17 results
Has the supplementation of oligosaccharides in the first year of life an effect on the incidence of atopic diseases and infections from the 5th until the 7th year of life in healthy children
Primary ObjectivesThe primary objectives are to evaluate in infants who are hospitalized with RSV infection:• the pharmacokinetics of JNJ-53718678 after multiple oral doses;• the safety and tolerability of JNJ-53718678 when administered for 7 days.…
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…
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…
Primary Objectives:The primary objectives are to evaluate in infants who are hospitalized with RSV infection:- the pharmacokinetics of JNJ-53718678 after multiple oral doses;- the safety and tolerability of JNJ-53718678 when administered for 7 days.…
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…
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…
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…
Primary Objective:To evaluate the efficacy of taldefgrobep alfa in participants who are already taking a stable dose of nusinersen or risdiplam or have a history of onasemnogene abeparvovec-xioi, compared to placebo, measured by change in the 32…
To determine the efficacy of treatment with mitapivat compared with placebo, as assessed by the reduction in transfusion burden in pediatric subjects with pyruvate kinase deficiency (PK deficiency) who are regularly transfused.
OBJECTIVES - Phase 2 (not to be conducted in NL):Phase 2 Primary Objective:- Identify a setrusumab dosing strategy in subjects with OI Phase 2 Secondary Objectives:- Evaluate the PK of setrusumab doses in subjects with OI- Determine the PD effects…
- 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…
Primary objective:* To demonstrate the efficacy of alpelisib as measured by the proportion of participants randomized to alpelisib with a response at Week 24 in at least one of the following groups:* Group 1 (>= 18 yr-old)* Group 2 (6 - 17 yr…
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…
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…
To determine the efficacy of treatment with mitapivat compared with placebo, as assessed by the increase in Hb concentrations in pediatric subjects with PK deficiency who are not regularly transfused
The primary efficacy objective for this study is to demonstrate non-inferiority of ocrelizumab compared with fingolimod