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A Phase 3 Efficacy and Safety Study of Ataluren
(PTC124 ®) in Patients with Nonsense Mutation Cystic
Fibrosis

To evaluate the ability of ataluren to improve pulmonary function relative to placeboTo determine the effect of ataluren on:1. Pulmonary symptoms2. General well-being3. Health-related Quality Life (HRQL)

A randomized, double-blind, placebo controlled study of canakinumab in patients with Hereditary Periodic Fevers (TRAPS, HIDS, or crFMF), with subsequent randomized withdrawal/ dosing frequency reduction and open-label long term treatment epochs

Primary: The primary objective of the randomized treatment epoch and for the overall study is to demonstrate that subcutaneous canakinumab administered every 4 weeks is superior to placebo in achieving a clinically meaningful reduction of disease…

Taste, Speech and oro-facial relations following partial tongue reduction: A single-center, longitudinal, functional outcome study in patients with Beckwith-Wiedemann Syndrome that have undergone tongue reduction surgery

The aim of this study is to investigate the long-term outcomes in patients with BWS that have been surgically treated at our institution with the help of simple, non-invasive, objective study tools. We hope to gain valuable information regarding the…

Oocyte vitrification in women at risk of ovarian failure; an observational study with follow-up of children

To determine safety and efficacy of oocyte vitrification.

Intracytoplasmatic Sperm Injection using testicular spermatozoa in men with azoospermia; an observational study with follow-up of the offspring.

To evaluate the efficacy of TESE and to describe the risks of the TESE procedure for the men with a four year follow-up of their offspring.

Multi-center, randomized, double-blind, placebo-controlled phase 2 study to assess the safety, tolerability and early signs of efficacy of tid orally administered BAY63-2521 in adult deltaF508
homozygous Cystic Fibrosis patients

The aim of this trial is to test in a first clinical study the above mentioned hypothesis that BAY63-2521 can at least partially correct deltaF508-CFTR function. Therefore, we plan to include patients with Cystic Fibrosis, who are homozygous for the…

A 2-part, randomized, double-blind, placebo-controlled, sequential group, dose-escalation study to assess the safety, tolerability and pharmacokinetics of single and multiple ascending oral doses of GLPG2737 in healthy male sub.

The purpose of the study is to investigate how safe GLPG2737 is and how well GLPG2737 is tolerated. It will also be investigated how quickly and to what extent GLPG2737 is absorbed into, distributed in, and eliminated from the body (this is called…

A phase 3 extension study of Ataluren (PTC124) in patients with Nonsense Mutation Cystic Fibrosis

Primary objectiveTo evaluate the long-term safety of 10-, 10-, 20-mg/kg ataluren in patients with nonsense mutation cystic fibrosis (nmCF), who previously participated in pivotal study PTC124-GD-021-CF, as determined by adverse events and laboratory…

Optimal timing and technique for surgical correction of peri-orbital osseous deformities in congenital craniofacial disorders

Primary objective:To determine the most optimal technique and timing of surgery for correction of peri-orbital osseous deformities (hypertelorism, vertical orbital dystopia, and/or midface hypoplasia) for each specific condition (1. syndromic…

A Randomized, Placebo- and Active-Controlled, Double-Blind, 4-way Crossover Design, Thorough ECG Study of VX-770 in Healthy Adult Subjects

Part A:To evaluate the safety and tolerability of increasing doses of VX-770 up to 450 mg (q12h) in healthy male subjects.Part B:To determine if therapeutic or supratherapeutic systemic exposure to multiple doses of VX 770 prolongs the mean…

Behavioral and psychosocial effects of rapid genetic counseling and testing in newly diagnosed breast cancer patients: a multicenter study

The proposed study will focus on newly diagnosed breast cancer patients who, prior to receiving treatment, are identified on the basis of age or family history of breast or ovarian cancer as having at least a 10% risk of carrying a mutation in the…

Oocyte vitrification in women at risk of ovarian failure; an observational study with follow-up of children

To determine safety of oocyte vitrification.

Bèta-TCP (bèta-tricalcium phosphate) as a bone substitute in patients undergoing repair of the alveolar cleft in CLP(Cleft Lip Palate) patients.

This current study aims to investigate the use of Curios bèta-TCP, a synthetic tricalcium phosphate for repair of the alveolar cleft in patients with clefts of lip, alveolus and palate.The specific properties of Progentix TCP result in optimal and…

A three-arm, randomized, double-blind, placebo-controlled study of the efficacy and safety of two trough-ranges of everolimus as adjunctive therapy in patients with tuberous sclerosis complex (TSC) who have refractory partial-onset seizures

Primary: To compare the reduction in frequency of partial-onset seizures on each of two trough ranges of everolimus (3-7 ng/mL and 9-15 ng/mL) versus placebo in patients with TSC who are taking one to three AEDs. Key secondary: Ability to completely…

CLEfts and FEeding Difficulties (CLEFED-1): the effect and timing of surgical closure in children with cleft palate.

The main objectives of this study are feeding techniques and skills and weight gain. Secondary objectives are NS feeding *feeding sequelae*, extent of the cleft, associated malformations, upper respiratory infections / pneumonia, medication (child…

Nasoalveolar Molding with nasal stent to improve nasal symmetry in unilateral cleft patients

Objective: permanent improvement of the symmetry of the external nose in patients with unilateral cleft

Prospective study in congenital giant melanocytic naevi after curettage and laser ablation with the Erbium YAG laser (left-right comparison)

Proof that laser treatment will be better than the routine method using curettage. Besides it is claimed that laser treatment is less painfull.This will be assessed by photography, in which only black white (grey) evaluations are made. Colours are…

The Impact of Psychological and Medical Factors of Women having a Child with a Congenital Anatomical Malformation on Subsequent Pregnancy Decision-Making: a psychological evaluation.

To identify factors that have an influence on the reproductive decision-making for a subsequent pregnancy of women having a child with congential anatomical anomalies.

Improving an active lifestyle and sports participation in adolescents and young adults with meningomyelocele (MMC): Optimalisation and evaluation of an intervention.

The purpose of this study is to improve, implement and evaluate an intervention program to improve physical activity in daily life and sportsparticipation of adolescents and young adults with MMC. The purpose of the intervention "Active…

The use of β-TCP (bèta-tricalcium phosphate) as a filler of the autologous symphyseal bone graft harvest site in patients undergoing grafting of alveolar clefts in CLAP(Cleft Lip Alveolus Palate) patients

This current study aims to investigate the use of bèta-TCP, a synthetic tricalcium phosphate, as a filler of the autologous symphyseal bone graft harvest site in patients undergoing grafting of clefts of lip, alveolus and palate.The specific…