Clinical trials: Inborn Errors of Metabolism Disorders
Malaria
Safety of Primaquine + Artemether-lumefantrine in G6PD Deficient Males With an Asymptomatic Malaria Infection (SAFEPRIM)
The purpose of this study is to evaluate the tolerability and safety of increasing doses of primaquine in combination with artemether-lumefantrine in G6PD deficient males with an asymptomatic P. falciparum malaria infection.
- Trial name
- NCT02174900
- Testing
- Artemether-Lumefantrine (AL) + 0.25 mg/kg primaquine, Artemether-Lumefantrine (AL) combination, Artemether-Lumefantrine (AL) + 0.4 mg/kg primaquine
- Where
- Burkina Faso
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- London School of Hygiene and Tropical Medicine
Cerebral Small Vessel Diseases, Cadasil
Taiwan Associated Genetic and Nongenetic Small Vessel Disease
The TAG-SVD enrolled patients with clinical and neuroimaging features of cerebral small vessel disease (CSVD). All enrolled patients will receive next-generation sequence (NGS) with probes designed to target five candidate CSVD…
- Conditions
- Cerebral Small Vessel Diseases, Cadasil, HTRA1-Related Autosomal Dominant Cerebral Angiopathy, COL4A1-Related Brain Small Vessel Disease With Haemorrhage
- Trial name
- TAG-SVD (NCT05473637)
- Testing
- MRI
- Where
- Taiwan
- Volunteers
- 500 people, healthy volunteers welcome
- Sponsor
- National Taiwan University Hospital
Healthy
Comparison of Nikkomycin Z Bioavailability Under Fed and Fasting Conditions
The primary purpose of this study is to evaluate if eating a high fat meal versus not eating any food affects how the study drug (Nikkomycin Z) is absorbed into the body.
- Trial name
- NCT01647256
- Testing
- Nikkomycin Z
- Where
- United States
- Volunteers
- 5 people, healthy volunteers welcome
- Sponsor
- University of Arizona
Congenital Adrenal Hyperplasia, Exercise Capacity
Exercise Capacity, Physical Fitness, and Physical Activity Levels in Children With CAH
Congenital Adrenal Hyperplesia (CAH) is a group of automosal recessive disorders that develop due to a deficiency of one of the five enyzmes necessary for cortisol synthesis in the adrenal cortex.
- Conditions
- Congenital Adrenal Hyperplasia, Exercise Capacity, Physical Activity, Quality of Life
- Trial name
- NCT06449456
- Where
- Turkey (Türkiye)
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Hacettepe University
All Diagnosed Health Conditions, ADD/ADHD
At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions
We are the missing link in clinical trials, connecting patients and researchers seamlessly and conveniently using a mobile health platform to advance medical research.
- Conditions
- All Diagnosed Health Conditions, ADD/ADHD, Alopecia Areata, Ankylosing Spondylitis
- Trial name
- NCT01931644
- Where
- United States
- Volunteers
- 17,667 people, healthy volunteers welcome
- Sponsor
- Sanguine Biosciences
Familial Hypobetalipoproteinemia, Familial Hypobetalipoproteinemia - Heterozygous Form
In Vivo Metabolism of apoB-containing Lipoproteins in ANGPTL3 Deficient Subjects
Rationale. ANGPTL3 has been identified as important regulator of lipolysis as well as a determinant of plasma levels of apolipoprotein B-containing lipoproteins.
- Trial name
- NCT05569928
- Testing
- Administration of deuterated compounds for kinetic study
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Marcello Arca
Irritable Bowel
Effect of Rate Of Delivery on Colonic Fermentation of inuliN (EON)
To test whether slowing the rate of delivery of inulin with either psyllium or divided dosing of inulin, will reduce colonic gas production as compared to inulin combined with placebo.
- Trial name
- EON (NCT05619341)
- Testing
- psyllium, inulin
- Where
- United Kingdom
- Volunteers
- 17 people, healthy volunteers welcome
- Sponsor
- University of Nottingham
Galactose Single Point (GSP), Residual Liver Function
GSP as an Novel Indicator for Residual Liver Function in Patients With Fatty Liver
The primary objective is to determine the galactose single point (GSP) cutoff values to discriminate subjects with different hepatic function.
- Trial name
- GSP (NCT04546022)
- Testing
- Drug: G.S.P. Solution 400 mg/ml I.V. 1.25 ml/kg BW G.S.P. solution after fasting for 6 hours.
- Volunteers
- 210 people, healthy volunteers welcome
- Sponsor
- National Defense Medical Center, Taiwan
Immune Deficiency, Immune Dysregulation Disorder
Allergy and Immunology Natural History Study
This protocol is a natural history study designed to evaluate subjects (and some family members) with suspected or identified genetic diseases of allergic inflammation or Immune Dysregulation.
- Conditions
- Immune Deficiency, Immune Dysregulation Disorder, Allergic Inflammation
- Trial name
- NCT05584488
- Where
- United States
- Volunteers
- 10,000 people, healthy volunteers welcome
- Sponsor
- Columbia University
Rheumatic Diseases, Rheumatoid Arthritis (RA)
Clinical Assessment for Rheumatologic Disease - Research and Advancement in Safety and Efficacy
The CARe RAiSE project represents a pioneering translational initiative aimed at advancing precision medicine in the treatment of autoimmune rheumatic diseases.
- Conditions
- Rheumatic Diseases, Rheumatoid Arthritis (RA), Giant Cell Arteritis (GCA), Psoriatic Arthritis (PsA)
- Trial name
- CARe RAiSE (NCT07150000)
- Testing
- Ex Vivo Assay
- Where
- Germany
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- University of Bonn
Hyperuricemia, Gout
Effect of Quercetin Supplements on Healthy Males: a Four-Week Randomized Cross-Over Trial
The purpose of this study was to test the effect of chronic consumption of Quercetin 500 mg tablets on blood uric acid and other biomarkers in adult males.
- Conditions
- Hyperuricemia, Gout, Kidney Calculi, Diabetes
- Trial name
- NCT01881919
- Testing
- Treatment, Control
- Where
- United Kingdom
- Volunteers
- 22 people, healthy volunteers welcome
- Sponsor
- University of Leeds
Fed
Bioequivalence Study of Indomethacin Extended-Release Capsules 75 mg Under Fed Condition
This is a randomized, balanced, open Label, two-treatment, two-period, two-sequence, single dose, crossover pivotal study. The purpose of this study is to assess the bioequivalence between Test Product and the corresponding…
- Trial name
- NCT01722110
- Testing
- Indomethacin Extended-Release Capsules USP 75 mg, Indomethacin Extended Release Capsules USP 75 mg
- Where
- India
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- IPCA Laboratories Ltd.
Fibrosis
Assessment Of Liver and Spleen Fibrosis in Patients With Gaucher Disease Using Fibroscan
The investigated cohort will examine liver and spleen fibrosis in patients with Gaucher disease divided into two groups, naive GD patients and GD patients treated with ERT.
- Trial name
- NCT01898325
- Testing
- Fibroscan
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Rambam Health Care Campus
Duarte Galactosemia
Intervention and Outcomes in Duarte Galactosemia
The purpose of this study is to learn about Duarte galactosemia (DG). This study will examine the possible effects of Duarte galactosemia (DG) in children, and determine whether dietary exposure to milk in infancy or early…
- Trial name
- NCT02519504
- Where
- United States
- Volunteers
- 566 people, healthy volunteers welcome
- Sponsor
- Emory University
X-linked Adrenoleukodystrophy
Validation of a Prognostic Biomarker Using Brain Diffusion MRI in X-linked Adrenoleukodystrophy
CALD is an inflammatory demyelinating disease that causes severe motor and cognitive deficit leading to rapid death. Hematopoietic stem cell transplantation (HSCT) can halt neuroinflammation in CALD through the replacement of…
- Trial name
- CALDIFF (NCT05911919)
- Testing
- brainQuant module of brainTale-care platform
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Healthy, Lipodystrophy, Familial Partial
Lipodystrophy and Fat Metabolism During Exercise
Mandibular dysplasia with deafness and progeroid features (MDP) syndrome is a rare genetic metabolic disorder that causes lipodystrophy: the inability of the body to store subcutaneous adipose tissue (fat under the skin).
- Trial name
- FAT (NCT04056000)
- Testing
- Caffeine, High-carbohdyrate breakfast, 60-minutes of steady state exercise
- Where
- United Kingdom
- Volunteers
- healthy volunteers welcome
- Sponsor
- University of Exeter
Healthy
Bioavailability Study of Ondansetron Orally Disintegrating Tablets Under Fed Conditions
To compare the single-dose bioavailability of Ondansetron ODT 8 mg and Zofran 8 mg
- Trial name
- NCT00653458
- Testing
- Ondansetron, Zofran
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- Par Pharmaceutical, Inc.
Lactose Intolerance
Effects of Lactose on Fecal Microflora
The genetics of lactase divides the population into 2 phenotypes: Those who can(LP) and those who cannot(LNP)digest lactose. This division may help modify disease risks according to geographic population distribution.
- Trial name
- ELM (NCT00599859)
- Testing
- lactose
- Where
- Canada
- Volunteers
- 57 people, healthy volunteers welcome
- Sponsor
- Sir Mortimer B. Davis - Jewish General Hospital
Rheumatoid Arthritis, Osteoarthritis
Rheumatology Informatics System for Effectiveness
The RISE Registry is an enhanced version of the ACR's Rheumatology Clinical Registry (RCR) and allows for a simplified entry process, while establishing a best-in-class resource to manage your patient population and improve…
- Conditions
- Rheumatoid Arthritis, Osteoarthritis, Gout
- Trial name
- RISE (NCT02230943)
- Where
- United States
- Volunteers
- 100,000 people, healthy volunteers welcome
- Sponsor
- American College of Rheumatology
Fabry Disease
Lipidomics and Functional Analyses of Platelets in Fabry Disease
This study aims to evaluate whether platelets are biochemically and functionally altered in Fabry disease (FD) and therefore possibly implicated in FD manifestations such as cerebrovascular events.
- Trial name
- NCT02649660
- Where
- 2 sites in Switzerland
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Spital Linth