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 range
5 Years
Sex
MALE
See this in plain English?
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).
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
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)
. 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.
. 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.
6
Number of Participants With Treatment-emergent Adverse Events (TEAE)
Timeframe: From screening to End-of-Study (approximately 9.75 years)
7
Number of Participants With Clinically Significant Abnormal Urinalysis Values
Timeframe: From screening to End-of-Study (approximately 9.75 years)
8
Number of Participants With Clinically Significant Abnormal Serum Chemistry Values
Timeframe: From screening to End-of-Study (approximately 9.75 years)
9
Number of Participants With Clinically Significant Abnormal Hematology Values
Timeframe: From screening to End-of-Study (approximately 9.75 years)