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A Clinical Study of Two Doses of a Selective p38 MAP Kinase Inhibitor, VX-745, to Evaluate the Effects of 12-Week Oral Twice-Daily Dosing on Amyloid Plaque Load as Assessed by Quantitative Dynamic 11C-PiB Positive Emission Tomography (PET) Amyloid Scanning

The primary objective is to assess the effects on amyloid plaque burden of administration of VX-745 for 12-weeks, as assessed by Dynamic 11C-PiB (Carbon-11 labeled Pittsburgh Compound B) PET Amyloid Scanning in patients with Mild Cogntitive…

Treatment of central pain syndromes by caloric vestibular stimulation

Objective of the study: With this pilot study we attempt to explore the possibility to treat patients suffering from intractable neuropathic pain with CVS

An open-label study to evaluate the long-term safety and tolerability of AFQ056 in adolescent patients with Fragile X Syndrome

PrimaryTo evaluate the long-term safety and tolerability of AFQ056 in adolescent patients with FXS as assessed by:* Incidence and severity of adverse events and serious adverse events* Changes in vital signs, laboratory assessments, and ECGs*…

A randomized, double blind, placebo-controlled crossover study to investigate the effects of a selective serotonergic reuptake inhibitor on resting state fMRI in heathy volunteers.

We propose to conduct a study in healthy subjects where a serotonergic challenge is given in a placebo-controlled, crossover fashion. Before each challenge, participants will be measured 1 time to define their baseline resting state networks (RSNs)…

An open-label, one-sequence crossover interaction study investigating the effect of multiple doses of Lu AE58054 on cytochrome P450 3A4 and 2D6 activity measured by midazolam and dextromethorphan pharmacokinetics in healthy young men

To study how the registered medicine Midazolam is absorbed, broken-down and excreted by the body after or without administration of multiple doses of the test compound.To study how the registered medicine Dextromethorphan is absorbed, broken-down…

A 12-month Longitudinal, Prospective, Observational, Natural History Study of Patients with Sanfilippo Syndrome Type A (MPS IIIA)

1. Primary Objective(s) The primary objective(s) of this study are to:* Evaluate the course of disease progression in patients with MPS IIIA who are untreated with any investigational products to inform possible future treatment studies * Determine…

Robotic toys within occupational therapy for children with developmental disabilities.

Objective:This study has as its aim to examine the short-term effect of a robotic-facilitated play therapy within occupational therapy on the level of playfulness of children with developmental disabilities aged 2-16 years.

A Phase 2a, Proof-of-Concept, Open-Label Study to Evaluate the Pharmacodynamics, Pharmacokinetics, and Safety of Obicetrapib in Patients with Early Alzheimer*s Disease (Hetero/Homozygote APOE4 Carriers)

The primary objective of this study is to evaluate the pharmacodynamics (PD) (apolipoproteins/lipid particles and cholesterol efflux) of obicetrapib in cerebrospinal fluid (CSF) and plasma (apolipoproteins/lipid particles) in patients with early…

Phase 3b Open-Label, Multicenter, Safety Study of BIIB037 (aducanumab) in Subjects With Alzheimer*s Disease Who Had Previously Participated in the Aducanumab Studies 221AD103, 221AD301, 221AD302 and 221AD205

Primary objective:To evaluate the safety and tolerability of aducanumab over 100 weeks of treatment after a wash-out period imposed by discontinuation of feeder studies in participants who had previously received aducanumab (i.e., previously treated…

Open label extension study for patients with early Alzheimer's Disease (AD) enrolled in study ANAVEX2-73-AD-004

Primary objective:To continue assessing the safety and tolerability of ANAVEX2-73.Safety and Tolerability Measures:* Physical examination* Vital signs (heart rate, respiratory rate, systolic blood pressure [SBP], diastolic blood pressure [DBP],…

An open-label, single-arm, multicenter study of intracerebral administration of adeno-associated viral vectors serotype rh.10 carrying the human N-sulfoglycosamine sulfohydrolase (SGSH) cDNA for the treatment of mucopolysaccharidosis type IIIA (MPS IIIA)

Primary: To assess the efficacy of intracerebral delivery of AAVrh.10SGSH gene therapy (LYS-SAF302) in improving or stabilizing the neurodevelopmental status of MPS IIIA patients after 24 months (main cohort), compared to the expected evolution…

A MULTICENTER, LONG-TERM EXTENSION STUDY TO EVALUATE THE SAFETY, TOLERABILITY, AND EFFICACY OF AL002 IN PARTICIPANTS WITH ALZHEIMER*S DISEASE

This study has been transitioned to CTIS with ID 2023-506872-29-00 check the CTIS register for the current data. The primary objectives of this add-on Phase 2 LTE study (see Table 1) are to evaluate the long-term safety and tolerability of IMP at…

Evaluating the safety, tolerability, and pharmacodynamic properties of IW-6463 in healthy elderly subjects.

Primary* To evaluate the effect of IW-6463 on cerebral blood flow (CBF) in healthy elderly participants* To assess the safety and tolerability of IW-6463 vs placebo when administered to healthy elderly participants for up to 15 daysExploratory* To…

A Phase 2, Single-Arm Study of the Biomarker Effects of ALZ-801 in Subjects with Early Alzheimer*s Disease Who Are Carriers of the *4 Variant of the Apolipoprotein E Gene (APOE4/4 or APOE3/4)

This study has been transitioned to CTIS with ID 2024-515858-25-00 check the CTIS register for the current data. Objectives - Core StudyPrimary Objectives• To evaluate the effects of oral ALZ-801 in subjects with Early AD who have the APOE4/4 or…