KHENEREXT
Research type
Research Study
Full title
A Phase IIb open-label, multi-centre, extension study to explore the long-term safety and efficacy of KH176 in subjects with a genetically confirmed mitochondrial DNA tRNALeu(UUR) m.3243A>G mutation who have completed the Khenergyze study KH176-202.
IRAS ID
303083
Contact name
Gráinne Gorman
Contact email
Sponsor organisation
Khondrion B.V.
Eudract number
2020-000832-23
Duration of Study in the UK
0 years, 8 months, 0 days
Research summary
The KHENEREXT study is a follow-up to the KHENERGYZE study, and the aim of this study is to investigate the safety and efficacy of the treatment with KH176 following use over a one year period. The study is for patients who have been diagnosed with a mitochondrial disease caused by a specific mutation in the DNA and who have completed the KHENERGYZE study. An important difference with the previous study, the KHENERGYZE study, is that the KHENEREXT study is a so-called "open-label" study, which means that the subjects will receive active treatment at a known dose during the treatment period. After completion of this open label extension study, patients can continue to receive the study medication in the context of a so-called “compassionate use” program. This is a program in which participants can continue to receive the study medication even though it has not yet been admitted to the market. In total the participation in the KHENEREXT study will take approximately 13 months, consisting of approximately 1-week screening, 12 months / 52 weeks treatment and 4 weeks follow-up after last medication intake. The number of subjects is dependent on the number of patients who completed the KHENERGYZE study. The KHENEREXT study will be conducted at the same sites in the UK, Netherlands, Germany and Denmark.
REC name
North East - Tyne & Wear South Research Ethics Committee
REC reference
21/NE/0151
Date of REC Opinion
21 Sep 2021
REC opinion
Further Information Favourable Opinion