Clinical trial NCT02455622: 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
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE4
Conditions: Hunter Syndrome
Lead sponsor: Takeda
Sponsor class: INDUSTRY
Collaborators: name: Takeda Development Center Americas, Inc.
First posted: 2015-05-28
Enrollment: 21
Sex: MALE
Maximum age (years): 5
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility 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.

Primary outcomes:
measure: Change in height
timeFrame: Screening to End-of-Study (5-10 years)
measure: Change in weight Z-scores
timeFrame: Screening to End-of-Study (5-10 years)
measure: Change in neurological examination to evaluate long-term safety in patient
timeFrame: Screening to End-of-Study (5-10 years)
measure: Number of participants with adverse events as measured by type, severity, and relationship to treatment
timeFrame: Screening to End-of-Study (5-10 years)
measure: Change in clinical laboratory testings as measured by urinalysis to evaluate long-term safety in patients
timeFrame: Screening to End-of-Study (5-10 years)
measure: Change in clinical laboratory testings as measured by serum chemistry to evaluate long-term safety in patients
timeFrame: Screening to End-of-Study (5-10 years)
measure: Change in clinical laboratory testings as measured by hematology to evaluate long-term safety in patients
timeFrame: Screening to End-of-Study (5-10 years)
Secondary outcomes:
measure: Urinary glycosaminoglycans (uGAG) levels normalized to urine creatinine
timeFrame: Screening to End-of-Study (5-10 years)
measure: Normalized uGAG divided by upper limit of normal for age (uGAG/ULN) every 12 months
timeFrame: Baseline to End-of-Study (5-10 years)
measure: Joint mobility, as measured by Joint Range of Motion (JROM) scores, including global, upper-limb, and lower-limb joint scores
timeFrame: Screening to End-of-Study (5-10 years)
measure: Distance walked, as measured by the Six Minute Walk Test (6MWT)
timeFrame: Screening to End-of-Study (5-10 years)
measure: Quality of Life, as measured by the Hunter-Syndrome Functional Outcome in Clinical Understanding Scale
timeFrame: Screening to End-of-Study (5-10 years)
measure: Impact of illness on ability to function in daily life, as measured by the Childhood Health Assessment Questionnaire (CHAQ Parent Report)
timeFrame: Screening to End-of-Study (5-10 years)
measure: Adaptive behavior, as measured by the Vineland Adaptive Behavior Scales (VABS II)
timeFrame: Screening to End-of-Study (5-10 years)
measure: Change in anti-idursulfase antibodies in serum
timeFrame: Screening to End-of-Study (5-10 years)
Other outcomes:
Locations:
city: Chicago
country: United States
facility: Ann & Robert H. Lurie Children's Hospital of Chicago
state: Illinois
city: Santo Domingo
country: Dominican Republic
facility: Hospital Infantil Dr Robert Reid Cabral
city: Mainz
country: Germany
facility: Universitätsmedizin der Johannes Gutenberg-Universität Mainz
city: Kuala Lumpur
country: Malaysia
facility: Hospital Kuala Lumpur
city: Manila
country: Philippines
facility: Philippine General Hospital
city: Beograd
country: Serbia
facility: Mother and Child Health Care Institute of Serbia Dr Vukan Cupic
city: Bangkok
country: Thailand
facility: Chulalongkorn University
city: Hanoi
country: Vietnam
facility: National Pediatrics Hospital
Study officials:
affiliation: Shire
name: Study Director
role: STUDY_DIRECTOR
References: