The goal of this observational study is to develop and validate tools to measure disease course in patients with primary mitochondrial myopathy (PMM). The main…

Primary Mitochondrial Disease

~Estimated

~$10 – $750

Children's Hospital of PhiladelphiaNCT05250375

Phase 2

An Interventional, Randomised, Double-blind, Parallel-group, Placebo-controlled, Flexible-dose, Adaptive Study of the 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) follo…

Primary Mitochondrial Disease

~Estimated

~$10 – $7,500

Pharming Technologies B.V.NCT05650229

Neurometabolic Profile of Individuals With Primary Mitochondrial Disease

Primary Mitochondrial Disease (PMD) is a genetic neurometabolic disorder, leading to central nervous system degeneration and increased risk of early mortality.…

Primary Mitochondrial Disease

~Estimated

~$10 – $750

Children's Hospital of PhiladelphiaNCT06890520