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 range5 Years

SexALL

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,…

What they're measuring

Participants with adverse events (AEs)

Timeframe: Baseline to Week 27

The incidence of Potentially clinically significant abnormality (PCSA) analyses for clinical laboratory, vital signs, and ECG parameters during the TE period

Timeframe: Baseline to Week 27

The percent change of uGAGs

Timeframe: Baseline to Week 26

Trial details

NCT IDNCT05134571

SponsorGenzyme, a Sanofi Company

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2023-07-26

View on ClinicalTrials.gov More Mucopolysaccharidosis I trials

Plain-language summary

Who can participate

Age range5 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Participants with adverse events (AEs)

Timeframe: Baseline to Week 27

The incidence of Potentially clinically significant abnormality (PCSA) analyses for clinical laboratory, vital signs, and ECG parameters during the TE period

Timeframe: Baseline to Week 27

The percent change of uGAGs

Timeframe: Baseline to Week 26