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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…

Evaluating Prenatal Exome Sequencing Study

This study evaluates the impact of the various outcomes of prenatal exome sequencing (definitive diagnosis, probable diagnosis and incidental findings) on clinical decision making and on parental psychological wellbeing, compared between different…

A phase I, open-label, randomized, two period, two-way cross over study to assess the influence of a high-calorie, high fat meal on the bioavailability of a 40 mg extended release PHA 022121 oral formulation

In this study we will investigate the influence of a high-calorie, high-fat meal on the uptake, breakdown, and elimination of the study compound PHA-022121. We will also investigate how safe PHA-022121 is and how well it is tolerated when it is used…

A Multicenter Study to Assess the Safety, Tolerability, Pharmacokinetics, and Effect on
Seizures and Behavioral Symptoms of Multiple Individually Titrated Doses of Radiprodil
in Children with GRIN-related Disorder.

This study has been transitioned to CTIS with ID 2023-509672-42-00 check the CTIS register for the current data. Primary Objective:-To determine the long-term safety and tolerability of multiple individually titrated doses of radiprodil as an addon…

A Phase 1/2 Open-Label, Ascending Dose, Multicenter Study to
Evaluate the Safety and Preliminary Efficacy of AVB-101
Administered by Bilateral Intrathalamic Infusion in Subjects With
Frontotemporal Dementia With Progranulin Mutations (FTD-GRN)

Primary Objectiveo evaluate the safety and tolerability of a one-time, intrathalamic administration of AVB-101 in subjects with FTD-GRN.Secondary Objectiveo To evaluate the preliminary clinical and biomarker measures of efficacy of a one-time,…

A Multicenter, Randomized, Double-blind, Placebo-controlled, Phase 1b/2a Study of WVE-003 Administered Intrathecally in Patients With Huntington*s Disease

Primary objective:x To evaluate the safety and tolerability of WVE-003 in patients with Huntington's disease (HD)Secondary objectives:x To characterize the pharmacokinetics (PK) of WVE-003 in plasma x To characterize the concentration of WVE-…

A Phase I, two parallel arms, open label, randomized, two period, two-way cross-over study to assess the steady-state pharmacokinetics of a 40 mg PHA-022121 XR tablet administered once daily with the steady-state pharmacokinetics of two 10 mg PHA-022121 IR capsules administered twice daily (Arm 1) and to assess the steady-state pharmacokinetics of 40 mg PHA-022121 XR tablet administered twice daily with the steady-state pharmacokinetics of four 10 mg PHA- 022121 IR capsules administered twice da

In this study we will investigate how quickly and to what extent a formulation of the study compound PHA-022121 is absorbed, transported, and eliminated from the body. There are two compositions of the study compound: one with an extended release…

A multi-centre, randomised, double-blind, placebo-controlled, Phase 2 study to investigate efficacy, safety and tolerability of SLN360 in participants with elevated lipoprotein(a) at high risk of atherosclerotic cardiovascular disease events.

The primary objective is to evaluate the effect of SLN360 on circulating levels of Lp(a) in participants with elevated Lp(a) at high risk of ASCVD events.The secondary objectives are to:• Evaluate safety and tolerability of SLN360 in participants…