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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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up predose at weeks 0, 5, 9, 13, 26, 40, 53, 79, and 106

Awards & highlights

EMBOLDEN Trial Summary

This trial is testing a possible treatment for children with a rare disease that affects the nervous system.

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.Check my eligibility

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.See study design

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.

EMBOLDEN Trial Eligibility Criteria

Inclusion Criteria

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

Select...

I have difficulty walking due to MLD.

Select...

I have been diagnosed with Metachromatic Leukodystrophy (MLD).

EMBOLDEN Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ predose at weeks 0, 5, 9, 13, 26, 40, 53, 79, and 106

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~predose at weeks 0, 5, 9, 13, 26, 40, 53, 79, and 106

This trial's timeline: 3 weeks for screening, Varies for treatment, and predose at weeks 0, 5, 9, 13, 26, 40, 53, 79, and 106 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Response in Group A: Time to Loss of Locomotion Measured by Progression to Gross Motor Function Classification in Metachromatic Leukodystrophy (GMFC-MLD) at Week 106

Secondary outcome measures

Area Under the Curve From the Time of Dosing to the Last Measurable Concentration (AUC0-t) of SHP611 in Serum

Area Under the Plasma Concentration Versus Time Curve Extrapolated to Infinity (AUC0-inf) of SHP611 in Serum

Change From Baseline in Cerebrospinal Fluid (CSF) Sulfatides Levels at Week 106 and End of Study (EOS)

+21 more

EMBOLDEN 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.

0 / 2Eligibility criteria met

Find a Location

Who is running the clinical trial?

ShireLead Sponsor

456 Previous Clinical Trials

97,256 Total Patients Enrolled

Takeda Development Center Americas, Inc.Industry Sponsor

56 Previous Clinical Trials

11,994 Total Patients Enrolled

Study DirectorStudy DirectorShire

1,207 Previous Clinical Trials

489,644 Total Patients Enrolled

Media Library

SHP611 (Enzyme Replacement Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT03771898 — Phase 2

Metachromatic Leukodystrophy Research Study Groups: SHP611

Metachromatic Leukodystrophy Clinical Trial 2023: SHP611 Highlights & Side Effects. Trial Name: NCT03771898 — Phase 2

SHP611 (Enzyme Replacement Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03771898 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are there opportunities to enroll in this research currently available?

"Clinicaltrials.gov informs us that this trial, originally posted on April 30th 2019 and updated most recently on March 23rd 2022, is no longer actively recruiting participants. However, there are 10 other trials in the same field currently accepting new patients."

Answered by AI

Is the eligibility criterion for this clinical trial limited to those under 30 years of age?

"This research study is open to youngsters aged between 6 months and 72 months. In addition, 10 studies are available for minors under 18 years old while 2 investigations can be accessed by those over 65."

Answered by AI

How many distinct locations are overseeing this trial?

"At present, this trial is taking place at Cincinnati Children's Hospital Medical Center in Ohio, Los Angeles Biomedical Research Institute at Harbor-UCLA in California and British Columbia Children's Hospital in Vancouver. Additionally, it can be found running at other 15 medical sites."

Answered by AI

Has SHP611 been approved by the United States Food and Drug Administration?

"Our team at Power determined the safety of SHP611 to be a 2, due to there being some evidence for safety but no data supporting efficacy."

Answered by AI

What are the eligibility criteria for participating in this research?

"This particular clinical trial seeks 36 young individuals between 6 months and 72 months of age suffering from arylsulfatase A deficiency. Criteria for inclusion are as follows: Group A participants must have GMFC-MLD levels 1 or 2; Group B need to score 3 on the same scale, while Groups C and F should show either 4 or 5/6 respectively. Furthermore, those in Groups D & E ought to be minimally symptomatic (Group D) or early symptomatic (Group E), with certain restrictions based upon their age range. Finally, all applicants should demonstrate that they can comply with the protocols established by this"

Answered by AI

Recent research and studies

clinicaltrials.govStudy

A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy

2019. "A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03771898.

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