N/A
Completed N=33
Screening Study to Identify Pediatric Patients With Hunter Syndrome Who Demonstrate Evidence of Central Nervous System (CNS) Involvement and Who Are Currently Receiving Treatment With Elaprase®
Hunter Syndrome
Source: ClinicalTrials.gov NCT00937794 ↗
Enrolled (actual)
33
Serious AEs
0.0%
Results posted
May 2016
Primary outcomePrimary: Number of Participants Who Were Screened For The Follow-On Study With an Investigational Agent — 17 participants
Summary
This study is being conducted to identify pediatric patients with Hunter syndrome who have neurodevelopmental disease characteristics, who are currently receiving treatment with Elaprase, and who may be suitable to participate in a clinical study with an investigational agent.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants Who Were Screened For The Follow-On Study With an Investigational Agent |
17 | — |
| PRIMARY Number of Participants With a Score of at Least 90 on The General Conceptual Ability (GCA) Sub-Scale of The Differential Ability Scale (DAS) |
5 | — |
Eligibility Criteria
Inclusion Criteria
- The patient is male and is ≥3 and <18 years of age
- The patient is currently receiving weekly IV infusions of Elaprase.
- The patient, patient's parent(s), or legally authorized guardian(s) has voluntarily signed an Institutional Review Board / Independent Ethics Committee-approved informed consent form after all relevant aspects of the study have been explained and discussed with the patient. The guardians' consent and subject's assent, as relevant, must be obtained.
Exclusion Criteria
- The patient has a CNS shunt.
- The patient has received a hematopoietic stem cell transplant.
- The patient is currently enrolled in a clinical trial.
- The patient has a significant medical or psychiatric comorbidity(ies) that might affect study data or confound the integrity of study results.
Data sourced from ClinicalTrials.gov (NCT00937794). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.