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Efficacy of KL1333 in Adult Patients With Primary Mitochondrial Disease

ConditionPrimary Mitochondrial Disease
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorPharming Technologies B.V.

About this trial

The primary objective of the FALCON study is to evaluate the efficacy of KL1333 on selected disease manifestations of primary mitochondrial disease (PMD) following 48 weeks of treatment. This objective involves evaluating the efficacy of KL1333 versus placebo on fatigue symptoms and impacts on daily living as well as on functional lower extremity strength and endurance. Additionally, the study evaluates the safety and tolerability of KL1333.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Age 18 years or older.

m.3243A>G associated MELAS-MIDD spectrum disorders,

single large scale mtDNA deletion associated KSS-CPEO spectrum disorders,

other multisystemic mtDNA-related disease (including MERRF).

Disqualifiers

Primary mitochondrial disease with predominant neurodegenerative phenotypes, such as, but not limited to, Leigh syndrome, Leber hereditary optic neuropathy (LHON) and Neuropathy ataxia-retinitis pigmentosa syndrome (NARP).

Primary mitochondrial disease nuclear DNA mutations or mutations causing mtDNA destabilisation. Genetic mtDNA variants of uncertain significance, likely pathogenic, or pathogenic mutations with degrees of heteroplasmy below what can be considered to definitely cause PMD.

General fatigue or muscle weakness due to causes other than mitochondrial disease, in the opinion of the investigator.

Significant cardiovascular disease (e.g., sustained or symptomatic arrhythmia; dilated heart chambers or reduced function; Mobitz II atrioventricular block or greater) OR abnormal ECG that is clinically significant, as determined by the investigator. Any QTcF > 450 msec for male patients and > 470 msec for female patients is exclusionary. In the case of an exclusionary QTcF, the ECG can be repeated twice and the average of 3 QTcF intervals should be used to determine the QTcF eligibility.

Trial design

Design model

Parallel

Treatments tested in this trial

  • KL1333

    Drug

    Twice daily

  • Placebo

    Drug

    Twice daily

Treatment groups

180 Participants
are divided into 2 treatment groups
Group A: KL1333Experimental treatment 1 intervention
Group B: Matching PlaceboPlacebo comparator 1 intervention

Trial outcomes

Primary outcomes

1

Change in-patient-reported fatigue symptoms and impacts on daily living measured by Patient-Reported Outcomes Measurement Information System (PROMIS®) Fatigue PMD Short Form

Change in t-score. Higher scores indicate greater fatigue severity.

Time frame
Baseline and 48 Weeks
2

Change in 30 Second Site-to-Stand Test.

Change in number of stands

Time frame
Baseline and 48 Weeks

Secondary outcomes

1

Change in Quality of Life in Neurological Disorders (Neuro-QoL) Lower Extremity Function (Mobility) Short Form

Change in t-score. Higher scores indicate greater functional ability.

Time frame
Baseline and 48 weeks
2

Change in Individual Activity Assessments - Interference

Assessing how much Primary Mitochondrial Disease symptoms get in the way of the ability to perform individually selected activities. A 5 point scale ranging from "No interference" to "Completely interferes"

Time frame
Baseline and 48 Weeks
3

Individual Activity Assessment - Change

Assessing the ability to perform individually selected activities compared to baseline. A 5 point scale ranging from "Much worse" to "Much better"

Time frame
48 weeks
4

Change in Patient Global Impression of Severity

Scale ranging from 1 (none) to 4 (severe).

Time frame
Baseline and 48 weeks

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Pharming Technologies B.V.

Lead sponsor

Source ClinicalTrials.gov
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