Clinical trials: Genetics of Drug Metabolism
Adenovirus, Anesthesia
Pharmacokinetics of Understudied Drugs Administered to Children Per Standard of Care
Understudied drugs will be administered to children per standard of care as prescribed by their treating caregiver and only biological sample collection during the time of drug administration will be involved.
- Conditions
- Adenovirus, Anesthesia, Anxiety, Anxiolysis
- Trial name
- PTN_POPS (NCT01431326)
- Testing
- The POPS study is collecting PK data on children prescribed the following drugs of interest per standard of care:
- Where
- 52 sites in United States, Canada and 3 more
- Volunteers
- 3,520 people
- Sponsor
- Daniel Benjamin
Mucopolysaccharidosis (MPS)
Safety, Tolerability, Ascending Dose and Dose Frequency Study of rhHNS Via an IDDD in MPS IIIA Patients
Sanfilippo syndrome, or Mucopolysaccharidosis (MPS) III, is a rare lysosomal storage disease (LSD) caused by loss in activity of 1 of 4 enzymes necessary for degradation of the glycosaminoglycan (GAG) heparan sulfate (HS) in…
- Trial name
- NCT01155778
- Testing
- Recombinant human heparan N-sulfatase (rhHNS)
- Where
- 2 sites in Netherlands, United Kingdom
- Volunteers
- 12 people
- Sponsor
- Shire
Elevated LDL Cholesterol
Oat and Cholesterol
The primary objective is to assess the effect of an oat ingredient provided over 4 weeks on serum LDL cholesterol in men and women with elevated LDL-cholesterol compared to a placebo.
- Trial name
- NCT03911427
- Testing
- Active oat beverage, Control rice beverage
- Where
- Canada
- Volunteers
- 207 people, healthy volunteers welcome
- Sponsor
- PepsiCo Global R&D
Hyperlipidemia, Hypercholesterolemia
A Study to Evaluate an Investigational Drug in Patients With Mixed Hyperlipidemia (0653A-071)(COMPLETED)
The purpose of this study is to assess the cholesterol lowering effects of an investigational drug in patients with mixed hyperlipidemia (high cholesterol and high triglycerides).
- Conditions
- Hyperlipidemia, Hypercholesterolemia, Hypertriglyceridemia
- Trial name
- NCT00093899
- Testing
- ezetimibe (+) simvastatin
- Volunteers
- 611 people
- Sponsor
- Organon and Co
Lipid Metabolism, Inborn Errors, Heart Disease
An Investigational Drug Study to Lower Non-Cholesterol Sterol Levels Associated With Sitosterolemia (0653-062)(COMPLETED)
This is a 6-month study with patients who have the rare disease, sitosterolemia which may result in heart-related diseases. These patients have unusually high absorption of non-cholesterol sterols, resulting in heart-related…
- Trial name
- NCT00092898
- Testing
- MK0653, ezetimibe
- Volunteers
- 30 people
- Sponsor
- Organon and Co
Hematologic Malignancies, Nonmalignant Diseases
Study of Thiotepa and TEPA Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients
Thiotepa is a chemotherapy drug used extensively in bone marrow transplantation. Thiotepa is a prodrug that undergoes metabolic conversion in the liver by CYP2B6 and CYP3A4 to its primary active metabolite, triethylene…
- Conditions
- Hematologic Malignancies, Nonmalignant Diseases, Immune Deficiency, Hemoglobinopathies
- Trial name
- NCT03609840
- Testing
- Thiotepa
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- University of California, San Francisco
Pompe Disease
Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease
This is a longitudinal natural history study of Infantile Pompe disease. The investigators will regularly collect and review medical information regarding the diagnosis of Pompe disease, response to enzyme replacement (ERT) using…
- Trial name
- NCT01665326
- Testing
- Observational
- Where
- United States
- Volunteers
- 400 people
- Sponsor
- Duke University
Functional Gastrointestinal Disorders
Effect of Controlled Diet Combined With a Fermented Milk Product on Gas-related Symptoms
To select among different dietary and clinical conditions the most appropriate to assess the impact of a fermented milk product on gas-related symptoms
- Trial name
- FLATBACK (NCT02936713)
- Testing
- Fermented milk product with probiotics (FMPP)
- Where
- Spain
- Volunteers
- 126 people, healthy volunteers welcome
- Sponsor
- Danone Global Research & Innovation Center
Hereditary Tyrosinemia, Type I
A Non-interventional, Post-Marketing Study to Describe Outcome of Nitisinone Treatment in HT-1 Patients
This is a prospective, non-interventional, non-comparative, multicenter study to collect data on HT-1 patients in China treated with Nitisinone in a routine clinical setting. No tests or examinations are mandated in the study.
- Trial name
- NCT06227429
- Testing
- Nitisinone
- Where
- 4 sites in China
- Sponsor
- Swedish Orphan Biovitrum
Hermansky-Pudlak Syndrome (HPS), Pulmonary Fibrosis
Pilot Study of a Multi-Drug Regimen for Severe Pulmonary Fibrosis in Hermansky-Pudlak Syndrome
This study will examine whether five drugs (pravastatin, Losartan, Zileuton, N-acetylcysteine and erythromycin) used together can slow the course of pulmonary fibrosis (scarring of the lung tissue) in patients with…
- Conditions
- Hermansky-Pudlak Syndrome (HPS), Pulmonary Fibrosis, Oculocutaneous Albinism, Platelet Storage Pool Deficiency
- Trial name
- NCT00467831
- Testing
- Losartan, Zileuton, N-Acetylcysteine
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Diabetes Mellitus
Observational Study to Assess Glycosylated Hemoglobin Changes After 6 Months of Treatment With Pioglitazone.
The purpose of this study is to asses changes in glycosylated hemoglobin, fasting blood lipids and genetic polymorphism's in peroxisomal proliferator activated receptors--gamma receptor after 6 months of pioglitazone, once daily…
- Trial name
- NCT00449553
- Testing
- Pioglitazone and sulphonylurea, Pioglitazone and metformin
- Where
- 4 sites in Denmark, Iceland and 2 more
- Volunteers
- 326 people
- Sponsor
- Takeda
Metabolism
Effect of Genetics on Metabolism of Efavirenz
This study will evaluate the effects of genetics on metabolism of the anti-HIV medicine efavirenz (Sustiva) and will see if Efavirenz interacts with bupropion (Zyban or Wellbutrin), a drug commonly used to treat depression and to…
- Trial name
- NCT00245986
- Testing
- Blood draw
- Where
- United States
- Volunteers
- 350 people, healthy volunteers welcome
- Sponsor
- National Institutes of Health Clinical Center (CC)
Dyslipidemia, Diabetes
Meta-analyses of the Effect of Important Food Sources of Sugars on Cardiometabolic Risk Factors
Fructose-containing sugars have been implicated in the epidemics of obesity, diabetes and related cardiometabolic disorders. This view is supported by lower quality evidence from ecological observations, animal models, and select…
- Conditions
- Dyslipidemia, Diabetes, Prediabetes, Dysglycemia
- Trial name
- NCT02716870
- Testing
- Fructose-containing Sugars
- Where
- Canada
- Volunteers
- 1 people, healthy volunteers welcome
- Sponsor
- University of Toronto
Acute Myeloid Leukemia, Chronic Lymphocytic Leukemia
Analysis of Genetic Factors Related to Predisposition and Prognosis of Hematological Malignancies in Israel
There are naturally occuring variations in the genetic makeup of all of us. Some of these variations may contribute to a change in susceptibility toward different diseases or change the prognosis.
- Conditions
- Acute Myeloid Leukemia, Chronic Lymphocytic Leukemia, Chronic Myelogenous Leukemia
- Trial name
- NCT00159003
- Where
- Israel
- Volunteers
- 100 people
- Sponsor
- Deborah Rund
Diabetes Mellitus, Type 2
Effect of a Postbiotic Intake on Glucose Control and Microbiota Composition of Type 2 Diabetic Subjects: a Randomized Controlled Trial.
The goal of this randomized clinical trial is to evaluate the effect of the administration of a postbiotic on glycemic control, insulin resistance and microbiota composition in subjects with type 2 diabetes.
- Trial name
- Diabet2Predict (NCT06448182)
- Testing
- Postbiotic
- Where
- 3 sites in Spain
- Volunteers
- 106 people
- Sponsor
- Clinica Universidad de Navarra, Universidad de Navarra
Fabry Disease
A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease
A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents with Fabry Disease.
- Trial name
- FLY (NCT06328608)
- Testing
- PRX-102 1 mg/kg every two weeks
- Where
- 12 sites in United States, Austria and 4 more
- Volunteers
- 22 people
- Sponsor
- Chiesi Farmaceutici S.p.A.
Morquio Disease
Establishment of Human Cellular Disease Models for Morquio Disease
Establishment of human cellular disease models for Morquio disease for an individualized therapy development having the capacity to address both hepatic and neurologic forms of the disease
- Trial name
- IPSMORQUIO (NCT03872713)
- Where
- Pakistan
- Volunteers
- 40 people
- Sponsor
- CENTOGENE GmbH Rostock
Cytomegalovirus Retinitis, HIV Infections
The Safety and Effectiveness of Ganciclovir Plus Interferon Beta in Preventing the Return of Cytomegalovirus (CMV) of the Eyes in Patients With AIDS
The use of ganciclovir (DHPG) in combination with interferon beta to prevent relapse of cytomegalovirus retinitis in patients with AIDS.
- Trial name
- NCT00002299
- Testing
- Interferon beta-1b, Ganciclovir
- Where
- 6 sites in United States
- Sponsor
- Hoffmann-La Roche
Gout
Validation Study of Physical Measurement of Tophi
The purpose of this study is to determine the inter- and intra-rater reproducibility of direct physical measurement of tophus nodules in subjects with gout.
- Trial name
- NCT00175006
- Testing
- Measurement of Tophi to validate procedure
- Volunteers
- 13 people
- Sponsor
- Takeda
Transthyretin (TTR)-Mediated Amyloidosis, Familial Amyloidotic Polyneuropathy (FAP)
The Study of an Investigational Drug, Revusiran (ALN-TTRSC), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Whose Disease Has Continued to Worsen Following Liver Transplant
The purpose of this study was to evaluate the safety and effectiveness of revusiran (ALN-TTRSC) in adults with transthyretin-mediated amyloidosis (ATTR), whose disease has continued to worsen after liver transplantation.
- Conditions
- Transthyretin (TTR)-Mediated Amyloidosis, Familial Amyloidotic Polyneuropathy (FAP), ATTR Amyloidosis, Familial Amyloid Neuropathies
- Trial name
- NCT02595983
- Testing
- Revusiran
- Where
- 6 sites in France, Germany and 4 more
- Volunteers
- 12 people
- Sponsor
- Alnylam Pharmaceuticals