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

NCT06406153 | Completed Phase 3

• 1 other identifier: CIN.LAR.AR.III.97
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 4

Brief Summary

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).

Conditions

Mucopolysaccharidosis Type 1

Keywords

Urinary Glycosaminoglycans; Laronidase; Mucopolysaccharidosis type I; Aldurazyme®

Outcome Measures

Primary Outcomes (1)

• mean uGAG

  urinary glycosaminoglycan

  Baseline, weeks 8, 10, 12, 20, 22, and 24

Secondary Outcomes (4)

• mean 6MWT

  Baseline, week 12, week 24

• mean predicted FVC

  Baseline, week 12, week 24

• mean enzyme activity level

  Weeks 11 and 23

• Number of participants with adverse events

  During the study period (screening visit up to week 24)

Study Arms (2)

YW17 (laronidase biosimilar)

EXPERIMENTAL

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

Biological: Laronidase; Drug: Antihistamine; Drug: Antipyretic

Aldurazyme®

ACTIVE COMPARATOR

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

Biological: Laronidase; Drug: Antihistamine; Drug: Antipyretic

Interventions

Laronidase BIOLOGICAL

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

Aldurazyme®; YW17 (laronidase biosimilar)

Antihistamine DRUG

An antihistamine is administered one hour before the infusion.

Aldurazyme®; YW17 (laronidase biosimilar)

Antipyretic DRUG

An antipyretic is administered one hour before the infusion.

Aldurazyme®; YW17 (laronidase biosimilar)

Eligibility Criteria

• Age: 5 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

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

You may not qualify if:

• 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

Sponsors & Collaborators

• Cinnagen: lead

Study Sites (4)

Clinical Research Development Unit of Akbar Hospital, Faculty of Medicine

Mashhad, Iran

Location

Growth and Development Research Center, Childrens Medical Center

Tehran, Iran

Location

Loghman Hospital

Tehran, Iran

Location

Mofid Childrens Hospital

Tehran, Iran

Location

Related Publications (1)

• Rabbani A, Alaei M, Asl SN, Setoodeh A, Shakiba M, Salehpour S, Eshraghi P, Salehpour O, Sabzvari A, Kafi H, Jarollahi A. Efficacy and safety of a biosimilar laronidase versus the reference laronidase in patients with mucopolysaccharidosis type I. Sci Rep. 2025 Aug 19;15(1):30427. doi: 10.1038/s41598-025-16351-4.

  PMID: 40830667 DERIVED

MeSH Terms

Conditions

Mucopolysaccharidosis I

Interventions

Iduronidase; Histamine Antagonists; Antipyretics

Condition Hierarchy (Ancestors)

Mucopolysaccharidoses; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lysosomal Storage Diseases; Mucinoses; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Glycoside Hydrolases; Hydrolases; Enzymes; Enzymes and Coenzymes; Histamine Agents; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Pharmacologic Actions; Chemical Actions and Uses; Physiological Effects of Drugs

Study Officials

• Ali Rabani, Professor

  Professor of Pediatric Endocrinology & Metabolism, Department of Pediatrics

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 27, 2024
• First Posted: May 9, 2024
• Study Start: September 10, 2022
• Primary Completion: August 12, 2023
• Study Completion: November 18, 2023
• Last Updated: August 6, 2024

Record last verified: 2024-08

Data Sharing

• IPD Sharing: Will not share

Locations

IR (4)