Clinical trials: Symptom Cluster, open to volunteers
Chronic Graft-versus-host-disease
A Study of Axatilimab at 3 Different Doses in Participants With Chronic Graft Versus Host Disease (cGVHD)
This is a Phase 2 study to evaluate the efficacy, safety, and tolerability of axatilimab at 3 different dose levels in participants with recurrent or refractory active chronic graft versus host disease (cGVHD) who have received…
- Trial name
- AGAVE-201 (NCT04710576)
- Testing
- Axatilimab
- Where
- 121 sites in United States, Australia and 14 more
- Volunteers
- 296 people
- Sponsor
- Syndax Pharmaceuticals
Angelman Syndrome
Use of Eye Tracking to Study Social Perception Abnormalities in Children With Angelman Syndrome
Angelman syndrome (AS) is a rare neurogenetic disorder that affects approximately 1 in 15,000 children - approximately 500,000 people worldwide.
- Trial name
- EYEANGEL (NCT06737718)
- Testing
- Eye tracking, Data collection from patients' medical files, questionnaires
- Where
- France
- Volunteers
- 60 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Migraine in Children, Complex Regional Pain Syndromes
Supraspinal Processing of Sensory Aspects of Pain
The goal of this basic science study is to learn about the brain mechanisms of chronic pain across different chronic pain syndromes in pediatric patients.
- Conditions
- Migraine in Children, Complex Regional Pain Syndromes, Musculoskeletal Pain, Functional Abdominal Pain Syndrome
- Trial name
- SCP (NCT05814497)
- Testing
- Multisensory Task, Graphesthesia, Divided attention
- Where
- United States
- Volunteers
- 255 people, healthy volunteers welcome
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Fragile X Syndrome, Autism Spectrum Disorder
Alpha Auditory Entrainment for Cognitive Enhancement and Sensory Hypersensitivity in Youth With Developmental Disorders
Fragile X Syndrome (FXS) is a complex neurodevelopmental disorder caused by a mutation on the X chromosome. Scientists have investigated FXS extensively in both humans and animals.
- Conditions
- Fragile X Syndrome, Autism Spectrum Disorder, Autistic Disorder, Asperger Syndrome
- Trial name
- ENTRAIN (NCT06227780)
- Testing
- Alpha Auditory Entrainment
- Where
- United States
- Volunteers
- 180 people, healthy volunteers welcome
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Myositis, Dermatomyositis
Environmental Risk Factors for the Anti-synthetase Syndrome
Background: Like other complex diseases, autoimmune diseases are the result of numerous causes, including genetic and environmental factors.
- Conditions
- Myositis, Dermatomyositis, Polymyositis, Juvenile Dermatomyositis
- Trial name
- NCT01276470
- Where
- 8 sites in United States
- Volunteers
- 580 people, healthy volunteers welcome
- Sponsor
- National Institute of Environmental Health Sciences (NIEHS)
Chronic Pain, Chronic Pain Syndrome
Interoceptive Awareness and Function in Adolescents With Chronic Pain
The main objectives of this study are to compare the levels of interoceptive awareness and self-reported function pre and post completion of an occupational therapy treatment plan.
- Conditions
- Chronic Pain, Chronic Pain Syndrome, Pain, Chronic
- Trial name
- NCT07325110
- Testing
- Occupational Therapy
- Where
- United States
- Volunteers
- 49 people
- Sponsor
- Connecticut Children's Medical Center
Neonatal Respiratory Distress Syndrome, PreTerm Neonate
MINImising Total Radiation EXposure in Preterm Infants
Being born too early (preterm birth) is the leading cause of death in children world-wide. In Australia, 97% of very preterm babies who are admitted to Neonatal Intensive Care Units need breathing support after birth to survive.
- Trial name
- MINI T-Rex (NCT06975189)
- Testing
- Lung ultrasound group, Standard Care
- Where
- Australia
- Volunteers
- 180 people
- Sponsor
- Western Health, Australia
Angelman Syndrome
Angelman Syndrome Natural History Study
The goal of this study is to conduct a prospective, longitudinal natural history study of children and adults with Angelman Syndrome using investigator-observed and parent-reported outcome measures to obtain data that will be…
- Trial name
- NCT04507997
- Where
- 11 sites in United States, Canada
- Volunteers
- 300 people
- Sponsor
- Boston Children's Hospital
Absence Epilepsy, Ketogenic Dieting
Ketogenic Diet for New-Onset Absence Epilepsy
The ketogenic diet is a medical therapy for epilepsy that is used nearly predominantly for refractory epilepsy (after 2-3 drugs have been tried and failed).
- Conditions
- Absence Epilepsy, Ketogenic Dieting, Epilepsy, Absence
- Trial name
- NCT04274179
- Testing
- Modified Atkins Diet, Absence epilepsy medications
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Johns Hopkins University
Medical Condition Requiring Extracorporeal Organ Support
The University of Freiburg Medical Center Extracorporeal Organ Support Device Registry
A prospective registry collecting pseudonymized clinical and treatment data from all patients treated with extracorporeal organ support (i.e., VV ECMO, VA ECMO, ECPR, Impella, IABP, and others) on the wards of the Department of…
- Trial name
- NCT05811780
- Where
- Germany
- Volunteers
- 1,500 people
- Sponsor
- University Hospital Freiburg
Congenital Adrenal Hyperplasia, Classic Congenital Adrenal Hyperplasia
A Study in Pediatric Participants With Congenital Adrenal Hyperplasia (Balance-CAH)
The purpose of this study is to evaluate the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) of atumelnant treatment in pediatric participants with classic congenital adrenal hyperplasia (CAH).
- Trial name
- NCT07159841
- Testing
- Atumelnant
- Where
- 52 sites in United States, Argentina and 11 more
- Volunteers
- 153 people
- Sponsor
- Crinetics Pharmaceuticals Inc.
Epstein-Barr Virus Infections, Primary Immune Deficiency Disorder
EBV-specific Cytotoxic T-lymphocytes (CTLs) for Refractory EBV Infection
Related donor Epstein-Barr Virus (EBV) specific cytotoxic T cells (CTLs) manufactured with the Miltenyi CliniMACS Prodigy Cytokine Capture System will be administered in children, adolescents and young adults with refractory EBV…
- Trial name
- NCT03266653
- Testing
- cytotoxic t-lymphocytes
- Where
- 8 sites in United States
- Volunteers
- 20 people
- Sponsor
- New York Medical College
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
Shwachman-Diamond Syndrome, SDS
Shwachman-Diamond Syndrome Global Patient Survey and Partnering Platform
The Shwachman-Diamond Syndrome Global Patient Survey and Collaboration Program (SDS-GPS) is an opportunity for patients and their families - from anywhere in the world - to share their experience living with SDS via a safe…
- Conditions
- Shwachman-Diamond Syndrome, SDS, IBMF, Congenital Neutropenia
- Trial name
- SDS-GPS (NCT06999954)
- Where
- United States
- Volunteers
- 8,000 people
- Sponsor
- Shwachman-Diamond Syndrome Alliance Inc
Viral Infection, Hematopoietic Stem Cell Transplantation (HSCT)
Adoptive T Lymphocyte Administration for Chronic Norovirus Treatment in Immunocompromised Hosts
This is a Phase I dose-escalation study to evaluate the safety of norovirus -specific T-cell (NST) therapy for chronic norovirus infection in participants following hematopoietic stem cell transplantation (HSCT) or who are…
- Conditions
- Viral Infection, Hematopoietic Stem Cell Transplantation (HSCT), Primary Immunodeficiency Disorders (PID)
- Trial name
- ATLANTIC (NCT04691622)
- Testing
- Norovirus -specific T-cell (NST) therapy
- Where
- 3 sites in United States
- Volunteers
- 48 people
- Sponsor
- Children's National Research Institute
Down Syndrome
Rebound Therapy on Ventilatory Functions in Down Syndrome
The purpose of this study is to examine the effects of rebound therapy on ventilatory functions in children with Down Syndrome.
- Trial name
- NCT07425353
- Testing
- Chest Physical Therapy Protocol, Rebound Therapy Protocol
- Volunteers
- 40 people
- Sponsor
- Beni-Suef University
Multi-system Atrophy - Parkinsonian Type, Orthostatic Hypotension, Dysautonomic
Feasibility Study Assessing the Effect of Carbidopa/Levodopa Ratio on Orthostatic Hypotension in Multiple System Atrophy - Parkinsonian Type and Parkinson Disease.
This study is aimed at patients with multi-system atrophy - parkinsonian type (P-MSA) or Parkinson's disease (PD) receiving dopaminergic drugs and suffering from orthostatic hypotension (OH).
- Conditions
- Multi-system Atrophy - Parkinsonian Type, Orthostatic Hypotension, Dysautonomic, Parkinson Disease
- Trial name
- CARBIDOH (NCT06831500)
- Testing
- Administration of Carbidopa/levodopa
- Where
- 2 sites in Switzerland
- Volunteers
- 36 people
- Sponsor
- Julien Bally
Sepsis, Antibiotic Reaction
PK/PD of Ceftazidime Avitbatan Sodium in Children With Severe Infection
Ceftazidime avitbatan sodium is a broad-spectrum antibiotic with an antimicrobial spectrum covering resistant gram-negative bacteria. Its use in pediatric intensive care for severe infections is not unusual.
- Trial name
- NCT04961580
- Testing
- Ceftazidime Avitbatan Sodium
- Where
- China
- Volunteers
- 30 people
- Sponsor
- Children's Hospital of Fudan University
Fragile X Syndrome (FXS), Autism Spectrum Disorder
Group CBT in Adolescents With Fragile X Syndrome and in Adolescents With Autism Spectrum Disorder
Fragile X Syndrome (FXS) is a rare genetic syndrome, caused by a mutation in the FMR1 gene located on the X chromosome. It is considered the leading hereditary cause of intellectual disability (ID) and the primary cause of Autism…
- Trial name
- NCT06677866
- Testing
- Cognitive behavioral therapy, Waiting List
- Where
- Italy
- Volunteers
- 20 people
- Sponsor
- Bambino Gesù Hospital and Research Institute
Acute Respiratory Distress Syndrome in Children
Assessment of Airway Opening Pressure in Invasively Ventilated Children
Acute respiratory distress syndrome (ARDS) in children is associated with significant morbidity and mortality. Current studies seek to individualize the management of children by defining several phenotypes, based until now…
- Trial name
- AOP-KID (NCT07262541)
- Testing
- Measurement of airway opening pressure
- Where
- France
- Volunteers
- 50 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
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