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A Multi-Centre, Randomised, Double Blind, Placebo Controlled, Parallel Group Study of TA 8995 in Patients with Mild Dyslipidaemia, Alone and In Combination with Statin Therapy

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…

A Phase 3, Randomized, Double Blind, Placebo Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation

Primary ObjectiveTo evaluate the efficacy of lumacaftor in combination with ivacaftor throughat Week 24 in subjects with cystic fibrosis (CF) who are homozygous for the F508del CFTR mutation on the CF transmembrane conductance regulator (CFTR)…

Intranasal administration of oxytocin in children with Prader-Willi Syndrome. A randomized, double-blind, placebo-controlled cross-over trial. Effects on satiety and food intake,
and social behaviour.;Amendment: Six months open-label extension to the original study

To evaluate the effects of intranasal oxytocin compared to placebo administration on appetite, satiety and food intake.Also effects on social behavior, BMI, body composition, IGF-I levels and genetic differences (deletion / mUPD). To assess oxytocin…

A Phase 1, Randomized, Double-Blind, Placebo Controlled, Single Dose Escalation Trial to Evaluate the Safety, Tolerability and Pharmacokinetics of Migalastat Hydrochloride given Intravenously to Healthy Volunteers with an Open-Label, Randomized, Two-Way Crossover Arm

Primary Objectives:* To investigate the pharmacokinetics of migalastat following a single 2hr IV infusion in healthy subjects.* To investigate the safety and tolerability of a single migalastat HCl 2hr IV infusion in healthy subjects.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…

A Phase 3, Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation

primary objective: To evaluate the long-term safety and tolerability of lumacaftor in combination with ivacaftor in subjects with cystic fibrosis (CF), homozygous or heterozygous for the F508del-cystic fibrosis transmembrane conductance regulator (…

A randomized, double-blind, placebo-controlled, parallel group study to evaluate the efficacy and safety of AFQ056 in adolescent patients with Fragile X Syndrome

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…

The effects of music therapy on expressive and communicative skills in relation to change in behavioral problems for patients with Huntington's Disease

Research questions:1. Does music therapy improve the expressive and communicative skills of patients with HD due to (specific) music therapy treatment? 2. Does music therapy change problem behavior of patients with HD?

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…

Topical rapamycin to treat fibrofolliculomas in Birt-Hogg-Dubé syndrome

Primary Objective: To determine whether topical application of rapamycin can lead to reduction in size and/or number of fibrofolliculomas in BHD patients and may prevent the growth of new ones. Secondary Objectives: Safety, formula acceptability and…

The efficacy of a physical intervention study for the improvement of the rest-activity rhythm and working memory in persons with Down*s syndrome suffering from Alzheimer*s disease. A pilot-study

The primary objective of the study is to examine the feasibility of measurements, program design and implementation adherence of a cardiovascular exercise program in persons with Down*s syndrome suffering from Alzheimer*s disease. Secondary…

A phase III, randomized, double blind, placebo-controlled clinical study to assess the efficacy and safety of GSK2402968 in subjects with Duchenne muscular dystrophy (DMD114044)

Primary: to assess efficacy of subcutaneous 6 mg/kg GSK2402968 versus placebo administered over 48 weeks in ambulant subjects with DMD. Secondary: Safety, tolerability, PK, quality of life.

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 outpatient phase 3 efficacy study of Ecopipam (PSYRX 101) in the symptomatic treatment of self-injurious behavior in subjects with Lesch-Nyhan disease.

The primary objective of this study is to assess the efficacy of Ecopipam to reduce self-injurious behaviors (SIB) in adults and children with Lesch-Nyhan Disease (LND) in an outpatient setting. The secondary objectives of this study are to assess…

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…

Genetic Susceptibility to Papilloma-induced Voice Disturbance

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

Nipple sensitivity after prophylactic nipple-sparing mastectomy compared to healthy BRCA1/2 gene mutation carriers.

The aim of this study is to objectify nipple-areola complex sensitivity in women who underwent unilateral or bilateral prophylactic NSM in Erasmus MC-Daniel den Hoed Cancer Center and their satisfaction with this operation, compared to healthy women…

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, 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…