Rare diseases
Investigational molecules
Locations
A safety study of romosozumab in children and adolescents with osteogenesis imperfecta
AustriaA study to evaluate the long-term safety and effectiveness of mRNA-3927 in patients with Propionic Acidemia
FranceA study of Enzomenib in adult patients with relapsed or refractory acute leukemia, including those with MLL rearrangement or NPM1 mutation.
BelgiumA Phase 3 Randomized, Double-blind,Placebo-controlled Study to Evaluate the Efficacy, Safety, and Tolerability of Maridebart Cafraglutide in Adult Participants with Obstructive Sleep Apnea not on Positive Airway Pressure Therapy and Living with Overweight or Obesity (MARITIME-OSA-2)
CzechiaN1T-MC-MALO: A Master Protocol for a Randomized, Controlled, Clinical Trial of Multiple Pharmacologic Agents in Adult Participants With Metabolic Dysfunction-Associated Steatotic Liver Disease Who Are at Increased Risk of Developing Major Adverse Liver Outcomes (SYNERGY-Outcomes); N1T-MC-TZ01 Tirzepatide in participants with high-risk MASLD; N1T-MC-RT01 Retatrutide in participants with high-risk MASLD
AustriaEvaluation of Safety, Pharmacokinetics, and Efficacy of RO7782493 and Panitumumab in Advanced Solid Tumors with KRAS G12D Mutation
FranceEvaluation of Lymphocyte-Sparing Radiotherapy and All-Trans Retinoic Acid in Lateralized Oropharyngeal, Laryngeal, and Hypopharyngeal Squamous Cell Carcinoma
BelgiumEvaluation of Long-Term Safety and Efficacy of Litifilimab in Adults with Active Systemic Lupus Erythematosus: A Phase 3 Multicenter, Randomized, Dose-Blind Study
BelgiumEvaluation of Retinol Palmitate Administration and Serum Levels in Preterm Infants with Bronchopulmonary Dysplasia: A Phase 2a Open-Label Study
GermanyPhase 1b-2 Study of Elacestrant and Abemaciclib in Patients with Brain Metastases from ER-Positive, HER2-Negative Breast Cancer
BelgiumPhase 1/2 Evaluation of Zilovertamab Vedotin in PD-1/L1 Refractory Locally Advanced or Metastatic Urothelial Carcinoma
DenmarkEvaluation of Safety and Efficacy of MK-2870 and Paclitaxel in Second-Line Treatment of Advanced/Metastatic Gastroesophageal Adenocarcinoma
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