21 results
Primary Objectives*To evaluate the safety and tolerability of multiple oral doses of GLPG1837 in subjects with CF and at least one copy of the S1251N mutation.Secondary Objectives*To assess changes in sweat chloride from baseline (Day 1) as the…
Primary objective of this study is to evaluate the clinical effect of a long term treatment (8 weeks) with oral B2-agonists in CF patients with residual CFTR function, especially on lung function (spirometry and airway resistance). Secondary…
To evaluate the effects of intranasal oxytocin versus placebo on social behaviour and also on eating behaviour in children with PWS.
The objective of this study is to determine whether influencing the protein pathway thought to underlie TSC by Everolimus treatment will improve cognitive abilities in these children.
The purpose of the study is to assess the efficacy and safety in adolescents of AFQ056 in treating behavioral symptoms characteristic of patients with FXS. Data from this study will be used to support the registration of AFQ056 in this population…
Primary objective of this pilot study is to evaluate the in vivo effect of a B2-agonists in CF patients with residual CFTR function, using dosages which are clinically used in patients with asthma. Secondary objective is to evaluate the difference…
The primary objective of this study is to evaluate the efficacy of different doses of TA 8995, a cholesteryl ester transfer protein (CETP) inhibitor, on the elevation of high density lipoprotein cholesterol (HDL C) and reduction of low density…
To determine the efficacy (as measured by objective tumor response) of single agent everolimus in the treatment of advanced malignancies or high risk polyps of patients known with PJS .
PrimaryTo evaluate the long-term safety and tolerability of AFQ056 in adolescent patients with FXS as assessed by:* Incidence and severity of adverse events and serious adverse events* Changes in vital signs, laboratory assessments, and ECGs*…
The aim of this project is to validate the hypothesis that intranasal insulin improves development and behaviour in children with Phelan-McDermid syndrome.
Does antagonism of the adenosine receptor by caffeine lead to an increased LPS-induced inflammatory reaction and an increase in (subclinical) tissuedamage?Does the C34T-polymorphism of the enzyme AMP-deaminase lead to a decreased inflammatory…
Please refer to protocol, section 1.2 "Rationale"
1. Primary Objectives• To evaluate the safety and tolerability of VX 440 monotherapy and VX 440 in dual and triple combination with VX-661 and IVA• To evaluate the efficacy of VX 440 monotherapy and VX 440 in dual and triple combination with VX 661…
Primary objective: To evaluate the safety and tolerability of SOBI003 at different dose levels.Secondary objectives:1. To characterize the pharmacokinetic (PK) properties of SOBI003 following single and repeated administration by the use of non-…
Primary aim: to confirm that treatment with bumetanide improves daily life functioning and reduces behavioral symptoms related to hyperexcitability in children and adolescents with TSC.Secondary aim: to identify neurophysiological and cognitive…
To evaluate the safety and tolerability of 4 different doses of GLPG2222 administered orally and q.d. for 29 days in adult subjects with CF whoare homozygous for the F508del CFTR mutation.Secondary objectives:To assess changes in biomarkers of CFTR…
Parts D and E: Primary Objectives:• To evaluate the safety and tolerability of VX-445 in TC with TEZ and IVA in subjects with cystic fibrosis (CF)• To evaluate the efficacy of VX-445 in TC with TEZ and IVA in subjects with CFSecondary Objectives• To…
The aim of this study is to investigate the effect of low-dose Lithium on stem cell dynamics, the number and size of polyps and, to assess safety outcomes of this drug in FAP patients.
Primary: To determine whether the outcome of the RAD51 assay (HR proficient or HR deficient) correlates with objective response rate (RR) of olaparib, in patients with recurrent high grade epithelial ovarian cancer.Secondary:• Compare the…
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…