Clinical trials: Metabolism Disorders
Urea Cycle Disorder
Observational Study That Will Collect Information on Patients With Urea Cycle Disorders (UCDs)
THRIVE is an observational study that will collect information on patients with UCDs. THRIVE will follow enrolled participants for up to 10 years.
- Trial name
- THRIVE (NCT01948427)
- Where
- United States
- Volunteers
- 203 people
- Sponsor
- Amgen
Infant, Obesity
The Intervention Nurses Start Infants Growing on Healthy Trajectories (INSIGHT) Study
This study will test an intervention program designed to provide developmentally appropriate guidance to parents of infants on responsive parenting and healthy lifestyle to see if that intervention will prevent rapid weight gain…
- Trial name
- INSIGHT (NCT01167270)
- Testing
- Child Safety Insights, Parenting Insights
- Where
- 2 sites in United States
- Volunteers
- 316 people, healthy volunteers welcome
- Sponsor
- Ian M. Paul, MD
Fanconi Anemia
FANCA Gene Transfer for Fanconi Anemia Using a High-safety, High-efficiency, Self-inactivating Lentiviral Vector
This is a Phase I/II clinical trial of gene therapy for treating Fanconi anemia using a self-inactivating lentiviral vector to functionally correct the defective gene.
- Trial name
- NCT03351868
- Testing
- Gene-modified autologous stem cells
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Shenzhen Geno-Immune Medical Institute
Healthy, Nervous System Disease
Functional and Metabolic Imaging Using Magnetic Resonance Imaging and Spectroscopy
Technical advances in magnetic resonance imaging (MRI) and magnetic resonance spectroscopy (MRS) have provided researchers with the opportunity to study changes of the central nervous system (CNS) and improve diagnosis and…
- Trial name
- NCT00001591
- Where
- United States
- Volunteers
- 450 people, healthy volunteers welcome
- Sponsor
- National Institutes of Health Clinical Center (CC)
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
GM1 Gangliosidosis, GM2 Gangliosidosis
Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2
This study is being conducted to better understand the natural course of GM1 gangliosidosis, GM2 gangliosidoses and Gaucher disease Type 2 (GD2).
- Conditions
- GM1 Gangliosidosis, GM2 Gangliosidosis, Gaucher Disease, Type 2, Tay-Sachs Disease
- Trial name
- RETRIEVE (NCT04470713)
- Where
- 17 sites in United States, Belgium and 8 more
- Volunteers
- 226 people
- Sponsor
- Idorsia Pharmaceuticals Ltd.
Celiac Disease, Eosinophilic Esophagitis
Prevalence of Eosinophilic Esophagitis in Children With Celiac Disease
Celiac disease (CD) and eosinophilic esophagitis (EE) are distinct diseases of the gastrointestinal tract with specific clinico-pathological characteristics.
- Trial name
- NCT02190513
- Volunteers
- 56 people
- Sponsor
- St. John Health System, Michigan
Malnutrition
Women's Work in Agriculture and Infant Nutrition
Background Over the last 10 years there has been an increase in the female agriculture labour force, in Pakistan, resulting in a feminisation of agriculture; which could have either a positive or negative impact on maternal and…
- Trial name
- WWAN (NCT03406182)
- Where
- 2 sites in Pakistan, United Kingdom
- Volunteers
- 1,161 people, healthy volunteers welcome
- Sponsor
- London School of Hygiene and Tropical Medicine
Iron Deficiency
Ferrous Sulfate Versus Iron Amino Acid Chelate
Iron deficiency and depleted levels of iron are the most prevalent nutritional deficiency and the leading cause of anemia in the world; this can occur at any age, but preschool children are at particular risk of developing it.
- Trial name
- NCT01412723
- Testing
- Ferrous sulfate as dietary supplementation, Iron amino acid chelated as dietary supplementation
- Where
- Colombia
- Volunteers
- 64 people
- Sponsor
- CES University
Rhabdomyolysis Linked to a Hereditary Disease of Metabolism
Evaluate the Effort Test as a Therapeutic Monitoring Tool in Acute Rhabdomyolyses
The prognosis of rhabdomyolyses related to hereditary diseases of metabolism is poor and treatments are only symptomatic. Rhabdomyolysis outbreaks are frequently precipitated by fever and fasting. They are unpredictable.
- Trial name
- EFFORHAB (NCT03802279)
- Testing
- Effort test, Functional tests on fibroblasts
- Where
- France
- Volunteers
- 27 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
MPS IIIB (Sanfilippo B Syndrome)
Evaluation of Blood Brain Barrier Integrity and Structural Abnormalities in MPS IIIB Patients Using Multimodal Magnetic Resonance Imaging
The purpose of the study is to characterize structural abnormalities in the brain and the integrity of the blood brain barrier in patients with mucopolysaccharidosis type IIIB (MPS IIIB).
- Trial name
- NCT02090179
- Where
- United Kingdom
- Volunteers
- 5 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Metabolic Cardiovascular Syndrome
Health Improvement After Pregnancy (HIP) Program Randomized Control Trial
Recent Canadian studies, public opinion polls and the Canadian Heart Health Strategy and Action Plan state that women's heart health is a key national priority; it should be addressed through improving heart health awareness and…
- Trial name
- HIP (NCT02077179)
- Testing
- HIP Program
- Where
- Canada
- Volunteers
- 43 people
- Sponsor
- Queen's University
Maturity-Onset Diabetes of the Young, Type 3, Maturity Onset Diabetes of the Young, Type 1
Switching From Insulin to Sulfonylurea in Diabetes Associated With Variants in MODY Genes
The purpose of this study is to switch from insulin to oral sulfonylurea in patients with apparent type 1 diabetes or maturity onset diabetes in the young that are insulin treated.
- Conditions
- Maturity-Onset Diabetes of the Young, Type 3, Maturity Onset Diabetes of the Young, Type 1, Childhood Diabetes Mellitus, Insulin-dependent Diabetes Mellitus
- Trial name
- SUtoChildT1D (NCT04239586)
- Testing
- Sulfonylurea
- Where
- Norway
- Volunteers
- 30 people
- Sponsor
- Haukeland University Hospital
Disability, Intellectual, Disability Psychological
The Role of Family and Individual Factors in Going Through Adolescence - Perspective of a Healthy Child.
The goal of this observational study is to investigate the specificity of the growing up process in young people with disabled siblings.
- Conditions
- Disability, Intellectual, Disability Psychological, Disability, Developmental, Diabetes
- Trial name
- NCT06156124
- Testing
- Psychological tests and psychosocial questionnaires
- Where
- Poland
- Volunteers
- 320 people, healthy volunteers welcome
- Sponsor
- University of Gdansk
Other Preterm Infants, Vitamin D Deficiency
The Effect of Vitamin D Administration to Premature Infants on Vitamin D Status and Respiratory Morbidity
This trial objective is to assess whether doubling the daily intake of vitamin D improves serum vitamin D levels and serves as primary prevention of respiratory infections and asthma in premature infants.
- Conditions
- Other Preterm Infants, Vitamin D Deficiency, Bronchiolitis, Asthma
- Trial name
- NCT02404623
- Testing
- Vitamin D
- Where
- Israel
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Soroka University Medical Center
Phenylketonuria
Effect of L-carnitine Supplementation on Phenylalanine and Brain-derived Neurotrophic Factor Levels in Infants and Children With Phenylketonuria
Effect of L-carnitine supplementation on phenylalanine and brain-derived neurotrophic factor levels in infants and children with phenylketonuria L-carnitine is mainly obtained from foods such as red meat and milk that are limited…
- Trial name
- NCT06901323
- Testing
- L-carnitine
- Where
- Egypt
- Volunteers
- 176 people, healthy volunteers welcome
- Sponsor
- Mansoura University
Sedentary Behavior, Affect
Effects of Multi-day Interruptions in Sitting on Type 2 Diabetes-relevant Outcomes in Children
The overall objective of this in-lab randomized controlled trial is to test the efficacy of multi-day interruptions in sedentary behavior vs.
- Conditions
- Sedentary Behavior, Affect, Metabolic Disturbance, Anxiety
- Trial name
- NCT04469790
- Testing
- SIT+WALK, EX
- Where
- United States
- Volunteers
- 188 people, healthy volunteers welcome
- Sponsor
- University of Southern California
Congenital Adrenal Hyperplasia
Androgen Reduction in Congenital Adrenal Hyperplasia, Phase 1
Children with congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency tend to have elevated circulating levels of androgens, which can accelerate skeletal maturation and adversely impact adult height.
- Trial name
- NCT02574910
- Testing
- Abiraterone acetate
- Where
- 4 sites in United States
- Volunteers
- 4 people
- Sponsor
- University of Texas Southwestern Medical Center
Alpha-Mannosidosis
Trial on Safety and Efficacy of Velmanase Alfa Treatment in Pediatric Patients With Alpha-Mannosidosis
The main objectives of the study are to evaluate safety and efficacy of repeated treatment with recombinant human alfa-mannosidase of patients with alfa-mannosidosis aged less than 6 years
- Trial name
- rhLaman-08 (NCT02998879)
- Testing
- Velmanase Alfa (e.g. Lamazym)
- Where
- 6 sites in Austria, Denmark and 3 more
- Volunteers
- 5 people
- Sponsor
- Chiesi Farmaceutici S.p.A.
Obese Adolescents, Obesity
A Study of XW003 Injection in Chinese Adolescents With Obesity
The aim of the study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of XW003 injection in Chinese adolescents with obesity
- Trial name
- NCT07143227
- Testing
- XW003 injection
- Where
- China
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Hangzhou Sciwind Biosciences Co., Ltd.
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