Ignite Creation Date:
2024-06-16 @ 11:51 AM
Last Modification Date:
2024-10-26 @ 3:31 PM
Study NCT ID:
NCT06451757
Status:
NOT_YET_RECRUITING
Last Update Posted:
2024-06-11
First Post:
2024-05-29
Brief Title:
KHENERFIN Study A Trial to Evaluate the Efficacy and Safety of Sonlicromanol in Primary Mitochondrial Diseases
Sponsor:
Khondrion BV
Organization:
Khondrion BV
Study Overview
Official Title:
A Phase III Randomised Double-blind Placebo-controlled Parallel-group Pivotal Trial to Assess the Efficacy and Safety of Sonlicromanol in Adult Subjects With a Genetically Confirmed Mitochondrial DNA tRNALeuUUR m3243AG Variant
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-06
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:
KH176-301
Brief Summary:
The KHENERFIN study is investigating whether the study medicine sonlicromanol is able to improve symptoms of fatigue and the impact of fatigue on daily life and whether sonlicromanol is able improve physical abilities of people living with mitochondrial disease such as balance control and lower limb skeletal muscle strength
For this study the effects of sonlicromanol are compared with those from a placebo study medication that looks like the actual study medicine but contains no active medicine The study medicine or placebo is a powder that is dissolved in water and must be taken twice daily during the treatment period of 52 weeks
Additionally the study evaluates the efficacy of sonlicromanol on selected secondary and exploratory outcome measures as well as the safety and tolerability of sonlicromanol after 52 weeks of treatment with sonlicromanol
For this study the effects of sonlicromanol are compared with those from a placebo study medication that looks like the actual study medicine but contains no active medicine The study medicine or placebo is a powder that is dissolved in water and must be taken twice daily during the treatment period of 52 weeks
Additionally the study evaluates the efficacy of sonlicromanol on selected secondary and exploratory outcome measures as well as the safety and tolerability of sonlicromanol after 52 weeks of treatment with sonlicromanol
Detailed Description:
The KHENERFIN study is investigating whether the study medicine sonlicromanol is able to improve symptoms of fatigue and the impact of fatigue on daily life and whether sonlicromanol is able improve physical abilities of people living with mitochondrial disease such as balance control and lower limb skeletal muscle strength Additionally the study evaluates the efficacy of sonlicromanol on selected secondary and exploratory outcome measures as well as the safety and tolerability of sonlicromanol This study is a placebo controlled double blind study the effects of sonlicromanol will be compared with a placebo study medication that looks like the actual study medicine but contains no active medicine Neither the participants nor the study team know who is receiving the study medicine or placebo and participants are not able to change which treatment they are assigned
During the screening period which lasts a maximum of 6 weeks it is assessed whether the potential participant meets all requirements to participate in the study Patients who complete the screening phase and are enrolled in the study are randomly by chance assigned to receive either the study medicine sonlicromanol or placebo no active medication Participants have an equal chance of receiving either sonlicromanol or a placebo A final follow-up visit is scheduled 2 weeks after taking the last dose of study medication Total study duration is approximately 60 weeks The study medicine as well as placebo is a powder that is dissolved in water and must be taken twice daily during the treatment period of 52 weeks A total of 150 patients with a confirmed mitochondrial DNA tRNALeuUUR 3243AG mutation will participate in this study
During the screening period which lasts a maximum of 6 weeks it is assessed whether the potential participant meets all requirements to participate in the study Patients who complete the screening phase and are enrolled in the study are randomly by chance assigned to receive either the study medicine sonlicromanol or placebo no active medication Participants have an equal chance of receiving either sonlicromanol or a placebo A final follow-up visit is scheduled 2 weeks after taking the last dose of study medication Total study duration is approximately 60 weeks The study medicine as well as placebo is a powder that is dissolved in water and must be taken twice daily during the treatment period of 52 weeks A total of 150 patients with a confirmed mitochondrial DNA tRNALeuUUR 3243AG mutation will participate in this study
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
True
Is an FDA AA801 Violation?:
None