Open Label Extension to Assess Long Term Safety and Efficacy of KL1333 in Patients With Primary Mitochondrial Disease
Pharming Technologies B.V. · with ICON plc · NCT07514338
Opens the official ClinicalTrials.gov record, with the study team’s contacts. Bored Studies doesn’t enroll anyone.
Key facts
- Compensation
- Not posted. Ask the study team before screening.
- Who can join
- Ages 18+ · All sexes
- Healthy volunteers
- Not accepted (a condition is required)
- Study type
- Interventional · Phase 2
- Target enrollment
- 140
- US sites recruiting
- 1
- Study start
- June 12, 2026
- Record last updated
- September 16, 2026
Status re-checked against ClinicalTrials.gov within the last 12 hours.
What the study is about
The purpose of this study is to investigate if the study medicine, KL1333, is safe, well-tolerated and effective long-term in improving the symptoms of fatigue and impacts on daily living and functional capacity (physical abilities) in people with PMD.
Being studied: Napazimone
Conditions: Mitochondrial Diseases
Eligibility criteria
Copied from the official record. The study team makes the final decision at screening.
Show full criteria
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.
Recruiting US locations
Akron Children's Hospital
Akron, OH 44308
Site contact: Bruce Cohen
Study contacts (from the official record)
- Audrey Simon, Sr. CPM · +31 71 524 7448 · a.simon@pharming.com