China Post-marketing Surveillance (PMS) Study of Aldurazyme®

Launched by GENZYME, A SANOFI COMPANY · Nov 14, 2021

Keywords

ClinConnect Summary

Study duration for each participant will be a total of 29 weeks which will include 2 weeks of screening, 26 weeks of treatment period and 1 week of follow-up period.

Gender

ALL

Eligibility criteria

• 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, within 30 days prior to study enrollment.
• • Received an investigational gene therapy.
• • The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.