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Assessing the Effect of Missing Doses (Off-Days) of Daily Medication in Patients Stable on Pharmacotherapy for ADHD Receiving Atomoxetine or OROS Methylphenidate: A Parallel Matched Group Clinical Study (On/Off Study)

The primary objective of the study is to assess the effect of missed doses of atomoxetine and OROS methylphenidate in ADHD patients who are stable on pharmacotherapy based on the patient*s daily behavior as assessed by the Daily Parent Report of…

A Multicenter, Double Blind, Randomized-Withdrawal Trial of Subcutaneous Golimumab, a Human Anti-TNF?? Antibody, in Pediatric Subjects with Active Polyarticular Course Juvenile Idiopathic Arthritis (JIA) Despite Methotrexate Therapy

The primary objective of this study is to assess the clinical efficacy of SC administration of golimumab in pediatric subjects (ages 2 to less than 18 years) with JIA manifested by more or equal to 5 joints with active arthritis despite MTX therapy…

'gamified' working memory training for addicted adolescents

The proposed research project will examine the potential benefits of a *gamified* working memory training for adolescents in addiction care. The primary objective is to test whether a gamified working memory training will lead to improved working…

Peppermint Oil for the treatment of Irritable Bowel Syndrome or Functional Abdominal Pain in Children:
the MINT study

The aim of the Mint study is to investigate the effectiveness of peppermint oil capsules compared to placebo capsules in reducing abdominal pain intensity in these children. In addition, we investigate the effect of peppermint oil capsules compared…

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…

A Phase III Multicenter, Randomized, Double-Blind, Double-Dummy Study To Evaluate Safety And Efficacy Of Ocrelizumab In comparison with Fingolimod in Children And Adolescents With Relapsing-Remitting Multiple Sclerosis

This study has been transitioned to CTIS with ID 2023-506516-40-00 check the CTIS register for the current data. The primary efficacy objective for this study is to demonstrate non-inferiority of ocrelizumab compared with fingolimod

A 2-Stage (Open-Label Followed by Randomized Double-Blind, Placebo-Controlled Stage), Phase 2 Trial of Setmelanotide in Patients with Specific Gene Variants in the Melanocortin-4 Receptor Pathway

Primary:• To evaluate the proportion of patients with obesity with genetic variants in a specific gene in the melanocortin-4 receptor (MC4R) pathway who achieve a clinically meaningful reduction in body weight in response to setmelanotide at the end…

EFFICACY, SAFETY, TOLERABILITY AND PHARMACOKINETICS OF TOFACITINIB FOR TREATMENT OF SYSTEMIC JUVENILE IDIOPATHIC ARTHRITIS (sJIA) WITH ACTIVE SYSTEMIC FEATURES IN CHILDREN AND ADOLESCENT SUBJECTS

Primary:* To assess the sustained efficacy of tofacitinib versus placebo in sJIA patients, as measured by time to sJIA flare in the double-blind randomized withdrawal phase.Secondary:* To assess efficacy of tofacitinib versus placebo in sJIA…

A Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Ambulatory and Non-Ambulatory Participants with Spinal Muscular Atrophy with Open-Label Extension (RESILIENT)

This study has been transitioned to CTIS with ID 2024-511852-42-00 check the CTIS register for the current data. Primary Objective:To evaluate the efficacy of taldefgrobep alfa in participants who are already taking a stable dose of nusinersen or…

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 Regularly Transfused, Followed by a 5-Year Open-label Extension Period

This study has been transitioned to CTIS with ID 2024-515024-37-00 check the CTIS register for the current data. To determine the efficacy of treatment with mitapivat compared with placebo, as assessed by the reduction in transfusion burden in…

EPIK-P2: A Phase II double-blind study with an upfront, 16-week randomized, placebo-controlled period, to assess the efficacy, safety and pharmacokinetics of alpelisib (BYL719) in pediatric and adult patients with PIK3CA-related overgrowth spectrum (PROS)

This study has been transitioned to CTIS with ID 2023-508530-34-00 check the CTIS register for the current data. Primary objective:* To demonstrate the efficacy of alpelisib as measured by the proportion of participants randomized to alpelisib with…

A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans Progressiva (PROGRESS)

This study has been transitioned to CTIS with ID 2023-504129-38-00 check the CTIS register for the current data. Primary:To determine the efficacy of INCB000928 for the prevention of new HO in participants with FOP.Key Secondary:To further evaluate…

DOUBLE BLIND, MULTICENTRE, RANDOMIZED, PLACEBO-CONTROLLED TRIAL TO EVALUATE SAFETY AND EFFICACY OF PITOLISANT IN CHILDREN FROM 6 TO LESS THAN 18 YEARS WITH NARCOLEPSY WITH/WITHOUT CATAPLEXY, FOLLOWED BY A PROLONGED OPEN-LABEL PERIOD

- To evaluate the efficacy of pitolisant ( 5, 10, 20,40mg/d in the Double Blind Period and 5, 10, 15, 20, 30, 40mg/d in the Open Label Period) in reducing residual Excessive Daytime Sleepiness (EDS) and the number of cataplectic episodes (for…

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 1-3 Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION363 in Amyotrophic Lateral Sclerosis patients with Fused in Sarcoma mutations (FUS-ALS)

This study has been transitioned to CTIS with ID 2024-512163-31-00 check the CTIS register for the current data. Primary objective: To evaluate the clinical efficacy of ION363 in clinical functioning and survival in FUS-ALS patients. Secondary…

A phase 3 randomized withdrawal, double-blind, placebo-controlled, multi-center study investigating the efficacy and safety of PF-04965842 in subjects aged 12 years and over, with moderate to severe atopic dermatitis with the option of rescue treatment in flaring subjects.

Primary Objective- To evaluate and compare the maintenance of effect of two doses of PF-04965842 (200 mg and 100 mg once daily [QD]) and placebo in subjects aged 12 and above with moderate to severe atopic dermatitis who respond to an initial open-…

Inspiratory muscle training in patients with nemaline myopathy

The primary objective is to determine the effect of a 8-week inspiratory muscle training program on respiratory muscle function in nemaline myopathy patients. The secondary objective is to determine respiratory muscle function in nemaline myopathy…

Effect of liraglutide for weight management in paediatric subjects with Prader-Willi Syndrome.
A randomised, placebo controlled, parallel group, multi-centre, multinational trial with a 16-week double-blind period and 36-week open-label period.

Primary objective- To compare the efficacy of liraglutide versus placebo on weight loss in obese paediatric subjects with PWS at 16 weeks and versus no treatment at 52 weeks.Secondary objectives - To compare the efficacy of liraglutide versus…