Early enzyme therapy may help kids with rare disease grow better

NCT ID NCT02455622

Completed Disease control Sponsor: Shire Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study followed 21 boys with Hunter syndrome who started taking Elaprase before age 6. Researchers tracked their height and weight for at least 5 years to see if the drug helps them grow more like other children. The study also monitored safety and compared growth data to untreated patients from a registry.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: Elaprase (idursulfase)
What this could lead to: If successful, this study could show that early treatment with Elaprase helps children with Hunter syndrome grow more normally.
What could go wrong: This is a small, completed Phase 4 study with only 21 participants, so results may not apply to all patients. Elaprase can cause infusion reactions and other side effects.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

mucopolysaccharidosis type 2

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

HUNTER SYNDROME

Contacts and locations

Locations

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States
Chulalongkorn University

Bangkok, 10330, Thailand
Hospital Infantil Dr Robert Reid Cabral

Santo Domingo, 10101, Dominican Republic
Hospital Kuala Lumpur

Kuala Lumpur, 50586, Malaysia
Mother and Child Health Care Institute of Serbia Dr Vukan Cupic

Belgrade, 11000, Serbia
National Pediatrics Hospital

Hanoi, Vietnam
Philippine General Hospital

Manila, 1000, Philippines
Universitätsmedizin der Johannes Gutenberg-Universität Mainz

Mainz, 55131, Germany

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