PL2
Sponsors
Actelion Pharmaceuticals Ltd., Janssen - Cilag International, Medical University Of Gdansk, Exelixis Inc.
Conditions
Aneurysms-osteoarthritis syndromeArterial Tortuosity syndromeDuchenne muscular dystrophy; Cardiomyopathy; TachycardiaFamilial thoracic aortic aneurysms and aortic dissectionsMarfan Syndrome
Diseases related to Marfan syndrome (rare diseases with aneurysms of the thoracic aorta and dissection of the aorta)Metastatic Medullary Thyroid CancerModerately to Severely Active Crohn's DiseaseMulti-system smooth muscle dysfunction syndrome
Phase 3
A Randomized, Multicenter, Double-Blind, Placebo-Controlled, Parallel-Group Study with Open-Label Extension Period to Assess the Efficacy and Safety of Selexipag as Add-On Treatment to Standard of Care in Children Aged ≥2 to <18 years with Pulmonary Arterial Hypertension
Active, not recruitingCTIS2022-501012-34-00
Start: 2020-02-27Target: 73Updated: 2025-11-03
A Phase 3 Study of the Efficacy, Safety, and Pharmacokinetics of Ustekinumab as Open-label Intravenous Induction Treatment Followed by Randomized Double-blind Subcutaneous Ustekinumab Maintenance in Pediatric Participants with Moderately to Severely Active Crohn’s Disease
CompletedCTIS2023-504978-38-00
Start: 2021-03-16End: 2025-03-03Target: 60Updated: 2024-09-24
Evaluation of the Efficacy of Valsartan in Slowing Down Aortic Root Dilatation in Children and Young Adults with Marfan-type Heritable Thoracic Aortic Diseases – Valsar-TAD, a randomised, double-blind, placebo-controlled multicentre trial
RecruitingCTIS2024-515059-39-00
Start: 2024-02-09Target: 180Updated: 2025-06-30
The efficacy and safety of Metoprolol as add-on treatment to standard of care in preventing cardiomyopathy in patients with Duchenne Muscular Dystrophy aged 8-17 years. A randomized, double-blind, placebo controlled study
CompletedCTIS2024-515057-19-00
Start: 2021-07-12End: 2025-10-06Target: 144Updated: 2024-12-16