Long-Term Safety and Efficacy of KL1333 in Adults with Primary Mitochondrial Disease
Belgium- Participants:18–64 years · 65+ years
- Substances:2-Isopropyl-3H-Naphtho(1,2-D)Imidazole-4,5-Dione
- Sponsor:Pharming Technologies B.V.
Rare diseases
Investigational molecules
Locations

Darwinweg 24, 2333 CR Leiden, The Netherlands
Pharming Technologies B.V. focuses clinical research on primary immunodeficiency disorders, with particular attention to common variable immunodeficiency and related immune dysregulation conditions. Its studies address the clinical burden of abnormal immune function in both adult and pediatric populations.
Research in this area includes ongoing work in patients with clinically significant immune dysfunction across multiple age groups.
Pharming Technologies B.V. runs clinical trials in conditions such as Activated PI3 kinase delta syndrome, Immunodeficiency common variable, Primary immunodeficiency syndrome, among others.
Clinical trials run by this sponsor – those enrolling participants first.
BelgiumDrug interaction study of leniolisib with a drug combination in healthy volunteers for Activated PI3K Delta Syndrome
GermanyA Study of Leniolisib Safety and Effectiveness for Immune System Problems in Patients with Common Variable Immunodeficiency
SpainLong-term safety and effectiveness study of leniolisib for patients with primary immunodeficiency and immune system problems
SpainStudy on the Effects of Leniolisib in Patients with Activated Phosphoinositide 3-Kinase Delta Syndrome
GermanyStudy on the Effects of Leniolisib in Children Aged 1 to 6 with Activated PI3K Delta Syndrome (APDS)
PortugalStudy of Leniolisib for Children Aged 4 to 11 with Activated PI3K Delta Syndrome
FranceStudy on Leniolisib for Patients with Activated Phosphoinositide 3-Kinase Delta Syndrome and Impaired Liver Function
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