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

To determine safety of oocyte vitrification.

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.

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…

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…

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…

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…

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 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…

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…

Treatment Effect of Bone Anchored Protraction in Cleft Patients

Bone-anchored maxillary protraction therapy improves facial profile in cleft children.

A two parts, open-label, single-dose mass balance and absolute bioavailability study with an oral regular dose of PHA-022121 and an oral (part 1) and intravenous (part 2) microtracer dose of 14C-PHA-022121 in healthy male subjects

In this study we will investigate how quickly and to what extent PHA-022121 is absorbed, transported, and eliminated from the body (this is called pharmacokinetics). PHA-022121 will first be given as a single oral dose, and then as an infusion in a…

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)

Study of expression of and immunity against neoantigens in patients with constitutional mismatch repair deficiency (CMMR-D) syndrome

In this explorative study, we aim to:• Analyze neoantigens expressed on different tumors of CMMR-D patients, to identify possible target antigens for DC vaccination studies.• Investigate the presence of pre-existing immune responses against…

A randomized, double-blind, placebo-controlled, single ascending dose study to examine the safety, tolerability and pharmacokinetics of orally administered PHA-022121 in healthy subjects.

Primary ObjectiveTo assess the tolerability and safety of single ascending oral doses of PHA-022121 administered after a standard meal and of a single 40 mg dose under fasted conditions in healthy adult subjects.To assess the PK characteristics of…

A Phase 2 Efficacy and Safety Study of Niraparib in Men with Metastatic Castration-
Resistant Prostate Cancer and DNA-Repair Anomalies

Primary objective:- To assess the efficacy of niraparib in subjects with measurable mCRPC and who have either biallelic DNA-repair anomalies in BRCA (BRCA 1 and BRCA 2 or germline BRCA. Secondary objectives:- To assess the efficacy of niraparib in…

An open-label, single-sequence crossover drug-drug interaction study to evaluate the effect of multiple oral doses of itraconazole on the pharmacokinetics of PHA-022121 in healthy subjects

The purpose of this study is to investigate the effect of multiple doses of itraconazole on how quickly and to what extent the new compound PHA-022121 is absorbed into the body and eliminated from the body (this is called pharmacokinetics). It will…

Newborns with Congenital Diaphragmatic hernia: inhaled Nitric Oxide versus intravenous Sildenafil: ;an international randomized controlled trial

To determine the most effective drug (iNO or IV sildenafil) for the treatment of PH in newborns with CDH and to reach evidence based dosing of this drug. Subsequently this drug will be added to the international guidelines for the treatment of…

AN OPEN-LABEL, FIXED-SEQUENCE, ASCENDING-DOSE, FIRST-IN-HUMAN STUDY TO ASSESS THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS, AND EFFICACY OF INTRAVENOUS INFUSIONS OF ATB200 CO-ADMINISTERED WITH ORAL AT2221 IN ADULT SUBJECTS WITH POMPE DISEASE

To evaluate the safety, tolerability, PK, PD and efficacy of intravenous (IV) ATB200 alone and when co-administered with oral AT2221.