; Therapeutic Use
- Sponsors Esteve
- 10 Jun 2016 Status changed from planning to recruiting.
- 01 Mar 2016 New trial record
- 25 Feb 2016 According to ESTEVE and UAB media release, company is planning to initiate this trial by the end of 2016. EGT-101 received orphan drug designation by the FDA and EMA in 2011.