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Trial details
A Phase 3, Open-label, Uncontrolled Study to Evaluate the Activity, Safety, Pharmacokinetics and Pharmacodynamics of Roxadustat for the Treatment of Anemia in Pediatric Participants with Chronic Kidney Disease
Current status:
Approved
|
Date registered:
07/03/2024
Trial version(s)
Current: 05/01/2024
Click here to view the tips and fields' descriptions
Main Information
Primary registry identifying number
LBCTR2024015495
Protocol number
1517-CL-1003
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
30/01/2024
Primary sponsor
Astellas
Primary sponsor: Country of origin
USA
Public title
A Phase 3, Open-label, Uncontrolled Study to Evaluate the Activity, Safety, Pharmacokinetics and Pharmacodynamics of Roxadustat for the Treatment of Anemia in Pediatric Participants with Chronic Kidney Disease
Acronym
-
Scientific title
A Phase 3, Open-label, Uncontrolled Study to Evaluate the Activity, Safety, Pharmacokinetics and Pharmacodynamics of Roxadustat for the Treatment of Anemia in Pediatric Participants with Chronic Kidney Disease
Acronym
-
Brief summary of the study: English
Roxadustat has been approved for the treatment of adult patients with symptomatic anemia associated with CKD. This study is part of a program to develop Roxadustat in the treatment of anemia in pediatric CKD patients. Since no data in pediatrics is currently available, per the Committee for Medicinal Products for Human Use scientific advice, a study duration of 52 weeks allows for adequate characterization of the safety and activity of Roxadustat. In the EU, this study is in accordance with the PIP approved by the PDCO.
Brief summary of the study: Arabic
تمت الموافقة على Roxadustat لعلاج المرضى البالغين الذين يعانون من فقر الدم العرضي المرتبط بمرض الكلى المزمن. هذه الدراسة هي جزء من برنامج لتطوير Roxadustat في علاج فقر الدم لدى مرضى CKD من الأطفال. نظرا لعدم توفر بيانات في طب الأطفال حاليا ، وفقا للمشورة العلمية للجنة المنتجات الطبية للاستخدام البشري ، فإن مدة الدراسة البالغة 52 أسبوعا تسمح بالتوصيف المناسب لسلامة ونشاط Roxadustat. في الاتحاد الأوروبي ، تتوافق هذه الدراسة مع PIP المعتمدة من قبل PDCO.
Health conditions/problem studied: Specify
Anemia in Pediatric Participants with Chronic Kidney Disease.
Interventions: Specify
Roxadustat will be administered 3 times a week according to weight-based pediatric dosing. A suitable, age-appropriate, roxadustat azo dye-free mini-tablet or tablet formulation will be utilized by participants who are able to swallow tablets and is to be diluted with purified water and administered via syringe orally or via a gastric tube as an aqueous dispersion for participants unable to swallow tablets. For ESA-treated participants, the appropriate starting dose of roxadustat will be based on prior ESA dose and the participant’s body weight. Titrations to the roxadustat starting dose will be based on current Hb level and change in Hb over the previous 4 weeks.
Key inclusion and exclusion criteria: Inclusion criteria
1. IRB/IEC approved written informed consent and privacy language as per national regulations (e.g., Health Insurance Portability and Accountability Act authorization for US study sites) must be obtained from the participant or participant’s parent or legal guardian, and if required, child assent, prior to any study-related procedures (including withdrawal of prohibited medication, if applicable). 2. Participant is aged 2 to < 18 years old at the screening/first study visit. 3. Participant has a diagnosis of anemia in CKD Kidney Disease Outcomes Quality Initiative stages 3 or 4 or 5. This can include participants not on dialysis or DD participants (including hemodialysis, peritoneal dialysis and hemodiafiltration participants). 4. Participants not on dialysis must have an estimated glomerular filtration rate (Schwartz formula) of < 60 mL/min per 1.73 m2. 5. ESA-treated participants should have a screening Hb level, assessed via HemoCue, between 10.0 and 12.0 g/dL; ESA-naïve participants can have a Hb level ≤ 11 g/dL. 6. Participant has a ferritin level > 100 ng/mL or a TSAT value > 20%. 7. Participant has an ALT and AST ≤ 2 x ULN and TBL ≤ 1.5 x ULN at enrollment visit. 8. Participant is treated with an ESA or is ESA-naïve, where ESA status is defined as: a. ESA-treated: Participant is taking a stable dose of an ESA for at least 4 weeks prior to screening. b. ESA-naïve: Participant has no prior ESA exposure OR participant’s total prior ESA exposure ≤ 3 weeks within the preceding 4 weeks from screening OR participant was previously treated with and discontinued an ESA ≥ 8 weeks prior to screening. 9. Female participant is not pregnant (see [Appendix 2]) and at least 1 of the following conditions apply: c. Not a WOCBP (see [Appendix 2]) d. WOCBP who agrees to follow the contraceptive guidance (see [Appendix 2]) from the time of informed consent through at least 4 weeks after final study intervention administration. 10. Female participant must agree not to breastfeed starting at screening and throughout the study and for 4 weeks post-last roxadustat dose. 11. Female participant must not donate ova starting at first administration of roxadustat and throughout the study period and for 4 weeks post-last roxadustat dose. 12. Male participants with female partner(s) of childbearing potential (including breastfeeding partner) must agree to use contraception (see [Appendix 2]) throughout the treatment period and for 4 weeks post-last roxadustat dose. 13. Male participants must not donate sperm during the treatment period and for 4 weeks post-last roxadustat dose. 14. Male participants with pregnant partner(s) must agree to remain abstinent or use a condom for the duration of the pregnancy throughout the study period and for 4 weeks post-last roxadustat dose. 15. Participant and/or participant’s parent or legal guardian agrees for the participant not to participate in another interventional study while participating in the present study.
Key inclusion and exclusion criteria: Gender
Both
Key inclusion and exclusion criteria: Age minimum
2
Key inclusion and exclusion criteria: Age maximum
18
Key inclusion and exclusion criteria: Exclusion criteria
1. Participant has received any investigational therapy within 28 days or 5 half-lives, whichever is longer, prior to screening. 2. Participant has any medical condition, including active, systemic or clinically significant infection, which in the opinion of the investigator or medical monitor, may pose a safety risk to a participant in this study, which may confound the safety or activity assessment or may interfere with study participation making the participant unsuitable for study. 3. Participant has a known or suspected hypersensitivity to roxadustat, related HIF-PHI, or any components of the formulation used. 4. Participant has uncontrolled hypertension as judged by the principal investigator in the 2 weeks prior to screening. 5. Participant has a known hematologic disease other than anemia secondary to renal disease, (e.g., history of sickle cell disease, sickle cell anemia, hemoglobin sickle cell disease, or hemoglobin sickle cell beta thalassemia). 6. Participant has untreated hypothyroidism. 7. Participant has severe hyperparathyroidism defined as serum PTH levels above 1000 pg/mL intact PTH within 4 weeks of screening. 8. Participant has a functioning kidney allograft. 9. Participant has a folate or B12 or carnitine deficiency. Acceptable if treated to normal values within 4 weeks of screening. 10. Participant has a known active malignancy or malignancy within 18 months before the screening visit. Radiation or chemotherapy must be completed at least 12 months before the screening visit. 11. Participant has a scheduled living donor organ transplantation date within 12 weeks of screening. 12. Participant has a whole blood or packed RBC transfusion during the 8 weeks prior to screening. 13. Participant has any current condition leading to active significant blood loss in the past 4 weeks. 14. Participant has a diagnosis of hemolytic uremic syndrome within 12 weeks prior to screening. a. Participant who has a previous diagnosis of atypical hemolytic syndrome must be relapse-free (stable Hb, normal platelet count, normal serum lactate dehydrogenase, and normal haptoglobin level) for more than 12 weeks prior to screening. 15. Participant has a history of chronic liver disease, including comorbidity with autosomal recessive polycystic kidney disease, cystinosis, and primary hyperoxaluria. 16. Participant had an episode of peritonitis within 30 days of screening. 17. Participant has active inflammation such as glomerulonephritis flare (i.e., lupus nephritis, IgA nephritis, rapidly progressive glomerulonephritis, membranoproliferative glomerulonephritis, antineutrophil cytoplasmic antibodies vasculitis) requiring pulse corticosteroid treatment or induction treatment with an immunosuppressive agent (i.e., cyclophosphamide, rituximab, or another monoclonal antibody) within 6 weeks of screening visit. Receipt of monoclonal antibody or biologic for maintenance treatment of underlying condition is acceptable. 18. Participant has a known history of human immunodeficiency virus infection.
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
Uncontrolled
Study phase
3
Study design: Purpose
Treatment
Study design: Assignment
Single
IMP has market authorization
Yes, Worldwide
IMP has market authorization: Specify the countries
China, Japan, Europe, Great Britan
Name of IMP
Roxadustat
Year of authorization
2018
Month of authorization
12
Type of IMP
Others
Type of IMP: Specify
Hemopoesis Stimulating Agent
Pharmaceutical class
Hypoxia-inducible factor, prolyl hydroxylase inhibitor (HIF-PHI).
Therapeutic indication
Symptomatic anemia associated with CKD.
Therapeutic benefit
In adult CKD participants with anemia not on dialysis, there was a statistically significant increase in Hb from baseline to weeks 28 through 36 in participants treated with roxadustat, showing superiority of roxadustat over placebo. The proportion of participants who achieved Hb response during the first 24 weeks without rescue therapy was higher in the roxadustat group compared with placebo. This difference was statistically significant and showed superiority of roxadustat over placebo. In addition, roxadustat was shown to be non-inferior to an ESA in adult CKD participants not on dialysis. In adult CKD participants with anemia on dialysis, the change from baseline in Hb without use of rescue therapy up to weeks 28 through 36 was similar between groups and noninferiority of roxadustat to ESA was confirmed. Similarly, the proportion of participants who achieved Hb response without use of rescue therapy up to weeks 28 through 36 was similar between groups and noninferiority of roxadustat to ESA was confirmed.
Biospecimen retention
None retained
Biospecimen description
NA
Target sample size
100
Actual enrollment target size
Date of first enrollment: Type
Anticipated
Date of first enrollment: Date
01/06/2024
Date of study closure: Type
Anticipated
Date of study closure: Date
31/12/2030
Recruitment status
Pending
Date of completion
IPD sharing statement plan
No
IPD sharing statement description
NA
Additional data URL
-
Summary Results
Secondary Identifying Numbers
Full name of issuing authority
Secondary identifying number
NA
NA
Sources of Monetary or Material Support
Name
Astellas
Secondary Sponsors
Name
NA
Contact for Public/Scientific Queries
Contact type
First name
Last name
Address
Country
Telephone
Email
Affiliation
Public
Aziz
Zoghbi
MCT Lebanon s.a.r.l.
Lebanon
01-- 612500 ext2040
Zog_Az@Mctcro. com
CRO
Scientific
Chebl
Mourani
Hotel Dieu de France Hospital
Lebanon
03 290 090
cheblmourani@gmail.com
PI
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
Pediatrician
Approved
Ethics Review
Ethics approval obtained
Approval date
Contact name
Contact email
Contact phone
Hotel Dieu de France
02/10/2023
Virginia El khoury
Virginia.elkhoury@usj.edu.lb
+961 1 421229
Countries of Recruitment
Name
Lebanon
Health Conditions or Problems Studied
Condition
Code
Keyword
Anemia in pediatrics with CKD
Anaemia in chronic diseases classified elsewhere (D63)
Anemia in Chronic Kidney Disease
Interventions
Intervention
Description
Keyword
Roxadustat
Hemopoiesis Stimulating Agent
Treatment
Primary Outcomes
Name
Time points
Measure
The Primary Objective is to Evaluate the activity of Roxadustat for the treatment of anemia in adolescents and children with CKD.
Weeks 20 to 24
Change in Hb level between baseline (before start of dosing) and average Hb level over treatment weeks 20 to 24. The 24-week treatment period is defined as 4 weeks of fixed dose treatment followed by 20 weeks of dose titration(s)
Key Secondary Outcomes
Name
Time points
Measure
Evaluate the PK and PD of roxadustat for the treatment of anemia in adolescents and children with CKD
-
PK parameters: Cmax, AUC, CL/F, Tmax ● PD assessments: Hb level and dose titration history at all timepoints
Evaluate the safety of roxadustat for the treatment of anemia in adolescents and children with CKD, including cardiovascular and thrombotic risks
-
● Number and percent of TEAEs ● Number and percent of treatment-emergent cardiovascular and thrombotic AEs ● Tabulations of safety assessments (e.g., clinical laboratory tests, vital signs, growth parameters and physical examinations)
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