Multicenter Study of HGT-1110 Administered Intrathecally in Children With Metachromatic Leukodystrophy (MLD)

Phase 1/2 Completed 24 enrolled Shire

Non-randomizedParallel-groupOpen-labelTreatment

Summary

The purpose of this study is to determine the safety of ascending doses of HGT-1110 administered by intrathecal (IT) injection for 38 weeks (20 injections) in children with metachromatic leukodystrophy (MLD).

Timeline

Start: 2012-02-02
Primary completion: 2017-01-20
Completion: 2017-01-20

Publications

I Dali C, Sevin C, Krageloh-Mann I, Giugliani R, Sakai N, Wu J, Wasilewski M. Safety of intrathecal delivery of recombinant human arylsulfatase A in children with metachromatic leukodystrophy: Results from a phase 1/2 clinical trial. Mol Genet Metab. 2020 Sep-Oct;131(1-2):235-244. doi: 10.1016/j.ymgme.2020.07.002. Epub 2020 Jul 16.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	HGT-1110	Protein / enzyme biologic	10 mg	Intrathecal
Subject	HGT-1110	Protein / enzyme biologic	30 mg	Intrathecal
Subject	HGT-1110	Protein / enzyme biologic	100 mg	Intrathecal

Multicenter Study of HGT-1110 Administered Intrathecally in Children With Metachromatic Leukodystrophy (MLD)

Summary

Timeline

Publications

Drugs

Indications