Clinical Trial Assessing KL1333
Research type
Research Study
Full title
A Randomised, Double-blind, Parallel-group, Placebo-controlled, Phase Ia/Ib, Multiple-site Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of KL1333 after a Single Oral Dose and Multiple Ascending Oral Doses in Healthy Subjects and Patients with Primary Mitochondrial Disease
IRAS ID
246516
Contact name
Robert Pitceathly
Contact email
Sponsor organisation
NeuroVive Pharmaceutical AB
Eudract number
2018-001794-24
Clinicaltrials.gov Identifier
18/SC/0286, REC
Duration of Study in the UK
1 years, 8 months, 8 days
Research summary
Mitochondria can be found in most cells and have a role of being the powerhouse of the cell via a complicated series of reactions. They produce energy in the form of adenosine triphosphate(ATP).
Primary mitochondrial diseases are a set of diseases caused by a dysfunction in these chains of reactions which vary in severity and body systems they affect, there are a varying degree of clinical manifestations of the effects of these aberrant reactions.
KL1333 is a new compound to treat this broad spectrum of potentially debilitating and life threatening diseases.
This study is to test the safety and tolerability of this compound by giving a single dose and a multiple ascending dose in healthy subjects and patients with primary mitochondrial disease.
The study will be a double-blind (neither the subjects or the researchers will know who is taking placebo or study drug), single and multiple oral dose conducted in 3 parts.
Part A will receive a single dose in healthy subjects over 2 treatment periods, the first will be after fasting 8 hours and the second treatment period will be after consuming a high fat breakfast.They will be confined to the unit for 5 days.
Part B will receive multiple doses in healthy subjects but the dose levels will be decided once data from Part A and available data from part B will be assessed. They will be confined to the unit for 14 days.
Part C will involve patients with primary mitochondrial diseases. With dosage levels to be decided once data from Part A and B is analysed.
Each group will consist of 8 people, 6 will receive the active drug and 2 will receive a placebo.REC name
South Central - Berkshire B Research Ethics Committee
REC reference
18/SC/0286
Date of REC Opinion
9 Nov 2018
REC opinion
Further Information Favourable Opinion