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

Cinnagen12 enrolled

Overview

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).

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.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Mucopolysaccharidosis Type 1

Interventions

LaronidaseBIOLOGICAL

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

AntihistamineDRUG

An antihistamine is administered one hour before the infusion.

AntipyreticDRUG

An antipyretic is administered one hour before the infusion.

Eligibility

Sex: ALLMin age: 5 YearsMax age: 18 Years

Medical Language ↔ Plain English

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

Locations (4)

Clinical Research Development Unit of Akbar Hospital, Faculty of Medicine

Mashhad, Iran

Growth and Development Research Center, Childrens Medical Center

Tehran, Iran

Loghman Hospital

Tehran, Iran

Mofid Childrens Hospital

Tehran, Iran

Outcomes

Primary Outcomes

mean uGAG

urinary glycosaminoglycan

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

Secondary Outcomes

mean 6MWT

6-minute walking test

Time frame: Baseline, week 12, week 24

mean predicted FVC

Forced vital capacity

Time frame: Baseline, week 12, week 24

mean enzyme activity level

Enzyme activity assay

Time frame: Weeks 11 and 23

Number of participants with adverse events

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.

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

Data from ClinicalTrials.gov

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