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Trial details
Patiromer for treatment of hyperkalaemia in children from birth to <6 years of age
Current status:
Approved
|
Date registered:
13/06/2023
Trial version(s)
Current: 14/12/2022
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Main Information
Primary registry identifying number
LBCTR2023055223
Protocol number
RLY5016-208p
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
14/12/2022
Primary sponsor
Vifor Pharma, Inc.
Primary sponsor: Country of origin
USA
Public title
Patiromer for treatment of hyperkalaemia in children from birth to <6 years of age
Acronym
Scientific title
A 2-Part, Open-Label, Phase 2, Multiple Dose Study to Evaluate the Pharmacodynamic Effects, Safety, and Tolerability of Patiromer in Children under 6 Years of Age with Hyperkalaemia (EMERALD 2)
Acronym
Brief summary of the study: English
This is a 2-part, multicentre, open-label, Phase 2, multiple dose, 2-age cohort (0 to <2 and 2 to <6 years of age) study, consisting of a 4-week pharmacodynamic (PD)/dose-ranging period followed by an optional 52-week safety-extension period to assess change in potassium levels from baseline to Day 28 following administration of different doses of patiromer administered in children 0 to <6 years of age with hyperkalaemia and To assess the safety and tolerability of patiromer in children 0 to <6 years of age with hyperkalaemia.The maximum study duration for a participant will be up to 58 weeks, including an up to 2 weeks screening period, a 4-week PD/dose-ranging period (Part 1), and a 52-week optional safety-extension period (Part 2).The study will include 2 cohorts: Cohort A (2 to <6 years of age) and Cohort B (0 to <2 years of age).
Brief summary of the study: Arabic
هذه دراسة مكونة من جزئين ، متعددة المراكز ، مفتوحة التسمية ، المرحلة 2 ، جرعة متعددة ، فئة عمرية 2 (من 0 إلى أقل من 2 و 2 إلى أقل من 6 سنوات من العمر) ، وتتألف من 4 أسابيع من الديناميكيات الدوائية (PD) / فترة الجرعة متبوعة بفترة تمديد أمان اختيارية مدتها 52 أسبوعًا لتقييم التغيير في مستويات البوتاسيوم من خط الأساس إلى اليوم 28 بعد إعطاء جرعات مختلفة من باتيرومير المعطى للأطفال من سن 0 إلى أقل من 6 سنوات مع فرط بوتاسيوم الدم ولتقييم السلامة وتحمل البيرومير لدى الأطفال من سن 0 إلى أقل من 6 سنوات مع فرط بوتاسيوم الدم ، وستصل مدة الدراسة القصوى للمشارك إلى 58 أسبوعًا ، بما في ذلك فترة فحص تصل إلى أسبوعين ، و 4 أسابيع PD / فترة نطاق الجرعة ( الجزء الأول) ، وفترة تمديد أمان اختيارية مدتها 52 أسبوعًا (الجزء 2). ستشمل الدراسة مجموعتين: الفوج أ (من 2 إلى أقل من 6 سنوات من العمر) والفوج ب (من 0 إلى أقل من سنتين من العمر).
Health conditions/problem studied: Specify
Hyperkalaemia
Interventions: Specify
The study will include 2 parts: 1) a 4-week PD/dose-ranging period in an interventional clinical trial setting, followed by 2) an optional 52-week safety-extension period. In the optional safety-extension period, the frequency and procedures of visits will adhere to the standard of care for paediatric subjects of the specific study site; however, at least every 3 months. Potassium values, patiromer dosing and compliance, and adverse events (AEs) will be reported. During this study, potassium values can be measured as serum, plasma, whole blood, or capillary blood potassium. However, any of the above methods need to be kept constant throughout the study, from screening visit to end of study.
Key inclusion and exclusion criteria: Inclusion criteria
1. Paediatric subjects (<6 years of age) with hyperkalaemia at screening. 2. Subject’s age should not reach 6 years during the 28 days of the PD/ dose-ranging period. 3. Subject is able to receive regular external feeding and medication, including via tubes, e.g., percutaneous endoscopic gastrostomy (PEG). 4. At screening/baseline, 2 potassium values need to be above the age-appropriate upper limit of normal (ULN) of the local laboratory. However, the average of the 2 potassium values from 2 separate sample collections (1 sample from screening/baseline and 1 sample not older than 30 days) needs to be above the age-appropriate ULN of the local laboratory plus 0.5 mEq/l (equivalent to 0.5 mmol/l). 5. In the opinion of the Investigator, the subject is expected to require treatment for hyperkalaemia for at least 28 days upon enrolment in the study. 6. If taking any renin-angiotensin aldosterone system inhibitors (RAASi), beta blockers, fludrocortisone, or diuretic medications, must be on a stable dose for at least 14 days prior to screening. 7. Parent(s) or legally acceptable representative(s) has provided the appropriate written informed consent, in accordance with local regulations. The assent of the child should also be obtained when appropriate or if requested by the Institutional Review Board (IRB)/Ethics Committee (EC)/Independent Ethics Committee (IEC). The written informed consent must be provided before any study-specific procedures are performed including screening procedures. 8. Parent(s) or legally authorised representative(s) or another appropriate person delegated by the legally authorised representatives must be available to help the study-site personnel ensure follow-up; accompany the participant to the study site on each assessment day according to the Schedule of Events (Table 1, Table 2) (e.g., able to comply with scheduled visits, treatment plan, laboratory tests and other study procedures); accurately and reliably dispense investigational product as directed.
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
1. Preterm birth infants with <37 weeks of gestation cannot be included in Cohort B. 2. Participants who due to their general condition, e.g., anaemia or low body weight are not suitable to have blood volume withdrawn as specified in the Schedule of Events (Table 1, Table 2). 3. Subjects with pseudo-hyperkalaemia due to haemolysis or to abnormally high numbers of platelets (above ULN), leukocytes (above ULN), or erythrocytes (above ULN) at screening based on results obtained from the local laboratory. 4. Any subject with evidence of potential potassium-related 12-lead electrocardiogram (ECG) changes (i.e., changes consistent with hyper- or hypokalaemia) at screening. 5. Any subject with serum magnesium <1.4 mg/dl at screening/baseline. 6. Any of the following renal conditions: maintenance haemodialysis or peritoneal dialysis, renal artery stenosis, and acute kidney injury (defined by 2012 Kidney Disease Improving Global Outcomes) or a history of acute renal insufficiency in the past 3 months. Note: CKD is not excluded. 7. A history of or current diagnosis of a severe gastrointestinal (GI) diagnosis or surgery that could affect GI transit of the drug (delayed gastric emptying), such as a severe swallowing disorder, severe gastroesophageal reflux, uncorrected pyloric stenosis, intussusception, any other intestinal obstruction (e.g., Hirschsprung disease, chronic intestinal pseudo-obstruction, clinically significant postsurgical abdominal adhesions) or any gut-shortening surgical procedure prior to screening. Pre-gastric above-mentioned pathologies may be disregarded in case of existence of a PEG tube, as the PEG tube will serve for nutrition and investigational product administration. 8. Liver enzymes (alanine aminotransferase, aspartate aminotransferase) more than 3 times the ULN at screening, based on the local laboratory, as well as subject’s respective age. 9. Active cancer, currently on cancer treatment, or history of cancer in the past 2 years (except for non-melanoma skin cancer). 10. Recipient of any organ transplant requiring treatment with immunosuppressive therapy or scheduled for kidney transplant procedure during the first 28 days after Day 1. 11. History of sudden infant death in a sibling. 12. Has severe hypoxaemia, respiratory acidosis, asphyxia, or hypotension 3 months before screening based on assessment of the Investigator. 13. Subjects treated with sodium polystyrene sulphonate, calcium polystyrene sulphonate, or sodium zirconium cyclosilicate within the last 48 hours prior to fulfilling the baseline potassium assessments requested in Inclusion Criterion 4. 14. Use of the following medications if doses have not been stable for at least 14 days prior to screening or if doses are anticipated to change during the 4-week PD/dose-ranging period: digoxin, bronchodilators, theophylline, heparins (including low molecular heparins), tacrolimus, mycophenolate mofetil, cyclosporine, trimethoprim, or cotrimoxazole. 15. Use of any investigational product for an unapproved indication within 30 days prior to screening or within 5 half-lives, whichever is longer. 16. Known hypersensitivity to patiromer or its components. 17. In the opinion of the Investigator, parent(s) or legal representative(s) inability to comply with the protocol. 18. In the opinion of the Investigator, any medical condition, uncontrolled systemic disease, or serious intercurrent illness that would significantly decrease study compliance or jeopardise the safety of the subject or potentially affect the quality of the data such as: hyperkalaemia at screening that requires emergency intervention; cardiovascular event or intervention within 3 months prior to screening; a haemodynamically unstable arrhythmia; hospitalisation for heart failure within the past 3 months; poorly controlled blood pressure; poorly controlled diabetes mellitus or frequent need for adjustment in insulin prescription or recent hospitalisation for treatment of hyper or hypoglycaemia. 19. If the child is being breastfed: a) There is suspicion of current alcohol or substance misuse/abuse in breastfeeding mother b) The breastfeeding mother is taking potassium supplements.
Type of Study
Type
Interventional
Type of intervention
Pharmaceutical
Trial scope
Other
Trial scope: Specify scope
Pharmacodynamic, Efficacy, Safety, Dose response, Tolerability
Study design: Allocation
Non-randomized controlled trial
Study design: Masking
Open (masking not used)
Study design: Control
N/A
Study phase
2
Study design: Purpose
Other
Study design: Specify purpose
Pharmacodynamic, Efficacy, Safety, Dose response
Study design: Assignment
Other
Study design: Specify assignment
cohort design
IMP has market authorization
Yes, Lebanon and Worldwide
IMP has market authorization: Specify the countries
USA, EU, Norway, Iceland, Lichtenstein, Switzerland and Australia, Eurasian Economic Union (EAEU; Russia)
Name of IMP
Patiromer
Year of authorization
2015
Month of authorization
10
Type of IMP
Others
Type of IMP: Specify
Potassium binder
Pharmaceutical class
Potassium binder
Therapeutic indication
treatment of hyperkalaemia in Pediatric population
Therapeutic benefit
This study will assess safety and dosing in this population and represents at the same time a potential treatment for hyperkalaemic paediatric subjects.
Biospecimen retention
None retained
Biospecimen description
NA
Target sample size
21
Actual enrollment target size
Date of first enrollment: Type
Anticipated
Date of first enrollment: Date
17/04/2023
Date of study closure: Type
Anticipated
Date of study closure: Date
30/04/2026
Recruitment status
Other
Recruitment status: Specify
Not Initiated
Date of completion
IPD sharing statement plan
Yes
IPD sharing statement description
The sponsor assures that the key design elements of this protocol will be posted in a publicly accessible database such as ClinicalTrials.gov. The clinical study report will be submitted to the regulatory authorities in Lebanon within one year of the end of the study (worldwide). The detailed obligations regarding the publication of any data, material results, or other information generated or created in relation to the study shall be set out in the agreement between each investigator and the sponsor.
Additional data URL
Summary Results
Secondary Identifying Numbers
Full name of issuing authority
Secondary identifying number
Sources of Monetary or Material Support
Name
Secondary Sponsors
Name
Contact for Public/Scientific Queries
Contact type
First name
Last name
Address
Country
Telephone
Email
Affiliation
Public
Aziz
Zoghbi
MCT-CRO,Berytech Technology and Health,5th Floor Damascus Road,Beirut,Lebanon
Lebanon
009611612 500
zog_az@mctcro. com
Regional Manager
Scientific
Chebl
Mourani
Hotel Dieu de France, Alfred Naccache Avenue, Achrafieh, Beirut, Lebanon
Lebanon
009613185632
cheblmourani@gmail.com
Principal Investigator
Scientific
Katia
El Taoum
AUBMC
Lebanon
+9618822363
ke27@aub.edu.lb
Principal Investigator
Centers/Hospitals Involved in the Study
Center/Hospital name
Name of principles investigator
Principles investigator speciality
Ethical approval
Hotel dieu de france
Chebl Mourani
Pediatric nephrology
Approved
American University of Beirut Medical Center
Katia El Taoum
Pediatric
Pending
Ethics Review
Ethics approval obtained
Approval date
Contact name
Contact email
Contact phone
Hotel Dieu de France
31/01/2023
Nancy Alam
nancy.alam@usj.edu.lb
+961 1 421 000 ext 2335
Countries of Recruitment
Name
United States of America
Health Conditions or Problems Studied
Condition
Code
Keyword
hyperkalaemia
Hyperkalaemia (E87.5)
hyperkalaemia
Interventions
Intervention
Description
Keyword
Patiromer for Cohort A (2 to <6 years of age)
The patiromer starting dose is based on Body Weight (Based on Outcomes and DSMB Recommendation).Titration during the study will depend on the potassium response of the subject.
Patiromer
Patiromer for Cohort B (0 to <2 years of age)
To ensure safety in Cohort B, dosing will begin with Cohort A (2 to <6 years of age). After 3 subjects from Cohort A have completed 4 weeks of treatment, the Data Safety and Monitoring Board (DSMB) will evaluate safety in these 3 subjects. If the safety profile is acceptable, enrolment will then proceed in Cohort B (0 to <2 years of age). The patiromer starting dose in Cohort B is based on body weight and DSMB recommendations, and it will be adapted during the study depending on the potassium response of the subject.
Patiromer
Primary Outcomes
Name
Time points
Measure
change in potassium levels
from baseline to Day 28
blood potassium
Key Secondary Outcomes
Name
Time points
Measure
change in potassium levels
from baseline to Day 3, Day 7, and Day 14 and end of the study Part 1 and during the optional safety-extension period.
blood potassium
treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
Part 1: Day 1 up to end of treatment (Day 28 ±3 Days); Part 2: Day 1 up to follow-up visit (Up to 54 weeks)]
NA
vital signs, 12-lead ECG, and clinical safety laboratory evaluations
From baseline to Day 3, Day 7, Day 14, Day 28 and during Part 2: up to 52 weeks
Change in resting heart rate (beats per minute) in systolic blood pressure (mmHg) in diastolic blood pressure (mmHg) in body temperature (Celsius) Normal and abnormal 12-lead electrocardiogram (ECG) findings
occurrence of blood potassium
From baseline to Day 3, Day 7, Day 14, Day 28 and during Part 2: up to 52 weeks
blood potassium
Occurrence of blood magnesium
From baseline to Day 3, Day 7, Day 14, Day 28 and during Part 2: up to 52 weeks
blood magnesium
Occurrence of serum calcium, phosphate, fluoride, creatinine, bicarbonate, and blood urea nitrogen levels
From baseline to Day 3, Day 7, Day 14, Day 28 and during Part 2: up to 52 weeks
blood test
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