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

Primary Purpose

Mucopolysaccharidosis I

Status

Completed

Phase

Phase 4

Locations

China

Study Type

Interventional

Intervention

Laronidase

About this trial

This is an interventional treatment trial for Mucopolysaccharidosis I focused on measuring clinical trial, enzyme replacement therapy, mucopolysaccharidosis I, laronidase

Eligibility Criteria

5 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

Investigational Site Number :1560003
Investigational Site Number :1560002
Investigational Site Number :1560004
Investigational Site Number :1560006
Investigational Site Number :1560001

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Aldurazyme (laronidase)

Arm Description

Aldurazyme (laronidase) treatment at approved dose and regimen, administered every week as an IV infusion

Outcomes

Primary Outcome Measures

Participants with adverse events (AEs)

Incidence of AEs, serious adverse events (SAEs) and adverse events of special interest (AESIs) including infusion associated reactions (IARs) during the treatment emergent (TE) period

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

The percent change of uGAGs

Secondary Outcome Measures

The percent change of uGAGs

The absolute change of uGAGs

The percent change of liver volume (Abdominal B type ultrasound examination)

Full Information

NCT ID

NCT05134571

First Posted

October 21, 2021

Last Updated

September 26, 2023

Sponsor

Genzyme, a Sanofi Company

1. Study Identification

Unique Protocol Identification Number

NCT05134571

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

September 2023

Overall Recruitment Status

Completed

Study Start Date

October 28, 2021 (Actual)

Primary Completion Date

July 26, 2023 (Actual)

Study Completion Date

July 26, 2023 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Genzyme, a Sanofi Company

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Product Manufactured in and Exported from the U.S.

Data Monitoring Committee

5. Study Description

Brief 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).

Detailed Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Mucopolysaccharidosis I

Keywords

clinical trial, enzyme replacement therapy, mucopolysaccharidosis I, laronidase

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 4

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

12 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Aldurazyme (laronidase)

Arm Type

Experimental

Arm Description

Aldurazyme (laronidase) treatment at approved dose and regimen, administered every week as an IV infusion

Intervention Type

Drug

Intervention Name(s)

Laronidase

Intervention Description

Solution for injection; Intravenous

Primary Outcome Measure Information:

Title

Participants with adverse events (AEs)

Description

Incidence of AEs, serious adverse events (SAEs) and adverse events of special interest (AESIs) including infusion associated reactions (IARs) during the treatment emergent (TE) period

Time Frame

Baseline to Week 27

Title

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

Time Frame

Baseline to Week 27

Title

The percent change of uGAGs

Time Frame

Baseline to Week 26

Secondary Outcome Measure Information:

Title

The percent change of uGAGs

Time Frame

Baseline to Week 2, Week 4, Week 8, Week 12 and Week 20

Title

The absolute change of uGAGs

Time Frame

Baseline to Week 2, Week 4, Week 8, Week 12, Week 20 and Week 26

Title

The percent change of liver volume (Abdominal B type ultrasound examination)

Time Frame

Baseline to Week 26

10. Eligibility

Sex

All

Minimum Age & Unit of Time

5 Years

Accepts Healthy Volunteers

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.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Clinical Sciences & Operations

Organizational Affiliation

Sanofi

Official's Role

Study Director

Facility Information:

Facility Name

Investigational Site Number :1560003

City

Beijing

ZIP/Postal Code

100034

Country

China

Facility Name

Investigational Site Number :1560002

City

Beijing

ZIP/Postal Code

100730

Country

China

Facility Name

Investigational Site Number :1560004

City

Guangzhou

ZIP/Postal Code

510623

Country

China

Facility Name

Investigational Site Number :1560006

City

Hangzhou

ZIP/Postal Code

310003

Country

China

Facility Name

Investigational Site Number :1560001

City

Wuhan

ZIP/Postal Code

430030

Country

China

12. IPD Sharing Statement

Plan to Share IPD

Yes

IPD Sharing Plan Description

Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

Learn more about this trial

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

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