Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy
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; Mucopolysaccharidosis II; MPS II
Intervention: Idursulfase (Biological)
Phase: Phase 4
Status: Recruiting
Sponsored by: Shire Human Genetic Therapies, Inc. Official(s) and/or principal investigator(s):
David AH Whiteman, MD, Principal Investigator, Affiliation: Shire Human Genetic Therapies, Inc.
Overall contact:
Tiffany Crump, Phone: 484-595-8850, Email: tcrump@shire.com
Summary
The objective of this study is to determine the safety of once weekly dosing of idursulfase
0. 5 mg/kg administered by intravenous (IV) infusion for male Hunter syndrome patients ≤ 5
years old.
Clinical Details
Official title: A Multi-Center, Open-Label Study Evaluating Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Enzyme Replacement Therapy
Study design: Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Primary outcome: Incidence of adverse events (including infusion-related adverse events), changes in 12-lead ECG, vital signs, standard laboratory parameters, and anti-idursulfase antibody status.
Secondary outcome: Mean change from Baseline to Week 53 in urinary GAG clearance (normalized for μg GAG/mg creatinine)Single-dose and repeat-dose pharmacokinetic parameters
Detailed description:
This study will provide a basis for evaluating the safety of idursulfase administered to
Hunter syndrome patients who are ≤ 5 years old. Additionally, this study will provide a
basis for evaluating the idursulfase single- and repeated-dose pharmacokinetic profiles as
well as the pharmacodynamic effect (as measured by urinary GAG excretion) in this pediatric
population. Additional exploratory measures will include abdominal ultrasound measurements
of liver and spleen volumes, assessments of growth with comparisons to normal population
growth data, assessments of annualized growth velocity, assessments of routine developmental
milestones using the Denver II, and assessments of clinical events, including the first
occurrence of certain hearing-related events (e. g., hearing loss, otitis media),
respiratory-related events (e. g., upper and lower respiratory infections), and specific
surgical procedures (e. g., adenoidectomy, placement of PE tubes).
All patients in this open-label study will receive once-weekly infusions of idursulfase at a
dose of 0. 5 mg/kg.
Eligibility
Minimum age: N/A.
Maximum age: 5 Years.
Gender(s): Male.
Criteria:
Inclusion Criteria:
- The patient has a diagnosis of Hunter syndrome based upon biochemical criteria either
documented in their medical history or established at Screening:
1. A deficiency in iduronate-2-sulfatase (I2S) enzyme activity of ≤ 10 % of the
lower limit of the normal range as measured in plasma, fibroblasts, or
leukocytes (based on normal range of measuring laboratory)
AND
2. A normal enzyme activity level of one other sulfatase as measured in plasma,
fibroblasts, or leukocytes (based on normal range of measuring laboratory).
- The patient is 5 years of age and under.
- The patient is male.
- The patient's parent(s), or patient's legal guardian must have voluntarily signed an
Institutional Review Board approved informed consent form after all relevant aspects
of the study have been explained and discussed with the patient's parent(s), or the
patient's legal guardian.
Exclusion Criteria:
- The patient has received treatment with another investigational therapy within 30
days prior to enrollment.
- The patient has clinically relevant medical condition(s) making implementation of the
protocol difficult.
- The patient has previously received idursulfase.
- The patient has known hypersensitivity to any of the components of idursulfase.
- The patient has had a tracheostomy.
Locations and Contacts
Tiffany Crump, Phone: 484-595-8850, Email: tcrump@shire.com
Klinika Chorob Metaboliczynch, Endokrynologii i Diabetologii, Warsaw 04-730, Poland; Recruiting
Anna Tylki-Szymanska, MD, Principal Investigator
National Taiwan University Hospital, Dept. of Pediatrics and Medical Genetics, Taipei 10016, Taiwan; Completed
Hospital de Clinicas de Porto Alegre, Servico de Genetica Medica, Porto Alegre, RS 90035-003, Brazil; Recruiting
Roberto Giugliani, MD, Principal Investigator
Additional Information
Related publications: Muenzer J, Gucsavas-Calikoglu M, McCandless SE, Schuetz TJ, Kimura A. A phase I/II clinical trial of enzyme replacement therapy in mucopolysaccharidosis II (Hunter syndrome). Mol Genet Metab. 2007 Mar;90(3):329-37. Epub 2006 Dec 20. Muenzer J, Wraith JE, Beck M, Giugliani R, Harmatz P, Eng CM, Vellodi A, Martin R, Ramaswami U, Gucsavas-Calikoglu M, Vijayaraghavan S, Wendt S, Puga AC, Ulbrich B, Shinawi M, Cleary M, Piper D, Conway AM, Kimura A. A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome). Genet Med. 2006 Aug;8(8):465-73. Erratum in: Genet Med. 2006 Sep;8(9):599. Wendt, Suzanne [corrected to Wendt, Susanne]; Puga, Antonio [corrected to Puga, Ana Cristina]; Conway, Ann Marie [corrected to Conway, Anne Marie].
Starting date: December 2007
Last updated: June 2, 2010
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