Clinical trials: Carbohydrates
Mucolipidosis Type IV
Mucolipidosis Type IV Natural History Study
The primary objectives of the study are: to describe the characteristics of the current international MLIV population; to define the median age at which patients with MLIV achieve or lose developmental milestones; to define the…
- Trial name
- NCT05782387
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Massachusetts General Hospital
Juvenile Idiopathic Arthritis
Treatment With Specific Carbohydrate Diet in Children With Juvenile Idiopathic Arthritis
The aim of this study is to explore if an already established diet with anti-inflammatory effect in paediatric inflammatory bowel disease would have an anti-inflammatory effect in children with JIA.
- Trial name
- JIA (NCT04205500)
- Testing
- Specific carbohydrate diet
- Where
- Sweden
- Volunteers
- 30 people
- Sponsor
- Uppsala University
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
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
The Coordination of Rare Diseases at Sanford (CoRDS) is an international patient registry that serves as a centralized resource for rare disease research.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- United States
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
Pompe Disease
Alglucosidase Alfa Pompe Safety Sub-Registry
To collect uniform and meaningful data on patients with Pompe disease who experience anaphylaxis, severe allergic reactions, and/or signals of severe cutaneous and/or systemic immune complex-mediated reactions following treatment…
- Trial name
- NCT01710813
- Testing
- alglucosidase alfa
- Where
- 18 sites in United States, Belgium and 4 more
- Volunteers
- 110 people
- Sponsor
- Genzyme, a Sanofi Company
Primary Hyperoxaluria Type 1 (PH1)
A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1
The purpose of this study is to evaluate the efficacy and safety of lumasiran in children and adults with primary hyperoxaluria type 1 (PH1).
- Trial name
- ILLUMINATE-A (NCT03681184)
- Testing
- Lumasiran
- Where
- 17 sites in United States, France and 6 more
- Volunteers
- 39 people
- Sponsor
- Alnylam Pharmaceuticals
Obesity, Water; Lack of
Preventing Early Childhood Obesity, Part 1: Family Spirit Nurture, 3-9 Months
This study aims to assess the impact of a brief home-visiting module, called "Family Spirit Nurture" (FSN), on American Indian (AI) parent feeding practices associated with increased risk for early childhood obesity, with a…
- Conditions
- Obesity, Water; Lack of, Breast Feeding, Tooth Erosion
- Trial name
- NCT03101943
- Testing
- Family Spirit Nurture (FSN), Control Program, Water Delivery
- Where
- United States
- Volunteers
- 136 people, healthy volunteers welcome
- Sponsor
- Johns Hopkins Bloomberg School of Public Health
Overweight, Obesity
Reducing Sugar-sweetened Beverage Consumption in Overweight Adolescents
The primary aim of this study is to examine the effect of a multi-component intervention, designed to reduce consumption of sugar-sweetened beverages, on weight gain, total energy intake, and diet quality in adolescents.
- Trial name
- BASH (NCT00381160)
- Testing
- Reduction of sugar-sweetened beverage consumption
- Where
- United States
- Volunteers
- 224 people, healthy volunteers welcome
- Sponsor
- Boston Children's Hospital
Mucopolysaccharidosis II
A Study of JR-141 in Patients With Mucopolysaccharidosis II (BR21)
A Phase II open-label, randomized, parallel group, 2 sites (Brazil), designed to evaluate the safety and efficacy of 3 doses of study drug for the treatment of the MPS II.
- Trial name
- NCT03359213
- Testing
- JR-141
- Where
- 2 sites in Brazil
- Volunteers
- 18 people
- Sponsor
- JCR Pharmaceuticals Co., Ltd.
Mucopolysaccharidosis III-A
Phase I/II Study of JR-441 in Patients With Mucopolysaccharidosis Type IIIA
A Phase I/ II, open-label, randomized, 2-arm study, designed to evaluate the safety and explore efficacy of the study drug in development for the treatment of MPS IIIA patients.
- Trial name
- NCT06095388
- Testing
- JR-441
- Where
- Germany
- Volunteers
- 15 people
- Sponsor
- JCR Pharmaceuticals Co., Ltd.
Sanfilippo Syndrome Type D
A Natural History Study of Sanfilippo Syndrome Type D
Sanfilippo syndrome type D is a ultra rare syndrome with limited available natural history data. This study is planned to document, through retrospective and prospective data collection, syndrome progression in children and young…
- Trial name
- MPSIIID (NCT05648851)
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Phoenix Nest
Primary Hyperoxaluria, Dent Disease
Rare Kidney Stone Consortium Patient Registry
The purpose of this study is to collect medical information from a large number of patients in many areas of the world with primary hyperoxaluria (PH), Dent disease, Cystinuria and APRT deficiency.
- Conditions
- Primary Hyperoxaluria, Dent Disease, Cystinuria, APRT Deficiency
- Trial name
- RKSC (NCT00588562)
- Where
- 4 sites in United States, Iceland
- Volunteers
- 730 people
- Sponsor
- Mayo Clinic
Late Onset Pompe Disease, Pompe Disease
Search for Serum/Plasma Biomarkers in Pompe's Disease
This study aims to analyze serum and plasma samples from patients with late onset Pompe disease treated and not treated with enzyme replacement therapy (ERT) to identify microRNA that could be specific of the disease.
- Trial name
- BIOPOMPE (NCT03045042)
- Testing
- Enzyme Replacement Agent
- Where
- Spain
- Volunteers
- 35 people, healthy volunteers welcome
- Sponsor
- Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau
Diabetes Mellitus, Type 1
What is the Dose Response of Varying Meal Content of Fat on Postprandial Glycaemia in Children With T1DM?
Background: Based on international evidence, current management of people with T1DM on intensive insulin therapy (IIT) use algorithms based on the meal carbohydrate content (MCC) to calculate the prandial insulin dose.
- Trial name
- REQINSOIL (NCT02371694)
- Testing
- Test drink
- Where
- Ireland
- Volunteers
- 30 people
- Sponsor
- Cork University Hospital
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.
Hypochondroplasia
An Interventional Study of Infigratinib in Children With Hypochondroplasia
ACCEL2/3 is a Phase 2/3 study. The purpose of the Phase 2 portion of the study (ACCEL2/3) is to evaluate the efficacy and safety of infigratinib in children with hypochondroplasia (HCH) receiving infigratinib at one of two doses…
- Trial name
- HCH (NCT06873035)
- Testing
- infigratinib 0.128 mg/kg/day, infigratinib 0.25 mg/kg/day
- Where
- 24 sites in United States, Australia and 8 more
- Volunteers
- 24 people
- Sponsor
- QED Therapeutics, a BridgeBio company
Muscle Damage
Effect of Whey Protein on Soccer Performance
Rationale: During exercise protein breakdown in muscles is larger than protein synthesis. This negative muscle protein balance leads to increased muscle damage and thereby to a reduced muscle tissue recovery.
- Trial name
- NCT01110122
- Testing
- Whey protein
- Where
- Netherlands
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- VU University of Amsterdam
Leigh Syndrome (Maternally Inherited, MILS), Leigh Syndrome (AR, AD, XR)
Drug Repurposing for Mitochondrial Disorders Using iPSCs Derived Neural Cells
In this project, the investigators are using iPSC lines derived from patients with Leigh syndrome that carry mutations in the mitochondrial (mtDNA) and in the nuclear DNA (nDNA) to reprogram them into neural progenitor cells and…
- Trial name
- cureMILS (NCT06967831)
- Testing
- skin biopsy, generation of iPSCs, blood drawing
- Where
- 2 sites in Germany
- Volunteers
- 80 people
- Sponsor
- Charite University, Berlin, Germany
Cold-water Immersion, Protein-carbohydrates Supplementation
Effects on CK, RPE, VAS Scale and Physical Performance of Three Recovery Methods in Elite Basketball Players
The aim of this study was to compare the effects of cold water intermittent ion (CWI), carbohydrate and protein supplementation (SUPPL), and active recovery (ACT) on creatine kinase (CK), perceptual and performance markers of…
- Conditions
- Cold-water Immersion, Protein-carbohydrates Supplementation, Active Recovery, Placebo
- Trial name
- NCT05805540
- Testing
- Cold-water immersion, Protein-carbohydrates supplementation, Active recovery
- Where
- Spain
- Volunteers
- 15 people, healthy volunteers welcome
- Sponsor
- University of Cadiz
Glycogen Storage Disease Type IA
A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)
The primary objectives of this study are to evaluate the efficacy of DTX401 to reduce or eliminate dependence on exogenous glucose replacement therapy to maintain euglycemia and to maintain or improve the quality of glucose…
- Trial name
- NCT05139316
- Testing
- DTX401, Oral prednisolone
- Where
- 20 sites in United States, Brazil and 7 more
- Volunteers
- 52 people
- Sponsor
- Ultragenyx Pharmaceutical Inc