Ignite Creation Date:
2024-10-26 @ 3:42 PM
Last Modification Date:
2024-10-26 @ 3:42 PM
Study NCT ID:
NCT06643442
Status:
NOT_YET_RECRUITING
Last Update Posted:
None
First Post:
2024-06-03
Brief Title:
Repurposing Empagliflozin for DMD-associated Cardiomyopathy in Children 6-18 Years of Age
Sponsor:
None
Organization:
None
Study Overview
Official Title:
Repurposing Empagliflozin for Duchenne Muscular Dystrophy - Associated Cardiomyopathy a Pharmacokinetics Safety and Proof-of-concept Study Among Children 6-18 Years of Age
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
REDMeD
Brief Summary:
This study aims at exploring the use of empagliflozin in children and adolescents 6-18 years old with Duchenne muscular distrophy DMD - associated cardiomyopathy This molecule is effective in reducing hospitalizations and mortality in adults with heart failure and is used in adolescents with type 2 diabetes mellitus but little is known on children and adolescents with heart failure Particularly the best dose to use in this population is currently unknown This trial aims to
1 define a dose rationale for this indication and age group pharmacokinetic study
2 assess and monitor safety
3 assess ease-of-swallow
4 explore middle-term 3-6 months efficacy and efficacy markers
Participants will be asked to attend 5 study visits over 6 months and one end-study visit 2-12 weeks thereafter Visit 1 will entail an 8h day-hospital stay while Visits 2 3 4 and 5 as well as the end-study visit will be outpatient clinics approximately 2h Participants will be asked to take the studied drug once daily during the 6 months of the study period
No comparison group is foreseen for this study
1 define a dose rationale for this indication and age group pharmacokinetic study
2 assess and monitor safety
3 assess ease-of-swallow
4 explore middle-term 3-6 months efficacy and efficacy markers
Participants will be asked to attend 5 study visits over 6 months and one end-study visit 2-12 weeks thereafter Visit 1 will entail an 8h day-hospital stay while Visits 2 3 4 and 5 as well as the end-study visit will be outpatient clinics approximately 2h Participants will be asked to take the studied drug once daily during the 6 months of the study period
No comparison group is foreseen for this study
Detailed Description:
Cardiac disease represents the main life-limiting condition in Duchenne muscular dystrophy DMD It is important to recognize and address this early in the disease course Because of lack of DMD specific drugs present attitudes for established DMD-related cardiomyopathy ground on current treatment for heart failure Unfortunately however current heart failure therapy in Paediatrics is still unsatisfactory with high in-hospital 7-26 and 5-year mortality 30-50 Furthermore mortality rate for DMD cardiomyopathy is worse than similarly aged idiopathic dilated cardiomyopathy DCM patients
Among the recent improvements in adult heart failure management the sodium glucose transporter type 2 inhibitors SGLT2i dapagliflozin and empagliflozin were found to reduce cardiovascular death or worsening heart failure by 25 on top of optimal medical therapy Indeed since 2021 they have been recommended as part of standard heart failure therapy
In the past paediatric heart failure trials often failed mainly because of suboptimal dose or inappropriate formulations and endpoints
This phase IIa open-label trial is designed to characterize pharmacokinetics primary outcome ease-of-swallow safety and explore potential efficacy markers secondary outcomes of empagliflozin in 12 children and adolescents with DMD-related cardiomyopathy so to inform the design and performance of subsequent state-of-the-art high-quality efficacy trials
Participants will receive empagliflozin during 6 months They will have 5 visits one end-study visit and 7 to 8 pharmacokinetic samples The timing of these samples will be optimized exploiting contemporary modeling and simulation techniques
Safety evaluation will occur throughout the study while ease-of-swallow will be evaluated at Visit 1 and efficacy markers at Visits 1 4 and 5
Pharmacokinetic modeling will characterize primary and secondary pharmacokinetic parameters and allow to define the optimal paediatric dose informing both current compassionate-care use and the design of future efficacy trials
Among the recent improvements in adult heart failure management the sodium glucose transporter type 2 inhibitors SGLT2i dapagliflozin and empagliflozin were found to reduce cardiovascular death or worsening heart failure by 25 on top of optimal medical therapy Indeed since 2021 they have been recommended as part of standard heart failure therapy
In the past paediatric heart failure trials often failed mainly because of suboptimal dose or inappropriate formulations and endpoints
This phase IIa open-label trial is designed to characterize pharmacokinetics primary outcome ease-of-swallow safety and explore potential efficacy markers secondary outcomes of empagliflozin in 12 children and adolescents with DMD-related cardiomyopathy so to inform the design and performance of subsequent state-of-the-art high-quality efficacy trials
Participants will receive empagliflozin during 6 months They will have 5 visits one end-study visit and 7 to 8 pharmacokinetic samples The timing of these samples will be optimized exploiting contemporary modeling and simulation techniques
Safety evaluation will occur throughout the study while ease-of-swallow will be evaluated at Visit 1 and efficacy markers at Visits 1 4 and 5
Pharmacokinetic modeling will characterize primary and secondary pharmacokinetic parameters and allow to define the optimal paediatric dose informing both current compassionate-care use and the design of future efficacy trials
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None