SNPMiner Trials: Clinical Trial Report
Report for Clinical Trial NCT02292654
Developed by Shray Alag, 2019.
SNP Clinical Trial Gene
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.
1 Interventions
Name: Olipudase alfa
Description: Pharmaceutical form: powder for concentrate for solution for infusion Route of administration: intravenous infusionType: Drug
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
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