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

Hunter Syndrome Clinical Trial

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

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
• 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
• 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.

Related Conditions & MeSH terms

• Hunter Syndrome
• Mucopolysaccharidosis II

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)

External Links

•   To obtain more information on the study, click here/on this link