Early enzyme therapy may help kids with rare disease grow better
NCT ID NCT02455622
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.
Disclaimer
Read more
Show less
This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for HUNTER SYNDROME are added.
By submitting, you agree to our Terms of use
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
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