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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT03888716
Other study ID # KL1333 2018-102
Secondary ID
Status Completed
Phase Phase 1
First received
Last updated
Start date March 18, 2019
Est. completion date March 16, 2021

Study information

Verified date October 2021
Source Abliva AB
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

This will be a double blind, randomised, placebo controlled, single and multiple oral dose study conducted in 3 parts: Part A, Part B and Part C. Part A and Part B include healthy volunteers only and will be completed before Part C including patients with primary mitochondrial disease will be initiated. The starting dose in the first cohort of Part A will be 25 mg. The dose level in the additional cohorts will be decided following review of data of the previous cohorts.


Description:

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Study Design


Related Conditions & MeSH terms


Intervention

Drug:
KL1333
25 and 100 mg KL1333 encapsulated tablets
Placebo Oral Tablet
25 and 100 mg placebo encapsulated tablets

Locations

Country Name City State
United Kingdom Covance Leeds Leeds West Yorkshire
United Kingdom UCL London

Sponsors (1)

Lead Sponsor Collaborator
Abliva AB

Country where clinical trial is conducted

United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Other NAD+/NADH concentrations and ratio (part B and C) Biomarker Days 10 and 15
Other FGF21 and GDF15 concentrations (part B and C) Biomarker Days 10 and 15
Other Lactate/pyruvate concentrations and ratio (part B and C) Biomarker Days 10 and 15
Other Newcastle Mitochondrial Disease Adult Scale (NMDAS) (part C) Assessment of mitochondrial disease. The NMDAS is a validated clinical rating scale designed to capture the natural history of mitochondrial disease. The NMDAS includes 3 domains: current function, system specific involvement, and current clinical, assessed on 6-point Likert-type scale from 0 to 5, as well as a fourth section including a score for the 12-Item Short Form Survey-Version 2. Day 10
Other Clinician Global Impression (CGI) (part C) Assessment of illness severity, global improvement or change, and therapeutic response.The CGI is a 3-item observer-rated scale that measures illness severity, global improvement or change, and therapeutic response. The CGI is rated on a 7-point Likert-type scale, with the severity of illness scale using a range of responses from 1 (normal) to 7 (amongst the most severely ill patients). Day 10
Other Patient Global Impression-Improvement (PGI-I) (part C) Patient reported assessment of severity of illness. The PGI-I is a patient-rated scale using a 7-point Likert-type scale, with the severity of illness scale using a range of responses from 1 (normal) to 7 (amongst the most severely ill patients). An item about the patient's worst symptom will be included in the assessment for this study. Day 10
Other Daily Fatigue Impact Severity (D-FIS) (part C) Assessment of fatigue. The D-FIS is a patient-rated scale developed to assess the symptom of fatigue as part of an underlying chronic disease or condition. The D-FIS includes 8 items assessed on 5-point Likert-type scale from 0 (no problem) to 4 (extreme problem). Day 10
Other Quality of Life in Neurological Disorders Fatigue Scale (Neuro-QoL Fatigue) (part C) Assessment of fatigue. The Neuro-QoL Fatigue is one of several scales that make up the Quality of Life in Neurological Disorders measurement system. It is a reliable and validated brief 19-item survey of fatigue, completed by the subject, with a recall period of the past 7 days. The 19 items are scored from 1 (never) to 5 (always) and consequently, Neuro-QoL Fatigue total scores range from 19 to 95, with higher scores indicating greater fatigue and greater impact of mitochondrial disease on activities. Day 10
Other 30 Second Sit-to-Stand Test (part C) Test Day 10
Primary Safety: incidence and severity of AEs Day 15
Primary Safety: incidence of laboratory abnormalities, based on haematology, clinical chemistry, and urinalysis test results Day 15
Primary Safety: 12 lead ECG parameters Day 15
Primary Safety: Number of participants with clinically significant abnormal vital signs measurements Day 15
Primary Safety: Number of participants with clinically significant abnormal physical examinations Day 15
Secondary PK: area under the curve, AUC0 8 Day 1
Secondary PK: AUC over a dosing interval (AUC0 t) Days 1 and 10
Secondary PK: temporal change parameter (TCP; AUC0 t/AUC0-8) Days 1 and 10
Secondary PK: Cmax Day 1
Secondary PK: time of the Cmax (Tmax) Day 1
Secondary PK: minimum observed plasma concentration (Cmin) Days 1 and 10
Secondary PK: apparent plasma terminal elimination half life (t1/2) Days 1 and 10
Secondary PK: mean residence time (MRT) Days 1 and 10
Secondary PK: apparent total plasma clearance (CL/F) Days 1 and 10
Secondary PK: apparent volume of distribution during the terminal phase (Vz/F) Days 1 and 10
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