Clinical trials: CHILD Syndrome
CTNNB1 Gene Mutation
Genotype-phenotype Correlations in Children and Adults With CTNNB1 Mutation
The aim of this study is to explore and define the correlations between genotype and phenotype in patients with CTNNB1 mutations as well as monitor and survey the natural history of the CTNNB1 syndrome.
- Trial name
- Gen-Phe CTNNB1 (NCT04812119)
- Testing
- The Modified Checklist for Autism in Toddlers, Revised with Follow-Up (M-CHAT-R/F), The Autism Spectrum Quotient-Children's Version (AQ-Child), The Autism-Spectrum Quotient (AQ)-Adolescent Version (AQ-Adolescent)
- Where
- 3 sites in Australia, Slovenia
- Volunteers
- 100 people
- Sponsor
- University Medical Centre Ljubljana
Generalized Myasthenia Gravis
Evaluating the Pharmacokinetics, Pharmacodynamics, and Safety of Efgartigimod Administered Intravenously in Children With Generalized Myasthenia Gravis
The purpose of this trial is to investigate the PK, PD, safety, and activity of efgartigimod IV in children and adolescents aged from 2 to less than 18 years of age with gMG.
- Trial name
- ADAPT Jr (NCT04833894)
- Testing
- Efgartigimod IV
- Where
- 25 sites in United States, Austria and 10 more
- Volunteers
- 15 people
- Sponsor
- argenx
Neurofibromatosis Type 1, Recurrent Childhood Brain Stem Glioma
Pomalidomide in Treating Younger Patients With Recurrent, Progressive, or Refractory Central Nervous System Tumors
This phase I trial studies the side effects and best dose of pomalidomide in treating younger patients with tumors of the brain or spine (central nervous system) that have come back or are continuing to grow.
- Conditions
- Neurofibromatosis Type 1, Recurrent Childhood Brain Stem Glioma, Recurrent Childhood Visual Pathway Glioma, Recurrent Primary Central Nervous System Neoplasm
- Trial name
- NCT02415153
- Testing
- Laboratory Biomarker Analysis, Pharmacological Study, Pomalidomide
- Where
- 12 sites in United States
- Volunteers
- 29 people
- Sponsor
- National Cancer Institute (NCI)
Congenital Disease
Kids Intestinal Dysfunction in Congenital Heart Disease: the Kind Heart Study
Approximately 40,000 infants are born each year in the United States with congenital heart defects (CHD), and heart defects are the leading cause of birth defect-related deaths in the United States.
- Trial name
- NCT02824705
- Testing
- Congenital Heart Disease Surgery
- Where
- United States
- Volunteers
- 53 people
- Sponsor
- University of Arizona
Acquired Immunodeficiency Syndrome, Tuberculosis
Pharmacokinetics of Lopinavir/Ritonavir Superboosting in Infants and Young Children Co-infected With HIV and TB
The purpose of this study is to determine the lopinavir levels in blood of HIV and TB infected children (3-15kg) when given lopinavir/ritonavir in a 1:1 ratio with rifampicin containing TB regimen and its safety.
- Trial name
- NCT02348177
- Testing
- lopinavir with ritonavir in 1:1 ratio, Lopinavir/ritonavir 4:1
- Where
- 5 sites in South Africa
- Volunteers
- 96 people
- Sponsor
- Drugs for Neglected Diseases
Growth Hormone Deficiency, Turner Syndrome
Long-term Safety and Effectiveness of Growth Hormone With GHD, TS, CRF, SGA , ISS and PWS in Children
The purpose of this study is to evaluate the long-term safety and effectiveness of growth hormone (Eutropin Inj./Eutropin plus Inj.) treatment with GHD (Growth Hormone Deficiency), TS (Turner Syndrome),CRF (Chronic Renal…
- Conditions
- Growth Hormone Deficiency, Turner Syndrome, Chronic Renal Failure, Small for Gestational Age
- Trial name
- LGS (NCT01604395)
- Where
- South Korea
- Volunteers
- 6,000 people
- Sponsor
- LG Chem
Autism Spectrum Disorder, Developmental Delay
A Salivary miRNA Diagnostic Test for Autism
The goal of this study is to validate a panel of miRNAs that distinguish children with autism spectrum disorder (ASD) from their non-ASD peers with a positive MCHAT-R.
- Trial name
- NCT02832557
- Testing
- Salivary collection, Vineland Adaptive Behavior Scale-III Assessment, Medical history questionaire
- Where
- United States
- Volunteers
- 998 people, healthy volunteers welcome
- Sponsor
- State University of New York - Upstate Medical University
Congenital Heart Defect, Hypoplastic Left Heart Syndrome
Physical Activity Promotion in Children and Adolescents With Single Ventricle Physiology (MedBike)
Single ventricular (SV) heart was a uniformly fatal condition before the advances in surgical treatment in 1980. In the present era, 5-year survival rate with SV is 75%, with some centers, including the Stollery Children's…
- Conditions
- Congenital Heart Defect, Hypoplastic Left Heart Syndrome, Physical Activity
- Trial name
- MedBike (NCT04056416)
- Testing
- MedBIKE
- Where
- Canada
- Volunteers
- 4 people
- Sponsor
- University of Alberta
Irritable Bowel Syndrome, Functional Abdominal Pain Syndrome
Hypnotherapy vs. Probiotics in Children With IBS and Functional Abdominal Pain
Current study aims to investigate the influence of gut-directed hypnotherapy and probiotic nutritional supplement (SymbioLact B) on gastrointestinal symptoms in children with functional abdominal pain or irritable bowel syndrome…
- Trial name
- NCT02613078
- Testing
- Gut-Directed Hypnotherapy, Nutritional Supplement, Self-Monitoring
- Where
- Germany
- Volunteers
- 60 people
- Sponsor
- University Hospital Tuebingen
Human Immunodeficiency Virus, Children
Pharmacokinetics of Once Daily Darunavir/Ritonavir in HIV-infected Children
Darunavir/ritonavir is one of the preferred antiretroviral agents as part of combination antiretroviral therapy for treatment of HIV-infected adults according to international guidelines.
- Trial name
- DAPHNE (NCT02285478)
- Where
- Netherlands
- Volunteers
- 12 people
- Sponsor
- Radboud University Medical Center
Celiac Disease in Children, Celiac Disease
Experiences of Disclosure in Adolescents With Coeliac Disease
Being diagnosed with coeliac disease means that individuals can no longer consume things containing gluten, such as bread or cake, or they can become very ill.
- Conditions
- Celiac Disease in Children, Celiac Disease, Celiac, Disclosure
- Trial name
- NCT04240340
- Testing
- semi-structured interview
- Where
- United Kingdom
- Volunteers
- 7 people
- Sponsor
- University of Oxford
Hereditary Angioedema, Pediatric
Berotralstat Treatment in Children With Hereditary Angioedema
The purpose of this study is to evaluate the pharmacokinetics (PK), safety and effectiveness of berotralstat to determine the appropriate weight-based dose for pediatric participants 2 to < 12 years of age for prophylactic…
- Trial name
- APeX-P (NCT05453968)
- Testing
- Berotralstat
- Where
- 15 sites in Austria, Canada and 8 more
- Volunteers
- 29 people
- Sponsor
- BioCryst Pharmaceuticals
CHILD Syndrome
Effects of Alexander Technique in Children With Upper Cross Syndrome.
This randomized clinical trial will involve 50 participants aged 13 to 16, recruited from Faisal Hospital Faisalabad and different schools nearby.
- Trial name
- NCT07248748
- Testing
- Alexander technique, Corrective exercises
- Where
- Pakistan
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Riphah International University
Nephrotic Syndrome in Children
Steroid Sensitive Nephrotic Syndrome in Children
Idiopathic nephrotic syndrome (INS) is one of the most common glomerular pathologies in children and corticosteroid therapy is its most effective treatment.
- Trial name
- NCT03878914
- Testing
- Corticosteroids
- Where
- 2 sites in United States, China
- Volunteers
- 34 people
- Sponsor
- Wayne State University
PANS, PANDAS
PEDI Pediatric Autoimmune Neuropsychiatric Disorder Associated With Streptococcus Study
The objective of the Pediatric Epidemiological Data and Incidence (PEDI) PANDAS study is to demonstrate the feasibility of enrollment and retention of subjects in a study to determine the incidence and natural history of children…
- Trial name
- PANDAS (NCT06811935)
- Where
- 2 sites in United States
- Volunteers
- 30 people
- Sponsor
- University of Wisconsin, Madison
Obstructive Sleep Apnea
Validity of a Home Respiratory Polygraphy for the Diagnosis of Obstructive Sleep Apnea in Children (DINISAS)
To assess the diagnostic validity and cost-effectiveness of a home respiratory polygraphy (HRP) performed at home compared with the standard polysomnography (PSG) in children with clinically suspected Obstructive Sleep Apnea…
- Trial name
- DINISAS (NCT03449550)
- Testing
- Home Respiratory Polygraphy (HRP), Standard Polysomnography (PSG)
- Where
- Spain
- Volunteers
- 320 people
- Sponsor
- Hospital Universitario Araba
Congenital Amegakaryocytic Thrombocytopenia, Diamond-blackfan Anemia
Busulfan, Antithymocyte Globulin, and Fludarabine Followed By a Donor Stem Cell Transplant in Treating Young Patients With Blood Disorders, Bone Marrow Disorders, Chronic Myelogenous Leukemia in First Chronic Phase, or Acute Myeloid Leukemia in First Remission
RATIONALE: Drugs used in chemotherapy, such as busulfan and fludarabine, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing.
- Conditions
- Congenital Amegakaryocytic Thrombocytopenia, Diamond-blackfan Anemia, Fanconi Anemia, Leukemia
- Trial name
- NCT00305708
- Testing
- anti-thymocyte globulin, busulfan, fludarabine phosphate
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- University of California, San Francisco
Fetal Alcohol Spectrum Disorder
Parenting Training for Children With FASD
Prenatal alcohol exposure can lead to a myriad of adverse developmental outcomes in children, and is the leading cause of mental disability in Canada.
- Trial name
- SF-FASD (NCT02210455)
- Testing
- Strongest Families FASD intervention, Psychoeducation
- Where
- Canada
- Volunteers
- 119 people
- Sponsor
- IWK Health Centre
HIV Infections
A Prospective Cohort of Children With HIV Infection
To evaluate clinical and immunological outcome of children treated with HAART.
- Trial name
- NCT00476606
- Testing
- Zidovudine, Stavudine, Didanosine, Lamivudine, Nevirapine, Efavirenz, LPV/r, Saquinavir, Indinavir, Ritonavir, Nelfinavir
- Where
- 3 sites in Thailand
- Volunteers
- 500 people
- Sponsor
- The HIV Netherlands Australia Thailand Research Collaboration
Ewing's Sarcoma, Osteosarcoma
Aflac ST0901 CHOANOME - Sirolimus in Solid Tumors
The best treatment for recurrent cancers or those that do not respond to therapies is not known. Typically, patients with these cancers receive a combination of cancer drugs (chemotherapy), surgery, or radiation therapy.
- Conditions
- Ewing's Sarcoma, Osteosarcoma, Astrocytoma, Atypical Teratoid/Rhabdoid Tumor
- Trial name
- Aflac ST0901 (NCT01331135)
- Testing
- sirolimus
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Emory University
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