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

NCT06406153 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2024-08-06

No results posted yet for this study

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

Interventions

BIOLOGICAL

Laronidase

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

DRUG

Antihistamine

An antihistamine is administered one hour before the infusion.

DRUG

Antipyretic

An antipyretic is administered one hour before the infusion.

Sponsors & Collaborators

  • Cinnagen

    lead INDUSTRY

Principal Investigators

  • Ali Rabani, Professor · Professor of Pediatric Endocrinology & Metabolism, Department of Pediatrics

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
CROSSOVER

Eligibility

Min Age
5 Years
Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-09-10
Primary Completion
2023-08-12
Completion
2023-11-18

Countries

  • Iran

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06406153 on ClinicalTrials.gov