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

• Register: ClinicalTrials.gov
• Last refreshed on: 2 August 2023
• Main ID: NCT02455622
• Date of registration: 20/05/2015
• Prospective Registration: Yes
• Primary sponsor: Takeda
• Public title: Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of Age
• Scientific 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
• Date of first enrolment: October 28, 2015
• Target sample size: 21
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT02455622
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 4

Countries of recruitment

Dominican Republic, Germany, Malaysia, Oman, Philippines, Saudi Arabia, Serbia, Thailand, United States, Vietnam

Contacts

• Name: Study Director
• Affiliation: Shire

Key inclusion & exclusion criteria

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.



Age minimum: N/A
Age maximum: 6 Years
Gender: Male

Health Condition(s) or Problem(s) studied

Hunter Syndrome

Intervention(s)

Drug: Elaprase for intravenous (IV) infusion

Primary Outcome(s)

Change in neurological examination to evaluate long-term safety in patient [Time Frame: Screening to End-of-Study (5-10 years)]

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)]

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)]

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)]

Change in height [Time Frame: Screening to End-of-Study (5-10 years)]

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)]

Change in weight Z-scores [Time Frame: Screening to End-of-Study (5-10 years)]

Secondary Outcome(s)

Urinary glycosaminoglycans (uGAG) levels normalized to urine creatinine [Time Frame: Screening to End-of-Study (5-10 years)]

Change in anti-idursulfase antibodies in serum [Time Frame: Screening to End-of-Study (5-10 years)]

Adaptive behavior, as measured by the Vineland Adaptive Behavior Scales (VABS II) [Time Frame: Screening to End-of-Study (5-10 years)]

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)]

Distance walked, as measured by the Six Minute Walk Test (6MWT) [Time Frame: Screening to End-of-Study (5-10 years)]

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)]

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)]

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)]

Secondary ID(s)

2014-004804-31

SHP-ELA-401

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Takeda Development Center Americas, Inc.