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

Sponsor

Genzyme, a Sanofi Company (Industry)

Overall Status

Recruiting

CT.gov ID

NCT05134571

Collaborator

(none)

Enrollment

Location

Arm

21.8

Anticipated Duration (Months)

0.5

Patients Per Site Per Month

Study Details

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).

Condition or Disease	Intervention/Treatment	Phase
Mucopolysaccharidosis I	Drug: Laronidase	Phase 4

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.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

12 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

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

Actual Study Start Date :

Oct 28, 2021

Anticipated Primary Completion Date :

Aug 24, 2023

Anticipated Study Completion Date :

Aug 24, 2023

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Aldurazyme (laronidase) Aldurazyme (laronidase) treatment at approved dose and regimen, administered every week as an IV infusion	Drug: Laronidase Solution for injection; Intravenous

Arm

Intervention/Treatment

Experimental: Aldurazyme (laronidase)

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

Drug: Laronidase

Solution for injection; Intravenous

Outcome Measures

Primary Outcome Measures

Participants with adverse events (AEs) [Baseline to Week 27]
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 [Baseline to Week 27]
The percent change of uGAGs [Baseline to Week 26]

Secondary Outcome Measures

The percent change of uGAGs [Baseline to Week 2, Week 4, Week 8, Week 12 and Week 20]
The absolute change of uGAGs [Baseline to Week 2, Week 4, Week 8, Week 12, Week 20 and Week 26]
The percent change of liver volume (Abdominal B type ultrasound examination) [Baseline to Week 26]

Eligibility Criteria

Criteria

Ages Eligible for Study:

5 Years and Older

Sexes Eligible for Study:

All

Accepts 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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Investigational Site Number :1560001	Wuhan		China	430030

Sponsors and Collaborators

Genzyme, a Sanofi Company

Investigators

Study Director: Clinical Sciences & Operations, Sanofi

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Genzyme, a Sanofi Company

ClinicalTrials.gov Identifier:

NCT05134571

Other Study ID Numbers:

LPS16578
U1111-1260-3947

First Posted:

Nov 26, 2021

Last Update Posted:

May 4, 2022

Last Verified:

Apr 8, 2022

Individual Participant Data (IPD) Sharing Statement:

Yes

Plan to Share IPD:

Yes

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.:

Yes

Keywords provided by Genzyme, a Sanofi Company

clinical trial

enzyme replacement therapy

mucopolysaccharidosis I

laronidase

Additional relevant MeSH terms:

Mucopolysaccharidoses

Mucopolysaccharidosis I

Study Results

No Results Posted as of May 4, 2022