Clinical trials

2

Search and review clinical trials. Use filters to narrow results by trial status, phase, treatment, biological sex and sponsor.

Condition / disease
Location
Status: Recruiting

Open Label Extension to Assess Long Term Safety and Efficacy of KL1333 in Patients With Primary Mitochondrial Disease

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.

Participants needed: 140
Trial details
Phase: Phase 2Age: 18+Biological sex: AllType: InterventionalSponsor: Pharming Technologies B.V.Updated: Sep 16, 2026Locations: 23
Eligibility criteria

Completed the FALCON study (age 18 years or older), and in the opinion of the in... [+4]

The subject is, in the investigator's opinion, unlikely to comply with the proto... [+1]

Status: Recruiting

Efficacy of KL1333 in Adult Patients With Primary Mitochondrial Disease

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.

Participants needed: 180
Trial details
Phase: Phase 2Age: 18+Biological sex: AllType: InterventionalSponsor: Pharming Technologies B.V.Updated: Jun 26, 2026Locations: 55
Eligibility criteria

Age 18 years or older. [+19]

Primary mitochondrial disease with predominant neurodegenerative phenotypes, suc... [+20]