Intrathecal SHP611 for Metachromatic Leukodystrophy
(EMBOLDEN Trial)

Recruiting in Palo Alto (17 mi)

+29 other locations

Age: < 18

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Shire

No Placebo Group

Prior Safety Data

Trial Summary

What is the purpose of this trial?This trial is testing if SHP611, injected into the fluid around the brain and spinal cord, can help children with Metachromatic Leukodystrophy (MLD) keep their ability to move and speak for a longer time. The study will also check how well children tolerate this treatment over a long period.

Eligibility Criteria

This trial is for children with Metachromatic Leukodystrophy (MLD) who have movement issues due to the disease. They must be diagnosed with MLD, able to follow the study plan, and meet specific age and GMFC-MLD criteria. Children can't join if they've had certain treatments like bone marrow transplants or gene therapy, are enrolled in another drug study, or have conditions that make it unsafe to participate.

Inclusion Criteria

I have difficulty walking due to MLD.

The participant's level of functioning must meet specific criteria for each group during the screening.

Your white blood cells do not have enough ASA activity compared to the normal range in the lab.

+5 more

Exclusion Criteria

I have had a bone marrow, stem cell transplant, or gene therapy.

You are too small for the SOPH-A-PORT Mini S Access Port to be used.

Any other medical condition or serious comorbid illness that in the opinion of the investigator would preclude participation in the study

+15 more

Participant Groups

The trial tests SHP611 given intrathecally (injected into spinal fluid) over approximately two years. It aims to see if this treatment helps children maintain their ability to move independently and assess its impact on movement and speech functions.

1Treatment groups

Experimental Treatment

Group I: SHP611Experimental Treatment1 Intervention

Participants will receive 150 milligrams (mg) of SHP611 intrathecally (IT) via intrathecal drug delivery device (IDDD) or lumbar puncture (LP) once weekly for 106 weeks in six groups (Group A, B, C, D, E, and F) based on participant's age and motor dysfunction.