SNPMiner Trials by Shray Alag


SNPMiner SNPMiner Trials (Home Page)


Report for Clinical Trial NCT02292654

Developed by Shray Alag, 2020.
SNP Clinical Trial Gene

A Phase 1/2, Multi-Center, Open-Label, Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Exploratory Efficacy of Olipudase Alfa in Pediatric Patients Aged <18 Years With Acid Sphingomyelinase Deficiency

Primary Objective: To evaluate the safety and tolerability of olipudase alfa administered intravenously in pediatric patients every 2 weeks for 52 weeks. Secondary Objective: To characterize the pharmacokinetic profile and evaluate the pharmacodynamics and exploratory efficacy of olipudase alfa administered intravenously in pediatric patients every 2 weeks for 52 weeks.

NCT02292654 Sphingomyelin Lipidosis
MeSH:Niemann-Pick Disease, Type A Niemann-Pick Diseases Niemann-Pick Disease, Type C Lipidoses

1 Interventions

Name: Olipudase alfa

Description: Pharmaceutical form: powder for concentrate for solution for infusion Route of administration: intravenous infusion
Type: Drug
Group Labels: 1

GZ402665


Primary Outcomes

Measure: Number of adverse events

Time: From screening through Week 64

Measure: Clinically significant changes in laboratory parameters (complete blood count (CBC), clinical chemistry, and urinalysis)

Time: From screening through Week 64

Measure: Clinically significant changes in physical examinations (vital signs, electrocardiogram (ECG), doppler echocardiography, and liver ultrasound doppler)

Time: From screening through Week 64

Secondary Outcomes

Measure: Maximum concentration (Cmax)

Time: With the first infusion at 0.3, 1.0 and 3.0 mg/kg and at Week 52

Measure: Area under the curve until the last measurable concentration (AUClast)

Time: With the first infusion at 0.3, 1.0 and 3.0 mg/kg and at Week 52

Measure: Area under the curve extrapolated to infinity (AUC)

Time: With the first infusion at 0.3, 1.0 and 3.0 mg/kg and at Week 52

Measure: Half-life (t1/2)

Time: With the first infusion at 0.3, 1.0 and 3.0 mg/kg and at Week 52

Measure: Clearance (CL)

Time: With the first infusion at 0.3, 1.0 and 3.0 mg/kg and at Week 52

Measure: Volume of distribution (Vss)

Time: With the first infusion at 0.3, 1.0 and 3.0 mg/kg and at Week 52

Measure: Change in sphingomyelin levels

Time: From Day 1 through Week 64

Measure: Change in sphingomyelin metabolite levels

Time: From Day 1 through Week 64

Purpose: Treatment

Allocation: N/A

Single Group Assignment


There are 2 SNPs

SNPs


1 L302P

- The patients is homozygous for SMPD1 gene mutations R496L, L302P, and fs330 or any combination of these 3 mutations. --- R496L --- --- L302P ---


2 R496L

- The patients is homozygous for SMPD1 gene mutations R496L, L302P, and fs330 or any combination of these 3 mutations. --- R496L ---



HPO Nodes