Clinical trials: Carbohydrates
Inherited Metabolic Disorders (IMD)
MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT)
This study is designed to assess the safety and efficacy of using MGTA-456 in patients with Inherited Metabolic Disorders (IMD) undergoing stem cell transplantation.
- Trial name
- NCT03406962
- Testing
- MGTA-456
- Where
- 2 sites in United States
- Volunteers
- 8 people
- Sponsor
- Magenta Therapeutics, Inc.
Leigh Syndrome
Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome
EPI-743 in Leigh syndrome participants that participated in previous EPI743-12-002 (NCT01721733) study.
- Trial name
- NCT02352896
- Testing
- EPI-743
- Where
- 4 sites in United States
- Volunteers
- 30 people
- Sponsor
- PTC Therapeutics
Hyperbilirubinemia, Jaundice
Demographic, Metabolic, and Genomic Description of Neonates With Severe Hyperbilirubinemia
The purpose of this study is to compare the demographic, metabolic, and genomic characteristics of patients who develop severe hyperbilirubinemia to patients who never developed a significant bilirubin level.
- Trial name
- NCT00383318
- Testing
- Gene mutation sample
- Where
- United States
- Volunteers
- 450 people, healthy volunteers welcome
- Sponsor
- Pediatrix
Epilepsy
Use of the Atkins Diet for Children With Intractable Epilepsy: A Comparison of Daily Carbohydrate Limits
This study is designed to further investigate the use of a modified Atkins diet for children with intractable, daily seizures. It is a follow-up to a study recently completed by our institution.
- Trial name
- NCT00181090
- Testing
- Modified Atkins diet
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Johns Hopkins University
Mucopolysaccharidoses
Validation and Reliability of Iris Cameras in Mucopolysaccharidoses
To assess the reliablity and validity of 2 new iris cameras in the assement of corneal opacification in mucopolysaccharidoses
- Trial name
- NCT05354219
- Testing
- Imaging
- Where
- United Kingdom
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Manchester University NHS Foundation Trust
Pompe Disease
Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease
A Phase 1/2, open-label, multicenter, multiple dose escalation study of BMN 701 administered by intravenous infusion every 2 weeks over a 24-week treatment period to patients with late-onset Pompe disease.
- Trial name
- NCT01230801
- Testing
- BMN 701
- Where
- 10 sites in United States, Australia and 3 more
- Volunteers
- 22 people
- Sponsor
- BioMarin Pharmaceutical
Mucopolysaccharidosis Type IIIB, MPS IIIB (Sanfilippo B Syndrome)
Early Access Treatment With Tralesinidase Alfa in Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B)
This is an intermediate-size patient population Early Access Program (EAP) providing access to intracerebroventricular (ICV) tralesinidase alfa (TA) for participants with mucopolysaccharidosis type IIIB (MPS IIIB, Sanfilippo…
- Trial name
- EAP (NCT07733856)
- Testing
- Tralesinidase alfa (TA)
- Where
- United States
- Sponsor
- Spruce Biosciences
Achondroplasia
A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With Achondroplasia
This is a Phase 3, multicenter, double-blind, randomized, placebo-controlled study to evaluate the efficacy and safety of infigratinib in children and adolescents with achondroplasia (ACH) who have completed at least 26 weeks of…
- Trial name
- PROPEL3 (NCT06164951)
- Testing
- Infigratinib 0.25 mg/kg/day
- Where
- 27 sites in United States, Argentina and 8 more
- Volunteers
- 114 people
- Sponsor
- QED Therapeutics, a BridgeBio company
Glycogen Storage Disease Type IV, Adult Polyglucosan Body Disease
GBE Deficiency (GSD IV and APBD) Natural History Study
Collection and review of clinical information related to glycogen branching enzyme (GBE) deficiency, diagnosed as Glycogen Storage Disease Type IV (GSD IV) or Adult Polyglucosan Body Disease (APBD generated during clinic visits.
- Conditions
- Glycogen Storage Disease Type IV, Adult Polyglucosan Body Disease, GSD4, GSD IV
- Trial name
- NCT02683512
- Testing
- No intervention
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- Duke University
Neurological Regression, Myoclonus
Natural History of Glycosphingolipid Storage Disorders and Glycoprotein Disorders
Study description: This is a natural history study that will evaluate any patient with enzyme or DNA confirmed GM1 or GM2 gangliosidosis, sialidosis or galactosialidosis.
- Conditions
- Neurological Regression, Myoclonus, Cherry Red Spot, Brain Atrophy
- Trial name
- NCT00029965
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Overweight, Obesity
Meta-analyses of the Effect of 'Catalytic' Doses of Fructose and Its Epimers on Carbohydrate Metabolism
Despite advances in the prevention and treatment of type 2 diabetes, its prevalence continues to rise worldwide. There is a need for new modalities to improve metabolic control in individuals with type 2 diabetes and those who…
- Conditions
- Overweight, Obesity, Dysglycemia, Prediabetes
- Trial name
- NCT02776722
- Testing
- fructose, allulose, tagatose, or sorbose
- Where
- Canada
- Volunteers
- 1 people, healthy volunteers welcome
- Sponsor
- University of Toronto
Dietary Habits, Diet, Healthy
Sip and Snack Better (SSB) Study: Improving Added Sugar in Adolescents
Teens consume more added sugar than any other age group. Too much added sugar is associated with poor diet quality, obesity risk, and negative cardiometabolic outcomes.
- Trial name
- NCT07027865
- Testing
- Sip & Snack Better (SSB) Intervention, Sip & Snack Better (SSB) Tech-Only Comparison
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Temple University
Mucopolysaccharidoses
Carotid Structure and Function in MPS Syndromes: A Multicenter Study of the Lysosomal Disease Network
Mucopolysaccharidosis (MPS) syndromes are disorders characterized by enzyme deficiencies, and they have been linked to heart health complications.
- Trial name
- NCT01586871
- Where
- 2 sites in United States
- Volunteers
- 1,147 people
- Sponsor
- University of Minnesota
MPS IVA, MPS VI
Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI
This is a Phase I/II, single arm, open label study of vosoritide therapy provided subcutaneously at 15 ug/kg/day for 96 weeks to 6 patients with MPS IVA or VI.
- Trial name
- NCT05845749
- Testing
- Vosoritide Injection [Voxzogo]
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- University of California, San Francisco
Hurler Syndrome, Inherited Metabolic Disorder
BPX-501 T Cells Infused Post Stem Cell Transplant in Pediatrics With Non-Malignant Disorders Ineligible for BPU004 Study
Providing access of BPX-501 gene modified T cells and rimiducid to pediatric patients who do not meet the eligibility criteria of the BP-U-004 study.
- Conditions
- Hurler Syndrome, Inherited Metabolic Disorder, Lysosomal Storage Disorder, Metachromatic Leukodystrophy
- Trial name
- NCT03639844
- Testing
- rivogenlecleucel, rimiducid
- Where
- 2 sites in United States
- Sponsor
- Bellicum Pharmaceuticals
Obesity
Differences in Nutrient Use During Exercise Between Children of Varying Body Mass Indices and Fitness Levels
Children who have previously participated in the study "MI Energy" will be invited to participate in "SUPER kids". Investigators want to better understand differences in nutrient use (e.g., fatty acid and carbohydrate) during…
- Trial name
- SUPERkids (NCT04819308)
- Where
- United States
- Volunteers
- 95 people, healthy volunteers welcome
- Sponsor
- Arkansas Children's Hospital Research Institute
Diabetes Mellitus, Type 2
A Research Study to See How Much CagriSema Lowers Blood Sugar and Body Weight Compared to Placebo in Children and Adolescents With Type 2 Diabetes
The purpose of this clinical study is to look into how well a study medicine called CagriSema helps children and adolescents living with diabetes lower their blood sugar and body weight.
- Trial name
- REIMAGINEYOUNG (NCT07282613)
- Testing
- CagriSema (Cagrilintide B and Semaglutide I)
- Where
- 58 sites in United States, Argentina and 8 more
- Volunteers
- 80 people
- Sponsor
- Novo Nordisk A/S
Mucopolysaccharidosis II
Long-term Follow-Up for RGX-121
RGX-121-5101 is an observational long-term follow-up study to the RGX-121-101 or RGX-121-1102 studies where participants received RGX-121, a gene therapy intended to deliver a functional copy of the iduronate-2-sulfatase gene…
- Trial name
- NCT04597385
- Testing
- Long-term Follow-Up
- Where
- 4 sites in United States, Brazil
- Volunteers
- 27 people
- Sponsor
- REGENXBIO Inc.
G6PD Deficiency
Evaluation of a Diagnostic to Identify Glucose-6-phosphate Dehydrogenase (G6PD) Deficiency in Brazil
Th objectives of this study are: To determine the performance of G6PD tests in detecting G6PD activity and hemoglobin (Hb) compared to a reference assay To assess the comprehension of the G6PD test packaging and labelling among…
- Trial name
- NCT04033640
- Testing
- SD Biosensor STANDARD G6PD Test, Pointe Scientific Test Kit, HemoCue System
- Where
- 2 sites in Brazil
- Volunteers
- 1,754 people, healthy volunteers welcome
- Sponsor
- PATH
Rare Genetic Muscle Diseases, Muscular Dystrophy, Duchenne
Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases
Since 2017, more than 250 analyses performed at the Molecular Genetics Laboratory of the Timone Enfant Hospital have yielded negative results in patients with rare genetic muscle diseases.
- Conditions
- Rare Genetic Muscle Diseases, Muscular Dystrophy, Duchenne, Muscular Dystrophy, Becker, Congenital Myopathy
- Trial name
- ARNseq-Musc (NCT06833489)
- Testing
- ARN extraction from muscle biopsies
- Where
- France
- Volunteers
- 50 people
- Sponsor
- Assistance Publique Hopitaux De Marseille