China Post-marketing Surveillance (PMS) Study of Aldurazyme® (NCT05134571) | Clinical Trial Compass
CompletedPhase 4
China Post-marketing Surveillance (PMS) Study of Aldurazyme®
China12 participantsStarted 2021-10-28
Plain-language summary
This is a single treatment arm study that is open-label to be conducted in Chinese participants with MPS I.
Trial Objectives are to evaluate the safety and tolerability of Aldurazyme in Chinese MPS I participants, and to evaluate the efficacy of Aldurazyme on the percent change of urinary glycosaminoglycans (uGAGs) from baseline to Week 26. The study will also evaluate the effect on uGAG level and liver volume (hepatomegaly) after 26 weeks, with Aldurazyme treatment in Chinese MPS I participants.
Treatment duration will include: 2 weeks of screening, 26 weeks of treatment and 1 week of follow-up period. During the treatment period, weekly visits are designed to accommodate weekly administration of Aldurazyme (laronidase).
Who can participate
Age range
5 Years
Sex
ALL
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:
* Chinese participants have a documented diagnosis of MPS I confirmed by measurable clinical signs and symptoms of MPS I and fibroblast or leukocyte IDUA (iduronidase) activity \<10% of normal.
* Participants have to be able to stand independently and walk a minimum of 5 meters in 6 minutes.
* A female participant is eligible to participate if she is not pregnant or breastfeeding, and at least one of the following conditions applies:
* Is not a woman of childbearing potential (WOCBP).
* OR
* Is a WOCBP and agrees to use an acceptable contraceptive method during the intervention period and at a minimum until 7 days after the last dose of study intervention.
* A WOCBP must have a negative highly sensitive pregnancy test (urine or serum as required by local regulations) within the screening period before the first dose of study intervention.
* If a urine test cannot be confirmed as negative (eg, an ambiguous result), a serum pregnancy test is required. In such cases, the participant must be excluded from participation if the serum pregnancy result is positive.
* Contraceptive/barrier method is not applicable for male participants.
Exclusion Criteria:
* Prior tracheostomy or bone marrow transplantation or hematopoietic stem cell transplantation.
* Have a plan to undergo bone marrow transplantation or hematopoietic stem cell transplantation within half a year after enrollment.
* Received an investigational drug, or device, other than Aldurazyme,…
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
Participants with adverse events (AEs)
Timeframe: Baseline to Week 27
2
The incidence of Potentially clinically significant abnormality (PCSA) analyses for clinical laboratory, vital signs, and ECG parameters during the TE period