Clinical trials: Brain Malformation
Hereditary Ataxia
Clinical and Molecular Correlations in Spinocerebellar Ataxia Type 10 (SCA10)
OBJECTIVES: I. Clinically evaluate members from families with a dominantly inherited ataxia and collect blood, skin and muscle samples for detailed molecular studies. II.
- Trial name
- NCT00004306
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Office of Rare Diseases (ORD)
Tourette Syndrome
Gilles de la Tourette Syndrome (TS) and Seasonality
This retrospective and prospective observational study intends to evaluate the correlation between seasons and the variation of the syndromic structure of the Tourette.
- Trial name
- NCT04076852
- Testing
- Administration of questionnaires
- Where
- Italy
- Volunteers
- 39 people
- Sponsor
- I.R.C.C.S Ospedale Galeazzi-Sant'Ambrogio
Upper Extremity Impairments, Cerebral Palsy (CP)
Obi Medical Robot: Evaluating Effectiveness Related to Usability
This mixed-methods usability study evaluates the effectiveness of the Obi Gen 3 robotic feeding device in meeting user needs among providers, caregivers, and patients.
- Conditions
- Upper Extremity Impairments, Cerebral Palsy (CP), ALS - Amyotrophic Lateral Sclerosis, Rett Syndrome
- Trial name
- NCT07151950
- Testing
- One-week trial of Obi Medical device, which provides robotic assistance with feeding to persons with upper extremity impairments who are unable to self-feed in the traditional manner using utensils
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Desin LLC
Tourette Disorder, Chronic Tic Disorder
Augmentation of Brief Habit Reversal Training With D-cycloserine or Placebo
Expert reviews and practice parameter papers recommend behavior therapy as a first-line intervention for youth with chronic tic disorders (CTDs) with mild-to-moderate tic severity.
- Trial name
- DCS+HRT (NCT02582515)
- Testing
- D-cycloserine
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- University of California, Los Angeles
Fetal Growth Retardation, Fetal Neurodevelopmental Disorder
Evaluation of Anterior Middle Brain Structures With Cerebrovascular Flow in Fetuses With Fetal Growth Restriction
The study will be conducted to examine the effects of abnormal growth pattern and cerebrovascular blood flow measurements on fetal frontal lobe development with the advancing ultrasound technology.
- Trial name
- NCT06215690
- Testing
- Ultrasound measurements
- Where
- Turkey (Türkiye)
- Volunteers
- 90 people, healthy volunteers welcome
- Sponsor
- Ankara Etlik City Hospital
Lennox Gastaut Syndrome
An Extended Access Program (EAP) for Participants Who Have Completed Rufinamide Study E2080-G000-303
This is an extended access study for participants who have completed Rufinamide Study E2080-G000-303 to continue to have access to rufinamide until it becomes commercially available in Poland or until no participants remain in…
- Trial name
- NCT03778424
- Testing
- Rufinamide
- Where
- 2 sites in Poland
- Sponsor
- Eisai Inc.
Fabry Disease
Natural History and Structural Functional Relationships in Fabry Renal Disease Treatment Outcomes(Changes)in Fabry Renal Disease Study
The investigators will perform a study with two major components. The first is a natural history study of untreated Fabry patients.
- Trial name
- LDN6702 (NCT01581424)
- Where
- 2 sites in United States
- Volunteers
- 50 people
- Sponsor
- University of Minnesota
Autism Spectrum Disorder, Cerebral Palsy
READYorNot[TM] Brain-Based Disabilities Trial
The purpose of this study is to find out if there is a benefit to using the MyREADY Transition[TM] BBD App for brain-based disabilities, compared to not using it.
- Conditions
- Autism Spectrum Disorder, Cerebral Palsy, Epilepsy, Fetal Alcohol Spectrum Disorders
- Trial name
- NCT03852550
- Testing
- e-health application
- Where
- 9 sites in Canada
- Volunteers
- 52 people
- Sponsor
- McMaster University
Tuberous Sclerosis Complex, Epilepsy
Stopping TSC Onset and Progression 2: Epilepsy Prevention in TSC Infants
This phase I/II clinical trial is an open-label clinical trial design to verify safety and dosing for TAVT-18 (sirolimus) powder for oral solution in TSC infants (N=5).
- Trial name
- STOP2 (NCT04595513)
- Testing
- TAVT-18 (sirolimus)
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Epilepsy, EEG With Periodic Abnormalities
Clinical Validation of zEEG, a Wireless EEG Recording System for Pediatric and Adult Patients With Epilepsy
The purpose of this study is to compare the standard clinical electroencephalography (EEG) device with a new portable wireless EEG device, further referred to as zEEG, made by ZETO®.
- Conditions
- Epilepsy, EEG With Periodic Abnormalities, EEG With Abnormally Slow Frequencies, EEG; Paroxysms, Occipital, Epilepsy of Childhood
- Trial name
- NCT05123469
- Testing
- Electroencephalography
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- University of Texas at Austin
Gaucher Disease Type 3
Study to Evaluate the Efficacy and Safety of Nizubaglustat (AZ-3102) in Patients With Gaucher Disease Type 3 (GD3)
A 96-week, open-label, Phase 2 study to evaluate the efficacy and safety of Nizubaglustat (AZ-3102) in patients with Gaucher disease type 3 (GD3).
- Trial name
- NCT07715084
- Testing
- AZ-3102
- Where
- 7 sites in India, Pakistan and 1 more
- Volunteers
- 20 people
- Sponsor
- Azafaros B.V.
PolG, Primary Mitochondrial Myopathy
Mitochondrial Video Assessment Source Material Study
Source material collection of videos to develop the Mitochondrial Video Assessment (MVA) for patients with mitochondrial myopathies.
- Trial name
- NCT06180421
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- The Emmes Company, LLC
Tourette Syndrome, Tic Disorder, Chronic Motor or Vocal
Open-label MNS for Tourette Syndrome
A recent report (Morera Maiquez et al 2020) described reduced tic severity in people with Tourette syndrome during 1-minute epochs of median nerve stimulation (MNS) at 10 Hz.
- Trial name
- NCT05016765
- Testing
- Active, self-directed electrical stimulation of the median nerve
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Washington University School of Medicine
Tourette Syndrome, Chronic Tic Disorder
VoIP Delivered Behavior Therapy for Tourette Syndrome
The purpose of this research is to examine the preliminary efficacy, feasibility, and acceptability of Voice over Internet Protocol delivered behavior therapy for Tourette Syndrome through in a randomized waitlist-controlled…
- Trial name
- NCT02247206
- Testing
- Behavior Therapy for Tics (CBIT)
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- University of Wisconsin, Milwaukee
Tourette's Syndrome
Ecopipam Treatment of Tourette's Syndrome in Subjects 7-17 Years
Tourette's Syndrome is a neurological disease characterized by motor and vocal tics. It has been hypothesized that abnormal interactions of dopamine with its receptors may cause the tics.
- Trial name
- NCT02102698
- Testing
- Ecopipam
- Where
- 11 sites in United States
- Volunteers
- 40 people
- Sponsor
- Emalex Biosciences Inc.
Hypotonia-Cystinuria Syndrome, Muscle Hypotonia
PREPL in Health and Disease
Evaluation of PREPL activity in healthy controls and known or possible PREPL deficient patients
- Conditions
- Hypotonia-Cystinuria Syndrome, Muscle Hypotonia, Healthy Volunteers, Dwarfism, Growth Hormone Deficiency
- Trial name
- PHD (NCT02263781)
- Testing
- Blood draw, muscle biopsy
- Where
- Belgium
- Volunteers
- 125 people, healthy volunteers welcome
- Sponsor
- Universitaire Ziekenhuizen KU Leuven
Tuberous Sclerosis Complex, Epilepsy
The Effectiveness and Safety of Resective Epilepsy Surgery for TRE
A prospective cohort studies to identify clinical seizure control, cognitive changes, and safety in resective epilepsy surgery in patients with TSC-related drug-resistant epilepsy.
- Trial name
- NCT04198181
- Testing
- Surgery
- Where
- China
- Volunteers
- 200 people
- Sponsor
- Beijing Children's Hospital
Malignant Tumor, Benign Neoplasm
Twenty-five Years of Advanced RadioTherapy at San Raffaele Hospital
This retrospective observational study aims to evaluate the efficacy and toxicity of advanced radiotherapy (ART) techniques, including intensity-modulated radiotherapy (IMRT), image-guided radiotherapy (IGRT), and stereotactic…
- Conditions
- Malignant Tumor, Benign Neoplasm, Parkinson Disease, Trigeminal Neuralgia
- Trial name
- 25-ART-HSR (NCT07894900)
- Testing
- Advanced radiotherapy techniques
- Where
- Italy
- Volunteers
- 10,000 people
- Sponsor
- IRCCS San Raffaele
Urea Cycle Disorder, Ornithine Transcarbamylase Deficiency
Liver Disease in Urea Cycle Disorders
This is a multi-center, cross-sectional study to assess risk for liver fibrosis and hepatic injury in individuals with urea cycle disorders (UCDs) using serum biomarkers, Fibroscan, and MRE.
- Conditions
- Urea Cycle Disorder, Ornithine Transcarbamylase Deficiency, Citrullinemia 1, ARGI Deficiency
- Trial name
- NCT04612764
- Where
- 5 sites in United States
- Volunteers
- 62 people
- Sponsor
- Baylor College of Medicine
Phenylketonuria
Nutritional and Neurotransmitter Changes in PKU Subjects on BH4
HYPOTHESIS: The investigators hypothesize that KuvanTM therapy could influence nutritional and body composition parameters and neurotransmitter concentrations in pediatric and adult PKU subjects.
- Trial name
- BH4&PKU (NCT00688844)
- Testing
- KuvanTM Therapy
- Where
- 2 sites in United States
- Volunteers
- 58 people, healthy volunteers welcome
- Sponsor
- Emory University
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