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. - KHENERGYZE

Khondrion B.V.0 sites27 target enrollmentSeptember 10, 2019

Overview

Phase: Phase 1
Intervention: Not specified
Conditions: A genetically confirmed mitochondrial desoxyribonucleic acid (DNA) transfer ribonucleic acid (tRNA)Leu(UUR) m.3243A>G mutation (including but not limited to MELAS, MIDD and mixed compositions).
Sponsor: Khondrion B.V.
Enrollment: 27
Status: Active, not recruiting
Last Updated: 3 years ago

No summary available.

Registry

who.int

Start Date

September 10, 2019

End Date

TBD

Last Updated

3 years ago

Study Type

Interventional clinical trial of medicinal product

Sex

All

Sponsor

Khondrion B.V.

•1\.Males and females aged 18 years or older at screening.
•2\.Ability and willingness to provide written Informed Consent prior to screening evaluations.
•3\.Confirmed mitochondrial DNA tRNALeu(UUR) m.3243A\>G mutation (heteroplasmy \= 20%, urinary epithelial cells).
•4\.Positive NMDAS score \>10 at Screening.
•5\. Three or more clinical features, with no other causative unifying diagnosis, found to commonly occur in subjects with a m.3243A\>G mutation:
•\- Deafness
•\- Developmental delay
•\- Diabetes Mellitus
•\- Epilepsy
•\- Gastrointestinal complaints

•1\. Surgery of gastro\-intestinal tract that might interfere with absorption.
•2\. Treatment with an investigational product within 3 months or 5 times the half\-life of the investigational product (whichever is longer) prior to the first dose of the study medication.
•3\. Documented history of ventricular tachycardia (HR\>110 beats/min).
•4\. History of acute heart failure, (family) history of unexplained syncope or congenital long and short QT syndrome or sudden death.
•5\. Clinically relevant abnormal laboratory, vital signs or physical or mental health
•a) Aspartate aminotransferase (ASAT) or alanine aminotransferase (ALAT) \> 3 x upper limit of normal (ULN), or bilirubin \> 3 x ULN at screening. If a patient has ASAT or ALAT \> 3 x ULN but \< 3\.5 x ULN, re\-assessment is allowed at the investigator’s discretion.
•b) Estimated glomerular filtration rate \= 60 mL/min according to the CKD\-EPI formula at screening.
•c) Systolic Blood pressure \> 150 mmHg at screening or baseline.
•d) All other clinically relevant parameters at screening or baseline as judged by the investigator.
•6\. Clinically relevant abnormal ECG or cardiac functioning, defined as ST\-segment elevation \> 1 mm in I, II, III, aVL, aVF, V3, V4, V5, V6; \> 2 mm in V1, V2; QTc \> 450 ms for male subjects; QTc: \> 470ms for female subjects (local, machine read), T\-top inversion in \>1 consecutive lead.