Clinical trial NCT06406153: Efficacy and Safety of YW17 (Laronidase-CinnaGen) Compared to Aldurazyme® in MPS I Patients
Official 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)"
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Mucopolysaccharidosis Type 1
Keywords: Urinary Glycosaminoglycans
Laronidase
Mucopolysaccharidosis type I
Aldurazyme®
Lead sponsor: Cinnagen
Sponsor class: INDUSTRY
First posted: 2024-05-09
Enrollment: 12
Sex: ALL
Minimum age (years): 5
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

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).

Detailed 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.

Eligibility criteria: 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

Primary outcomes:
measure: mean uGAG
timeFrame: Baseline, weeks 8, 10, 12, 20, 22, and 24
Secondary outcomes:
measure: mean 6MWT
timeFrame: Baseline, week 12, week 24
measure: mean predicted FVC
timeFrame: Baseline, week 12, week 24
measure: mean enzyme activity level
timeFrame: Weeks 11 and 23
measure: Number of participants with adverse events
timeFrame: During the study period (screening visit up to week 24)
Other outcomes:
Locations:
city: Mashhad
country: Iran, Islamic Republic of
facility: Clinical Research Development Unit of Akbar Hospital, Faculty of Medicine
city: Tehran
country: Iran, Islamic Republic of
facility: Growth and Development Research Center, Childrens Medical Center
city: Tehran
country: Iran, Islamic Republic of
facility: Loghman Hospital
city: Tehran
country: Iran, Islamic Republic of
facility: Mofid Childrens Hospital
Study officials:
affiliation: Professor of Pediatric Endocrinology & Metabolism, Department of Pediatrics
name: Ali Rabani, Professor
role: PRINCIPAL_INVESTIGATOR
References: