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Genetic Susceptibility to Papilloma-induced Voice Disturbance

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

Bone density as outcome parameter in haemophilia treatment

• Compare bone density between patients with mild haemophilic and those with severe haemophilia*.• Compare the relationship between treatment regimen: prophylactic (high-dose vs. intermediate dose) and on-demand and bone density.• Examine the…

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…

A randomised, multicentre, double-blind, placebo-controlled, crossover trial determining the efficacy of dry powder mannitol in improving lung function in subject with Cystic Fibrosis aged six to seventeen years.

The purpose of this trial is primarily to assess the efficacy of inhaled mannitol compared with a true placebo in subjects with cystic fibrosis aged 6 to 17 years.We hypothesize that inhaled mannitol will improve the overall health and hygiene of…

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, Crossover Study to Evaluate the
Efficacy and Safety of Ivacaftor and VX-661 in Combination With Ivacaftor in Subjects Aged
12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-CFTR Mutation, and a
Second Allele With a CFTR Mutation Predicted to Have Residual Function

To evaluate the efficacy of VX-661 in combination with ivacaftor and ivacaftormonotherapy through 8 weeks of treatment in subjects with cystic fibrosis (CF)who are heterozygous for the F508del mutation on the CF transmembraneconductance regulator (…

A Phase 1, Randomized, Double-blind, Placebo-Controlled, Single- and Multiple-Dose Escalation Study Evaluating Safety and Pharmacokinetics of VX-440 Alone and in Combination with VX-661/Ivacaftor, and Bioavailability and Food Effect of VX-440 in Healthy Adult Subjects

The purpose of Part A is to investigate how safe the study compound VX-440 is and how well the study compound istolerated. The study will also investigate how quickly and to what extent the compound is absorbed into and eliminatedfrom the body (this…

A comparative phase2 study assessing the efficacy of triheptanoin, an anaplerotic therapy in Huntington's Disease (HD)

The primary objective of TRIHEP 3 is to evaluate the efficacy of triheptanoin in (i) increasing the short term energy response in the metabolic profile of the brain of early affected HD patients, as captured by 31P-MRS, and (ii) slowing atrophy in…

Dietary treatment of children with Angelman Syndrome and refractory epilepsy

Primary objective: - 50% seizure reduction or more at 3 months of dietary treatment .Secondary objectives: -level of ketosis on KD and MAD.-number of patients maintaining >50% seizure reduction on MAD.-number of withdrawals (not able to…

A Phase I/II Open Label Study in MPS IIIB Subjects to Investigate the Safety, Biodistribution, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously

To evaluate the safety and tolerability of IV administration of SBC-103 in subjects with MPS lllB, Sanfilippo B.

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 Phase 1, Randomized, Double blind, Placebo Controlled, Single and Multiple Dose Escalation Study Evaluating Safety and Pharmacokinetics of VX 152 Alone and in Combination with VX 661/Ivacaftor, and Bioavailability and Food Effect of VX 152 in Healthy Adult Subjects

The purpose of Part A is to investigate how safe the study compound VX-152 is and how well the study compound is tolerated. The study will also investigate how quickly and to what extent the compound is absorbed into and eliminated from the body (…

A Double-Blind, Placebo-Controlled, Dose-Escalating, Phase 1 Study to Determine the Safety, Tolerability, Pharmacokinetics and Effect on Circulating Alpha-1 Antitrypsin Levels of ARC-AAT in Healthy Volunteer Subjects and in Patients with Alpha-1 Antitrypsin Deficiency (AATD)

The primary objective of the study is Study to determine the Safety, Tolerability, Pharmacokinetics and Effect on Circulating Alpha-1 Antitrypsin Levels of ARC-AAT in Patients with Alpha-1 Antitrypsin Deficiency (AATD)

National observational study to monitor the new guideline concerning treatment of atypical hemolytic uremic syndrome

Monitoring and evaluation of the Dutch guideline for treatment of aHUS in children and adults during two years.

A Multi-Center, Open-Label Study Evaluating the Safety, Tolerability, and
Efficacy of Pridopidine in Patients with Huntington's Disease

Primary Objective: The primary objective of this study is to evaluate safety and tolerability of pridopidine in patients with HD.Secondary Objectives: The secondary objectives of the study are to assess the effects of long-term, open-label dosing…

Randomized, double-blind, placebo-controlled oxytocin trial in children with Prader-Willi syndrome.
Effects on social behaviour.

To evaluate the effects of intranasal oxytocin versus placebo on social behaviour and also on eating behaviour in children with PWS.

The Effects of Transcutaneous Vagus Nerve Stimulation on the Pupil Diameter, Heart Rate and Heart Rate Variability in Epilepsy Patients and Healthy Subjects.

Primary objective: The main objective of this pilot study is to gain more insight into the effects of tVNS: to assess if the ABVN and consecutively the NTS are really stimulated by tVNS. Therefore this study investigates the effects of tVNS on the…

Thyroid hormone analog therapy of patients with severe psychomotor retardation caused by mutations in the MCT8 thyroid hormone transporter: The Triac Trial.

1. Normalization of the abnormal serum TH parameters and thereby improving the clinical condition of the AHDS patients2. Observation of changes in cognitive and motor function.

A Multicenter, Open-Label Study of Sebelipase Alfa in Patients with Lysosomal Acid Lipase deficiency

The primary objective of this study is to evaluate the safety of sebelipase alfa in a more broad population of subjects with LAL Deficiency than have been previously studied.The secondary objectives of this study are (1) to evaluate effects of…

Assessment of real life and trained inhalation of Tobramycin Inhalation Powder (TIP) and Tobramycin Inhalation Solution (TIS) TIPTIS study.

Primary objective of the study is to record TIP inhalations on video at home to study the association between inhalation manoeuver and cough. Secondary objectives are: To study the inhalation maneuvers of patients using TIP in the home situation; to…