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

CinnaGen

Status and phase: Completed; Phase 3

Conditions: Mucopolysaccharidosis Type 1

Treatments: Biological: Laronidase; Drug: Antihistamine; Drug: Antipyretic

Study type: Interventional

Funder types: Industry

Identifiers

NCT06406153

CIN.LAR.AR.III.97

Details and patient eligibility

About

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).

Full description

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.

Enrollment: 12 patients

Sex: All

Ages: 5 to 18 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Crossover Assignment

Masking: None (Open label)

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12 participants in 2 patient groups

YW17 (laronidase biosimilar)

Experimental group

Description:

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

Treatment:

Drug: Antipyretic

Drug: Antihistamine

Biological: Laronidase

Aldurazyme®

Active Comparator group

Description:

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

Treatment:

Drug: Antipyretic

Drug: Antihistamine

Biological: Laronidase