N/A
N=26
An Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients
Hunter Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT00882921 ↗Enrolled (actual)
26
Serious AEs
61.5%
Results posted
Jul 2014
Primary outcome: Primary: Infusion-Related Adverse Event (IRAE) Rates Between IgG Anti-idursulfase Antibody Positive (Ab+) and Anti-idursulfase IgG Antibody Negative (Ab-) Patients — 0.0121; 0.0042; 0.0055; 0.0026 IRAE/Week — p=0.1309
Study Design & Population
- Study type
- Observational
- Phase
- N/A
- Interventions
- Idursulfase (Biological)
- Age
- Pediatric, Adult, Older Adult · 5+ yrs
- Sex
- Male
- Sponsor
- Shire
- Primary completion
- Feb 2013
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Infusion-Related Adverse Event (IRAE) Rates Between IgG Anti-idursulfase Antibody Positive (Ab+) and Anti-idursulfase IgG Antibody Negative (Ab-) Patients |
0.0121; 0.0042; 0.0055; 0.0026 | 0.1309 |
| SECONDARY Change From Baseline in uGAG Levels to 109 Weeks |
-74.07 | — |
Summary
The objective of this study is to evaluate the effect of anti-idursulfase antibodies on idursulfase safety (measured by infusion related adverse events) between patients who develop anti-idursulfase antibodies and patients who do not after long-term idursulfase enzyme replacement therapy (ERT).
Eligibility Criteria
Inclusion Criteria
Patients must meet all of the following criteria to be considered eligible for enrollment:
- The patient is male and enrolled in the HOS (i.e., meets the entry criteria of a documented diagnosis of Hunter syndrome)
- The patient is ≥ 5 years-old
- The patient is on idursulfase treatment or scheduled to begin idursulfase treatment within 30 days of study enrollment
- The patient, patient's parent(s), or patient's legally authorized guardian must have voluntarily signed an Institutional Review Board (IRB)/Independent Ethics Committee (IEC)-approved informed consent form after all relevant aspects of the study have been explained and discussed with the patient, patient's parent(s), or patient's legally authorized guardian.
Exclusion Criteria
Patients who meet any of the following criteria are not eligible for this study:
- The patient has received biologic/ERT products other than idursulfase, or other investigational product(s) for any reason within 30 days prior to study entry.
- The patient has a life expectancy of < 2 years
- The patient is unable to comply with the protocol, e.g., has a clinically relevant medical condition making implementation of the protocol difficult; has an uncooperative attitude; is unable to return for safety evaluations; or is otherwise unlikely to complete the study, as determined by the Investigator.
Data sourced from ClinicalTrials.gov (NCT00882921). 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.