Clinical trials: Brain Malformation
Tourette Syndrome, Tourette's Disorder
Assessment of Children With Tic Onset in the Past 6 Months
The purpose of this research is to study why most children who have tics never develop Tourette syndrome but some do. In other words, we aim to find features that may predict whose tics will go away and whose tics will continue…
- Conditions
- Tourette Syndrome, Tourette's Disorder, Chronic Motor or Vocal Tic Disorder, Transient Tic Disorder
- Trial name
- NewTics (NCT01177774)
- Where
- United States
- Volunteers
- 99 people
- Sponsor
- Washington University School of Medicine
Malformation Brain
Neuroradiology Assesses Chiari Malformation's Impact on Airways, Cranial Base, and Sleep Disorders in Children.
The severity of sleep disorders in patients with Chiari malformations can vary. The investigators propose to establish a correlation between the severity of sleep-disordered breathing (SDB) and the quantitative neuroradiological…
- Trial name
- CHDSB (NCT05913297)
- Testing
- Magnetic Resonance, Polysomnographic evaluation
- Where
- Italy
- Volunteers
- 138 people, healthy volunteers welcome
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Infantile Globoid Cell Leukodystrophy
The Natural History of Infantile Globoid Cell Leukodystrophy
The purpose of this natural history study is to understand more about the progression of infantile Krabbe disease, a very rare genetic disease. There is very little published longitudinal data with only anecdotal cases.
- Trial name
- NCT00983879
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Zymenex A/S
Cerebral Palsy, Dystonia
Whole Body Vibration for Dystonia Cerebral Palsy
Health bones allow us to move and walk freely without pain. Physical activities have been shown to relate to healthy bone growth.
- Trial name
- WBVT (NCT03779308)
- Testing
- whole body vibration therapy
- Where
- Hong Kong
- Volunteers
- 10 people
- Sponsor
- The Hong Kong Polytechnic University
Bardet-Biedl Syndrome
Genetics and Clinical Characteristics of Bardet-Biedl Syndrome
This study will evaluate patients with a rare inherited condition called Bardet-Biedl syndrome . The purpose of the study is to learn more about the genetics and clinical characteristics of this disorder.
- Trial name
- NCT00078091
- Where
- United States
- Volunteers
- 194 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Niemann-Pick Diseases
Induced Pluripotent Stem Cells for Niemann Pick Disease
Establishment of individualized human cellular disease models based on induced pluripotent stem cells that reflect the broad heterogeneous phenotypic spectrum of Niemann Pick disease
- Trial name
- IPSNPABC (NCT03883750)
- Where
- Pakistan
- Volunteers
- 40 people
- Sponsor
- CENTOGENE GmbH Rostock
Mucopolysaccharidoses, Progressive Pseudorheumatoid Dysplasia
Pediatric Arthropathy Beyond Inflammation: Clinical Spectrum and Diagnostic Approach at Assiut University Children Hospital
To identify the most common underlying causes of non-inflammatory arthropathy in children presenting to Assiut University Children Hospital for through clinical evaluation, laboratory testing, and imaging, in order to improve…
- Conditions
- Mucopolysaccharidoses, Progressive Pseudorheumatoid Dysplasia, Farber Disease, Osteogenesis Imperfecta
- Trial name
- NCT07173010
- Volunteers
- 35 people
- Sponsor
- Assiut University
Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis
Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases
The purpose of this study is to determine a safe dose of BPX-501 gene modified T cells infused after a haplo-identical stem cell transplant to facilitate engraftment and the safety of Rimiducid (AP1903) on day 7 to prevent GVHD.
- Conditions
- Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome, Hemoglobinopathies
- Trial name
- NCT02231710
- Testing
- BPX-501 and Rimiducid
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Bellicum Pharmaceuticals
Lennox-Gastaut Syndrome
Phase III Randomized, Double-Blind, Placebo-Controlled Study of Oral Topiramate for Lennox-Gastaut Syndrome
OBJECTIVES: I. Evaluate the safety and efficacy of oral topiramate in patients with Lennox-Gastaut syndrome.
- Trial name
- NCT00004776
- Testing
- topiramate
- Volunteers
- 10 people
- Sponsor
- National Center for Research Resources (NCRR)
Huntington's Disease
HDClarity: a Multi-site Cerebrospinal Fluid Collection Initiative to Facilitate Therapeutic Development for Huntington's Disease
HDClarity will seek at least 2500 research participants at different stages of Huntington's disease (HD). The primary objective is to collect a high quality CSF sample for evaluation of biomarkers and pathways that will enable…
- Trial name
- HDClarity (NCT02855476)
- Where
- 40 sites in United States, Canada and 8 more
- Volunteers
- 2,500 people, healthy volunteers welcome
- Sponsor
- University College, London
Hypothalamic Obesity, Hypothalamic Tumor
Efficacy and Safety of Phentermine/Topiramate in Youth With Hypothalamic Obesity
Hypothalamic obesity (HO) refers to the substantial weight gain that often complicates hypothalamic brain tumors. Children with this treatment-recalcitrant form of obesity have excess rates of metabolic sequelae compared to…
- Conditions
- Hypothalamic Obesity, Hypothalamic Tumor, Craniopharyngioma
- Trial name
- NCT06299891
- Testing
- Phentermine / Topiramate Extended Release Oral Capsule [Qsymia]
- Where
- 2 sites in United States
- Volunteers
- 24 people
- Sponsor
- Seattle Children's Hospital
Tourette's Disorder
Omega-3 Fatty Acids in Tourette's Disorder
This study examines the safety and efficacy of omega-3 fatty acids (fish oil) for the treatment of Tourette's Disorder.
- Trial name
- NCT01315327
- Testing
- Omega-3 Fatty Acids, Olive oil
- Where
- United States
- Volunteers
- 33 people
- Sponsor
- NYU Langone Health
Lennox-Gastaut Syndrome
A Placebo-Controlled, Double-Blind Comparative Study of E2080 in Lennox-Gastaut Syndrome Patients (Study E2080-J081-304)
To confirm that the combination therapy of rufinamide has superior efficacy compared to placebo in patients with Lennox-Gastaut syndrome.
- Trial name
- NCT01146951
- Testing
- Rufinamide (E2080)
- Where
- 23 sites in Japan
- Volunteers
- 66 people
- Sponsor
- Eisai Limited
Gaucher Disease, Type I, Gaucher Disease, Type III
Gaucherite - A Study to Stratify Gaucher Disease
The purpose of this research is to review data already collected and to collect new data from adults and children in England with Gaucher Disease to determine clinical factors which predict severity and response to therapy of…
- Trial name
- Gaucherite (NCT03240653)
- Testing
- Stratified response to Enzyme Therapy
- Where
- 8 sites in United Kingdom
- Volunteers
- 250 people
- Sponsor
- Cambridge University Hospitals NHS Foundation Trust
Late Infantile Metachromatic Leukodystrophy
Long-term Metazym Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)
This is a single center, open-label study of patients with late infantile MLD. All patients were previous treated 26 weeks in the phase I trial (EudraCT number: 2006-005341-11, NCT00418561).
- Trial name
- NCT00633139
- Testing
- Recombinant human Arylsulfatase A (rhASA)
- Where
- Denmark
- Volunteers
- 13 people
- Sponsor
- Shire
Hyperhomocysteinemia, Thrombotic, Cbs-Related, Ischemic Stroke
Methylenetetrahydrofolate Reductase Deficiency in Ischemic Stroke
Adult onset epileptic seizures is rare and often associated with metabolic disorders, drugs and intracranial pathologies such as ischemia, hemorrhage or space-occupying lesions.
- Conditions
- Hyperhomocysteinemia, Thrombotic, Cbs-Related, Ischemic Stroke, Epilepsy
- Trial name
- NCT04287881
- Testing
- Methylenetetrahydrofolate reductase (MTHFR) deficiency
- Where
- 2 sites in Turkey (Türkiye)
- Volunteers
- 32 people
- Sponsor
- Istanbul Medeniyet University
Congenital Adrenal Hyperplasia, Hypogonadotropic Hypogonadism
Growing up With the Young Endocrine Support System (YESS!)
Transition from paediatric to adult endocrinology is a challenge for adolescents, families and doctors. Up to 25% of young adults with chronic endocrine disorders are lost to follow-up ('drop-out') once the young adult moves out…
- Conditions
- Congenital Adrenal Hyperplasia, Hypogonadotropic Hypogonadism, Growth Hormone Deficiency, Combined Pituitary Hormone Deficiency
- Trial name
- YESS (NCT04252001)
- Testing
- YESS! game, Snake '97, Transition-toolkit
- Where
- 7 sites in Belgium, Netherlands and 2 more
- Volunteers
- 160 people, healthy volunteers welcome
- Sponsor
- dr. Laura C. G. de Graaff-Herder
Hurler Syndrome (MPS I), Hurler-Scheie Syndrome
Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
The goal of this research study is to establish chimerism and avoid graft-versus-host-disease (GVHD) in patients with inherited metabolic disorders.
- Conditions
- Hurler Syndrome (MPS I), Hurler-Scheie Syndrome, Hunter Syndrome (MPS II), Sanfilippo Syndrome (MPS III)
- Trial name
- NCT01372228
- Testing
- hematopoietic stem cell infusion
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Talaris Therapeutics Inc.
Cerebral Palsy
Integrated Management of Pronation Deformity in Cerebral Palsy
Pronation deformity is a very common problem in children with cerebral palsy. This deformity is usually has neural and non-neural aspects.
- Trial name
- NCT03472261
- Testing
- Botulinum toxin type A, Upper extremity orthoses, Home exercise program
- Where
- Turkey (Türkiye)
- Volunteers
- 35 people
- Sponsor
- Kocaeli University
Preterm Birth, Ventilator Lung; Newborn
Cerebral Oxygenation in Preterm Neonates with Respiratory Support During Skin-to-skin Care on the First Day After Birth
This is a prospective observational pilot study investigating if skin-to-skin care (SSC) has an influence on cerebral oxygenation and perfusion measured with near-infrared spectroscopy (NIRS) compared to incubator care in…
- Trial name
- Kangaroo (NCT04865302)
- Testing
- SSC
- Where
- Austria
- Volunteers
- 43 people
- Sponsor
- Medical University of Graz
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