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Trial details
Long Term Extension Study in Patients With Primary Hyperoxaluria
Current status:
Approved
|
Date registered:
20/02/2021
Trial version(s)
Current: 02/12/2020
Click here to view the tips and fields' descriptions
Main Information
Primary registry identifying number
LBCTR2020124677
Protocol number
DCR-PHXC-301
MOH registration number
NCT04042402
Trial already registered with the MoPH
Study registered at the country of origin
Study registered in clinicaltrials.gov
Type of registration
Prospective
Date of registration in national regulatory agency
02/08/2019
Primary sponsor
Dicerna Pharmaceuticals, Inc.
Primary sponsor: Country of origin
USA
Public title
Long Term Extension Study in Patients With Primary Hyperoxaluria
Acronym
Scientific title
An Open-Label Roll-Over Study to Evaluate the Long-Term Safety and Efficacy of DCR-PHXC Solution for Injection (Subcutaneous Use) in Patients With Primary Hyperoxaluria
Acronym
“PHYOX3”
Brief summary of the study: English
The proposed study is designed to provide patients previously enrolled in Phase 1 and 2 studies of DCR-PHXC long-term access to DCR-PHXC, and to evaluate the long-term safety and efficacy of DCR-PHXC in patients with PH.
Brief summary of the study: Arabic
تم تصميم الدراسة المقترحة لتزويد المرضى المسجلين سابقًا في دراسات المرحلتين الأولى والثانية من DCR-PHXC بالوصول طويل الأمد إلى DCR-PHXC ، ولتقييم سلامة وفعالية DCR-PHXC على المدى الطويل في مرضى PH.
Health conditions/problem studied: Specify
Primary Hyperoxaluria Type 1 (PH1) Primary Hyperoxaluria Type 2 (PH2) Kidney Diseases Urologic Diseases Genetic Disease
Interventions: Specify
Drug: DCR-PHXC Multiple fixed doses of DCR-PHXC by subcutaneous (SC) injection. Other Name: Nedosiran
Key inclusion and exclusion criteria: Inclusion criteria
Key Inclusion Criteria: •Participant successfully completed a Dicerna Pharmaceuticals, Inc. study of DCR PHXC. OR Participant is the sibling of a participant who successfully completed a Dicerna Pharmaceuticals, Inc. study of DCR PHXC. Siblings must be younger than 18 years of age and must have genetically confirmed PH. •For participants rolling over from a multidose study of DCR-PHXC, enrollment should occur within a window of 25 to 60 days from the last dose of study intervention. Estimated GFR at screening ≥ 30 mL/min normalized to 1.73 m2 body surface area (BSA), calculated using Chronic Kidney Disease Epidemiology Collaboration (CKD EPI) formula in participants aged ≥ 18 years (Levey & Stevens, 2010), or the formula by Schwartz in participants aged 6 to 16 years (Schwartz et al., 2009; National Kidney Foundation, 2002). In Japan, the formula by Uemura et al. will be used for participants aged 6 to 17 years (Uemura et al., 2014).
Key inclusion and exclusion criteria: Gender
Both
Key inclusion and exclusion criteria: Age minimum
6
Key inclusion and exclusion criteria: Age maximum
99
Key inclusion and exclusion criteria: Exclusion criteria
Key exclusion criteria include: • Renal or hepatic transplantation; prior or planned within the study period • Current dialysis • Documented evidence of clinical manifestations of systemic oxalosis (including pre-existing 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
Other
Trial scope: Specify scope
Long Term safety and Efficacy
Study design: Allocation
Single Arm Study
Study design: Masking
Open (masking not used)
Study design: Control
N/A
Study phase
3
Study design: Purpose
Treatment
Study design: Assignment
Single
IMP has market authorization
No
Name of IMP
DCR PHXC
Type of IMP
Others
Type of IMP: Specify
Pharmaceutical Chemical
Pharmaceutical class
A synthetic double-stranded (hybridized duplex) ribonucleic acid (RNA) oligonucleotide conjugated to N-acetyl-D-galactosamine (GalNAc) amino-sugar residues.
Therapeutic indication
Primary Hyperoxaluria.
Therapeutic benefit
At present, no therapies are approved by regulatory authorities for the treatment of patients with PH. DCR-PHXC treatment has the potential benefit to reduce or eliminate the excess oxalate production in the liver and thus avoid the need for a combined liver and kidney transplantation in patients not already on renal replacement therapy.
Biospecimen retention
None retained
Biospecimen description
Blood and Urine Samples
Target sample size
50
Actual enrollment target size
Date of first enrollment: Type
Anticipated
Date of first enrollment: Date
01/02/2021
Date of study closure: Type
Anticipated
Date of study closure: Date
30/12/2023
Recruitment status
Other
Recruitment status: Specify
Enrolling by Invitation
Date of completion
30/12/2023
IPD sharing statement plan
No
IPD sharing statement description
Participants will be assigned a unique identifier by the Sponsor. Any participant records or datasets that are transferred to the Sponsor will contain the identifier only; participant names or any information which would make the participant identifiable will not be transferred.
Additional data URL
Summary Results
Secondary Identifying Numbers
Full name of issuing authority
Secondary identifying number
US NCT Number
NCT04042402
Sources of Monetary or Material Support
Name
Dicerna pharmaceuticals inc. 75 Hayden Avenue Suite 400 Lexington, MA 02421, USA
Secondary Sponsors
Name
NA
Contact for Public/Scientific Queries
Contact type
First name
Last name
Address
Country
Telephone
Email
Affiliation
Public
Chadi
Safa
lebanon. Baabda. Chiah. Ain el remeneh
Lebanon
0096171251819
Chadi.safa@clinart.net
Clinart
Scientific
Chebl
Mourani
Alfred Naccache Blvd, External Viewing Tower, Floor 4, Room 9403
Lebanon
03 290090
cheblmourani@gmail.com
HDF
Centers/Hospitals Involved in the Study
Center/Hospital name
Name of principles investigator
Principles investigator speciality
Ethical approval
1. Hotel Dieu de France
Chebl Mourani
Pediatric Nephrologist
Approved
2. Saint George University Hospital
Pauline Abu jaoude
Nephrologist
Approved
Ethics Review
Ethics approval obtained
Approval date
Contact name
Contact email
Contact phone
Hotel Dieu de France
30/03/2020
Nancy Choukair Alam
nancy.alam@usj.edu.lb
: +961 1 421 000
Saint George Hospital University Medical Center
09/07/2020
Sandra Berberi
smberbari@stgeorgehospital.org
+961 1 44 16 30
Countries of Recruitment
Name
France
Netherlands
Germany
United Kingdom
United States of America
Lebanon
Spain
Italy
Australia
Canada
Japan
Health Conditions or Problems Studied
Condition
Code
Keyword
Primary Hyperoxaluria
2-Propanol (T51.2)
Kidney Diseases
Interventions
Intervention
Description
Keyword
DCR-PHXC
Multiple fixed doses of DCR-PHXC by subcutaneous (SC) injection
Nedosiran
Primary Outcomes
Name
Time points
Measure
To evaluate the effect of DCR PHXC on estimated glomerular filtration rate
Annual change from baseline
estimated glomerular filtration rate
Key Secondary Outcomes
Name
Time points
Measure
The incidence and severity of treatment-emergent adverse events (TEAE) and SAEs associated with abnormal 12 lead electrocardiogram (ECG) readings
TEAEs and SAEs are evaluated monthly for 3 years
Electrocardiogram (ECG)
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
Download as PDF
Save a PDF copy of the summary of the trial