Trial Profile
A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Efficacy of Intracerebroventricular AX 250 in Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)
Status:
Completed
Phase of Trial:
Phase I/II
Latest Information Update: 01 Feb 2023
Price :
$35
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At a glance
- Drugs Tralesinidase alfa (Primary)
- Indications Mucopolysaccharidosis III
- Focus Adverse reactions; First in man; Therapeutic Use
- Sponsors Allievex Corporation; BioMarin Pharmaceutical
- 17 Jan 2023 Results (n=22) assessing the safety, tolerability, and efficacy of tralesinidase alfa administered via an implanted i.c.v. device to patients with Sanfilippo syndrome type B and also the pharmacokinetics and pharmacodynamics results of our clinical investigation of tralesinidase alfa in the treatment of children with MPS IIIB over a 48-week period, published in the Journal of Clinical Investigation.
- 08 Jul 2020 Status changed from active, no longer recruiting to completed.
- 05 Jul 2020 This trial has been discontinued in Spain, according to European Clinical Trials Database record.