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NCT Number: NCT06406153

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

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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Key information

Conditions

Mucopolysaccharidosis Type 1 Carbohydrate Metabolism, Inborn Errors Congenital, Hereditary, and Neonatal Diseases and Abnormalities Connective Tissue Diseases Genetic Diseases, Inborn Lysosomal Storage Diseases Metabolic Diseases Metabolism, Inborn Errors Mucinoses Mucopolysaccharidoses Mucopolysaccharidosis I Nutritional and Metabolic Diseases Skin and Connective Tissue Diseases

Age range

5 year–18 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Clinical Research Development Unit of Akbar Hospital, Faculty of Medicine, Mashhad, Iran

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Laronidase

Biological

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

Antihistamine

Drug

An antihistamine is administered one hour before the infusion.

Antipyretic

Drug

An antipyretic is administered one hour before the infusion.

Primary outcomes

  1. mean uGAG

    Time frame: Baseline, weeks 8, 10, 12, 20, 22, and 24

    urinary glycosaminoglycan

Secondary outcomes

  1. mean 6MWT

    Time frame: Baseline, week 12, week 24

    6-minute walking test

  2. mean predicted FVC

    Time frame: Baseline, week 12, week 24

    Forced vital capacity

  3. mean enzyme activity level

    Time frame: Weeks 11 and 23

    Enzyme activity assay

  4. Number of participants with adverse events

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

    All reported AEs are classified based on the Medical Dictionary for Regulatory Activities (MedDRA) terms and are graded according to the Common Terminology Criteria for Adverse Events (CTCAE) v5.0.

Sponsors and collaborators

Lead sponsor

Cinnagen

Industry

Registry information

Official study title

A Phase III, Single Arm, Cross-over, Multicenter Clinical Trial to Compare Efficacy and Safety of YW17(Laronidase; CinnaGen) Versus Laronidase (Aldurazyme®; Genzyme, BioMarin) in Patients With Mucopolysaccharidosis Type I (MPS I)"

Important dates

Study start
2022
Primary completion
2023
Study completion
2023
First posted
May 9, 2024
Registry last updated
Aug 6, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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