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Bumetanide in Autism Medication and Biomarker study

The primary objective of the proposed study is to investigate whether bumetanide therapy indeed reduces autistic symptomatology. Important secondary goals of this project are to determine whether bumetanide will improve specific behavioral,…

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…

A Phase 2A Multicenter, Randomized, Double Blind, Placebo-Controlled, Parallel-Group Safety and Tolerability Trial of PQ912 in Subjects with early Alzheimer's Disease

To assess the safety and tolerability of multiple doses of PQ912 compared with placebo in subjects with early stage of Alzheimers Disease.

Assessment of Safety, Tolerability, and Pharmacodynamic Effects of LY2886721 in Patients with Mild Cognitive Impairment Due to Alzheimer*s Disease or Mild Alzheimer's Disease

Assessment of Safety, Tolerability, and Pharmacodynamic Effects of LY2886721 in Patients withMild Cognitive Impairment Due to Alzheimer*s Disease or Mild Alzheimer's Disease

A Placebo-controlled, Double-blind, Parallel-group, Bayesian Adaptive Randomization Design and Dose Regimen-finding Study to Evaluate Safety, Tolerability, and Efficacy of BAN2401 in Subjects With Early Alzheimer*s Disease

Objectives:Primary Objectives:1. To evaluate the efficacy of BAN2401 compared to placebo by establishing the ED90 (as defined in theprotocol) for BAN2401 on the derived Composite Clinical Score at 12 months of treatment in subjects withEarly…

A randomized, double-blind, placebo-controlled, parallel group study to evaluate the efficacy and safety of AFQ056 in adolescent patients with Fragile X Syndrome

The purpose of the study is to assess the efficacy and safety in adolescents of AFQ056 in treating behavioral symptoms characteristic of patients with FXS. Data from this study will be used to support the registration of AFQ056 in this population…

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 Phase 2b, double-blind, randomized, placebo-controlled study of RVT-101 in subjects with dementia with Lewy bodies (DLB).

Primary• To assess the effects of RVT-101 versus placebo on global function as measured by the Clinician*s Interview-Based Impression of Change Plus Caregiver Input (CIBIC+) after 24 weeks of treatment• To assess the effects of RVT-101 versus…

Bumetanide to Ameliorate Tuberous Sclerosis Complex Hyperexcitable Behaviors

Primary aim: to confirm that treatment with bumetanide improves daily life functioning and reduces behavioral symptoms related to hyperexcitability in children and adolescents with TSC.Secondary aim: to identify neurophysiological and cognitive…

A Long-Term Extension Study of the Safety and Tolerability of RVT-101 in Subjects with Dementia with Lewy Bodies (DLB)

To assess the long-term safety and tolerability of 35 mg and 70 mg RVT-101 in subjects with DLB

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…

Bumetanide for the Autism Spectrum Clinical Effectiveness Trial

The primary objective of the study is to test the effectiveness of bumetanide across the whole spectrum of sensory processing disorders within ASD, ADHD and/or epilepsy. We also want to determine which subgroups (high/low IQ, comorbidities or not)…

Randomized, double-blind, placebo-controlled, two way crossover, single centre study evaluating the acute and chronic effect of clonazepam on cognitive tests and patient-reported outcome measures in patients with ARID1B-related intellectual disability

* To test the hypothesis that clonazepam administration has acute beneficial effects compared to placebo on body sway, adaptive tracking, smooth eye pursuit, tapping frequency and the animal fluency test.* To test the hypothesis that multiple-doses…

A Double-Blind, Placebo-Controlled Proof-of-Concept Study of a Selective p38 MAP Kinase Alpha Inhibitor, Neflamapimod, Administered for 24 Weeks in Subjects with Mild Alzheimer*s Disease.

The primary objective is to evaluate the effects of administration of neflamapimod (VX-745) for 24-weeks on immediate and delayed recall aspects of episodic memory, as assessed by the Hopkins Verbal Learning Test * Revised (HVLT-R) in subjects with…

A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Pharmacodynamics and Pharmacokinetics of TW001 in Alzheimer Patients

Primary objective:The main objective of the present trial are:• to evaluate the effect of TW001 on oxidative stress biomarkers• to evaluate the safety of TW001 in patients with Alzheimer*s DiseaseSecondary objective:The secondary objective of the…

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…

A randomized, placebo-controlled, double-blind, parallel-group Phase 2a exploratory study with placebo run-in to investigate PK/PD effects, safety, tolerability and pharmacokinetics of REM0046127 oral suspension compared with placebo in subjects with mild to moderate Alzheimer's disease

• To assess the tolerability and safety of the investigational medicinal product

A 12-month, open-label, multicenter, randomized, safety, efficacy, pharmacokinetic (PK) and pharmacodynamic (PD) study of two regimens of anti-CD40 monoclonal antibody, CFZ533 vs. standard of care control, in adult de novo liver transplant recipients with a 12-month additional follow-up and a long-term extension (CONTRAIL I).

This study will allow the assessment of the ability of CFZ533 to replace calcineurin inhibitors (CNIs) in terms of anti-rejection efficacy, while providing potentially better renal function with an expected similar safety and tolerability profile.…

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…