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Trial details
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
Current status:
Approved
|
Date registered:
27/01/2022
Trial version(s)
Current: 08/10/2021
Click here to view the tips and fields' descriptions
Main Information
Primary registry identifying number
LBCTR2021104866
Protocol number
DCR-PHXC-203
MOH registration number
Trial already registered with the MoPH
Study registered at the country of origin
Type of registration
Prospective
Date of registration in national regulatory agency
04/11/2019
Primary sponsor
Dicerna Pharmaceuticals
Primary sponsor: Country of origin
United States of America
Public title
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
Acronym
Scientific title
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
Acronym
Brief summary of the study: English
This is a Phase 2, multi-dose (3.5 mg/kg), open-label, single-arm, uncontrolled, multicenter study of nedosiran in pediatric participants (birth to 5 years of age) with genetically confirmed PH type 1 (PH1), type 2 (PH2), or type 3 (PH3) with relatively intact renal function based upon eGFR and serum creatinine. Participants will receive monthly SC doses (3.5 mg/kg) of nedosiran over 6 months. The primary objective of this study is to characterize the safety of nedosiran in pediatric participants (birth to 5 years of age) with PH1, PH2, and PH3. The secondary objective of this study is to characterize the efficacy of nedosiran in pediatric participants (birth to 5 years of age) with PH1, PH2, and PH3. The efficacy of nedosiran in lowering Uox will be assessed via monthly spot urine samples. Participants completing this study may be eligible for long-term treatment with nedosiran in Study DCR-PHXC-301.
Brief summary of the study: Arabic
هذه مرحلة 2 ، جرعات متعددة (3.5 مجم / كجم) ، علامة مفتوحة ، ذراع واحدة ، غير خاضعة للرقابة ، دراسة متعددة المراكز عن nedosiran في المشاركين في طب الأطفال (من الولادة حتى سن 5 سنوات) مع نوع PH مؤكد وراثيًا من النوع 1 (PH1) ، النوع 2 (PH2) ، أو النوع 3 (PH3) مع وظيفة كلوية سليمة نسبيًا على أساس معدل الترشيح الكبيبي (eGFR) وكرياتينين المصل. سيحصل المشاركون على جرعات شهرية من SC (3.5 مجم / كجم) من nedosiran على مدى 6 أشهر. الهدف الأساسي من هذه الدراسة هو توصيف سلامة nedosiran عند الأطفال المشاركين (من الولادة إلى 5 سنوات من العمر) مع PH1 و PH2 و PH3. الهدف الثانوي لهذه الدراسة هو توصيف فعالية nedosiran في الأطفال المشاركين (من الولادة إلى 5 سنوات من العمر) مع PH1 و PH2 و PH3. سيتم تقييم فعالية nedosiran في خفض Uox من خلال عينات البول الموضعية الشهرية. قد يكون المشاركون الذين أكملوا هذه الدراسة مؤهلين للعلاج طويل الأمد باستخدام nedosiran في دراسة DCR-PHXC-301.
Health conditions/problem studied: Specify
Primary Hyperoxaluria Type 1, 2 & 3.
Interventions: Specify
monthly SC doses (3.5 mg/kg) of nedosiran over 6 months.
Key inclusion and exclusion criteria: 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
Key inclusion and exclusion criteria: Gender
Both
Key inclusion and exclusion criteria: Age minimum
0
Key inclusion and exclusion criteria: Age maximum
5
Key inclusion and exclusion criteria: 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)
Type of Study
Type
Interventional
Type of intervention
Pharmaceutical
Trial scope
Safety
Trial scope: Specify scope
Study design: Allocation
Single Arm Study
Study design: Masking
Open (masking not used)
Study design: Control
Dose comparison
Study phase
2
Study design: Purpose
Treatment
Study design: Assignment
Single
IMP has market authorization
No
Name of IMP
Nedosiran
Type of IMP
Others
Type of IMP: Specify
pharmaceutical product
Pharmaceutical class
DCR-PHXC consists of the drug substance (DCR-L1360) in WFI) DCR-L1360 is a synthetic double-stranded (hybridized duplex) RNA oligonucleotide conjugated to GalNAc aminosugar residues. After SC administration, the GalNAc sugars conjugated to the RNA oligonucleotide bind to asialoglycoprotein receptors (ASGR) to deliver DCR-L1360 to hepatocytes.
Therapeutic indication
DCR-PHXC (Nedosiran sodium) reduces the level of mRNA encoding the dominant form of the LDH enzyme, specifically, the LDHA isoenzyme. Lactate dehydrogenase catalyzes the cytosolic conversion of glyoxylate to oxalate in the liver and this biochemical reaction is believed to be critical for oxalate generation for all 3 genetic forms of PH.
Therapeutic benefit
DCR-PHXC (Nedosiran sodium) reduces the level of mRNA encoding the dominant form of the LDH enzyme, specifically, the LDHA isoenzyme. Lactate dehydrogenase catalyzes the cytosolic conversion of glyoxylate to oxalate in the liver and this biochemical reaction is believed to be critical for oxalate generation for all 3 genetic forms of PH.
Biospecimen retention
Samples with DNA**
Biospecimen description
blood samples, urine samples and buccal cells.
Target sample size
5
Actual enrollment target size
5
Date of first enrollment: Type
Anticipated
Date of first enrollment: Date
06/12/2021
Date of study closure: Type
Anticipated
Date of study closure: Date
02/01/2023
Recruitment status
Pending
Date of completion
09/12/2022
IPD sharing statement plan
No
IPD sharing statement description
N/A
Additional data URL
Summary Results
Secondary Identifying Numbers
Full name of issuing authority
Secondary identifying number
Dicerna Pharmaceuticals
N/A
Sources of Monetary or Material Support
Name
Dicerna Pharmaceuticals
Secondary Sponsors
Name
Premier Research
Contact for Public/Scientific Queries
Contact type
First name
Last name
Address
Country
Telephone
Email
Affiliation
Public
Sarah
Kharsa
Beirut
Lebanon
+96181209199
sarah.kharsa@clinart.net
Clinart MEA
Scientific
Nancy
Choucair
Beirut
Lebanon
+9611421000
nancy.alam@usj.edu.lb
Hotel Dieu De France Ethics Committee
Centers/Hospitals Involved in the Study
Center/Hospital name
Name of principles investigator
Principles investigator speciality
Ethical approval
Hotel Dieu De France Hospital
Chebl Mourani
Pediatric Nephrology
Approved
Ethics Review
Ethics approval obtained
Approval date
Contact name
Contact email
Contact phone
Hotel Dieu de France
07/07/2021
Nancy Choucair
nancy.alam@usj.edu.lb
01-421000
Countries of Recruitment
Name
Lebanon
United States of America
United Kingdom
France
Poland
Turkey
Health Conditions or Problems Studied
Condition
Code
Keyword
Primary Hyperoxaluria
Nephrotic syndrome, other (N04.8)
hyperoxaluria
Interventions
Intervention
Description
Keyword
Nedosiran
DCR-PHXC 170 mg/mL Solution for Injection
Nedosiran
Primary Outcomes
Name
Time points
Measure
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
6 months
Change from Baseline in 12-lead ECG, physical examination findings, vital sign assessments, and clinical laboratory tests
Key Secondary Outcomes
Name
Time points
Measure
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
6 months
Percent and absolute change from Baseline to Month 6 in spot urinary oxalate-to-creatinine ratio
Trial Results
Summary results in Lebanon
Study results globally
Date of posting of results summaries
Date of first journal publication of results
Results URL link
Baseline characteristics
Participant flow
Adverse events
Outcome measures
URL to protocol files
Link(s) to publications related to the study
Changes History
Change
Date
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