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Enzyme Replacement Therapy

Enzyme Replacement Therapy for Hunter Syndrome

Phase 1 & 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 has received and tolerated a minimum of 12 months of treatment with weekly IV infusions of Elaprase and has received 80% of the total planned infusions within the last 6 months

Be younger than 65 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from start of study drug administration up to follow-up (169 months)

Awards & highlights

Study Summary

This trial will collect long-term safety data for kids with Hunter syndrome and cognitive impairment who are receiving two different types of enzyme replacement therapy.

Who is the study for?

This trial is for pediatric patients with Hunter syndrome and cognitive impairment who have been treated with Elaprase IV infusions for at least a year. They must have tolerated the treatment well, completed prior study requirements, and not be part of another clinical study. Children unable to follow the protocol or at high risk from anesthesia are excluded.Check my eligibility

What is being tested?

The trial is testing the long-term safety and outcomes of combining intrathecal Idursulfase-IT with intravenous Elaprase in children. It's an extension of a previous study, aiming to understand how these treatments work over time in managing Hunter syndrome.See study design

What are the potential side effects?

Potential side effects may include reactions related to infusion such as swelling or pain at injection site, allergic responses, issues related to anesthesia during administration, and general risks associated with enzyme replacement therapies.

Eligibility Criteria

Inclusion Criteria

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

Select...

I have been on Elaprase for 12 months and missed less than 20% of doses in the last 6 months.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from start of study drug administration up to follow-up (169 months)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from start of study drug administration up to follow-up (169 months)

This trial's timeline: 3 weeks for screening, Varies for treatment, and from start of study drug administration up to follow-up (169 months) for reporting.

Treatment Details

Study Objectives

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

Primary outcome measures

Number of Participants With Treatment-emergent Adverse Events (TEAEs)

Secondary outcome measures

Area Under the Curve Extrapolated to Infinity (AUC0-infinity) of Idursulfase Administered as Intrathecal and in Conjunction With Elaprase

Area Under the Curve From the Time of Dosing to the Last Measureable Concentration (AUC0-t) of Idursulfase Administered as Intrathecal and in Conjunction With Elaprase

Change From Baseline in CSF Biomarkers

+11 more

Trial Design

1Treatment groups

Experimental Treatment

Group I: Idursulfase-ITExperimental Treatment2 Interventions

Idursulfase-IT will be administered once monthly and weekly IV infusions of Elaprase at the dose used in study HGT-HIT-045 via intrathecal drug delivery device (IDDD).

0 / 1Eligibility criteria met

Find a Location

Who is running the clinical trial?

ShireLead Sponsor

456 Previous Clinical Trials

97,267 Total Patients Enrolled

13 Trials studying Hunter Syndrome

1,784 Patients Enrolled for Hunter Syndrome

Shire DirectorStudy DirectorTakeda

6 Previous Clinical Trials

3,976 Total Patients Enrolled

Shire PhysicianStudy DirectorShire

2 Previous Clinical Trials

238 Total Patients Enrolled

Media Library

Elaprase (Enzyme Replacement Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT01506141 — Phase 1 & 2

Hunter Syndrome Research Study Groups: Idursulfase-IT

Hunter Syndrome Clinical Trial 2023: Elaprase Highlights & Side Effects. Trial Name: NCT01506141 — Phase 1 & 2

Elaprase (Enzyme Replacement Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT01506141 — Phase 1 & 2

Frequently Asked Questions

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

May I partake in this clinical trial per the guidelines?

"This clinical trial accepts those between the ages of 3 and 18 that have been diagnosed with Hunter Syndrome. A maximum of 25 people can participate in this research."

Answered by AI

Is the eligibility criteria for this clinical trial limited to individuals aged 18 and above?

"This trial has a narrow age range requirement of 3 to 18 years old. Presently, 413 clinical trials exist for individuals below the legal age while 1,520 studies are available to patients above 65."

Answered by AI

Has this study been conducted before?

"To date, 6 trials of Idursulfase-IT have been conducted in 15 cities and 16 nations. The original trial was sponsored by Shire back in 2010 and included 25 participants who completed both phases 1 & 2 of the drug approval process. Subsequently, 7 additional studies were executed."

Answered by AI

Is recruitment currently taking place for this experimental research?

"Unfortunately, this trial is no longer recruiting new participants. It was first posted on August 1st 2010 and had its final update in April 26th 2022. If you are still searching for trials to participate in, there are 1959 clinical studies looking for people with hunter syndrome and 6 recruiting patients for Idursulfase-IT treatment."

Answered by AI

Can you elaborate on the experiments conducted with Idursulfase-IT?

"At this juncture, 6 Idursulfase-IT clinical trials are ongoing with 2 in Phase 3. Chapel Hill, North carolina is host to several of these studies while 39 other medical centres around the nation have active involvement as well."

Answered by AI

How many locales are administering this experiment?

"This clinical trial is currently enrolling participants in a variety of different medical centres, such as Seattle Children's Hospital (Washington), Ann & Robert H Lurie Childrens Hospital of Chicago (Illinois) and University of North carolina at Chapel Hill (North Carolina)."

Answered by AI

What is the aggregate figure of those participating in this investigation?

"Unfortunately, the current trial is not enrolling patients. It was initially posted on August 1st 2010 and last updated on April 26th 2022. However, there are 1959 studies actively accepting participants with hunter syndrome as well as 6 trials for Idursulfase-IT that require volunteers at this moment in time."

Answered by AI

Recent research and studies

Molecular Genetics and MetabolismJournal

A Phase I/II Clinical Trial of Enzyme Replacement Therapy in Mucopolysaccharidosis II (hunter Syndrome)

Muenzer, Joseph, Muge Gucsavas-Calikoglu, Shawn E. McCandless, Thomas J. Schuetz, and Alan Kimura. 2007. “A Phase I/II Clinical Trial of Enzyme Replacement Therapy in Mucopolysaccharidosis II (hunter Syndrome)”. Molecular Genetics and Metabolism. Elsevier BV. doi:10.1016/j.ymgme.2006.09.001.

Genetics in MedicineJournal

A Phase II/III Clinical Study of Enzyme Replacement Therapy with Idursulfase in Mucopolysaccharidosis II (hunter Syndrome)

Muenzer, Joseph, James E. Wraith, Michael Beck, Roberto Giugliani, Paul Harmatz, Christine M. Eng, Ashok Vellodi, et al.. 2006. “A Phase II/III Clinical Study of Enzyme Replacement Therapy with Idursulfase in Mucopolysaccharidosis II (hunter Syndrome)”. Genetics in Medicine. Elsevier BV. doi:10.1097/01.gim.0000232477.37660.fb.

Genetics in MedicineJournal

Long-term Open-label Phase I/II Extension Study of Intrathecal Idursulfase-it in the Treatment of Neuronopathic Mucopolysaccharidosis II

Muenzer, Joseph, Suresh Vijayaraghavan, Margot Stein, Shauna Kearney, Yuna Wu, and David Alexanderian. 2022. “Long-term Open-label Phase I/II Extension Study of Intrathecal Idursulfase-it in the Treatment of Neuronopathic Mucopolysaccharidosis II”. Genetics in Medicine. Elsevier BV. doi:10.1016/j.gim.2022.04.002.

Genetics in MedicineJournal