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A Phase 3, Randomized, Double-Blind Trial of Two Formulations of Setmelanotide (Daily and Weekly) with a Crossover to Open-Label Once Weekly Setmelanotide in Patients with Specific Gene Defects in the Melanocortin-4 Receptor Pathway Who Are Currently on a Stable Dose of the Once Daily Formulation

Primary: To compare the pharmacokinetics (PK) of the once daily (QD) and once weekly (QW) formulations of setmelanotideSecondary Objectives:To assess the safety of the QW formulation of setmelanotide with up to 6 months (26 weeks) of drug…

An Open-Label, Multicenter, Extension Trial to Evaluate the Long-Term Safety and Efficacy of Apitegromab in Patients with Type 2 and Type 3 Spinal Muscular Atrophy Who Completed Previous Investigational Trials of Apitegromab

This study has been transitioned to CTIS with ID 2024-511654-42-00 check the CTIS register for the current data. -Evaluate the long-term safety and tolerability of apitegromab in patients with Type 2 and Type 3 SMA-Evaluate the long-term efficacy of…

A PROSPECTIVE, RANDOMIZED, OPEN-LABEL PHASE 2 STUDY TO EVALUATE THE SUPERIORITY OF INOTUZUMAB OZOGAMICIN MONOTHERAPY VERSUS ALLR3 FOR INDUCTION TREATMENT OF CHILDHOOD HIGH RISK FIRST RELAPSE B-CELL PRECURSOR ACUTE LYMPHOBLASTIC LEUKAEMIA

This study has been transitioned to CTIS with ID 2023-509810-13-00 check the CTIS register for the current data. Primary Objectives Efficacy: To demonstrate the superiority of InO monotherapy vs ALLR3 induction in paediatric participants between 1…

Evaluation of QbTest on a smartphone- A Clinical Investigation

The objective of the current study is to evaluate QbTest on a smartphone (QbMT), in terms of: 1. The feasibility of QbMT as a smartphone software that incorporates the Quantified behavioral Test (QbTest *)2. The validity of QbMT in individuals…

A TWO-PART, SEAMLESS, MULTI-CENTER, RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE-BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7204239 IN COMBINATION WITH RISDIPLAM (RO7034067) IN PATIENTS WITH SPINAL MUSCULAR ATROPHY

This study has been transitioned to CTIS with ID 2023-506761-65-00 check the CTIS register for the current data. The purpose of this Phase II/III study is to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of…

An international prospective umbrella trial for children with atypical teratoid/rhabdoid tumours (ATRT)
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A randomized phase III study evaluating the non-inferiority of three courses of high-dose chemotherapy (HDCT) compared to focal radiotherapy as consolidation therapy

This study has been transitioned to CTIS with ID 2022-501456-28-00 check the CTIS register for the current data. Primary objectives Part A:To test the non-inferiority, as evaluated by OS, of three courses of HDCT compared to focal RT plus…

Prospective Analysis of Pharmacokinetic Infliximab data in Paediatric Inflammatory Bowel Disease patients

This study has been transitioned to CTIS with ID 2023-507352-72-00 check the CTIS register for the current data. The primary study objective of our study is to assess the efficacy of an IFX intensified induction scheme vs. a standard dosing schedule…

Clinical Survey Study to Assess Physical Function and the Incidence of Hypoglycemia in Patients 1 Year of Age and Over with GSD III.

The primary objective of this study is to evaluate the incidence of hypoglycemia in adult and pediatric patients with GSD III. The secondary objective of this study is to evaluate the sensitivity of various muscle strength and function measures to…

Validation and development of new dichoptic VR-gaming method to treat childhood amblyopia; Vedea Amblyopia Therapy (VAT)

The VAT project is set out to prove that this approach is superior and that the Quality of Life of children with amblyopia might be significantly improved in comparison to occlusion therapy.

Clinical validation of the NOVEOS immunoassay for allergy diagnostics

The aim is to evaluate the clinical performance of the system and verify the claims of reduced interference with biotin and CCDs. The measurements on NOVEOS will be mirrored in this study against the clinical diagnosis, the outcome of skin prick…

A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy

Primary Objective:Double-blind period : Evaluate the effect of SRP-4045 and SRP-4053 (combined-active group) compared to placebo on ambulation, endurance, and muscle function, as measured by the 6MWTSecondary Objectives:- Double-blind period:…

Decoding personalized nutritional, microbiome and host patterns impacting clinical and prognostic features in Crohn*s disease

By means of this study we want to find out whether there are certain characteristics that can influence or predict the course of Crohn's disease. We want to do this by collecting data on diet, inflammation levels in the blood, complaints and…

StoMakker: Improving the quality of life of children receiving an ileostomy, colostomy or continent urostomy by offering access to a peer support platform, age dependent information provision and games in a smartphone application

The main objective of this study is to investigate whether access to a peer support platform, age-dependent information provision and games in a smartphone application increases self-reported quality of life, for children receiving a stoma.

SARSLIVA 2.0: Dense saliva sampling for studying SARS-CoV-2 transmission dynamics - a household study

primary objectives:SARSLIA 2.0:1. To study SARS-CoV-2 transmission dynamics within households using saliva, with home self-sampling, storage and transport to the laboratory.2. To study the emergence of SARS-CoV-2 in saliva from asymptomatic or pre-…

Evaluation of the additional effect of continuous ultrasound bladder monitoring in urotherapy for children with functional daytime urinary incontinence. The SENS-U trial

To evaluate cost effectiviness of the SENS-U (continuous ultrasonic bladder monitoring) in urotherapy for children with functional daytime urinary incontinence.

An 18-month, open-label, single-arm safety extension study of an age-and bodyweight-adjusted oral finerenone regimen, in addition to an ACEI or ARB, for the treatment of children and young adults from 1 to 18 years of age with chronic kidney disease and proteinuria

This study has been transitioned to CTIS with ID 2023-504885-50-00 check the CTIS register for the current data. A study to learn more about how safe the study treatment finerenone is inlong-term use when taken with an ACE inhibitor or angiotensin…

Connection Interrupted: Genetic causes and clinical characteristics of hereditary optic neuropathies

The goal of this study is to identify the genetic causes underlying hereditary optic neuropathies and establish genotype-phenotype correlations. This will lead to more reliable prognosis predictions, better genetic counselling and hopefully form a…

A multi-center, open-label study to determine the dose and safety of oral asciminib in pediatric patients with Philadelphia chromosome positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP), previously treated with one or more tyrosine kinase inhibitors.

This study has been transitioned to CTIS with ID 2023-508129-28-00 check the CTIS register for the current data. The primary objective of this study is to characterize the pharmacokinetic (PK) profile of asciminib in pediatric patients, with the…

Alveolar cleft repair using osteoinductive ceramics in children with unilateral cleft lip and palate

To provide for evidence that calcium phosphate based scaffolds generate equal amounts of residual bone volume compared to the use of autologous bone grafts in unilateral alveolar cleft grafting. A secondary objective is to prove that calcium…

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Fixed-Dose, Multicenter Study to Examine the Efficacy and Safety of ZX008 in Subjects with CDKL5 Deficiency Disorder Followed by an Open-Label Extension

This study has been transitioned to CTIS with ID 2023-506269-78-00 check the CTIS register for the current data. Objectives for Part 1: Primary (Efficacy): To demonstrate that the efficacy of fenfluramine (ZX008) 0.8 mg/kg/day is superior to placebo…