rhASA

DRUG2 trials

Sponsors

Shire Human Genetic Therapies Inc.

Conditions

Late Metachromatic Leukodystrophy (MLD)

Phase 1

An Open-Label Extension of Study HGT-MLD-070 Evaluating Long Term Safety and Efficacy of Intrathecal Administration of HGT-1110 in Patients with Metachromatic Leukodystrophy

Active, not recruitingCTIS2024-514403-34-00

Shire Human Genetic Therapies Inc.Late Metachromatic Leukodystrophy (MLD)

Start: 2012-10-10Target: 10Updated: 2025-09-30

Phase 2

A Global, Multicenter, Single-arm, Matched External Control Study of Intrathecal SHP611 in Subjects with Late Infantile Metachromatic Leukodystrophy

Active, not recruitingCTIS2024-514402-31-00

Shire Human Genetic Therapies Inc.Late Metachromatic Leukodystrophy (MLD)

Start: 2019-09-09Target: 10Updated: 2025-09-23