recombinant human arylsulfatase A (TAK-611) / Takeda - LARVOL DELTA

EMBOLDEN: A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy (clinicaltrials.gov) - P2 | N=36 | Active, not recruiting | Sponsor: Shire | Trial completion date: Oct 2025 ➔ Jan 2026

Trial completion date • Metabolic Disorders

EMBOLDEN: A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy (clinicaltrials.gov) - P2 | N=36 | Active, not recruiting | Sponsor: Shire | Trial completion date: Mar 2025 ➔ Oct 2025

Trial completion date • Metabolic Disorders

An Efficacy and Safety Study of HGT-1110 in Participants With Metachromatic Leukodystrophy (clinicaltrials.gov) - P1/2 | N=24 | Active, not recruiting | Sponsor: Shire | Trial completion date: Dec 2024 ➔ Sep 2025 | Trial primary completion date: Dec 2024 ➔ Sep 2025

Trial completion date • Trial primary completion date • Metabolic Disorders

An Efficacy and Safety Study of HGT-1110 in Participants With Metachromatic Leukodystrophy (clinicaltrials.gov) - P1/2 | N=24 | Active, not recruiting | Sponsor: Shire | Trial completion date: Apr 2023 ➔ Dec 2024 | Trial primary completion date: Apr 2023 ➔ Dec 2024

Trial completion date • Trial primary completion date • Metabolic Disorders

A Study of Intrathecal SHP611 in Participants With Late Infantile Metachromatic Leukodystrophy (clinicaltrials.gov) - P2 | N=36 | Active, not recruiting | Sponsor: Shire | Trial completion date: Aug 2022 ➔ Mar 2025 | Trial primary completion date: Aug 2022 ➔ Mar 2023

Trial completion date • Trial primary completion date • Metabolic Disorders

A Study of Intrathecal SHP611 in Participants With Late Infantile Metachromatic Leukodystrophy (clinicaltrials.gov) - P2; N=42; Active, not recruiting; Sponsor: Shire; Recruiting ➔ Active, not recruiting

Clinical • Enrollment closed • Metabolic Disorders

Intravenous arylsulfatase A in metachromatic leukodystrophy: a phase 1/2 study. (PubMed, Ann Clin Transl Neurol) - P1, P1/2, P2 | "IV rhASA was generally well tolerated. There was no evidence of efficacy in preventing motor and cognitive deterioration, suggesting that IV rhASA may not cross the blood-brain barrier in therapeutic quantities. The relative stability of peripheral nerve function during the study indicates that rhASA may be beneficial if delivered to the appropriate target site and supports the development of rhASA for intrathecal administration in MLD."

Journal • P1/2 data • Cognitive Disorders • Lysosomal Storage Diseases • Metabolic Disorders • Rare Diseases

Safety of intrathecal delivery of recombinant human arylsulfatase A in children with metachromatic leukodystrophy: Results from a phase 1/2 clinical trial. (PubMed, Mol Genet Metab) - P1/2 | "Intrathecal rhASA was generally well tolerated at doses up to 100 mg EOW. These preliminary data support further development of rhASA as a therapy for patients with MLD."

Clinical • Journal • P1/2 data • Lysosomal Storage Diseases • Metabolic Disorders • Rare Diseases

Open-Label Extension Study Evaluating Safety and Efficacy of HGT-1110 in Patients With Metachromatic Leukodystrophy (clinicaltrials.gov) - P1/2; N=24; Enrolling by invitation; Sponsor: Shire; N=18 ➔ 24; Trial primary completion date: Feb 2018 ➔ Oct 2021

Enrollment change • Trial primary completion date • Biosimilar

Pharmacokinetic Modeling of Intrathecally Administered Recombinant Human Arylsulfatase A (TAK-611) in Children with Metachromatic Leukodystrophy (MLD). (PubMed, Clin Pharmacol Ther) - "Estimated parameters suggested rapid distribution of TAK-611 from CSF into the putative brain tissue compartment, with persistence in the brain between doses (median distributive and terminal half-lives in the CNS: 1.02 and 477 hours, respectively). This model provides a valuable basis for understanding the pharmacokinetic distribution of TAK-611, and for pharmacokinetic/pharmacodynamic analyses of functional outcomes."

Clinical • Journal • PK/PD data

A Study of Intrathecal SHP611 in Participants With Late Infantile Metachromatic Leukodystrophy (clinicaltrials.gov) - P2; N=35; Recruiting; Sponsor: Shire; Not yet recruiting ➔ Recruiting

Enrollment open

Research study to determine the effects of an investigational drug, SHP611 on patients with with Late Infantile Metachromatic Leukodystrophy (MLD) specially the gross motor function, using the Gross Motor Function Classification in Metachromatic Leukodystrophy (GMFC-MLD) compared with matched historical control data in children with MLD. (clinicaltrialsregister.eu) - P2; N=35; Sponsor: Shire Human Genetic Therapies, Inc.

Clinical • New P2 trial