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Phase 3, Multicenter, Randomized, Double-Masked, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Tinlarebant in the Treatment ofStargardt Disease in Adolescent Subjects

This study has been transitioned to CTIS with ID 2024-513483-26-00 check the CTIS register for the current data. To assesses the efficacy of Tinlarebant in slowing the rate of growth of atrophic lesion(s) in adolescent subjects with STGD1.

A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study with an Open-Label Extension to Evaluate the Efficacy and Safety of Oral Rilzabrutinib (PRN1008) in Adults and Adolescents with Persistent or Chronic Immune Thrombocytopenia (ITP)

Primary Efficacy Objective (Blinded Treatment Period)• To demonstrate the efficacy of rilzabrutinib versus placebo in participants with refractory/relapsed ITP, based on the durability of platelet response during the last12 weeks of the 24-week…

A PHASE II, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL GROUP STUDY TO EVALUATE THE SAFETY, EFFICACY, AND PHARMACODYNAMICS OF 52 WEEKS OF TREATMENT WITH BASMISANIL IN PARTICIPANTS AGED 2 TO 14 YEARS OLD WITH DUP15Q SYNDROME FOLLOWED BY A 2-YEAR OPTIONAL OPEN-LABEL EXTENSION

Part 1:To evaluate the effects of 52 weeks of treatment with basmisanil on core symptom domains of Dup15q syndrome (language and social skills) and dailyfunctioning. Part 2:• To evaluate the tolerability and safety of up to 3 years of treatment with…

An Operationally Seamless, Randomized Phase 2/3 Study Consisting of a Phase 2 Single Blind, Dose-Evaluation Phase and a Phase 3 Double-Blind, Placebo-controlled Phase to Assess the Efficacy and Safety of Setrusumab in Subjects with Osteogenesis Imperfecta

This study has been transitioned to CTIS with ID 2024-510919-29-00 check the CTIS register for the current data. OBJECTIVES - Phase 2 (not to be conducted in NL):Phase 2 Primary Objective:- Identify a setrusumab dosing strategy in subjects with OI…

A Phase 3, Multicenter, Randomized, Platform Study of p19 Inhibition of the IL-23
Pathway to Establish Efficacy in Pediatric Crohn*s Disease;ISA title: Efficacy, Safety, and Pharmacokinetics of Guselkumab in Pediatric Participants with
Moderately to Severely Active Crohn*s Disease

This study has been transitioned to CTIS with ID 2023-504735-41-00 check the CTIS register for the current data. Primary for Platform-Level Exploratory Analysis1. To evaluate whether treatment with IL-23 inhibitors is superior to adult placebo in…

A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Subjects With Pyruvate Kinase Deficiency Who Are Not Regularly Transfused, Followed by a 5-Year Open-label Extension Period

This study has been transitioned to CTIS with ID 2024-515025-28-00 check the CTIS register for the current data. To determine the efficacy of treatment with mitapivat compared with placebo, as assessed by the increase in Hb concentrations in…