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Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of Age

Primary Purpose

Hunter Syndrome

Status
Active
Phase
Phase 4
Locations
International
Study Type
Interventional
Intervention
Elaprase for intravenous (IV) infusion
Sponsored by
Takeda
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hunter Syndrome

Eligibility Criteria

undefined - 6 Years (Child)MaleDoes not accept healthy volunteers

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.

Sites / Locations

  • Ann & Robert H. Lurie Children's Hospital of Chicago
  • Hospital Infantil Dr Robert Reid Cabral
  • Universitätsmedizin der Johannes Gutenberg-Universität Mainz
  • Hospital Kuala Lumpur
  • Philippine General Hospital
  • Mother and Child Health Care Institute of Serbia Dr Vukan Cupic
  • Chulalongkorn University
  • National Pediatrics Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Enrolled Patients

Arm Description

Patients who are receiving treatment with Elaprase in this study (SHP-ELA-401), who are <6 years of age, and were previously treatment-naïve. Patients who are not enrolled in this study (SHP-ELA-401) but are enrolled in the Hunter Outcome Survey (HOS) patient registry and were < 6 years of age at start of Elaprase treatment. While not enrolled in the present Study SHP-ELA-401, their height and weight data from HOS will be utilized in the Primary Growth Analysis for this study.

Outcomes

Primary Outcome Measures

Change in height
Change in weight Z-scores
Change in neurological examination to evaluate long-term safety in patient
Number of participants with adverse events as measured by type, severity, and relationship to treatment
Change in clinical laboratory testings as measured by urinalysis to evaluate long-term safety in patients
Change in clinical laboratory testings as measured by serum chemistry to evaluate long-term safety in patients
Change in clinical laboratory testings as measured by hematology to evaluate long-term safety in patients

Secondary Outcome Measures

Urinary glycosaminoglycans (uGAG) levels normalized to urine creatinine
Normalized uGAG divided by upper limit of normal for age (uGAG/ULN) every 12 months
Joint mobility, as measured by Joint Range of Motion (JROM) scores, including global, upper-limb, and lower-limb joint scores
Distance walked, as measured by the Six Minute Walk Test (6MWT)
Quality of Life, as measured by the Hunter-Syndrome Functional Outcome in Clinical Understanding Scale
Impact of illness on ability to function in daily life, as measured by the Childhood Health Assessment Questionnaire (CHAQ Parent Report)
Adaptive behavior, as measured by the Vineland Adaptive Behavior Scales (VABS II)
Change in anti-idursulfase antibodies in serum
number and percentage of patients testing anti-idursulfase antibody positive and negative at each time point

Full Information

First Posted
May 20, 2015
Last Updated
July 19, 2023
Sponsor
Takeda
Collaborators
Takeda Development Center Americas, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT02455622
Brief Title
Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of Age
Official Title
A Long-Term, Open-Label, Multicenter, Phase IV Study to Assess Longitudinal Changes on Height and Weight in Patients With MPS II Who Are Receiving Elaprase and Started Treatment With Elaprase at <6 Years of Age
Study Type
Interventional

2. Study Status

Record Verification Date
July 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
October 28, 2015 (Actual)
Primary Completion Date
July 9, 2025 (Anticipated)
Study Completion Date
July 9, 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Takeda
Collaborators
Takeda Development Center Americas, Inc.

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hunter Syndrome

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
21 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Enrolled Patients
Arm Type
Experimental
Arm Description
Patients who are receiving treatment with Elaprase in this study (SHP-ELA-401), who are <6 years of age, and were previously treatment-naïve. Patients who are not enrolled in this study (SHP-ELA-401) but are enrolled in the Hunter Outcome Survey (HOS) patient registry and were < 6 years of age at start of Elaprase treatment. While not enrolled in the present Study SHP-ELA-401, their height and weight data from HOS will be utilized in the Primary Growth Analysis for this study.
Intervention Type
Drug
Intervention Name(s)
Elaprase for intravenous (IV) infusion
Other Intervention Name(s)
Idursulfase
Intervention Description
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.
Primary Outcome Measure Information:
Title
Change in height
Time Frame
Screening to End-of-Study (5-10 years)
Title
Change in weight Z-scores
Time Frame
Screening to End-of-Study (5-10 years)
Title
Change in neurological examination to evaluate long-term safety in patient
Time Frame
Screening to End-of-Study (5-10 years)
Title
Number of participants with adverse events as measured by type, severity, and relationship to treatment
Time Frame
Screening to End-of-Study (5-10 years)
Title
Change in clinical laboratory testings as measured by urinalysis to evaluate long-term safety in patients
Time Frame
Screening to End-of-Study (5-10 years)
Title
Change in clinical laboratory testings as measured by serum chemistry to evaluate long-term safety in patients
Time Frame
Screening to End-of-Study (5-10 years)
Title
Change in clinical laboratory testings as measured by hematology to evaluate long-term safety in patients
Time Frame
Screening to End-of-Study (5-10 years)
Secondary Outcome Measure Information:
Title
Urinary glycosaminoglycans (uGAG) levels normalized to urine creatinine
Time Frame
Screening to End-of-Study (5-10 years)
Title
Normalized uGAG divided by upper limit of normal for age (uGAG/ULN) every 12 months
Time Frame
Baseline to End-of-Study (5-10 years)
Title
Joint mobility, as measured by Joint Range of Motion (JROM) scores, including global, upper-limb, and lower-limb joint scores
Time Frame
Screening to End-of-Study (5-10 years)
Title
Distance walked, as measured by the Six Minute Walk Test (6MWT)
Time Frame
Screening to End-of-Study (5-10 years)
Title
Quality of Life, as measured by the Hunter-Syndrome Functional Outcome in Clinical Understanding Scale
Time Frame
Screening to End-of-Study (5-10 years)
Title
Impact of illness on ability to function in daily life, as measured by the Childhood Health Assessment Questionnaire (CHAQ Parent Report)
Time Frame
Screening to End-of-Study (5-10 years)
Title
Adaptive behavior, as measured by the Vineland Adaptive Behavior Scales (VABS II)
Time Frame
Screening to End-of-Study (5-10 years)
Title
Change in anti-idursulfase antibodies in serum
Description
number and percentage of patients testing anti-idursulfase antibody positive and negative at each time point
Time Frame
Screening to End-of-Study (5-10 years)

10. Eligibility

Sex
Male
Maximum Age & Unit of Time
6 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Group 1: Prospective Patient Group The patient is male. The patient is Elaprase-naïve at study entry. 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: 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 The patient has a documented mutation in the I2S gene. OR 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). The patient will be <6 years of age at the start of Elaprase treatment. 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. The patient is male. The patient is enrolled in HOS. The patient was <6 years of age at the start of Elaprase treatment. The patient received Elaprase weekly treatment for at least 5 years. The patient had a height assessment and a weight assessment documented within 3 months before or after Elaprase treatment start. The patient has had annual height and weight assessments from start of Elaprase through age 10 years. The patient, patient's parent(s), or legally authorized guardian(s) agree(s) to data collection. 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 The patient has received treatment with any investigational drug or device within the 30 days prior to study entry. The patient has received or is receiving treatment with idursulfase-IT. The patient has received growth hormones, a cord blood infusion, or a bone marrow transplant at any time. The patient has received blood product transfusions within 90 days prior to Screening. 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Study Director
Organizational Affiliation
Shire
Official's Role
Study Director
Facility Information:
Facility Name
Ann & Robert H. Lurie Children's Hospital of Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
Hospital Infantil Dr Robert Reid Cabral
City
Santo Domingo
ZIP/Postal Code
10101
Country
Dominican Republic
Facility Name
Universitätsmedizin der Johannes Gutenberg-Universität Mainz
City
Mainz
ZIP/Postal Code
55131
Country
Germany
Facility Name
Hospital Kuala Lumpur
City
Kuala Lumpur
ZIP/Postal Code
50586
Country
Malaysia
Facility Name
Philippine General Hospital
City
Manila
ZIP/Postal Code
1000
Country
Philippines
Facility Name
Mother and Child Health Care Institute of Serbia Dr Vukan Cupic
City
Beograd
ZIP/Postal Code
11000
Country
Serbia
Facility Name
Chulalongkorn University
City
Bangkok
ZIP/Postal Code
10330
Country
Thailand
Facility Name
National Pediatrics Hospital
City
Hanoi
Country
Vietnam

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing Plan Description
Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement
IPD Sharing Access Criteria
IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
IPD Sharing URL
https://vivli.org/ourmember/takeda/
Links:
URL
https://clinicaltrials.takeda.com/study-detail/5f6b5fe94db2bf003ab47b2e
Description
To obtain more information on the study, click here/on this link

Learn more about this trial

Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of Age

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