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

NCT00607386 | Completed Phase 4 Results

• 2 other identifiers: HGT-ELA-0382007-006044-22
• Study Type: interventional
• Target: 28
• Locations: 3 countries
• Sites: 3

Brief 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.

Conditions

Hunter Syndrome; Mucopolysaccharidosis II; MPS II

Keywords

Hunter syndrome; hunters syndrome; hunter's syndrome; hunter disease; hunters disease; hunter's disease; MPS II; MPSII; MPS2; MPS 2; mucopolysaccharides; lysosomal storage disease; lysosomal storage disorder; chronic ear infection; enlarged adenoids; mps symptoms; mps diagnosis; 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; hunter syndrome therapy; hunter's disease treatment; mps society

Outcome Measures

Primary Outcomes (1)

• Safety Evaluation

  An adverse event (AE) was defined as any untoward medical occurrence in a clinical investigation participant administered as a pharmaceutical product that did not necessarily have a causal relationship with this treatment. A serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. Number of participants with AEs occurred after start of study treatment until 30 days after the last infusion of idursulfase, were reported.

  From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks

Secondary Outcomes (9)

• Mean Change From Baseline to Week 53 in Normalized Urinary Glycosaminoglycan (GAG) Levels

  Baseline, Weeks 18, 36 and 53

• Single- and Repeat-Dose Pharmacokinetics - Maximum Observed Serum Concentration (Cmax)

  Weeks 1 and 27

• Single- and Repeat-Dose Pharmacokinetics - Time of Maximum Observed Serum Concentration (Tmax)

  Weeks 1 and 27

• Single- and Repeat-Dose Pharmacokinetics - Area Under the Serum Concentration-Time Curve From Time 0 to the Final Time Point With a Concentration of at Least Lower Limit of Quantitation (AUClast)

  Weeks 1 and 27

• Single- and Repeat-Dose Pharmacokinetics - Area Under the Serum Concentration-Time Curve From Time 0 to Infinity (AUCinf)

  Weeks 1 and 27

Study Arms (1)

Idursulfase

OTHER

Open-label treatment with idursulfase

Biological: Idursulfase

Interventions

Idursulfase BIOLOGICAL

Solution for intravenous infusion, 0.5 mg/kg weekly

Also known as: Elaprase

Idursulfase

Eligibility Criteria

• Age: Up to 5 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• The patient has a diagnosis of Hunter syndrome based upon biochemical criteria either documented in their medical history or established at Screening:
• 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
• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Shire: lead
• Covance: collaborator
• PharmaNet: collaborator
• PRA Health Sciences: collaborator

Study Sites (3)

Hospital de Clinicas de Porto Alegre, Servico de Genetica Medica

Porto Alegre, Rio Grande do Sul, 90035-903, Brazil

Location

Instytut Pomnik Centrum Zdrowia Dziecka, Klinika Chorob Metaboliczynch, Endokrynologii i Diabetologii

Warsaw, 04-730, Poland

Location

National Taiwan University Hospital, Dept. of Pediatrics and Medical Genetics

Taipei, 10016, Taiwan

Location

Related Publications (3)

• 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. doi: 10.1016/j.ymgme.2006.09.001. Epub 2006 Dec 20.

  PMID: 17185020 BACKGROUND

• 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. doi: 10.1097/01.gim.0000232477.37660.fb.

  PMID: 16912578 BACKGROUND

• Pano A, Barbier AJ, Bielefeld B, Whiteman DA, Amato DA. Immunogenicity of idursulfase and clinical outcomes in very young patients (16 months to 7.5 years) with mucopolysaccharidosis II (Hunter syndrome). Orphanet J Rare Dis. 2015 Apr 24;10:50. doi: 10.1186/s13023-015-0265-2.

  PMID: 25902842 RESULT

MeSH Terms

Conditions

Mucopolysaccharidosis II; Sudden Infant Death; Lysosomal Storage Diseases

Interventions

idursulfase

Condition Hierarchy (Ancestors)

X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Heredodegenerative Disorders, Nervous System; Mucopolysaccharidoses; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Mucinoses; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Death, Sudden; Death; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Infant Death

Results Point of Contact

• Title: Study Director
• Organization: Shire

ClinicalTransparency@shire.com

+1 866 842 5335

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 22, 2008
• First Posted: February 5, 2008
• Study Start: December 31, 2007
• Primary Completion: July 8, 2011
• Study Completion: July 8, 2011
• Last Updated: June 8, 2021
• Results First Posted: November 7, 2013

Record last verified: 2021-05

Locations

PL (1); TW (1); BR (1)