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KL1333 for Mitochondrial Disease (FALCON Trial)

Phase 2
Waitlist Available
Led By Grainne Gorman, MD, PhD
Research Sponsored by Abliva AB
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Presence of mitochondrial myopathy defined as myopathy (proximal muscle weakness) or limited exercise tolerance
Confirmed PMD diagnosis caused by a known pathogenic gene mutation or deletion of the mitochondrial genome according to ACMG/AMP criteria, with multisystemic disease expressions
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 48 weeks
Awards & highlights

FALCON Trial Summary

This trial aims to test a new drug for primary mitochondrial disease, to see if it helps with fatigue, daily living, and strength/endurance. It also checks for safety.

Who is the study for?
Adults over 18 with primary mitochondrial disease (PMD) characterized by muscle weakness or limited exercise tolerance, chronic fatigue, and a confirmed genetic diagnosis. Participants must be clinically stable, able to attend appointments, willing to maintain diet and therapy regimens, not pregnant, and agree to contraception requirements.Check my eligibility
What is being tested?
The FALCON study is testing the effectiveness of KL1333 on fatigue symptoms and daily life impact in PMD patients after 48 weeks compared to a placebo. It also measures lower extremity strength/endurance while assessing safety and tolerability of KL1333.See study design
What are the potential side effects?
While specific side effects are not listed here for KL1333 or the placebo, typically such trials monitor for any adverse reactions ranging from mild discomforts like headaches or nausea to more serious issues affecting organ function.

FALCON Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have muscle weakness or struggle with physical activity due to mitochondrial myopathy.
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I have a confirmed PMD diagnosis with symptoms affecting multiple systems in my body.
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I have been feeling extremely tired for more than 3 months.
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I am stable except for my mitochondrial disease symptoms.
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I am not pregnant and follow the required birth control measures.
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I am 18 years old or older.
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I have been on stable doses of vitamins or supplements for my mitochondrial disease for 3 months.
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I often feel very tired.
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I am willing to stop taking idebenone for the study.
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I can attend all study appointments as scheduled.

FALCON Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~48 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and 48 weeks for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Change in 30 Second Site-to-Stand Test.
Change in-patient-reported fatigue symptoms and impacts on daily living measured by Patient-Reported Outcomes Measurement Information System (PROMIS®) Fatigue PMD Short Form
Secondary outcome measures
Change from baseline in Glycated haemoglobin (HbA1c) for subjects with diabetes
Change in Clinician Global Impression of Severity
Change in Individual Activity Assessments - Interference
+6 more

FALCON Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: KL1333Experimental Treatment1 Intervention
Twice daily
Group II: Matching PlaceboPlacebo Group1 Intervention
Twice daily
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
KL1333
2019
Completed Phase 1
~90

Find a Location

Who is running the clinical trial?

Abliva ABLead Sponsor
2 Previous Clinical Trials
86 Total Patients Enrolled
2 Trials studying Mitochondrial Diseases
86 Patients Enrolled for Mitochondrial Diseases
Grainne Gorman, MD, PhDPrincipal InvestigatorWellcome Centre for Mitochondrial Research, Newcastle University
Amel Karaa, MDPrincipal InvestigatorMassachusetts General Hospital
4 Previous Clinical Trials
583 Total Patients Enrolled
2 Trials studying Mitochondrial Diseases
264 Patients Enrolled for Mitochondrial Diseases

Media Library

KL1333 (Other) Clinical Trial Eligibility Overview. Trial Name: NCT05650229 — Phase 2
Mitochondrial Diseases Research Study Groups: KL1333, Matching Placebo
Mitochondrial Diseases Clinical Trial 2023: KL1333 Highlights & Side Effects. Trial Name: NCT05650229 — Phase 2
KL1333 (Other) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05650229 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Has the U.S. Food and Drug Administration accepted KL1333 for therapeutic usage?

"The safety rating for KL1333 is 2, as this phase two trial has some evidence suggesting it's harmless but not yet any data corroborating its effectiveness."

Answered by AI

How many people have volunteered to take part in this research project?

"Affirmative. Clinicaltrials.gov's data shows that this trial is currently enrolling patients, with the initial posting dating to December 13th 2022 and last updated on April 19th 2023. The study requires 180 participants from 7 different research sites."

Answered by AI

How widely is this scientific experiment being implemented in Canada?

"Currently, this trial is enrolling patients in 7 different sites around the world, such as Madrid, Houston and Barcelona. To reduce unnecessary travel costs for participants it would be wise to choose the closest available location if you decide to join."

Answered by AI

Are there any vacancies available to participate in this trial?

"That is correct. Clinicaltrials.gov reveals that this study, initially posted on December 13th 2022, is currently enrolling participants. The researchers plan to recruit 180 people from 7 clinical trial sites."

Answered by AI

What end results are the researchers hoping to demonstrate through this clinical trial?

"This clinical trial, taking place over the course of 48 weeks, plans to evaluate patient-reported fatigue symptoms and daily living through PROMIS® Fatigue PMD Short Form. Secondary objectives include a Global Impression of Change Scale ranging from 2 (much better) to -2 (much worse), as well as changes in t-score on Neuro-QoL Lower Extremity Function for mobility and an Individual Activity Interference scale with ratings from "No interference" to "Completely interferes"."

Answered by AI
~98 spots leftby Oct 2025