Clinical trial NCT00882921: An Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients
Official title: A Multi-Center Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients Enrolled in the Hunter Outcome Survey (HOS) Receiving Idursulfase Enzyme Replacement Therapy
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hunter Syndrome
Keywords: MPS 2
MPSII
hunter's disease treatment
enzyme replacement therapy
hunter's disease
iduronate 2 sulfatase
mps diagnosis
mps symptoms
iduronate sulfatase
MPS II treatment
chronic ear infection
hunters syndrome
mps ii therapy
MPS2
hunter's syndrome
Hunter syndrome
elaprase
lysosomal storage disease
hunter syndrome therapy
mps society
hunter syndrome treatment
hunter's syndrome treatment
mucopolysaccharides
mps ii
idursulfase
hunters disease
enlarged adenoids
ert treatment
hunter disease
lysosomal storage disorder
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2009-04-17
Enrollment: 26
Sex: MALE
Minimum age (years): 5
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with Hunter syndrome

Brief 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).

Detailed description: This study is being conducted to satisfy post-marketing commitments to monitor anti-idursulfase antibody development in Hunter syndrome patients after long-term idursulfase enzyme replacement therapy. The study will be conducted as a sub-study within the Hunter Outcome Survey (HOS). Hunter syndrome patients in the HOS who have previously received idursulfase as well as treatment-naive patients who will begin idursulfase treatment within 30 days of study enrollment will be included.

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.

Primary outcomes:
measure: Infusion-Related Adverse Event (IRAE) Rates Between IgG Anti-idursulfase Antibody Positive (Ab+) and Anti-idursulfase IgG Antibody Negative (Ab-) Patients
timeFrame: Baseline to 109 Weeks
Secondary outcomes:
measure: Change From Baseline in uGAG Levels to 109 Weeks
timeFrame: Baseline to 109 Weeks
Other outcomes:
Locations:
city: Oakland
country: United States
facility: Children's Hospital & Research Center Oakland
state: California
city: Minneapolis
country: United States
facility: Children's Hospitals and Clinics of Minnesota, Division of Genetics
state: Minnesota
city: Porto Alegre
country: Brazil
facility: Hospital de Clinicas de Porto Alegre, Servico de Genetica Medica
state: RS
city: Birmingham
country: United Kingdom
facility: Birmingham Children's Hospital
city: London
country: United Kingdom
facility: Great Ormond Street Hospital
city: Manchester
country: United Kingdom
facility: Central Manchester University Hospitals, St. Mary's Hospital
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References:
citation: Giugliani R, Harmatz P, Jones SA, Mendelsohn NJ, Vellodi A, Qiu Y, Hendriksz CJ, Vijayaraghavan S, Whiteman DA, Pano A. Evaluation of impact of anti-idursulfase antibodies during long-term idursulfase enzyme replacement therapy in mucopolysaccharidosis II patients. Mol Genet Metab Rep. 2017 Feb 21;12:2-7. doi: 10.1016/j.ymgmr.2017.01.014. eCollection 2017 Sep.