Clinical trials: Brain Malformation, open to volunteers
Phenylketonuria (PKU)
A Study of MZE782 in Adults With PKU
The purpose of this study is to evaluate the safety, tolerability, and efficacy of MZE782 in adults with Phenylketonuria (PKU).
- Trial name
- CIPheR (NCT07694440)
- Testing
- MZE782
- Where
- 9 sites in United States, France and 1 more
- Volunteers
- 38 people
- Sponsor
- Maze Therapeutics
Acute Liver Failure, Wilson Disease
Daily Versus Alternate Day Plasma Exchange in Wilson Disease With Acute Liver Failure in Children
Wilson disease in children has a varied presentation. Wilson disease with acute liver failure is associated with very high mortality and morbidity.
- Trial name
- NCT06698991
- Testing
- Plasma Exchange, Standard Medical Treatment
- Where
- India
- Volunteers
- 20 people
- Sponsor
- Institute of Liver and Biliary Sciences, India
Gut-Brain Disorders, GERD Without Erosive Esophagitis
Neuroregulators for the Treatment of Diseases Associated With Esophageal-brain-gut Axis Communication Abnormalities.
Gastroesophageal reflux disease (GERD) poses a challenging medical condition to manage, with up to 40% of patients showing refractory to standard medical intervention, which usually begins with a proton pump inhibitor (PPI).
- Trial name
- NCT06778824
- Testing
- Takepron, Zoloft 50Mg Tablet, Imipramine Pill
- Where
- Taiwan
- Volunteers
- 610 people
- Sponsor
- Hualien Tzu Chi General Hospital
Friedreich Ataxia, Cardiomyopathies
A Study to Investigate the Safety, Tolerability, and Pharmacokinetics of ATI-105 in Healthy Adults
The goal of this clinical trial is to learn how ATI-105 administered to healthy participants at works in the body at different doses and with or without food.
- Conditions
- Friedreich Ataxia, Cardiomyopathies, Central Nervous System Diseases, Neuromuscular Diseases
- Trial name
- NCT07869654
- Testing
- ATI-105
- Where
- United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Adjuvia Therapeutics, Inc.
Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia
Palliative Care Needs of Children With Rare Diseases and Their Families
The palliative care needs of family caregivers of children with rare diseases and their children are largely unmet, including the need for support to prepare for future medical decision making.
- Conditions
- Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia, Asparagine Synthetase Deficiency, CHARGE Syndrome
- Trial name
- FACE-Rare (NCT06938542)
- Testing
- Family Centered pediatric palliative care for family caregivers of children with rare diseases.
- Where
- United States
- Volunteers
- 480 people, healthy volunteers welcome
- Sponsor
- Children's National Research Institute
Huntington Disease
A Randomized Study of SPK-10001 Gene Therapy in Participants With Huntington's Disease
The main goal of this study is to evaluate the safety, tolerability, and preliminary efficacy of SPK-10001 in participants with Huntington's Disease.
- Trial name
- NCT06826612
- Testing
- SPK-10001
- Where
- 5 sites in United States
- Volunteers
- 53 people
- Sponsor
- Hoffmann-La Roche
Brain Arteriovenous Malformations
In Situ Injection of Anti-angiogenics in Patients With Brain Arteriovenous Malformations Not Eligible for Exclusion Treatment
Brain arteriovenous malformations (bAVMs) are rare aggressive vascular malformations affecting mostly young and healthy adults. The most frequent revealing condition (in almost 50% of cases) is an intra-cerebral hemorrhage, which…
- Trial name
- BLITZ (NCT07075757)
- Testing
- Single in situ intra-arterial injection of bevacizumab
- Where
- 5 sites in France
- Volunteers
- 20 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Cardiovascular Abnormalities, Arterial Blood Pressure
Multicenter Study on Cardiovascular and Metabolic Complications in Patients With Biochemically Silent Pheochromocytomas and Paragangliomas
The aim of the study is to characterise the cardiovascular and metabolic complications pre- and post-surgery of patients with biochemically negative PPGL and to compare them with normal individuals and patients with secreting…
- Conditions
- Cardiovascular Abnormalities, Arterial Blood Pressure, Heart Rate, Congestive Heart Failure Chronic
- Trial name
- NCT07316075
- Where
- Greece
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Laikο General Hospital, Athens
Fabry Disease
A Study of Patients With Fabry Disease (US Specific)
This is an observational study to evaluate the effects of treatment on long-term effectiveness, safety, and health-related quality of life (HRQOL) in patients with Fabry disease, with a main focus on migalastat.
- Trial name
- NCT06906367
- Testing
- migalastat HCl, ERT
- Where
- 8 sites in United States
- Volunteers
- 450 people
- Sponsor
- Amicus Therapeutics
Cerebral Arteriovenous Malformation, Intracranial Arterial Diseases
Cerebral Arteriovenous Malformation With Aneurysm: Epidemiology, Clinical Features, and Prognosis
The study is a multicenter, prospective cohort study designed to assess the natural history, clinical safety and efficacy of various treatment strategies in patients with cerebral arteriovenous malformations (AVMs) and…
- Conditions
- Cerebral Arteriovenous Malformation, Intracranial Arterial Diseases, Intra Cerebral Hemorrhage, Intracranial Aneurysm
- Trial name
- NCT07353736
- Where
- China
- Volunteers
- 630 people
- Sponsor
- Duan Chuanzhi
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
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
Tourette Syndrome, Chronic Tic Disorder
Internet-based Behavior Therapy for Adults With Tourette Syndrome
This study protocol outlines a parallel-group, randomized controlled trial (RCT) designed to evaluate the effectiveness of Internet-delivered behavior therapy (BT) based on exposure with response prevention (ERP) for adults with…
- Trial name
- TICNET (NCT06271083)
- Testing
- Exposure with response prevention, Brief psychoeducation with general psychological support
- Where
- Sweden
- Volunteers
- 110 people
- Sponsor
- Karolinska Institutet
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
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
Huntington's Disease
Enroll -HD: A Prospective Registry Study in a Global Huntington's Disease Cohort
Enroll-HD is a longitudinal, observational, multinational study that integrates two former Huntington's disease (HD) registries-REGISTRY in Europe, and COHORT in North America and Australasia-while also expanding to include sites…
- Trial name
- NCT01574053
- Where
- 183 sites in United States, Argentina and 18 more
- Volunteers
- 35,000 people, healthy volunteers welcome
- Sponsor
- CHDI Foundation, Inc.
Gait Impairment, Cerebral Palsy (CP)
Clinical Investigation to Validate the Safety and Performance of the ABLE Kids Device in Pediatric Patients With Neurological Gait Disorders in a Clinical Setting
The primary objective of the study is to validate the safety and clinical performance of the ABLE Kids device in pediatric patients with neurological gait disorders or muscular weakness during a 10-session gait training program…
- Conditions
- Gait Impairment, Cerebral Palsy (CP), Neuromuscular Diseases (NMD), Spinal Cord Injuries (SCI)
- Trial name
- NCT07745712
- Testing
- ABLE Kids
- Where
- Spain
- Volunteers
- 15 people
- Sponsor
- ABLE Human Motion S.L.
Fabry Disease
Biomarkers and Cardiac Imaging Diagnostic Assay for Monitoring Patients With Fabry Disease
Fabry disease (FD) is a rare X-linked lysosomal storage disorder caused by deficient activity of the enzyme α-Gal A resulting from mutations affecting the GLA gene.
- Trial name
- NCT05698901
- Testing
- Agalsidase beta
- Where
- Taiwan
- Volunteers
- 150 people
- Sponsor
- Mackay Memorial Hospital
Intermediate AMD
A Clinical Trial Evaluating the Safety and Efficacy of a New Light Combination Therapy Addressing Intermediate AMD
The proposed clinical investigation wants primary to validate the safety of the innovative light therapy approach and in second priority provide insight and confirmations on therapeutic effect.
- Trial name
- retinaSEES-PoC (NCT06557369)
- Testing
- reSEES
- Where
- Italy
- Volunteers
- 30 people
- Sponsor
- Oculox Technologies SA
Migraine Disease, Patent Foramen Ovale
Migraine Headaches Elimination With Patent Foramen Ovale-Directed Therapy
Migraine is a common and often disabling condition, but its exact causes are not fully understood. Some people with migraines have a small opening in the heart wall called a patent foramen ovale (PFO).
- Trial name
- COMFORT-PFO (NCT07629635)
- Testing
- Prasugrel 5mg, Transcatheter Closure of Patent Foramen Ovale
- Where
- United States
- Volunteers
- 32 people
- Sponsor
- Columbia University