Efficacy and Safety of YW17 (Laronidase-CinnaGen) Compared to Aldurazyme® in MPS I Patients

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

12 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-09-10

Study Completion Date

2023-11-18

Brief Summary

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The purpose of this phase III study is to assess the efficacy and safety of YW17 produced by CinnaGen Company compared to Aldurazyme® in mucopolysaccharidosis type I (MPS I) patients.

All patients receive Aldurazyme® for 12 weeks, followed by YW17 for another 12 weeks.

The primary outcome is the assessment of the maintenance of the mean uGAG levels at the end of each medication administration. The secondary outcomes are the assessment of 6-minute walking test (6MWT), predicted forced vital capacity (FVC), enzyme activity assay, and adverse events (AEs).

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Detailed Description

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This is a phase III, single-sequence, cross-over study to assess the efficacy and safety of YW17 produced by CinnaGen Company in comparison with Aldurazyme® in MPS I patients.

All patients receive 0.58 mg/kg of Aldurazyme® for 12 weeks and then receive 0.58 mg/kg of YW17 for another 12 weeks.

Premedication with antipyretics and/or antihistamines is administered for all patients one hour before the infusion.

The primary outcome is to compare the mean uGAG levels at weeks 8, 10, and 12 (related to Aldurazyme®) with the mean uGAG levels at weeks 20, 22, and 24 (related to YW17).

The secondary outcomes, including 6MWT and FVC are assessed at the beginning and the end of each medication administration. Enzyme activity is assessed at the end of each medication administration. Safety assessments are performed during the study.

Conditions

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Mucopolysaccharidosis Type 1

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

CROSSOVER

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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YW17 (laronidase biosimilar)

YW17 (2.9 mg/5 mL) produced by CinnaGen Company, is administered 0.58 mg/kg weekly.

Group Type EXPERIMENTAL

Laronidase

Intervention Type BIOLOGICAL

Laronidase as Aldurazyme® (the first 12 weeks) or YW17 (the second 12 weeks) is administered.

Antihistamine

Intervention Type DRUG

An antihistamine is administered one hour before the infusion.

Antipyretic

Intervention Type DRUG

An antipyretic is administered one hour before the infusion.

Aldurazyme®

Aldurazyme® (2.9 mg/5 mL), is administered 0.58 mg/kg weekly.

Group Type ACTIVE_COMPARATOR

Laronidase

Intervention Type BIOLOGICAL

Laronidase as Aldurazyme® (the first 12 weeks) or YW17 (the second 12 weeks) is administered.

Antihistamine

Intervention Type DRUG

An antihistamine is administered one hour before the infusion.

Antipyretic

Intervention Type DRUG

An antipyretic is administered one hour before the infusion.

Interventions

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Laronidase

Laronidase as Aldurazyme® (the first 12 weeks) or YW17 (the second 12 weeks) is administered.

Intervention Type BIOLOGICAL

Antihistamine

An antihistamine is administered one hour before the infusion.

Intervention Type DRUG

Antipyretic

An antipyretic is administered one hour before the infusion.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Age 5-18
* Diagnosed with MPS I
* Signing informed consent form

Exclusion Criteria

* Prior bone marrow transplantation or being a candidate for receiving haematopoietic stem cell transplantation (HSCT)
* Prior tracheotomy
* Being naïve to laronidase
* Acute hydrocephalus
* Abnormal renal function determined by measuring serum creatinine and blood urea nitrogen (BUN) levels
* Any severe organic disease that is not associated with MPS I
* Known hypersensitivity to laronidase or components of the laronidase solution
* Presence of any medical condition or other circumstances that could significantly interfere with study compliance
* Pregnancy and lactation
* Administration of any investigational drug within 30 days before study enrollment

Minimum Eligible Age

5 Years

Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No