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SHP611-201
Recruitment Complete

A Study of Intrathecal SHP611 in Participants With Late Infantile Metachromatic Leukodystrophy (Embolden)

Clinicaltrials.gov
#NCT03771898
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About this clinical trial

The primary purpose of this study is to evaluate the effects of intrathecal (IT) administration of SHP611 on gross motor function, using the Gross Motor Function Classification in Metachromatic Leukodystrophy (GMFC-MLD) compared with matched historical control data in children with metachromatic leukodystrophy (MLD).

US
CA
DE
11+
Interventional Phase 2 clinical trial.

At a glance

What medical conditions were being studied?

Metachromatic Leukodystrophy (MLD)

What was the clinical trial testing?

SHP611

How many participants were enrolled?

36

When was the clinical trial conducted?

May 2019 - Oct 2025

How long was participation in the clinical trial?

Participants will be in the study for approximately 2 years (26 months).

Key requirements

Sexes

All

Age

Up to 6 Years

Healthy volunteers?

No

Entry criteria

Boys and girls can take part.
Must be infants/children between 6 and 72 months of age.
Must have been diagnosed Metachromatic Leukodystrophy (MLD).
Must have a walking disorder or weakness resulting from MLD.
Cannot be weighing less than 5 kg.
Additional entry criteria will be discussed with the study doctor.
Clinicaltrials.gov

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