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Effectiveness of methylphenidate in children and adults with Smith Magenis syndrome and attention-deficit/hyperactivity disorder: An N-of-1 series

The primary objective is to determine the efficacy of methylphenidate on ADHD symptoms in Smith Magenis syndrome.The secondary objectives include the efficacy of methylphenidate on emotion dysregulation and specific goals that are important to the…

Serotonergic modulation of population receptive fields

The primary objective is to determine the effects of serotonergic stimulation, via the administration of psilocybin, on population receptive fields in different modalities and to relate these effect to specific computational aspects of the divisive…

Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C (NPC): A Phase III, randomized, placebo-controlled, double-blind, crossover study

This study has been transitioned to CTIS with ID 2023-510278-14-00 check the CTIS register for the current data. Primary Objective:In all jurisdictions except the United States (US), the primary objective is to evaluate the efficacy of N-Acetyl-L-…

A Phase 3, Double-blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy and Safety of Mitapivat in Subjects With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (ENERGIZE)

This study has been transitioned to CTIS with ID 2024-512745-16-00 check the CTIS register for the current data. Study AG348-C-017 is a Phase 3, double-blind, randomized, placebo-controlled, multicenter study designed to demonstrate the clinical…

Leflunomide and Hydroxychloroquine combination therapy
for primary Sjögren*s Syndrome

This study has been transitioned to CTIS with ID 2024-510904-36-00 check the CTIS register for the current data. To assess clinical efficacy and safety of Leflunomide/Hydroxychloroquine in pSS patients in a phase IIb placebo-controlled randomized…

A phase 3, randomized, placebo-controlled, double-blind study of vimseltinib to assess the efficacy and safety in patients with tenosynovial giant cell tumor (MOTION)

This study has been transitioned to CTIS with ID 2024-513624-42-00 check the CTIS register for the current data. Primary Objective: • To evaluate anti-tumor activity of vimseltinib using Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 by…

LIBRETTO-432: A Placebo-controlled Double-Blinded Randomized Phase 3 Study of Adjuvant Selpercatinib following Definitive Locoregional Treatment in Participants with Stage IB-IIIA RET fusion-Positive NSCLC

This study has been transitioned to CTIS with ID 2023-506784-33-00 check the CTIS register for the current data. To compare EFS of participants in the primary analysis population with Stage II-IIIA RET fusion-positive NSCLC treated with…

A Phase 3, Double-blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy and Safety of Mitapivat in Subjects With Transfusion-Dependent Alpha- or Beta-Thalassemia (ENERGIZE-T)

This study has been transitioned to CTIS with ID 2024-512747-23-00 check the CTIS register for the current data. Study AG348-C-018 is a Phase 3, double-blind, randomized, placebo-controlled, multicenter study designed to demonstrate the clinical…

Adjuvant immunotherapy with anti-PD-1 monoclonal antibody Pembrolizumab (MK-3475) versus placebo after complete resection of high-risk Stage III melanoma: A randomized, double-blind Phase 3 trial of the EORTC Melanoma Group.

This study has been transitioned to CTIS with ID 2023-509136-25-00 check the CTIS register for the current data. To prospectively assess whether post-operative adjuvant therapy with pembrolizumab improves recurrence-free survival (RFS) as compared…

A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients with Glycogen Storage Disease Type Ia

This study has been transitioned to CTIS with ID 2023-508750-25-00 check the CTIS register for the current data. Primary: To evaluate the efficacy of DTX401 to reduce or eliminate dependence on exogenous glucose replacement therapy needed to…