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Trial details
Trial details
A Phase 2 Open-Label Study to Evaluate the Safety and Efficacy of DCR-PHXC in Patients With Primary Hyperoxaluria Type 1 or 2 and Severe Renal Impairment, With or Without Dialysis
Current status:
Approved
|
Date registered:
29/02/2024
Trial version(s)
Current: 21/11/2022
Click here to view the tips and fields' descriptions
Main Information
Primary registry identifying number
LBCTR2022125202
Protocol number
DCR-PHXC-204
MOH registration number
NCT04580420
Trial already registered with the MoPH
Study registered at the country of origin
Type of registration
Prospective
Date of registration in national regulatory agency
21/01/2021
Primary sponsor
Dicerna Pharmaceuticals
Primary sponsor: Country of origin
United States of America
Public title
A Phase 2 Open-Label Study to Evaluate the Safety and Efficacy of DCR-PHXC in Patients With Primary Hyperoxaluria Type 1 or 2 and Severe Renal Impairment, With or Without Dialysis
Acronym
Scientific title
A Phase 2 Open-Label Study to Evaluate the Safety and Efficacy of DCR-PHXC in Patients With Primary Hyperoxaluria Type 1 or 2 and Severe Renal Impairment, With or Without Dialysis
Acronym
PHYOX7
Brief summary of the study: English
This is a repeat dose, uncontrolled, open-label, Phase 2 study of DCR-PHXC in patients with primary hyperoxaluria type 1 (PH1) or type 2 (PH2) and severe renal impairment, with or without dialysis.
Brief summary of the study: Arabic
هذه جرعة متكررة ، غير خاضعة للرقابة ، مفتوحة التسمية ، دراسة المرحلة 2 لـ DCR-PHXC في المرضى مع فرط أوكسالات البول الأولي من النوع 1 (PH1) أو النوع 2 (PH2) والضعف الكلوي الشديد ، مع أو بدون غسيل الكلى.
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 include: • Genetically confirmed PH1 or PH2 • Estimated glomerular filtration rate (eGFR) < 30 mL/min normalized to 1.73 m2 body surface area (BSA) • Mean of 2 Pox values > 20 µmol/L during Screening • For participants receiving hemodialysis or peritoneal dialysis, total duration of hemodialysis or peritoneal dialysis must be less than or equal to 18 months and stable dialysis regimen for at least 2 weeks prior to Screening • Willing to adhere to a low oxalate diet and avoid high doses of vitamin C
Key inclusion and exclusion criteria: Gender
Both
Key inclusion and exclusion criteria: Age minimum
0
Key inclusion and exclusion criteria: Age maximum
99
Key inclusion and exclusion criteria: Exclusion criteria
Key exclusion criteria include: • Hepatic transplantation, prior to or planned in the 6 months from Day 1. Renal transplantation planned in the 6 months from Day 1. Prior renal transplantation is allowed. • Documented evidence of clinical manifestations of systemic oxalosis
Type of Study
Type
Interventional
Type of intervention
Pharmaceutical
Trial scope
Therapy
Trial scope: Specify scope
Study design: Allocation
Single Arm Study
Study design: Masking
Open (masking not used)
Study design: Control
N/A
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
RNA therapy
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
30
Actual enrollment target size
Date of first enrollment: Type
Anticipated
Date of first enrollment: Date
30/03/2023
Date of study closure: Type
Anticipated
Date of study closure: Date
30/11/2026
Recruitment status
Pending
Date of completion
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
NCT04580420
Sources of Monetary or Material Support
Name
Dicerna Pharmaceuticals, Inc. 75 Hayden Ave. Lexington, MA 02421 US (617) 621-8097
Secondary Sponsors
Name
N/A
Contact for Public/Scientific Queries
Contact type
First name
Last name
Address
Country
Telephone
Email
Affiliation
Public
Chadi
Safa
ebanon. Baabda. Chiah. Ain el remeneh
Lebanon
009617125 1819
csafa@ctifact.com
CTI
Scientific
Chebl
Mourani
Alfred Naccache Blvd, External Viewing Tower, Floor 4, Room 9403
Lebanon
03 290090
cheblmourani@g mail.com
HDF
Centers/Hospitals Involved in the Study
Center/Hospital name
Name of principles investigator
Principles investigator speciality
Ethical approval
Hotel Dieu De France Hospital
Dr Chebl Mourani
Pediatric Nephrologist
Approved
Ethics Review
Ethics approval obtained
Approval date
Contact name
Contact email
Contact phone
Hotel Dieu de France
15/11/2022
Nancy El Alam
nancy.alam@usj.edu.lb
+961 1 421 000
Countries of Recruitment
Name
United States of America
Germany
France
Spain
Italy
Lebanon
United Kingdom
United Arab Emirates
Romania
Health Conditions or Problems Studied
Condition
Code
Keyword
Primary Hyperoxaluria
2-Propanol (T51.2)
N/A
Interventions
Intervention
Description
Keyword
Nedosiran
IMP
N/A
Primary Outcomes
Name
Time points
Measure
Key Secondary Outcomes
Name
Time points
Measure
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
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