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Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT02455622
Other study ID # SHP-ELA-401
Secondary ID 2014-004804-31
Status Active, not recruiting
Phase Phase 4
First received
Last updated
Start date October 28, 2015
Est. completion date July 9, 2025

Study information

Verified date July 2023
Source Takeda
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

This long-term study will provide Elaprase treatment to children enrolled in this study and will utilize data from both enrolled patients and Hunter Outcome Survey (HOS) patient registry data to conduct the primary growth analysis to assess changes in height and weight in patients with Mucopolysaccharidosis II (Hunter syndrome) MPS II.


Recruitment information / eligibility

Status Active, not recruiting
Enrollment 21
Est. completion date July 9, 2025
Est. primary completion date July 9, 2025
Accepts healthy volunteers No
Gender Male
Age group N/A to 6 Years
Eligibility Inclusion Criteria: - Group 1: Prospective Patient Group 1. The patient is male. 2. The patient is Elaprase-naïve at study entry. 3. The patient must have a documented diagnosis of MPS II. Of the 3 criteria below, the combinations (3a AND 3b) or (3a AND 3c) will be accepted as diagnostic of MPS II: 1. The patient has a deficiency in I2S enzyme activity of =10% of the lower limit of the normal range as measured in plasma, fibroblasts, or leukocytes (based on the reference laboratory's normal range). AND 2. The patient has a documented mutation in the I2S gene. OR 3. The patient has a normal enzyme activity level of one other sulfatase as measured in plasma, fibroblasts, or leukocytes (based on the normal range of measuring laboratory). 4. The patient will be <6 years of age at the start of Elaprase treatment. 5. The patient, patient's parent(s) or legally authorized guardian(s) 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. Consent of the patient's parent(s) or legally authorized guardian(s) and the patient's assent, as relevant, must be obtained. Group 2: Retrospective Data Inclusion Criteria: Retrospective Patient Group patients will be enrolled in HOS and not Study SHP-ELA-401; however, their growth data may be included in the analysis for Study SHP-ELA-401 if the following data inclusion criteria are met. 1. The patient is male. 2. The patient is enrolled in HOS. 3. The patient was <6 years of age at the start of Elaprase treatment. 4. The patient received Elaprase weekly treatment for at least 5 years. 5. The patient had a height assessment and a weight assessment documented within 3 months before or after Elaprase treatment start. 6. The patient has had annual height and weight assessments from start of Elaprase through age 10 years. 7. The patient, patient's parent(s), or legally authorized guardian(s) agree(s) to data collection. 8. The patient, patient's parent(s), or legally authorized guardian(s) must have signed an IRB/IEC-approved informed consent form after all relevant aspects of the HOS study have been explained and discussed. Consent of the patient's parent(s) or legally authorized guardian(s) and the patient's assent, as relevant, must be obtained. Exclusion Criteria: - Group 1: Prospective Patient Group 1. The patient has received treatment with any investigational drug or device within the 30 days prior to study entry. 2. The patient has received or is receiving treatment with idursulfase-IT. 3. The patient has received growth hormones, a cord blood infusion, or a bone marrow transplant at any time. 4. The patient has received blood product transfusions within 90 days prior to Screening. 5. The patient is unable to comply with the protocol as determined by the Investigator. Group 2: Retrospective Data Exclusion Criteria: HOS patients that meet the following criteria are not eligible to be included into the Study SHP-ELA-401 Primary Growth Analysis: 1. Patient was treated with growth hormone or other medications or interventions intended to promote growth in the time period covered by the analysis.

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
Elaprase for intravenous (IV) infusion
Patients enrolled in this study will receive once-weekly IV infusions of Elaprase at a dose of 0.5 mg/kg and will be followed for a minimum of 5 years after initiation of Elaprase treatment, or until they reach their 10th birthday, whichever is longer.Height and weight data from HOS will be utilized in the Primary Growth Analysis for this study.

Locations

Country Name City State
Dominican Republic Hospital Infantil Dr Robert Reid Cabral Santo Domingo
Germany Universitätsmedizin der Johannes Gutenberg-Universität Mainz Mainz
Malaysia Hospital Kuala Lumpur Kuala Lumpur
Philippines Philippine General Hospital Manila
Serbia Mother and Child Health Care Institute of Serbia Dr Vukan Cupic Beograd
Thailand Chulalongkorn University Bangkok
United States Ann & Robert H. Lurie Children's Hospital of Chicago Chicago Illinois
Vietnam National Pediatrics Hospital Hanoi

Sponsors (2)

Lead Sponsor Collaborator
Takeda Takeda Development Center Americas, Inc.

Countries where clinical trial is conducted

United States,  Vietnam,  Dominican Republic,  Germany,  Malaysia,  Philippines,  Serbia,  Thailand, 

Outcome

Type Measure Description Time frame Safety issue
Primary Change in height Screening to End-of-Study (5-10 years)
Primary Change in weight Z-scores Screening to End-of-Study (5-10 years)
Primary Change in neurological examination to evaluate long-term safety in patient Screening to End-of-Study (5-10 years)
Primary Number of participants with adverse events as measured by type, severity, and relationship to treatment Screening to End-of-Study (5-10 years)
Primary Change in clinical laboratory testings as measured by urinalysis to evaluate long-term safety in patients Screening to End-of-Study (5-10 years)
Primary Change in clinical laboratory testings as measured by serum chemistry to evaluate long-term safety in patients Screening to End-of-Study (5-10 years)
Primary Change in clinical laboratory testings as measured by hematology to evaluate long-term safety in patients Screening to End-of-Study (5-10 years)
Secondary Urinary glycosaminoglycans (uGAG) levels normalized to urine creatinine Screening to End-of-Study (5-10 years)
Secondary Normalized uGAG divided by upper limit of normal for age (uGAG/ULN) every 12 months Baseline to End-of-Study (5-10 years)
Secondary Joint mobility, as measured by Joint Range of Motion (JROM) scores, including global, upper-limb, and lower-limb joint scores Screening to End-of-Study (5-10 years)
Secondary Distance walked, as measured by the Six Minute Walk Test (6MWT) Screening to End-of-Study (5-10 years)
Secondary Quality of Life, as measured by the Hunter-Syndrome Functional Outcome in Clinical Understanding Scale Screening to End-of-Study (5-10 years)
Secondary Impact of illness on ability to function in daily life, as measured by the Childhood Health Assessment Questionnaire (CHAQ Parent Report) Screening to End-of-Study (5-10 years)
Secondary Adaptive behavior, as measured by the Vineland Adaptive Behavior Scales (VABS II) Screening to End-of-Study (5-10 years)
Secondary Change in anti-idursulfase antibodies in serum number and percentage of patients testing anti-idursulfase antibody positive and negative at each time point Screening to End-of-Study (5-10 years)
See also
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Completed NCT03292887 - Hunter Outcome Survey (HOS)
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Completed NCT00920647 - A Safety and Dose Ranging Study of Idursulfase (Intrathecal) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Hunter Syndrome Who Have Central Nervous System Involvement and Are Receiving Treatment With Elaprase® Phase 1/Phase 2
Completed NCT01449240 - Collection and Study of Cerebrospinal Fluid in Patients With Hunter Syndrome
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Completed NCT01506141 - An Extension Study of HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Idursulfase-IT in Conjunction With Elaprase in Pediatric Participants With Hunter Syndrome and Cognitive Impairment Phase 1/Phase 2