What side effects are associated with this type of intervention?

Intrathecal administration of treatments, such as SHP611 for Metachromatic Leukodystrophy (MLD), can have side effects including headaches, nausea, dizziness, and potential infections at the injection site. Other possible side effects may include back pain, fever, and in rare cases, neurological complications such as seizures or meningitis. It is important for patients to discuss these potential risks with their healthcare provider to weigh the benefits and risks of the treatment.

What prior FDA approvals exist?

Currently, there are no specific FDA-approved treatments for Metachromatic Leukodystrophy (MLD) that involve intrathecal administration similar to SHP611. The primary focus of treatment for MLD has been on managing symptoms and supportive care. Research and clinical trials, such as the one involving SHP611, are ongoing to explore new therapeutic options that could potentially prolong motor function and improve quality of life for patients with MLD.

What other types of research exist?

Currently, there are no specific alternatives to SHP611 mentioned for Metachromatic Leukodystrophy (MLD) in the provided context. However, exploring treatments used in similar neurodegenerative conditions, such as intrathecal administration of other therapeutic agents, gene therapy, or stem cell therapy, might offer potential alternatives. Consulting with a healthcare provider specializing in MLD or neurodegenerative diseases is recommended to explore the most up-to-date and suitable treatment options available in 2024.

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Enzyme Replacement Therapy

Intrathecal SHP611 for Metachromatic Leukodystrophy (EMBOLDEN Trial)

Phase 2

Waitlist Available

Research Sponsored by Shire

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

The participant must have a gait disorder due to spastic ataxia or weakness attributable to MLD

The participant must have a documented diagnosis of MLD

Must not have

History of bone marrow transplant (BMT), hematopoietic stem cell transplantation (HSCT), or gene therapy

Drug therapy requiring substances known to be incompatible with the materials of construction

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline, week 106

Awards & highlights

No Placebo-Only Group

Summary

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.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

Possible side effects of SHP611 may include reactions at the injection site, potential infection risks from repeated spinal injections, allergic responses, and other complications related to intrathecal administration.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have difficulty walking due to MLD.

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I have been diagnosed with Metachromatic Leukodystrophy (MLD).

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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

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I am not on medications that react with the trial's drug materials.

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I am at risk of abnormal bleeding due to my condition or treatment.

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I have a genetic disorder affecting my enzyme activity, not MLD.

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I have previously been treated with SHP611.

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My first MLD symptoms were related to behavior or thinking.

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I have spine issues that could make surgery risky.

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I might have an infection.

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I weigh more than 11 pounds.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline, week 106

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline, week 106

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, week 106 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Percent Probability of Free of Loss of Locomotion in the Last Time Interval Up to 2 Years (Week 106) Based on GMFC-MLD for SHP611 Group A and GLIA-MLD Matched External Control

Secondary study objectives

Change From Baseline in Cerebrospinal Fluid (CSF) Sulfatides Levels at Week 106

Change From Baseline in GMFM-88 Total Score at Week 106

Group A: Number of Participants Who Maintained Their Gross Motor Function Evaluated by Using the GMFC-MLD at Week 106 Compared With Matched External Control Group Data

+18 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

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.

0 / 11Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Metachromatic Leukodystrophy (MLD) often involve enzyme replacement therapies that aim to address the deficiency of arylsulfatase A, the enzyme lacking in MLD patients. Intrathecal administration, such as with SHP611, involves injecting the enzyme directly into the spinal fluid, allowing it to bypass the blood-brain barrier and reach the central nervous system more effectively. This method is crucial for MLD patients as it targets the brain and spinal cord, where the disease causes the most damage, potentially prolonging motor function and improving quality of life.