Nedosiran in Pediatric Patients from Birth to 5 Years of Age with PH1, PH2, or PH3 and Relatively Intact Renal Function

A Phase 2 Open-Label Multicenter Study to Evaluate the Safety, Pharmacokinetics, and Efficacy of Nedosiran in Pediatric Patients from Birth to 5 Years of Age with Primary Hyperoxaluria and Relatively Intact Renal Function - PHYOX8

Status

Not yet recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2021-001083-16-ES

Enrollment

Registered

2021-06-23

Start date

2021-08-18

Completion date

Unknown

Last updated

2021-08-24

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Primary Hyperoxaluria MedDRA version: 20.1 Level: PT Classification code 10020703 Term: Hyperoxaluria System Organ Class: 10038359 - Renal and urinary disorders

Interventions

Product Name: Nedosiran Product Code: DCR-PHXC Pharmaceutical Form: Solution for injection INN or Proposed INN: DCR-L1360 Current Sponsor code: DCR-PHXC Other descriptive name: Nedosiran Concentration

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Estimated glomerular filtration rate (eGFR) at Screening = 30 mL/min normalized to 1.73 m2 body surface area (BSA). 2. Average spot Uox-to-creatinine ratio at Screening above 2 times the 95th percentile for age based on Matos et al, 1999: > 0.44 mol/mol in participants 0.34 mol/mol in participants from 6 months to 0.26 mol/mol in participants 12 months to 0.20 mol/mol in participants from 2 to 0.16 mol/mol in participants from 3 to 5 years Are the trial subjects under 18? yes Number of subjects for this age range: 15 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: 1. Renal or hepatic transplantation (prior or planned within the study period) 2. Plasma oxalate (Pox) > 30 µmol/L at Screening 3.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)

Design outcomes

Primary

Measure	Time frame
Main Objective: To assess the safety of nedosiran in neonates, infants, and young children with PH and relatively intact renal function based upon eGFR and serum creatinine;Secondary Objective: 1. To characterize the efficacy of nedosiran in neonates, infants, and young children with PH and relatively intact renal function based upon eGFR and serum creatin 2. To characterize the PK of nedosiran in neonates, infants, and young children with PH and relatively intact renal function based upon eGFR and serum creatinineine 3. 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 4. To evaluate the effect of nedosiran on eGFR in neonates, infants, and young children with PH and relatively intact renal function based upon eGFR and serum creatinine;Primary end point(s): - The incidence and nature of TEAEs and SAEs - Change from Baseline in 12-lead ECG, physical examination findings, vital sign assessments, and clinical laboratory tests;Timepoint(s) of evaluation of this end point: Descriptive statistics will be provided for absolute values and changes from baseline in physical examination findings, vital sign measurements, ECGs, and clinical laboratory results at each timepoint.	—

Measure

Time frame

Main Objective: To assess the safety of nedosiran in neonates, infants, and young children with PH and relatively intact renal function based upon eGFR and serum creatinine;Secondary Objective: 1. To characterize the efficacy of nedosiran in neonates, infants, and young children with PH and relatively intact renal function based upon eGFR and serum creatin 2. To characterize the PK of nedosiran in neonates, infants, and young children with PH and relatively intact renal function based upon eGFR and serum creatinineine 3. 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 4. To evaluate the effect of nedosiran on eGFR in neonates, infants, and young children with PH and relatively intact renal function based upon eGFR and serum creatinine;Primary end point(s): - The incidence and nature of TEAEs and SAEs - Change from Baseline in 12-lead ECG, physical examination findings, vital sign assessments, and clinical laboratory tests;Timepoint(s) of evaluation of this end point: Descriptive statistics will be provided for absolute values and changes from baseline in physical examination findings, vital sign measurements, ECGs, and clinical laboratory results at each timepoint.

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Secondary

Measure	Time frame
Secondary end point(s): 1. Percent and absolute change from Baseline to Month 6 in spot urinary oxalate-to-creatinine ratio 2. Plasma PK parameters for nedosiran and/or its metabolites, including Cmax, AUCt and AUC8 (if estimable) 3. Percentage of participants with spot urinary oxalate-to-creatinine ratio = the ULN and = 1.5 x ULN at any time point through Month 6 4. Change from Baseline in eGFR at Month 6 (only in participants = 12 Months of age at Screening);Timepoint(s) of evaluation of this end point: 1. The percent and absolute change in average spot urinary oxalate-to creatinine ratio from Baseline to Day 180 will be summarized 2. Pharmacokinetic analyses will be described in the SAP finalized before database lock. The population PK analysis will be presented separately from the main CSR 3. The percentage of participants with spot urinary oxalate-to-creatinine ratio = the ULN and = 1.5 x ULN at any time point through Month 6 will be summarized. 4. The percent change from baseline in eGFR at Month 6 (only in participants = 12 Months of age at Screening) will be summarized.	—

Measure

Time frame

Secondary end point(s): 1. Percent and absolute change from Baseline to Month 6 in spot urinary oxalate-to-creatinine ratio 2. Plasma PK parameters for nedosiran and/or its metabolites, including Cmax, AUCt and AUC8 (if estimable) 3. Percentage of participants with spot urinary oxalate-to-creatinine ratio = the ULN and = 1.5 x ULN at any time point through Month 6 4. Change from Baseline in eGFR at Month 6 (only in participants = 12 Months of age at Screening);Timepoint(s) of evaluation of this end point: 1. The percent and absolute change in average spot urinary oxalate-to creatinine ratio from Baseline to Day 180 will be summarized 2. Pharmacokinetic analyses will be described in the SAP finalized before database lock. The population PK analysis will be presented separately from the main CSR 3. The percentage of participants with spot urinary oxalate-to-creatinine ratio = the ULN and = 1.5 x ULN at any time point through Month 6 will be summarized. 4. The percent change from baseline in eGFR at Month 6 (only in participants = 12 Months of age at Screening) will be summarized.

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Countries

Canada, France, Germany, Italy, Japan, Lebanon, Morocco, Poland, Spain, Turkey, United Kingdom, United States

Contacts

Public ContactClinical Trials Information

Dicerna Pharmaceuticals Inc

ahenderson@dicerna.com0001617612 6275

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026