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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…

Intranasal administration of oxytocin in children with Prader-Willi Syndrome. A randomized, open-label, cross-over trial of different treatment regimens of oxytocin administration. Effects on eating behaviour and social behaviour.

To evaluate the effects of long-term intranasal oxytocin on social behaviour in children with PWS and to compare the effects of different doses and frequencies of oxytocin administration

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…

An open-label, two-period, fixed sequence study to evaluate the effects of multiple oral doses of AFQ056 on the pharmacokinetics of a monophasic oral contraceptive in healthy female volunteers

* To examine how multiple oral doses of the test compound (an experimental medication) will be taken up by the body, metabolized and excreted by the body in combination with the oral contraception pill which consists of ethinyl-estradiol and…

Genetic Susceptibility to Papilloma-induced Voice Disturbance

To discover human genes which are likely to confer susceptibility for recurrent respiratory papillomatosis

Efficacy and safety of treatment with Cinacalcet in patients with Primary Hyperparathyroidism due to a MEN-1 mutation

1. What are the effects of cinacalcet on clinical and biochemical parameters, including bone turnover markers, in patients with primary hyperparathyroidism due to a MEN-I mutation?2. What are the effects of cinacalcet on bone mineral density and…

A Randomized, Multicenter, Multinational, Phase 3B, Open-Label, Parallel-Group Study of Fabrazyme (agalsidase beta) in Treatment-Naive Male Pediatric Patients with Fabry Disease Without Severe Symptoms

The objectives of this open-label study are to evaluate the efficacy (GL-3 clearance), pharmacokinetics (PK), and safety parameters (including immunogenicity) for 2 alternative dose regimens of Fabrazyme (0.5 mg/kg every 2 weeks [q2w] and 1.0 mg/kg…

Suicidality in Huntington*s disease - a qualitative add-on study

To explore how HD mutation carriers experience suicidal ideation, plans, or previous suicide attempts (from now on jointly referred to as suicidality), how they cope with suicidality, and which ideas and wishes HD mutation carries have on how…

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*…

The effect of intranasal insulin on development and behaviour of children with Phelan-McDermid syndrome

The aim of this project is to validate the hypothesis that intranasal insulin improves development and behaviour in children with Phelan-McDermid syndrome.

Single-center, open label, multiple dose study to investigate the pharmacokinetics of RO5186582 given BID over 28 days, and in addition, the excretion and metabolism of [13C]- labelled IV microdoses and an oral [14C]-labelled dose of RO5186582 in Healthy Male Volunteers

Primary:* To determine the absolute oral bioavailability and further characterize the PK of RO5186582 using a stable isotope technique.* To explore the routes and rates of elimination of [14C] labelled RO5186582.Secondary:* To identify and quantify…

An open-label study of two single oral doses of galantamine, examining the pharmacokinetics, safety, and tolerability in children with Down syndrome

The primary objective is to determine the pharmacokinetic characteristics of galantamine after 2 single oral doses in children with Down syndrome. Secondary objectives are to evaluate safety and tolerability.

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 effect of polyethylene glycol treatment on gut health in Cystic Fibrosis, a pilot study

Main objective: to assess the effects of PEG treatment on bile acid homeostasis in cystic fibrosis patients. Secondary objective: to assess the effects of PEG treatment on gastrointestinal symptoms and quality of life, and on gut health-related…

The glutamate/GABA balance as novel therapeutic target for psychotic and cognitive symptoms in 22q11.2 deletion syndrome

The objective of this study is to examine the effect of riluzole on the glutamate/GABA balance in the brain in patients with 22q11.2DS. The secondary objective is to examine the effects of riluzole on psychotic symptoms and cognitive functioning. In…

The influence of pancreatic function on the pharmacokinetics of ivacaftor in patients with cystic fibrosis

Our primary objective of the study is:- To evaluate if pancreas sufficiency leads to a higher absorption and exposure of ivacaftor in CF patients than pancreas insufficiency. This will be done by comparing pharmacokinetic parameters (AUC, Cmax, Tmax…

Ethanol submandibular duct ligation for drooling in children with neurodisabilities

the primary aim of this study is to evaluate the safety (adverse events and complaints) of submandibular duct ligation right after intraductal ethanol infusion into the submandibular salivary gland (Ethanol two-duct ligation [E-2DL] in order to…

A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Gating or Residual Function Mutation (F/G and F/RF Genotypes)

Primary objective (Parts A and B):To evaluate the long-term safety and tolerability of VX-445/TEZ/IVA in subjects with CF who are heterozygous for the F508del mutation and a gating (F/G) or residual function (F/RF) mutation.Secondary objectives (…

AN OPEN-LABEL, MULTICENTER STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS AND PHARMACODYNAMICS OF RO7248824 IN PARTICIPANTS WITH ANGELMAN SYNDROME

This study has been transitioned to CTIS with ID 2024-514797-45-00 check the CTIS register for the current data. PRIMARY OBJECTIVE: - To assess the (long-term) safety and tolerability profile of RO7248824.SECONDARY OBJECTIVE: - To investigate the…