Trial record 18 of 35 for:    SIDS

Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy

This study has been completed.
Sponsor:
Collaborators:
Covance
PharmaNet
PRA International
Information provided by (Responsible Party):
Shire
ClinicalTrials.gov Identifier:
NCT00607386
First received: January 22, 2008
Last updated: February 19, 2014
Last verified: February 2014
  Purpose

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.


Condition Intervention Phase
Hunter Syndrome
Mucopolysaccharidosis II
MPS II
Biological: Idursulfase
Phase 4

Study Type: Interventional
Study Design: Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Shire:

Primary Outcome Measures:
  • Safety Evaluation [ Time Frame: 53 weeks ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Mean Change From Baseline to Week 53 in Normalized Urinary Glycosaminoglycan (GAG) Levels (μg/mg Creatinine) [ Time Frame: 53 weeks ] [ Designated as safety issue: No ]
    Analysis of urinary GAG levels was performed at baseline, Week 18, Week 36, and Week 53 as an assessment of the pharmacodynamic effects of Elaprase (idursulfase) in this patient population.

  • Single- and Repeat-Dose Pharmacokinetics - Maximum Observed Serum Concentration (Cmax) [ Time Frame: Weeks 1 and 27 ] [ Designated as safety issue: No ]
  • Single- and Repeat-Dose Pharmacokinetics - Time of Maximum Observed Serum Concentration (Tmax) [ Time Frame: Weeks 1 and 27 ] [ Designated as safety issue: No ]
  • Single- and Repeat-Dose Pharmacokinetics - Area Under the Serum Concentration-Time Curve From Time 0 to the Final Time Point With a Concentration ≥ LOQ (AUClast) [ Time Frame: Weeks 1 and 27 ] [ Designated as safety issue: No ]
  • Single- and Repeat-Dose Pharmacokinetics - Area Under the Serum Concentration-Time Curve From Time 0 to Infinity (AUCinf) [ Time Frame: Weeks 1 and 27 ] [ Designated as safety issue: No ]
  • Single- and Repeat-Dose Pharmacokinetics - Elimination Half-Life (t1/2) [ Time Frame: Weeks 1 and 27 ] [ Designated as safety issue: No ]
  • Single- and Repeat-Dose Pharmacokinetics - Mean Residence Time From Time 0 to Infinity (MRTinf) [ Time Frame: Weeks 1 and 27 ] [ Designated as safety issue: No ]
  • Single- and Repeat-Dose Pharmacokinetics - Clearance (CL) [ Time Frame: Weeks 1 and 27 ] [ Designated as safety issue: No ]
  • Single- and Repeat-Dose Pharmacokinetics - Volume of Distribution at Steady State (Vss) [ Time Frame: Weeks 1 and 27 ] [ Designated as safety issue: No ]

Enrollment: 28
Study Start Date: December 2007
Study Completion Date: July 2011
Primary Completion Date: July 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Idursulfase
Open-label treatment with idursulfase
Biological: Idursulfase
Solution for intravenous infusion, 0.5 mg/kg weekly
Other Name: Elaprase

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

Ages Eligible for Study:   up to 5 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00607386

Locations
Brazil
Hospital de Clinicas de Porto Alegre, Servico de Genetica Medica
Porto Alegre, RS, Brazil, 90035-903
Poland
Instytut Pomnik Centrum Zdrowia Dziecka, Klinika Chorob Metaboliczynch, Endokrynologii i Diabetologii
Warsaw, Poland, 04-730
Taiwan
National Taiwan University Hospital, Dept. of Pediatrics and Medical Genetics
Taipei, Taiwan, 10016
Sponsors and Collaborators
Shire
Covance
PharmaNet
PRA International
Investigators
Study Director: Arian Pano, MD, MPH Shire Human Genetic Therapies, Inc.
Principal Investigator: Roberto Giugliani, MD, PhD Hospital de Clinicas de Porto Alegre
Principal Investigator: Wuh-Liang Hwu, MD, PhD National Taiwan University Hospital
Principal Investigator: Anna Tylki-Szymanska, MD, PhD Instytut Pomnik Centrum Zdrowia Dziecka
  More Information

Publications:
Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT00607386     History of Changes
Other Study ID Numbers: HGT-ELA-038
Study First Received: January 22, 2008
Results First Received: September 3, 2013
Last Updated: February 19, 2014
Health Authority: United States: Food and Drug Administration
Taiwan: Department of Health
Brazil: National Health Surveillance Agency
Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products

Keywords provided by Shire:
MPS2
MPS 2
mps 2
Hunter syndrome
hunters syndrome
hunter's syndrome
hunter disease
hunters disease
hunter's disease
MPS II
MPSII
mps ii
mucopolysaccharides
lysosomal storage disease
lysosomal storage disorder
chronic ear infection
enlarged adenoids
mps symptoms
mps diagnosis
mps ii therapy
MPS II therapy
MPS II treatment
ert treatment
elaprase
idursulfase
iduronate sulfatase
iduronate 2 sulfatase
enzyme replacement therapy
hunter syndrome treatment
hunter's syndrome treatment

Additional relevant MeSH terms:
Mucopolysaccharidoses
Mucopolysaccharidosis II
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
Metabolic Diseases
Mental Retardation, X-Linked
Mental Retardation
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Genetic Diseases, X-Linked
Heredodegenerative Disorders, Nervous System

ClinicalTrials.gov processed this record on April 21, 2014