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An Open-Label Study to Evaluate the Pharmacokinetics, Pharmacodynamics, and Safety of Bempedoic
Acid in Pediatric Patients (6 to 17 Years of Age) with Heterozygous Familial Hypercholesterolemia

This study has been transitioned to CTIS with ID 2024-515864-30-00 check the CTIS register for the current data. Primary Objectives:• To assess the pharmacokinetics (PK) of bempedoic acid (ETC-1002 and ESP15228) in pediatric patients (6 to 17 years…

A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy in Subjects With Cystic Fibrosis

This study has been transitioned to CTIS with ID 2024-514173-22-00 check the CTIS register for the current data. Primary ObjectiveTo evaluate the long-term safety and tolerability of VNZ/TEZ/D-IVA in subjects with CFSecondary ObjectiveTo evaluate…

Neurological, cardiovascular, visual and neurocognitive performance in pediatric HIV-1- infected patients as compared to healthy controls NOVICE - III a follow up study

This study is a follow-up study of the NOVICE I & NOVICE II (METC 2012_093 and METC 2016_173). The objective of the study is to both cross-sectionally and longitudinally, after an interval of 10 years, gain more insight into the presence,…

Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C (NPC): A Phase III, randomized, placebo-controlled, double-blind, crossover study

This study has been transitioned to CTIS with ID 2023-510278-14-00 check the CTIS register for the current data. Primary Objective:In all jurisdictions except the United States (US), the primary objective is to evaluate the efficacy of N-Acetyl-L-…

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…

A Phase 1/2 Dose-Escalation and Dose-Expansion Study of the Safety and Efficacy of Anti-CD7 Allogeneic CAR-T cells (WU-CART-007) in Patients with Relapsed or Refractory T-cell Acute Lymphoblastic Leukemia (T-ALL)/Lymphoblastic Lymphoma (LBL)

Primary Objectives• To characterize the safety, tolerability, dose-limiting toxicities (DLT), and maximum tolerated dose (MTD) or maximum administered dose (MAD) (if no MTD is defined) and define the Recommended Phase 2 Dose (RP2D) of WU-CART-007 in…

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…

Psychophysics for the Detection of Temporal Fine Structure Performance in Normal Hearing and Cochlear Implant Users

The aim of this current proposal is to compare test methods for determining TFS performance, to establish detection thresholds for both CI users and normal hearing subjects and to produce normative data for the computer model and machine learning…

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…

A Phase 1-3 Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION363 in Amyotrophic Lateral Sclerosis patients with Fused in Sarcoma mutations (FUS-ALS)

This study has been transitioned to CTIS with ID 2024-512163-31-00 check the CTIS register for the current data. Primary objective: To evaluate the clinical efficacy of ION363 in clinical functioning and survival in FUS-ALS patients. Secondary…

The role of the relative aerobic load of walking and walking behaviour after spinal cord injury

The primary aim is to determine whether the relative aerobic load of walking is a determinant of the amount of walking in daily life in people with a chronic spinal cord injury with a walking function. Secondary goals are to gain insight into other…

An international prospective umbrella trial for children with atypical teratoid/rhabdoid tumours (ATRT)
including
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…

Clinical, virological, immunological, psychosocial and epidemiological consequences of the human MonkeypoX Virus outbreak, a PROspective observational cohort study (pro-MPX)

The aim of this study is to improve our understanding of clinical, virological, and psychosocial outcomes in patients with MPXVID. To get a better understanding of associated risk factors for MPXV infection, and to measure quality of life and stigma…

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…

A Randomised Open-Label Trial of Early, Very High-Titre Convalescent Plasma Therapy in Clinically Vulnerable Individuals with Mild COVID-19

To determine whether convalescent plasma collected from donors who have recovered from COVID-19 and who have a very high titre of anti-SARS-CoV-2 antibodies reduce the risk of hospitalization or death due to COVID-19 in patients with early symptoms…

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…

Natural history and biomarker identification in Spinocerebellar Ataxia type 7

Primary objective:- To identify (a combined set of) clinical and non-clinical markers most sensitive to disease progression in Dtuch SCA7 mutation carriers.Secondary objectives:- To quantify the annual change in disease-relevant clinical scales and…

Treating eating disorder symptoms with Eye Movement Desensitization and Reprocessing (EMDR)

This study will clarify to what extent adding the EMDR protocol in the treatment of ED will lead to a change in self-reported PTSD symptoms, injured self-image, clinical perfectionism and negative body perception as underlying transdiagnostic…