China post-marketing surveillance (PMS) study of Aldurazyme®

Phase 1

• Conditions: Mucopolysaccharidosis I; MedDRA version: 20.1Level: PTClassification code 10056886Term: Mucopolysaccharidosis ISystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

• Registration Number: EUCTR2023-001027-16-Outside-EU/EEA
• Lead Sponsor: Genzyme Europe B. V

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2023-11-22
• Last Update Posted: 27 November 2023

Recruitment & Eligibility

• Status: A
• Sex: All
• Target Recruitment: Not specified

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 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.

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

Exclusion Criteria

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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified