KHENERGYZE
Research type
Research Study
Full title
A Phase IIb double-blind, randomised, placebo-controlled, multi-centre, confirmative three-way cross-over study on cognitive function with two doses of KH176 in subjects with a genetically confirmed mitochondrial DNA tRNALeu(UUR) m.3243A>G mutation.
IRAS ID
263546
Contact name
Grainne Gorman
Contact email
Sponsor organisation
Khondrion B.V.
Eudract number
2019-000599-40
Duration of Study in the UK
1 years, 2 months, 3 days
Research summary
The KHENERGYZE study: A study to investigate the safety and effectiveness, and specifically the ability to process information, of KH176 in subjects with a mitochondrial DNA mutation.
Mitochondrial diseases, estimated prevalence 1 in 4,300 adults, is caused by pathogenic mutations in genes finally encoding for mitochondrial proteins. When mitochondria are defective, this may result in a wide range of serious and debilitating illnesses and symptoms can include fatigue, exercise intolerance, muscle weakness and ataxia, heart failure, deafness, blindness, stunted growth, and cognitive dysfunction including learning disabilities.
Despite advances in the understanding of mitochondrial disorders, treatment options are extremely limited and, to date, largely supportive. This trial will test whether a drug called KH176 has an effect on the way patients process knowledge and information (cognitive functioning). The effect of two different doses: 50 mg and 100 mg will be tested and compared with a placebo.
The Khenergyze trial is for patients with mitochondrial disease caused by a specific m.3243A>G mutation in the DNA.
Participants will be asked to take KH176 two times a day for 12 weeks, divided into 3 different periods of 4 weeks with 2 weeks of wash-out in between. The total treatment period is 16 weeks.
Taking part will also require participants to visit a research centre in Newcastle for eleven visits. Participants will also receive five telephone calls from the research team to check on the progress.
Each visit will last 1-13 hours. During these visits a number of tests will be done (including blood tests, memory tests, smell and hearing tests, assessment of balance and walking). This study is expected to benefit the patient population with this mitochondrial disease by furthering the development of a new therapy and providing more information to those studying potential treatments for mitochondrial diseases.REC name
North East - Newcastle & North Tyneside 1 Research Ethics Committee
REC reference
19/NE/0310
Date of REC Opinion
22 Jan 2020
REC opinion
Further Information Favourable Opinion