recombinant human heparan N-sulfatase (SHP610) / Takeda - LARVOL DELTA

Long-term safety and clinical outcomes of intrathecal heparan-N-sulfatase in patients with Sanfilippo syndrome type A. (PubMed, Mol Genet Metab) - P1/2 | "Overall, rhHNS IT was well tolerated in the extension study. However, rhHNS IT was unable to slow the neurocognitive decline of patients with MPS IIIA. This study was subsequently terminated early because pre-specified efficacy criteria were not met, and the study did not yield clinical proof of concept. (Clinicaltrials.gov Identifier NCT01299727)."

Clinical • Clinical data • Journal • Lysosomal Storage Diseases • MRI

A multicenter open-label extension study of intrathecal heparan-N-sulfatase in patients with Sanfilippo syndrome type A. (PubMed, Mol Genet Metab) - "Greater declines were observed in patients at the higher end of the age range, suggesting earlier intervention may increase the possibility of a response to treatment. rhHNS IT treatment remained generally well tolerated up to 96 weeks."

Clinical • Journal • CNS Disorders • Hunter Syndrome • Lysosomal Storage Diseases • Metabolic Disorders • Rare Diseases

Intrathecal heparan-N-sulfatase in patients with Sanfilippo syndrome type A: A phase IIb randomized trial. (PubMed, Mol Genet Metab) - P2b; "rhHNS IT treatment reduced heparan sulfate and GAG levels in treated patients. Though the primary neurocognitive endpoint was not met, important lessons in the design and endpoints for evaluation of cognitive and behavioral diseases resulted."

Clinical • Journal • P2b data

An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093. (clinicaltrials.gov) - P2; N=17; Terminated; Sponsor: Shire; Trial completion date: Oct 2021 ➔ Mar 2019; Active, not recruiting ➔ Terminated; Trial primary completion date: Oct 2021 ➔ Mar 2019; Completion of follow-up period

Clinical • Trial completion date • Trial primary completion date • Trial termination

Extension of Study HGT-SAN-055 Evaluating Administration of rhHNS in Patients With Sanfilippo Syndrome Type A (MPS IIIA) (clinicaltrials.gov) - P1/2; N=12; Terminated; Sponsor: Shire; Trial completion date: Oct 2021 ➔ Feb 2019; Active, not recruiting ➔ Terminated; Trial primary completion date: Oct 2021 ➔ Feb 2019; Completion of follow-up period

Clinical • Clinical data • Trial completion date • Trial primary completion date • Trial termination