Randomized, Open-Label, Phase II, Multicenter, Multi-Country Study to Evaluate Safety and Efficacy of Dasatinib 50 mg in First-Line Treatment of Early Chronic Phase Chronic Myeloid Leukemia

Phase 1

Recruiting

• Conditions: Early Chronic Phase Chronic Myeloid Leukemia; hyperoxaluria; N04.8

• Registration Number: LBCTR2019010169
• Lead Sponsor: Hikma Pharmaceuticals

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2021-09-10
• Study Completion: 2022-06-30
• Last Update Posted: 19 August 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 100

Inclusion Criteria

Key inclusion criteria include
• Estimated glomerular filtration rate (eGFR) at Screening = 30 mL/min normalized to 1.73 m2 body surface area (BSA).
• Average spot Uox-to-creatinine ratio at Screening above 2 times the 95th percentile for age based on Matos et al, 1999:
o > 0.44 mol/mol in participants < 6 months
o > 0.34 mol/mol in participants from 6 months to <12 months
o > 0.26 mol/mol in participants 12 months to < 2 years
o > 0.20 mol/mol in participants from 2 to < 3 years and
o > 0.16 mol/mol in participants from 3 to 5 years

Exclusion Criteria

Key exclusion criteria include
• Renal or hepatic transplantation (prior or planned within the study period)
• Plasma oxalate (Pox) > 30 µmol/L at Screening
• Documented evidence of clinical manifestations of severe systemic oxalosis (including preexisting retinal, heart, or skin calcifications, or history of severe bone pain, pathological fractures, or bone deformations)

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
ame: To characterize the safety of nedosiran in neonates, infants, and young children with PH and relatively intact renal function based upon eGFR and serum creatinine;Timepoints: 6 months;Measure: Change from Baseline in 12-lead ECG, physical examination findings, vital sign assessments, and clinical laboratory tests

Secondary Outcome Measures

Name	Time	Method
ame: To assess the efficacy of nedosiran in neonates, infants, and young children with PH and relatively intact renal function based upon eGFR and serum creatinine;Timepoints: 6 months;Measure: Percent and absolute change from Baseline to Month 6 in spot urinary oxalate-to-creatinine ratio