Shire Human Genetic Therapies Inc.

Address: 300 Shire Way

Sponsor Overview

assignment

Specializations

Metachromatic Leukodystrophy Research

Shire Human Genetic Therapies Inc., operating as Takeda, focuses its clinical research on late metachromatic leukodystrophy (MLD), a rare inherited lysosomal storage disorder affecting the nervous system. The studies center on children with late infantile MLD and address the progression of motor impairment associated with the disease.

Gross motor function decline
Locomotion preservation
Neurologic disease progression

The sponsor’s research activity in this area reflects an emphasis on rare neurodegenerative disease and the clinical consequences of impaired motor control in affected children.

Enzyme Replacement Therapy

The sponsor is involved in clinical investigation of enzyme replacement therapy for MLD, including treatment approaches designed to address the underlying metabolic defect in the disease. This work includes long-term follow-up in patients receiving HGT-1110 and ongoing assessment of therapeutic durability.

Metabolic correction
Long-term safety
Treatment persistence

These studies place a therapeutic focus on inherited disorders requiring sustained enzyme-based intervention.

Intrathecal Therapy

Clinical research supported by the sponsor includes intrathecal administration strategies for delivering therapy directly to the central nervous system. This route of administration is relevant to disorders in which access to the nervous system is essential for therapeutic effect.

Central nervous system delivery
Neurotherapeutic administration
Spinal fluid-based treatment

The sponsor’s interest in this area is closely tied to treatment of neurologic lysosomal disease with direct CNS-targeted delivery.

Pediatric Rare Disease

The funded trials are concentrated in pediatric rare disease, specifically children living with metachromatic leukodystrophy. The research addresses disease burden in early life and the need for therapies tailored to pediatric neurogenetic conditions.

Pediatric neurology
Inherited metabolic disease
Rare genetic disorder

This area of interest links genetic medicine with treatment development for severe childhood-onset neurologic illness.

Trial Name	Country	Status
Intrathecal Administration of Cebsulfase Alfa in Pediatric Patients with Late Infantile Metachromatic Leukodystrophy: A Single-Arm, Matched External Control Study	Belgium France Germany Greece Italy Spain	Not Recruiting
Long-Term Safety and Efficacy of Intrathecal Cebsulfase Alfa in Pediatric Patients with Late Metachromatic Leukodystrophy: An Extension of Study HGT-MLD-070	Czechia Denmark France Germany Italy	Not Recruiting