Trial Profile
A Phase 2 Open-Label Study to Evaluate Safety, Tolerability, and Efficacy of Intracerebroventricular BMN 190 in Pediatric Patients < 18 years of age with CLN2 Disease
Status:
Completed
Phase of Trial:
Phase II
Latest Information Update: 15 Mar 2023
Price :
$35
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At a glance
- Drugs Cerliponase alfa (Primary)
- Indications Neuronal ceroid lipofuscinosis
- Focus Adverse reactions; Pharmacodynamics; Therapeutic Use
- Sponsors BioMarin Pharmaceutical
- 19 May 2022 Status changed from active, no longer recruiting to completed.
- 01 Sep 2021 Interim results (n=14 ; as of Apr 2020) assessing safety and efficacy of cerliponase alfa from an expanded cohort including children less than 3 years presented at the 34th International Epilepsy Congress
- 21 Sep 2020 Planned primary completion date changed from 1 Nov 2021 to 1 Oct 2021.