Clinical trials: Genetic Conditions
Gout, Hyperuricemia
A Study to Assess the Efficacy, Safety, and Pharmacokinetics of ABP-671 in Patients With Gout or Hyperuricemia
This is a Phase 2a, multicenter, randomized, double-blind, placebo-controlled, dose-ranging study to evaluate the efficacy, safety, and pharmacokinetics study of 6 different dose regimens of ABP-671 compared with placebo.
- Trial name
- NCT04638543
- Testing
- ABP-671
- Where
- 5 sites in Australia
- Volunteers
- 60 people
- Sponsor
- Atom Therapeutics Co., Ltd
Mental Retardation, Developmental Disabilities
Healthy Lifestyles for People With Intellectual Disabilities
Relatively few health promotion and disease prevention programs have included or targeted people with disabilities, and even fewer have focused on individuals with intellectual disabilities.
- Conditions
- Mental Retardation, Developmental Disabilities, Intellectual Disabilities
- Trial name
- HLID (NCT00597948)
- Testing
- Healthy Lifestyles Curriculum
- Where
- United States
- Volunteers
- 131 people, healthy volunteers welcome
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Musculoskeletal Diseases, Genetic Disease
Turkish Version of the Affordance in the Home Environment for Motor Development-Toddler (AHEMD-T)
This study aimed to translate the Affordance in the Home Environment for Motor Development -Toddler (AHEMD-T) into Turkish and examine its psychometric properties.
- Conditions
- Musculoskeletal Diseases, Genetic Disease, Metabolic Disease, Delays Developmental
- Trial name
- NCT04556500
- Testing
- investigating the psychometric properties of Turkish Affordance in the Home Environment for Motor Development-Toddler (AHEMD-T)
- Where
- Turkey (Türkiye)
- Volunteers
- 139 people, healthy volunteers welcome
- Sponsor
- Hacettepe University
Chronic Kidney Disease(CKD), Genetic Kidney Disease
Genomic First Testing in Chronic Kidney Disease
This multi-center study examines the role of genetic testing in patients with chronic kidney disease (CKD) who are identified as being at risk for genetic kidney disease, based on Ontario Health's Provincial Genetic Program…
- Trial name
- NCT06794567
- Testing
- Genetic Testing
- Where
- Canada
- Volunteers
- 2,400 people, healthy volunteers welcome
- Sponsor
- Dervla Connaughton
Diabetes Mellitus, Type 2, PreDiabetes
Blood Markers of Early Pancreas Cancer
Identifying biomarkers of early pancreatic ductal adenocarcinoma (PDAC) could facilitate screening for individuals at higher than average risk and expedite the diagnosis in individuals with symptoms and substantially improve an…
- Conditions
- Diabetes Mellitus, Type 2, PreDiabetes, Pancreas Cyst, Chronic Pancreatitis
- Trial name
- NCT03568630
- Where
- United States
- Volunteers
- 1,250 people, healthy volunteers welcome
- Sponsor
- University of Nebraska
Wilson Disease
Study of Tetrathiomolybdate in Patients With Wilson Disease
OBJECTIVES: Evaluate the safety and efficacy of ammonium tetrathiomolybdate alone and compared with trientine therapy as initial treatment in patients with Wilson disease presenting neurologically.
- Trial name
- NCT00004339
- Testing
- tetrathiomolybdate, trientine
- Where
- United States
- Volunteers
- 90 people
- Sponsor
- National Center for Research Resources (NCRR)
Muscular Dystrophies, Scapular Dyskinesis
Evaluation of a Textile Scapula Orthosis
Shoulder instability due to muscle weakness is a common problem in disorders of the upper extremities. During arm motion, the scapula acts as a dynamic base for the humeral head.
- Conditions
- Muscular Dystrophies, Scapular Dyskinesis, Muscular Dystrophy, Facioscapulohumeral, Muscle Weakness
- Trial name
- ScapOrthosis (NCT04154098)
- Testing
- Scapula orthosis assistance, Manual scapula assistance, No assistance
- Where
- 2 sites in Germany, Switzerland
- Volunteers
- 8 people
- Sponsor
- Swiss Federal Institute of Technology
Vasomotor Symptoms as a Sex Hormone-dependent Disorder in Women and Men, Hot Flashes
A Study to Learn How Safe the Study Treatment Elinzanetant is and How it Moves Into, Through and Out of the Body When Given as Single Increasing Doses That Are Higher Than Normally Used Compared to Placebo and Moxifloxacin in Healthy Participants
Researchers are looking for a better way to treat vasomotor symptoms, a condition of having hot flashes. The condition is caused by hormonal changes and occurs primarily in women, but can also affect men.
- Conditions
- Vasomotor Symptoms as a Sex Hormone-dependent Disorder in Women and Men, Hot Flashes, Healthy Volunteers
- Trial name
- NCT05481528
- Testing
- Moxifloxacin, Elinzanetant (BAY3427080)
- Where
- Germany
- Volunteers
- 51 people, healthy volunteers welcome
- Sponsor
- Bayer
Chronic Myeloid Leukemia (CML), Acute Myelogenous Leukemia (AML)
CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant
The purpose of this study is to learn more about the effects of (classification determinant) CD34+ stem cell selection on graft versus host disease (GVHD) in children, adolescents, and young adults.
- Conditions
- Chronic Myeloid Leukemia (CML), Acute Myelogenous Leukemia (AML), Myelodysplastic Syndrome (MDS), Juvenile Myelomonocytic Leukemia (JMML)
- Trial name
- NCT02061800
- Testing
- CliniMACS CD34+ Reagent System, Thiotepa, Cyclophosphamide
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Diane George
Asthma, COPD
Acoustic Waveform Respiratory Evaluation
The study will evaluate the feasibility of using smartphone speakers and microphones to evaluate the caliber of the airways, detect airway obstruction, aid in airway disease diagnosis, and identify disease exacerbations.
- Conditions
- Asthma, COPD, Cystic Fibrosis, Ciliary Motility Disorders
- Trial name
- AWARE (NCT06512064)
- Testing
- AWARE
- Where
- 2 sites in United States
- Volunteers
- 800 people, healthy volunteers welcome
- Sponsor
- Indiana University
Neurofibromatosis Type 1
Reading Disability in Children With NF1
A national, multicenter, randomized, transverse clinical trial, estimating the existence of phonological deficits in children with NF1 children compared with control children without NF1 with the same reading level.
- Trial name
- NCT02397967
- Testing
- Neuropsychological assessments
- Where
- 6 sites in France
- Volunteers
- 181 people, healthy volunteers welcome
- Sponsor
- University Hospital, Toulouse
Congenital Adrenal Hyperplasia, Hyperplasia
Subcutaneous Hydrocortisone Children With Congenital Adrenal Hyperplasia
This is an open-label, non-randomized, single arm proof-of concept feasibility trial comparing standard oral hydrocortisone (HC) treatment with a six-week intervention of interval bolus delivery (pulsatile) of subcutaneous (SQ)…
- Conditions
- Congenital Adrenal Hyperplasia, Hyperplasia, Adrenal Hyperplasia, Congenital Disorders
- Trial name
- NCT03718234
- Testing
- Subcutaneous hydrocortisone
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- University of Minnesota
Gout, Chronic Kidney Disease (CKD)
A Phase 4 Safety and Efficacy Study to Evaluate Lesinurad 200 mg in Participants With Gout and Renal Impairment
This study evaluates the safety and efficacy of lesinurad administered with an XOI versus a placebo plus an XOI in gout participants who have moderate renal impairment and who are not at target level of serum urate (sUA).
- Trial name
- NCT03226899
- Testing
- Lesinurad, XOI, colchicine
- Where
- 116 sites in United States, Czechia and 2 more
- Volunteers
- 242 people
- Sponsor
- Ironwood Pharmaceuticals, Inc.
Mitochondrial Disease, Chronic Kidney Disease
Expanded Access Program for R007 (Probucol) Tablets in Adults With Mitochondrial Disease and Chronic Kidney Disease
The purpose of this expanded access program is to enable access to R007 (probucol) tablets for the compassionate treatment of adults with mitochondrial disease who also have chronic kidney disease.
- Trial name
- NCT06931834
- Testing
- Probucol
- Where
- United States
- Sponsor
- RiboNova Inc.
Stargardt Disease, Stargardt Macular Dystrophy
A Study About the Safety of a Single ASP2020 Eye Injection and if it Helps People With Vision Loss From Stargardt-type Eye Conditions
Macular dystrophies are a group of inherited eye conditions that affect the macula. The macula is in the center of the retina, the light sensitive part at the back of the eye.
- Conditions
- Stargardt Disease, Stargardt Macular Dystrophy, Stargardt-like Macular Dystrophy
- Trial name
- DEVOTE (NCT07734064)
- Testing
- ASP2020
- Where
- 4 sites in United States
- Volunteers
- 30 people
- Sponsor
- Astellas Institute for Regenerative Medicine
Pancreatic Ductal Adenocarcinoma, Pancreatic Cancer
Cholesterol Disruption in Combination With the Standard of Care in Patients With Advanced Pancreatic Adenocarcinoma
Cardiovascular diseases and cancers, the two leading causes of death in Canada, require cholesterol to sustain their progression.
- Conditions
- Pancreatic Ductal Adenocarcinoma, Pancreatic Cancer, Pancreas Cancer, Metastatic Cancer
- Trial name
- NCT04862260
- Testing
- Cholesterol metabolism disruption
- Where
- 2 sites in Canada
- Volunteers
- 3 people
- Sponsor
- CHU de Quebec-Universite Laval
To Determine Bioequivalence Under Fed Conditions
Bioequivalence Study of Doxycycline Monohydrate Tablets Under Fed Conditions
The purpose of this study is to compare the single-dose bioequivalence of Par and Oclassen doxycycline monohydrate 100 mg.
- Trial name
- NCT01380496
- Testing
- doxycycline monohydrate
- Where
- Canada
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Par Pharmaceutical, Inc.
Obesity
GLP-1 Response and Effect in Individuals With Obesity Causing Genetic Mutations
The obesity epidemic is attributable to dietary and behavioral trends acting on a person's genetic makeup to determine body mass and susceptibility to obesity-related diseases.
- Trial name
- NCT02082496
- Testing
- Liraglutide
- Where
- Denmark
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- University of Copenhagen
Asthma, COPD
Blood Collection From Individuals With Lung Disease for Genetic Studies (Qatar)
This is a research study where researchers are collecting blood to evaluate the genetic characteristics of individuals with chronic lung diseases, including asthma, COPD (chronic obstructive pulmonary disease), interstitial lung…
- Conditions
- Asthma, COPD, Interstitial Lung Disease, Cystic Fibrosis
- Trial name
- Q-BC (NCT00504361)
- Where
- Qatar
- Volunteers
- 600 people, healthy volunteers welcome
- Sponsor
- Weill Medical College of Cornell University
Vaccine-induced Thrombotic Thrombocytopenia Syndrome, Guillain-Barre Syndrome
Gene Sequencing as a Strategy for Identifying Genetic Factors Associated With Serious Adverse Events After Covid-19 Vaccines in Use in Brazil
This protocol proposes to investigate genetic factors that may be involved in the pathogenesis of adverse events of interest with selected covid-19 vaccines: vaccine-induced immune thrombotic thrombocytopenia, and neurological…
- Conditions
- Vaccine-induced Thrombotic Thrombocytopenia Syndrome, Guillain-Barre Syndrome, Acute Disseminated Encephalomyelitis, Transverse Myelitis
- Trial name
- NCT05630313
- Where
- Brazil
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- The Immunobiological Technology Institute (Bio-Manguinhos) / Oswaldo Cruz Foundation (Fiocruz)
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