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New pill aims to ease fatigue in rare mitochondrial disease

NCT ID NCT07514338

Disease control Sponsor: Pharming Technologies B.V. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Sep 17, 2026 · Updated 2 times

Summary

This study is for people who completed a previous trial of KL1333 (napazimone) for primary mitochondrial disease. It will test the drug's long-term safety and whether it helps with fatigue and daily activities. About 140 participants will take the pill twice daily for an extended period. The goal is to see if KL1333 can be a safe, long-term treatment option.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
KL1333 (napazimone) taken as a pill twice daily
What this could lead to
If it works, this could offer a long-term treatment option to reduce fatigue and improve daily living for people with primary mitochondrial disease.
What could go wrong
This is an early-phase extension study with no placebo group, so results may not prove effectiveness. Side effects or tolerability issues could limit its use.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 140 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jun 2026

Expected to finish

Mar 2029

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Completed the FALCON study (age 18 years or older), and in the opinion of the investigator and sponsor has been compliant with the study requirements * Willingness and ability to attend study appointments within the specified time windows * Willingness and ability to complete electronic patient-reported outcomes * Concomitant medications likely to remain stable throughout participation in the study where clinically possible * Willingness to suspend treatment with idebenone during the study Exclusion Criteria: * The subject is, in the investigator's opinion, unlikely to comply with the protocol, e.g., due to cognitive impairment, or is unsuitable for any reason. * Any medical, psychiatric, laboratory or other condition that may negatively affect the benefit-risk considerations of study participation or interfere with the interpretation of study results and, in the judgment of the investigator and/or the medical monitor, would make the subject inappropriate for entry into this study. Subjects not enrolling directly at the FALCON study completion visit (FALCON Week 48) or the safety follow-up visit (FALCON Week 53) study will be required to fulfill the additional exclusion criteria below during the screening visit: • General fatigue or muscle weakness due to causes other than mitochondrial disease, in the opinion of the investigator.

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Conditions

The condition(s) this trial relates to.

inborn mitochondrial metabolism disorder mitochondrial disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Mitochondrial diseases

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    23 sites in 9 countries. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Akron Children's Hospital

    RECRUITING

    Akron, Ohio, 44308, United States

  • Azienda Ospedaliera Universitaria Gaetano Martino Messina

    RECRUITING

    Messina, 98125, Italy

  • Azienda Ospedaliero Universitaria Pisana

    RECRUITING

    Pisa, 56126, Italy

  • CHU de NICE - Hôpital Archet 2

    RECRUITING

    Nice, France

  • Centre Hospitalier Universitaire (CHU) de Bordeaux - Groupe Hospitalier Pellegrin

    RECRUITING

    Bordeaux, France

  • Centre Hospitalier Universitaire d'Angers

    RECRUITING

    Angers, 49933, France

  • Centre Hospitalier Universitaire de Nantes

    RECRUITING

    Nantes, 44093, France

  • Centre Hospitalier Universitaire de Nice, Hopital Pasteur 2

    RECRUITING

    Nice, France

  • Charles University and General University Hospital

    RECRUITING

    Prague, 128 08, Czechia

  • Copenhagen Neuromuscular Center, Rigshospitalet

    RECRUITING

    Copenhagen, Denmark

  • Fondazione IRCCS Istituto Neurologico Carlo Besta

    RECRUITING

    Milan, 20133, Italy

  • Hopital Roger Salengro, CHRU de Lille

    RECRUITING

    Lille, France

  • Hopital Universitaire de Bruxelles (H.U.B)/ Academisch Ziekenhuis Brussel

    RECRUITING

    Brussels, Belgium

  • Hopitaux Universitaires de Strasbourg

    RECRUITING

    Strasbourg, 67091, France

  • Hospital Erasme

    RECRUITING

    Brussels, 1070, Belgium

  • Hospital General Universitario de Catalunya

    RECRUITING

    Barcelona, Spain

  • Hospital Universitario 12 de Octubre

    RECRUITING

    Madrid, Spain

  • Radboud University Medical Center

    RECRUITING

    Nijmegen, 6525, Netherlands

  • Universitaetsklinikum Halle

    RECRUITING

    Halle, 6120, Germany

  • Universitair Ziekenhuis Gent

    RECRUITING

    Ghent, Belgium

  • Universitair Ziekenhuis Leuven Gasthuisberg Campus

    RECRUITING

    Leuven, Belgium

  • University Hospital

    RECRUITING

    Ghent, 9000, Belgium

  • University Hospital

    RECRUITING

    Leuven, 3000, Belgium

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