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A phase IIa, open-label study of two doses of GLPG1837 in subjects with cystic fibrosis and the S1251N mutation

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…

A B2-agonist as a CFTR activator in CF - Part 2

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…

A B2-agonist as a CFTR activator in CF

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…

Pilot study of everolimus in the treatment of advanced malignancies in patients with Peutz-Jeghers syndrome

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 .

An open-label study to evaluate the long-term safety and tolerability of AFQ056 in adolescent patients with Fragile X Syndrome

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*…

AN OPEN-LABEL, SINGLE-ARM, PROOF-OF-CONCEPT STUDY TO EVALUATE THE SAFETY AND EFFICACY OF SINGLE AND MULTIPLE DOSES OF REGN1500 IN PATIENTS WITH HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA

Please refer to protocol, section 1.2 "Rationale"

The chemopreventive effect of Lithium on adenoma development in patients with familial adenomatous polyposis (FAP); a pilot study

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.

FAB: Functional Analysis of BRCAness

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…

An open, non-controlled, parallel, ascending multiple-dose, multicenter study to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of SOBI003 in pediatric MPS IIIA patients

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-…

Bumetanide to Ameliorate Tuberous Sclerosis Complex Hyperexcitable Behaviors

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…