異染性白質ジストロフィーの小児における脊髄内SHP611の研究

基本情報

NCT ID

NCT03771898

ステータス

実施中（募集終了）

試験のフェーズ

第2相

試験タイプ

介入

目標被験者数

36

治験依頼者名

Takeda

概要

The main aim of the study is to determine if SHP611 given by injection into the spinal fluid that surrounds the brain and spinal cord (intrathecal; IT) prolongs the time for children with Metachromatic Leukodystrophy (MLD) to retain the ability to move from place to place. Other aims of the study are to determine the effects of intrathecal administration of SHP611 on movement and speech functions and to learn how well SHP611 injected in the spinal fluid that surrounds the brain and spinal cord is tolerated. Study participants will receive SHP611 for about 2 years with the possibility of an extended treatment period.

対象疾患

介入

依頼者（Sponsor）

実施施設 (1)