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Enzyme Replacement Therapy

Idursulfase + Elaprase for Hunter Syndrome

Phase 2 & 3

Waitlist Available

Research Sponsored by Shire

Eligibility Criteria Checklist

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

Must have

Be younger than 65 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline through month 121

Awards & highlights

Study Summary

This trial allows patients who completed a previous study to continue receiving Elaprase, a treatment for Hunter syndrome, with or without idursulfase IT.

Who is the study for?

This trial is for pediatric patients with Hunter Syndrome who completed the HGT-HIT-094 study. They must have been regularly receiving Elaprase and their guardians should consent to continued treatment. Those with severe allergies, uncontrolled seizures, bleeding disorders, high blood pressure, or certain spinal abnormalities cannot participate.Check my eligibility

What is being tested?

The trial continues treatment from a previous study (HGT-HIT-094), where participants receive Elaprase along with Idursulfase IT if they hadn't before. It aims to assess long-term safety and outcomes of these treatments in children with Hunter Syndrome.See study design

What are the potential side effects?

Possible side effects include allergic reactions to the drug components or implant device materials, issues related to intrathecal administration such as infection risk at the port site, and general risks associated with enzyme replacement therapies.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline through month 121

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline through month 121

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline through month 121 for reporting.

Treatment Details

Study Objectives

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

Primary outcome measures

Change From Baseline in the Concentration of Glycosaminoglycan (GAG) in Cerebrospinal Fluid (CSF)

Change From Baseline in the Concentration of Glycosaminoglycan (GAG) in Urine

Maximum Observed Concentration (Cmax) of Idursulfase in Cerebrospinal Fluid (CSF)

+5 more

Secondary outcome measures

Change From Baseline in Age Equivalent Scores of the Bayley Scales of Infant Development, Third Edition (BSID-III) Domains

Change From Baseline in Age Equivalents Score of the Differential Ability Scales, Second Edition (DAS-II)

Change From Baseline in Age Equivalents Score of the Vineland Adaptive Behavior Scales, Second Edition (VABS-II) Sub Domains

+11 more

Trial Design

1Treatment groups

Experimental Treatment

Group I: Idursulfase-ITExperimental Treatment2 Interventions

Participants will receive 10 milligrams (mg) of idursulfase-IT intrathecally via intrathecal drug delivery device (IDDD) or lumbar puncture (LP) once every 28 days along with standard-of-care therapy with Elaprase for 480 weeks. Participants who are younger than 3 years of age will receive an adjusted dose of 7.5 mg (greater than [>] 8 months to 30 months of age) and 10 mg (>30 months to 3 years of age) of idursulfase-IT.

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

ShireLead Sponsor

456 Previous Clinical Trials

97,236 Total Patients Enrolled

13 Trials studying Hunter Syndrome

1,753 Patients Enrolled for Hunter Syndrome

Study DirectorStudy DirectorTakeda

1,207 Previous Clinical Trials

489,624 Total Patients Enrolled

14 Trials studying Hunter Syndrome

1,736 Patients Enrolled for Hunter Syndrome

Media Library

Elaprase (Enzyme Replacement Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT02412787 — Phase 2 & 3

Hunter Syndrome Research Study Groups: Idursulfase-IT

Hunter Syndrome Clinical Trial 2023: Elaprase Highlights & Side Effects. Trial Name: NCT02412787 — Phase 2 & 3

Elaprase (Enzyme Replacement Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02412787 — Phase 2 & 3

Frequently Asked Questions

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

What are researchers hoping to uncover through this study?

"The primary outcome of this clinical trial is to Maximum Observed Serum Concentration (Cmax) of Idursulfase, which will be evaluated over the course of Baseline through Month 121. Secondary objectives include Change From Baseline in Age Equivalents Score of the Vineland Adaptive Behavior Scales, Second Edition (VABS-II) Sub Domains and Change From Baseline in T-scores of the Core Subtests Differential Ability Scales, Second Edition (DAS-II). The mean age equivalent score will be obtained by averaging out the age-equivalent scores for the all the sub-"

Answered by AI

Is this the initial investigation of this type of medical treatment?

"Idursulfase-IT has a long research history, with the first trial taking place a decade ago in 2010. That initial study was sponsored by Shire and only included 25 patients. Following that initial research, Idursulfase-IT received Phase 1 & 2 drug approval. Presently, there are 6 different ongoing trials for Idursulfase-IT being conducted in 16 countries and across 15 cities."

Answered by AI

Are there other existing examples of Idursulfase-IT's efficacy?

"Idursulfase-IT was first studied a decade ago at Emanuel Hospital. Since then, there have been seven completed trials and six ongoing studies. Many of the ongoing clinical trials are based in the city of Chicago."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094

2015. "Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT02412787.