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The impact of communication possibilities on psychosocial difficulties in children with hearing loss

The primary objective of this study is to determine the effect of specific communicative abilities (speech recognition in noise; vocal emotion recognition and emotion expression) in children with hearing loss on their psychological (emotional and…

A Phase 1/2 Open-Label, Multicenter, Dose Ranging and Confirmatory Study to Assess the Safety, Tolerability and Efficacy of PBKR03 Administered to Pediatric Subjects with Early Infantile Krabbe Disease (Globoid Cell Leukodystrophy) (GALax-C)

This first-in-human study is planned to include both a dose ranging phase and a confirmatory phase, with the intention that all data collected throughout the study may support registration. The dose ranging phase will be conducted in cohorts of 3-4…

Investigating immune-epithelial cells in patients with cystic fibrosis

The primary objective of this study is to characterize the immune cell composition and epithelial-immune cell crosstalks in patients with CF. The endpoint will be constituted from several read-outs on peripheral blood mononuclear cells (PBMC's…

A Multicenter, Long-Term Open-Label Safety Study of Adjunctive Troriluzole in Subjects with Obsessive Compulsive Disorder

The primary objective of the study is to evaluate the long term safety and tolerability of troriluzole as adjunctive therapy in subjects with OCD who have had an inadequate response to standard of care (SOC) treatment for OCD and as defined in the…

A Phase 3, multicenter, randomized, double-blind, placebo-controlled study to assess the efficacy, safety, and tolerability of AVP-786 (deudextromethorphan hydrobromide [d6-DM]/quinidine sulfate [Q]) for the treatment of agitation in patients with dementia of the Alzheimer*s type.

The primary objective is to:* Evaluate the efficacy, safety, and tolerability of AVP-786 compared to placebo for thetreatment of agitation in patients with dementia of the Alzheimer*s typeThe secondary objectives are to:* Evaluate the effects of AVP…

A Dose-Escalation and Expansion Study of the Safety and Pharmacokinetics of XL092 as Single-Agent and Combination Therapy in Subjects with Inoperable Locally Advanced or Metastatic Solid Tumors

• Determine the maximum tolerated dose (MTD) and/or recommended dose (RD) for further evaluation of XL092 when administered alone and in combination with ICIs to subjects with advanced solid tumors• Evaluate preliminary efficacy of XL092 when…

A Phase 2 Basket Study of Tucatinib in Combination with Trastuzumab in Subjects with Previously Treated, Locally Advanced Unresectable or Metastatic Solid Tumors Driven by HER2 Alterations

Primary ObjectiveTo evaluate the antitumor activity of tucatinib given in combination with trastuzumab in subjects with previously treated, locally-advanced unresectable or metastatic human epidermal growth factor receptor 2 (HER2) overexpressing/…

Phase 3, Multicenter, Double-blind, Randomized, Placebo-controlled, Parallel Group Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Apremilast in Children From 5 to Less Than 18 Years of Age With Active Juvenile Psoriatic Arthritis (PEAPOD)

This study has been transitioned to CTIS with ID 2023-503435-17-00 check the CTIS register for the current data. Estimate the efficacy of apremilast compared with placebo in the treatment of Juvenile Psoriatic Arthritis (JPsA) in pediatric subjects…

A multicENter, randomized, open-label, parallel group, pilot study to evaluate the use of sacubitril/valsartan in HeartMate 3 LVAD recipients

The primary objective of this study is to assess the safety and tolerability of sacubitril/valsartan compared with standard of care used for treating BP in patients that have been implanted with the HM3 LVAD.

An open-label dose escalation study to estimate maximum tolerated dose (MTD), identify dose-limiting toxicities (DLTs) and study pharmacokinetics following a single dose of intracranially administered temozolomide-based SI-053 as an add-on to the current standard of care (SoC), in adult patients with newly diagnosed glioblastoma (GBM).

This study has been transitioned to CTIS with ID 2024-515128-35-00 check the CTIS register for the current data. The primary objective is:• To evaluate the safety and tolerability of a single local i.c. dose of SI-053 to establish the MTD and/or the…

Phase 3 Follow-up Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated with Variants in the RPGR gene

This study has been transitioned to CTIS with ID 2024-511411-25-00 check the CTIS register for the current data. Main objective:To assess the long-term safety and tolerability of AAV5-hRKp.RPGR in individuals with RPGR-XLRPTo assess the long-term…

An Open-label, Multinational, Multicenter, Intravenous
Infusion Study of the Efficacy, Safety, Pharmacokinetics,
and Pharmacodynamics of avalglucosidase alfa in
Treatment-naïve Pediatric Participants with Infantile-Onset Pompe Disease (IOPD)

This study has been transitioned to CTIS with ID 2024-513859-33-00 check the CTIS register for the current data. The overall objective is to assess efficacy, safety, pharmacokinetic (PK), pharmacodynamics (PD) of avalglucosidase alfa in male and…

A Randomised, Double-Blind, Placebo-Controlled Phase 3 Clinical Trial to Assess the Efficacy and
Safety of Lebrikizumab in Combination With Topical Corticosteroids in Adult and Adolescent Patients
With Moderate-To-Severe Atopic Dermatitis That Are Not Adequately Controlled With Cyclosporine or
For Whom Cyclosporine is Not Medically Advisable.

To evaluate the efficacy of lebrikizumab compared with placebo in patients not adequately controlled with cyclosporine or for whom cyclosporine is not medically advisable up to Week 16

An Open Label Study to Evaluate the Long-term Safety and Efficacy of Odevixibat (A4250) in Patients with Alagille Syndrome (ASSERT-EXT)

This study has been transitioned to CTIS with ID 2023-509028-17-00 check the CTIS register for the current data. To demonstrate a sustained effect of odevixibat on pruritus in patients with ALGS who have completed study A4250-012 (ASSERT)

Randomised, double-blind, placebo-controlled and parallel group
trial to investigate the effects of two doses (up-titration to a fixed
dose regimen) of oral BI 685509 on portal hypertension after 24
weeks treatment in patients with clinically significant portal
hypertension (CSPH) in compensated cirrhosis

The trial will compare two doses of BI 685509 (2 mg and 3 mg BID) with placebo, on top of standard of care, in patients with CSPH in compensated alcohol-related cirrhosis. The primary objective is to estimate the mean difference between treatment…

The nursing home as a possible risk factor for a decrease in physical activity in elderly people with dementia: relation with cognitive function and quality of life.

Primary Objective: What is the effect of a transfer from home to a nursing home environment on the level of physical activity in the elderly with dementia over time? Secondary Objective(s): Is there a relationship between a possible change in the…

Building participant-specific biomechanical models of the knee through the integration of dynamic computed tomography images and biomechanics lab data.

Our primary aim is to develop methods and assess the feasibility of creating a participant-specific biomechanical model of the knee that integrates biomechanics data with dynamic computed tomography (CT) imaging data to determine 3D tibiofemoral and…

An open-label trial of the long-term safety and tolerability of nintedanib per os, on top of standard of care, over at least 2 years, in children and adolescents with clinically significant fibrosing Interstitial Lung Disease (InPedILD®-ON)

The main objective of the trial is to assess the safety and tolerability of long-term treatment with nintedanib in pediatric patients with clinically significant fibrosing ILD.See section 2.1 and 2.2 of the protocol.

A Phase 2 study to assess the efficacy and safety of 2 dosage regimens of oral fidrisertib (IPN60130) for the treatment of fibrodysplasia ossificans progressiva in male and female paediatric and adult participants

This study has been transitioned to CTIS with ID 2024-511469-13-00 check the CTIS register for the current data. Main objective:• Evaluate the efficacy of IPN60130 monotherapy compared with placebo recipients in inhibiting new HO volume in adult and…

An exploratory pharmacokinetics and pharmacodynamics study of beta-lactam antibiotics in pediatric intensive care patients: is there a need for more precision? (EXPAT-Kids)

The aim of this study is to describe the PK/PD characteristics of frequently used beta-lactam antibiotics in PICU patients. The main objective is to identify whether current antibiotic dosing regimens of the selected beta-lactams achieve defined…