← Back to Search

Enzyme Replacement Therapy

Elaprase IV Infusion for Hunter Syndrome

Phase 4
Waitlist Available
Research Sponsored by Shire
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Retrospective Data Inclusion Criteria: The patient is male
Prospective Patient Group: The patient must have a documented diagnosis of MPS II. Acceptable diagnostic criteria include: a deficiency in I2S enzyme activity of ≤10% of the lower limit of the normal range as measured in plasma, fibroblasts, or leukocytes AND a documented mutation in the I2S gene OR a normal enzyme activity level of one other sulfatase as measured in plasma, fibroblasts, or leukocytes
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline to end-of-study (5-10 years)
Awards & highlights

Study Summary

This trial will provide Elaprase treatment to children with MPS II and compare their growth to data from the HOS patient registry.

Who is the study for?
This trial is for boys under 6 with Hunter syndrome (MPS II) who haven't had Elaprase before. They must have a confirmed diagnosis and agree to data collection. Boys can't join if they've had blood transfusions, growth treatments, or other recent investigational drugs.Check my eligibility
What is being tested?
The study tests the long-term effects of Elaprase IV infusions on height and weight in young boys with MPS II. It combines new patient data with existing records from the Hunter Outcome Survey to analyze growth patterns.See study design
What are the potential side effects?
Elaprase may cause allergic reactions, including hives, itching, difficulty breathing; infusion-related reactions like headache, fever; and possibly affect heart rate and blood pressure.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I am a male patient.
Select...
I have been diagnosed with MPS II based on specific enzyme activity tests or gene mutation.
Select...
I have never received Elaprase before.
Select...
I am under 6 years old and starting Elaprase treatment.
Select...
I am a male patient.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline to end-of-study (5-10 years)
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline to end-of-study (5-10 years) 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 in clinical laboratory testings as measured by hematology to evaluate long-term safety in patients
Change in clinical laboratory testings as measured by serum chemistry to evaluate long-term safety in patients
Change in clinical laboratory testings as measured by urinalysis to evaluate long-term safety in patients
+4 more
Secondary outcome measures
Adaptive behavior, as measured by the Vineland Adaptive Behavior Scales (VABS II)
Change in anti-idursulfase antibodies in serum
Distance walked, as measured by the Six Minute Walk Test (6MWT)
+5 more

Trial Design

1Treatment groups
Experimental Treatment
Group I: Enrolled PatientsExperimental Treatment1 Intervention
Patients who are receiving treatment with Elaprase in this study (SHP-ELA-401), who are <6 years of age, and were previously treatment-naïve. Patients who are not enrolled in this study (SHP-ELA-401) but are enrolled in the Hunter Outcome Survey (HOS) patient registry and were < 6 years of age at start of Elaprase treatment. While not enrolled in the present Study SHP-ELA-401, their height and weight data from HOS will be utilized in the Primary Growth Analysis for this study.

Find a Location

Who is running the clinical trial?

ShireLead Sponsor
456 Previous Clinical Trials
97,271 Total Patients Enrolled
13 Trials studying Hunter Syndrome
1,788 Patients Enrolled for Hunter Syndrome
Takeda Development Center Americas, Inc.Industry Sponsor
56 Previous Clinical Trials
12,009 Total Patients Enrolled
1 Trials studying Hunter Syndrome
5 Patients Enrolled for Hunter Syndrome
TakedaLead Sponsor
1,202 Previous Clinical Trials
4,178,241 Total Patients Enrolled
4 Trials studying Hunter Syndrome
18 Patients Enrolled for Hunter Syndrome

Media Library

Elaprase (Enzyme Replacement Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT02455622 — Phase 4
Hunter Syndrome Research Study Groups: Enrolled Patients
Hunter Syndrome Clinical Trial 2023: Elaprase Highlights & Side Effects. Trial Name: NCT02455622 — Phase 4
Elaprase (Enzyme Replacement Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02455622 — Phase 4

Frequently Asked Questions

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

How many participants have enrolled in this medical experiment?

"This medical trial is not currently accepting participants. It was initially published on October 28th 2015 and most recently updated on May 5th 2022. For those seeking out other studies, there are presently 20 trials actively enrolling patients with Hunter Syndrome and 6 trials for Elaprase via intravenous infusion that require volunteers."

Answered by AI

Has this experiment been conducted before in a similar capacity?

"Presently, 6 trials are underway to assess the effects of intravenous Elaprase infusions. The initial investigation into this therapy was undertaken in 2010 by Shire and included 25 individuals who successfully navigated through Phase 1 & 2 drug approval stages. Since then, an additional 7 research projects have been initiated around the world across 16 nations at 15 different locations."

Answered by AI

Has the FDA sanctioned Elaprase for intravenous administration?

"Elaprase for intravenous (IV) infusion has been approved, thus its safety was rated a 3."

Answered by AI

Have there been any past explorations utilizing Elaprase through intravenous delivery?

"Currently, 6 Elaprase trials that involve intravenous infusion are underway. Two of these medical studies have reached the Phase 3 stage and 39 locations across America offer this treatment. Notably, many of them are located in Chapel Hill, North carolina."

Answered by AI

Are there still spaces available in the clinical trial?

"Unfortunately, this project is not currently enrolling patients. The trial was initially opened on October 28th 2015 and the last edit to its information occurred May 5th 2022. Fortunately, at present there are 26 other studies actively seeking participants for clinical trials."

Answered by AI
Recent research and studies
clinicaltrials.govStudy
Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of Age
2015. "Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of Age". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT02455622.
~3 spots leftby Jul 2025
Unbiased ResultsWe believe in providing patients with all the options.
Your Data Stays Your DataWe only share your information with the clinical trials you're trying to access.
Verified Trials OnlyAll of our trials are run by licensed doctors, researchers, and healthcare companies.
Back to top