An Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients
Information source: Shire Human Genetic Therapies, Inc.
Information obtained from ClinicalTrials.gov on October 04, 2010
Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Hunter Syndrome
Phase: N/A
Status: Recruiting
Sponsored by: Shire Human Genetic Therapies, Inc. Official(s) and/or principal investigator(s):
Paul R Harmatz, MD, Principal Investigator, Affiliation: Children's Hospital & Research Center Oakland
Ed J Wraith, Principal Investigator, Affiliation: Centraol Manchester University Hospitals, St. Mary's Hospital
Chris Hendriksz, MD, Principal Investigator, Affiliation: Birmingham Children's Hospital
Porta Alegre, RS, Principal Investigator, Affiliation: Hospital de Clinicas de Porto Alegre
Ed J Wraith, Prof, Principal Investigator, Affiliation: Central Manchester University Hospitals, St. Mary's Hospital
Nancy Mendelsohn, MD, Principal Investigator, Affiliation: Children's Hospitals and Clinics of Minnesota
Overall contact:
Tiffany Crump, Phone: 484-595-8850, Email: tcrump@shire.com
Summary
The objective of this study is to evaluate the effect of anti-idursulfase IgG, IgM & IgE
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.
Clinical Details
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 design: Observational Model: Cohort, Time Perspective: Prospective
Primary outcome: To evaluate the relative risk of experiencing an infusion-related adverse event given anti-idursulfase antibody positive status relative to anti-idursulfase antibody negative status.
Secondary outcome: To measure the mean (and percent) difference in urinary GAG level between the groups of IgG anti-idursulfase antibody positive and anti-idursulfase IgG antibody negative patients.
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
Minimum age: 5 Years.
Maximum age: N/A.
Gender(s): Male.
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.
Locations and Contacts
Tiffany Crump, Phone: 484-595-8850, Email: tcrump@shire.com
Birmingham Children's Hospital, Birmingham B46NH, United Kingdom; Recruiting
Chris Hendriksz, MD, Phone: +44 121 333 9907, Email: Chris.hendriksz@bch.nhs.uk
Catherine Little, Phone: +44 7917 175 500, Email: Catherine.little@bch.nhs.uk
St. Mary's Hospital, Manchester M139WLUK, United Kingdom; Recruiting
Ed Wraith, Phone: 00441617012137, Ext: 8, Email: Ed.Wraith@cmft.nhs.uk
Sarah Phillippo, Phone: 01617019261, Email: Sarah.Phillippo@cmft.nhs.uk
Ed J Wraith, Principal Investigator
Children's Hospital & Research Center Oakland, Oakland, California 94609, United States; Recruiting
Paul Richard Harmatz, MD, Phone: 510-428-3885, Email: pharmatz@mail.cho.org
Jo Ann Johnson, Phone: 510-428-3885, Ext: 5421, Email: jajohnson@mail.cho.org
Paul R Harmatz, MD, Principal Investigator
Children's Hospitals and Clinics of Minnesota Division of Genetics, Minneapolis, Minnesota 55404, United States; Recruiting
Nancy Mendelsohn, MD, Phone: 612-813-6360, Email: Nancy.Mendelsohn@childrensmn.org
Kristi Jarvis, Phone: 612-813-6661, Email: Kristi.Jarvis@childrensmn.or
Nancy Mendelsohn, MD, Principal Investigator
Hospital de Clinicas de Porto Alegre, Servico de Genetica Medica, Porta Alegre, RS, Brazil; Recruiting
Roberto Giugliani, MD, Phone: +55 51 3316 8011, Email: rgiugliani@hcpa.ufrgs.br
Taiane A Vieira, Phone: +55 51 3333 2125, Email: tavieira@hcpa@ufrgs.br
Additional Information
Starting date: October 2008
Last updated: July 12, 2010
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