Clinical trials: Neurobehavioral Manifestation
Hunter Syndrome, Mucopolysaccharidosis II
Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy
The objective of this study is to determine the safety of once weekly dosing of idursulfase 0.5 mg/kg administered by intravenous (IV) infusion for male Hunter syndrome patients ≤ 5 years old.
- Conditions
- Hunter Syndrome, Mucopolysaccharidosis II, MPS II
- Trial name
- NCT00607386
- Testing
- Idursulfase
- Where
- 3 sites in Brazil, Poland and 1 more
- Volunteers
- 28 people
- Sponsor
- Shire
Brain Death
Non-randomized, Open-labeled, Interventional, Single Group, Proof of Concept Study With Multi-modality Approach in Cases of Brain Death Due to Traumatic Brain Injury Having Diffuse Axonal Injury
This is the proof of concept study with multi-modality approach (using intra-thecal bioactive peptides, stem cells, laser and transcranial IV laser and Median Nerve stimulation as adjuvants) in cases of brain death due to…
- Trial name
- NCT02742857
- Testing
- BQ-A Peptide Extract, Mesenchymal Stem Cells, Transcranial Laser Therapy
- Where
- India
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Bioquark Inc.
Arrhythmias, Cardiac, Palpitation
Ambulatory Leadless Electrocardiogram Recorder Trial Feasibility
The goal of this study is to compare the heart signals that are received by a new heart monitoring device called the HeartWatch to heart signals that are recorded by existing monitors used in cardiology clinics today.
- Conditions
- Arrhythmias, Cardiac, Palpitation, Syncope, Light Headedness
- Trial name
- ALERT (NCT05821465)
- Testing
- HeartWatch
- Where
- Canada
- Volunteers
- 144 people
- Sponsor
- HelpWear Inc.
X-Linked Adrenoleukodystrophy
Safety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With Adrenoleukodystrophy
This is a phase I/II, open label dose escalation study of multiple dose levels of NV1205 with a long-term treatment phase.
- Trial name
- NCT03196765
- Testing
- Sobetirome (NV1205)
- Where
- 13 sites in Argentina, Australia and 6 more
- Sponsor
- NeuroVia, Inc.
Mucopolysaccharidosis II
An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type II
Multicenter, open-label, single-group, designed to evaluate the long term efficacy and safety of study drug for the treatment of the MPS II.
- Trial name
- NCT04348136
- Testing
- JR-141
- Where
- 20 sites in Japan
- Volunteers
- 27 people
- Sponsor
- JCR Pharmaceuticals Co., Ltd.
Rett Syndrome
A Phase 3 Study of Fenfluramine Hydrochloride in Rett Syndrome
The purpose of this study is to investigate the efficacy of fenfluramine hydrochloride (HCl) versus placebo in study participants with Rett syndrome (RTT).
- Trial name
- NCT07503444
- Testing
- fenfluramine hydrochloride
- Where
- 40 sites in United States, Belgium and 10 more
- Volunteers
- 200 people
- Sponsor
- UCB BIOSCIENCES, Inc.
Developmental Delay, Skeletal Abnormalities
Biomarker for Sly Disease (MPS VII) (BioSly)
Development of a new MS-based biomarker for the early and sensitive diagnosis of Sly disease from blood (plasma)
- Conditions
- Developmental Delay, Skeletal Abnormalities, Hepatomegaly, Splenomegaly
- Trial name
- BioSly (NCT02298699)
- Where
- 5 sites in Egypt, Germany and 2 more
- Sponsor
- CENTOGENE GmbH Rostock
Trisomy 13, Trisomy 18
Prenatal Non-invasive Aneuploidy Test Utilizing SNPs Trial
This prospective blinded study will assess the diagnostic capability of an informatics enhanced SNP based technology (Parental Support) to identify pregnant women who are carrying a fetus with an aneuploidy from fee floating DNA…
- Conditions
- Trisomy 13, Trisomy 18, Trisomy 21, Aneuploidy
- Trial name
- PreNATUS (NCT01545674)
- Testing
- Blood Draw
- Where
- 29 sites in United States, Canada and 4 more
- Volunteers
- 937 people, healthy volunteers welcome
- Sponsor
- Natera, Inc.
Prader-Willi Syndrome
An Open-Label Study of DCCR Tablet in Patients With PWS
The primary objective of this study is to monitor the long-term safety of DCCR in PWS patients.
- Trial name
- NCT04086810
- Testing
- DCCR
- Sponsor
- Soleno Therapeutics, Inc.
Aneuploidy, Trisomy 21
Non-Invasive Chromosomal Evaluation of Trisomy Study
This study is being conducted to provide clinically annotated samples to support continued improvements in the Ariosa Test content, methodology, specimen processing and quality control.
- Conditions
- Aneuploidy, Trisomy 21, Trisomy 18, Trisomy 13
- Trial name
- NICHE (NCT02201862)
- Where
- 3 sites in United States
- Volunteers
- 2,000 people, healthy volunteers welcome
- Sponsor
- Cindy Cisneros
Pyruvate Dehydrogenase Complex Deficiency
Expanded Access Treatment Protocol With DCA for Patients With PDCD
Expanded Access (EA) will provide a transition to continue therapy for those patients who are currently in the open label extension of the Phase III study, SL 1009-01, while also allowing new patients diagnosed with PDCD who meet…
- Trial name
- NCT06931262
- Testing
- Dichloroacetate (DCA)
- Sponsor
- Saol Therapeutics Inc
Postoperative Pain
A Phase 3 Study to Evaluate the Safety and Efficacy of SKY0402 in Subjects Undergoing Total Knee Arthroplasty
The primary objective is to demonstrate that SKY0402 is superior when compared to bupivacaine HCl in the management of postoperative pain for patients undergoing total knee arthroplasty.
- Trial name
- TKA (NCT00745290)
- Testing
- Bupivacaine HCl, SKY0402
- Where
- 19 sites in United States
- Volunteers
- 245 people
- Sponsor
- Pacira Pharmaceuticals, Inc
Down Syndrome, Aneuploidy
Noninvasive Screening for Affected Pregnancies: Assay Development & Optimization in Affected Pregnancies
To collect samples for the purpose of developing and optimizing an in vitro noninvasive prenatal diagnostic (NIPD) test. The NIPD test employs circulating cell free (ccff) DNA extracted from whole blood samples collected from…
- Trial name
- CHARMM-AP (NCT01052688)
- Testing
- Maternal blood draw of 30 to 50ml
- Where
- 10 sites in United States
- Volunteers
- 150 people
- Sponsor
- Sequenom, Inc.
Mucopolysaccharidosis II
A Study of JR-141 in Patients With Mucopolysaccharidosis II (BR21)
A Phase II open-label, randomized, parallel group, 2 sites (Brazil), designed to evaluate the safety and efficacy of 3 doses of study drug for the treatment of the MPS II.
- Trial name
- NCT03359213
- Testing
- JR-141
- Where
- 2 sites in Brazil
- Volunteers
- 18 people
- Sponsor
- JCR Pharmaceuticals Co., Ltd.
Disorientation, Cognitive Impairment
Evaluation of Safe Use of SECURIDRAP® SELFIA®
Interventional, multicenter, prospective and non-comparative clinical investigation carried out in 9 French establishments in order to assess the safety of the SÉCURIDRAP® SELFIA® bedding by mesasuring all the adverse events…
- Conditions
- Disorientation, Cognitive Impairment, Behavior Disorders
- Trial name
- SECURIDRAP (NCT05510401)
- Testing
- SECURIDRAP® SELFIA®
- Where
- 10 sites in France
- Volunteers
- 108 people
- Sponsor
- Groupe Mulliez-Flory
Oral Health, Intellectual Disability
Supervised Brushing Programme for Intellectual Disabled Students
Intellectual disabled persons faced more dental problems. According to a medical review paper of international and local studies, this group has poorer dental hygiene, more severe gum disease and more untreated caries.
- Conditions
- Oral Health, Intellectual Disability, Randomized Controlled Trial
- Trial name
- NCT03234231
- Testing
- 3-month supervised tooth brushing programme, oral health education talk
- Volunteers
- 602 people
- Sponsor
- MD Equation
Bipolar I Disorder, Manic or Mixed
Evaluate the Safety of Depakote Extended Release Tablets in the Treatment of Mania Associated With Bipolar Disorder in Children and Adolescents
To determine if long-term treatment, up to 6 months, with Depakote ER is safe and effective in the treatment of bipolar I disorder in children and adolescents.
- Trial name
- NCT00195767
- Testing
- divalproex sodium
- Where
- United States
- Volunteers
- 150 people
- Sponsor
- Abbott
Communication Disorders
Adaptive and Individualized AAC
This project will test the feasibility of developing a smart augmentative or alternative communication (AAC) system that is effective in delivering communication capabilities that automatically adapt to the users' physical…
- Trial name
- NCT04247828
- Testing
- Experimental AAC, Generic AAC
- Where
- 2 sites in United States
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Altec Inc.
Healthy Volunteer, Down Syndrome
A Molecular and Functional Brain Imaging Study in Individuals With Down Syndrome and Healthy Controls Following Single Dose RG1662
This single-center, single-blind, placebo-controlled, parallel-group study with crossover component will evaluate the GABAAalpha5 receptor expression, occupancy and functional connectivity in the brains of individuals with Down…
- Trial name
- NCT01667367
- Testing
- RG1662
- Where
- United Kingdom
- Volunteers
- 13 people, healthy volunteers welcome
- Sponsor
- Hoffmann-La Roche
Pain, Post-operative
Placebo-Controlled Evaluation of Intranasal Dexmedetomidine for Postoperative Analgesia Following Bunionectomy
The primary objective of this study is to evaluate the analgesic efficacy of two dose levels of DEX-IN compared with placebo, using the summed pain intensity difference over the first 48 hours (SPID48) in subjects with acute…
- Trial name
- NCT02169336
- Testing
- Intranasal Dexmedetomidine
- Where
- 3 sites in United States
- Volunteers
- 95 people
- Sponsor
- Baudax Bio