Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Ye… (NCT02455622) | Clinical Trial Compass
CompletedPhase 4
Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of Age
United States, Dominican Republic, Germany21 participantsStarted 2015-10-28
Plain-language 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.
Who can participate
Age range5 Years
SexMALE
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
✓. 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.
Exclusion criteria
✕. 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.
What they're measuring
1
Height Overall
Timeframe: Prospective participants: From Baseline through End-of-Study (approximately 9.75 years); HOS participants followed up to a maximum of 9.5 years where participant data were accessed from the registry between the period of 08/01/07 to 02/06/22
2
Weight Overall
Timeframe: Prospective participants: From Baseline through End-of-Study (approximately 9.75 years); HOS participants followed up to a maximum of 9.5 years where participant data were accessed from the registry between the period of 08/01/07 to 02/06/22
3
Change From Baseline in Height Measured by Z-score
Timeframe: Prospective participants: From Baseline through End-of-Study (approximately 9.75 years); HOS participants followed up to a maximum of 9.5 years where participant data were accessed from the registry between the period of 08/01/07 to 02/06/22
4
Change From Baseline in Weight Measured by Z-score
Timeframe: Prospective participants: From Baseline through End-of-Study (approximately 9.75 years); HOS participants followed up to a maximum of 9.5 years where participant data were accessed from the registry between the period of 08/01/07 to 02/06/22
5
Number of Participants With Clinical Significant Abnormal Neurological Examination
Timeframe: From Screening to End-of-Study (approximately 9.75 years)
6
Number of Participants With Treatment-emergent Adverse Events (TEAE)