A Phase 4, single-arm, open-label safety and efficacy study of Aldurazyme® (laronidase) as enzyme replacement therapy in participants with Mucopolysaccharidosis I (MPS I) in China

Genzyme Europe B. V0 个研究点开始时间: 2023年11月22日最近更新: 2023年11月27日

适应症

试验速览

暂无简介。

•Chinese participants have a documented diagnosis of MPS I confirmed by measurable clinical signs and symptoms of MPS I and fibroblast or leukocyte IDUA 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).
•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.
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 12
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 4
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•Participants are excluded from the study if any of the following criteria apply:
•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, within 30 days prior to study enrollment.
•Received an investigational gene therapy.

发起方

Genzyme Europe B. V