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Trial details
Trial details
A Natural History Study of Patients with Genetically Confirmed Primary Hyperoxaluria Type 3 with a History of Stone Events
Current status:
Approved
|
Date registered:
09/12/2022
|
Date last updated:
07/07/2021
Trial version(s)
Current: 05/02/2021
Click here to view the tips and fields' descriptions
Main Information
Primary registry identifying number
LBCTR2021054742
Protocol number
DCR-PHXC-502
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
Primary sponsor
Dicerna Pharmaceuticals, Inc.
Primary sponsor: Country of origin
United States
Public title
A Natural History Study of Patients with Genetically Confirmed Primary Hyperoxaluria Type 3 with a History of Stone Events
Acronym
Scientific title
A Natural History Study of Patients with Genetically Confirmed Primary Hyperoxaluria Type 3 with a History of Stone Events
Acronym
Brief summary of the study: English
This is a natural history study of adults, adolescents, and children (≥ 2 years of age) with genetically confirmed primary hyperoxaluria type 3 (PH3) who have a history of stone events during the last 3 years and/or the presence of pre-existing stones detected by renal ultrasound at Screening.
Brief summary of the study: Arabic
دراسة التاريخ الطبيعي للمرضى المصابين بفرط أوكسالات البول الأولي المؤكد وراثيًا من النوع 3 مع تاريخ حالات الحصوات
Health conditions/problem studied: Specify
Primary Hyperoxaluria type 3
Interventions: Specify
this is a non-interventional study
Key inclusion and exclusion criteria: Inclusion criteria
Key inclusion criteria include • Genetically confirmed PH3 • History of stone events (defined as presence of calcifications in the urinary tract and/or kidney, their relative location, and the number and size of stones) during the last 3 years and/or presence of pre-existing stones detected by renal ultrasound at Screening • Uox > 0.7 mmol/24 hours normalized to 1.73 m2 BSA • eGFR at Screening ≥ 30 mL/min • Able to accommodate 24-hour urine collection
Key inclusion and exclusion criteria: Gender
Both
Key inclusion and exclusion criteria: Age minimum
2
Key inclusion and exclusion criteria: Age maximum
85
Key inclusion and exclusion criteria: Exclusion criteria
Exclusion Criteria Participants are excluded from the study if any of the following criteria apply: Medical Conditions 1. Prior hepatic transplantation; or planned transplantation within the study period 2. Currently receiving dialysis or anticipating requirement for dialysis during the study period
Type of Study
Type
Observational
Study model
Other
Study model: Specify model
natural history study
Study model: Explain model
Natural History of Patients with PH3 and a History of Stone Events
Time perspective
Retrospective
Time perspective: Explain time perspective
This is a non-interventional study that will last up to 2 years.
Target follow-up duration
1
Target follow-up duration: Unit
year
Number of groups/cohorts
0
Biospecimen retention
Samples with DNA**
Biospecimen description
blood and urine samples
Target sample size
480
Actual enrollment target size
1
Date of first enrollment: Type
Anticipated
Date of first enrollment: Date
15/06/2021
Date of study closure: Type
Anticipated
Date of study closure: Date
01/01/2024
Recruitment status
Pending
Date of completion
15/06/2023
IPD sharing statement plan
No
IPD sharing statement description
N/A, this is an observational trial.
Additional data URL
Summary Results
Secondary Identifying Numbers
Full name of issuing authority
Secondary identifying number
Hotel Dieu De France Ethics Committee
N/A
Sources of Monetary or Material Support
Name
Premier Research CRO
Secondary Sponsors
Name
Dicerna Pharmaceuticals INC.
Contact for Public/Scientific Queries
Contact type
First name
Last name
Address
Country
Telephone
Email
Affiliation
Public
Sarah
Kharsa
Beirut
Lebanon
+81-209199
sarah.kharsa@clinart.net
Clinart MEA
Scientific
Chadi
Safa
Beirut
Lebanon
+9613210603
chadi.safa@clinart.net
Clinart MEA
Centers/Hospitals Involved in the Study
Center/Hospital name
Name of principles investigator
Principles investigator speciality
Ethical approval
Hotel Dieu De France University Hospital
Dr. Chebl Mourani
Pediatrics
Approved
Saint George University Hospital Medical Center
Dr. Pauline Abou Jaoude
Pediatrics
Pending
Ethics Review
Ethics approval obtained
Approval date
Contact name
Contact email
Contact phone
Hotel Dieu de France
02/02/2021
Nancy Alam
nancy.alam@usj.edu.lb
+961 1 421 000 ext 2335
Countries of Recruitment
Name
Lebanon
United States of America
Canada
United Kingdom
France
Germany
Poland
Health Conditions or Problems Studied
Condition
Code
Keyword
Primary Hyperoxaluria
Nephrotic syndrome (N04)
N/A
Interventions
Intervention
Description
Keyword
Primary Outcomes
Name
Time points
Measure
The objective of this study is to collect data on stone formation and the degree of nephrocalcinosis in patients (≥ 2 years of age) with genetically confirmed PH3 and relatively intact renal function and to explore the potential relationship between Uox and new stone formation.
4
stone formation
Key Secondary Outcomes
Name
Time points
Measure
new stone formation and degree of nephrocalcinosis
N/A
N/A
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