Clinical trials: Multiple Anomalies
Gout
The the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of XNW3009 in Health Subject
XNW3009 is a small molecule hURAT1 inhibitor developed independently by Sinovent Pty Ltd., and is intended to treat gout-related hyperuricemia.
- Trial name
- NCT04040907
- Testing
- XNW3009
- Where
- Australia
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Evopoint Biosciences Inc.
Respiratory Failure, Respiratory Arrest
Postoperative Respiratory Abnormalities
The study aims to determine how historical cases of respiratory abnormalities are documented by clinicians in the electronic health records (EHR) of Memorial Hermann Healthcare System (MHHS) inpatient facilities.
- Conditions
- Respiratory Failure, Respiratory Arrest, Respiratory Distress Syndrome, Acute Respiratory Failure
- Trial name
- AI-ARF (NCT04079829)
- Testing
- Observational retrospective cohort to describe data validity; and Data reliability; and Completeness of the data
- Where
- United States
- Volunteers
- 50,000 people, healthy volunteers welcome
- Sponsor
- Efficacy Care R&D Ltd
Parkinson's Disease, Parkinsonian Disorders
Gait Analysis in Neurological Disease
The purpose of this study is to investigate whether speed-dependent measures of gait (so called gait signatures) can be identified in patients with neurological conditions that affect gait, particularly in subjects with…
- Conditions
- Parkinson's Disease, Parkinsonian Disorders, Atypical Parkinson Disease, Progressive Supranuclear Palsy
- Trial name
- NCT02994719
- Testing
- Anti-Parkinson medication, Deep Brain Stimulation
- Where
- United States
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Beth Israel Deaconess Medical Center
Atopic Dermatitis
A Study of BBT001 in Healthy Volunteers (HVs) and in Adult Patients With Atopic Dermatitis (AD)
This is a Phase 1, randomized, blinded, placebo controlled, single-ascending dose (SAD) and multiple-ascending dose (MAD) study of BBT001 in healthy volunteers (HVs) and adult patients with moderate to severe Atopic Dermatitis…
- Trial name
- NCT06808477
- Testing
- BBT001
- Where
- 14 sites in United States, Australia and 2 more
- Volunteers
- 237 people, healthy volunteers welcome
- Sponsor
- Bambusa Therapeutics
Smith Lemli Opitz Syndrome, CHILD Syndrome
Biochemical and Phenotypical Aspects of Smith-Lemli-Opitz Syndrome and Related Disorders of Cholesterol Metabolism
Background: Smith-Lemli-Opitz Syndrome (SLOS) is a genetic disorder. It can cause birth defects and developmental delays. There is no cure for SLOS or other inherited diseases related to cholesterol production or storage.
- Conditions
- Smith Lemli Opitz Syndrome, CHILD Syndrome, Lathosterolosis, Desmosterolosis
- Trial name
- NCT05047354
- Where
- United States
- Volunteers
- 250 people, healthy volunteers welcome
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Phenylketonuria, Healthy
Safety and Tolerability of SYNB1618 in Healthy Adult Volunteers and Adult Subjects With Phenylketonuria
This Phase 1/2a, first-in-human, oral single and multiple dose-escalation, randomized, double-blinded, placebo-controlled study is evaluating SYNB1618 in healthy volunteers (HV) and subjects diagnosed with phenylketonuria (PKU)…
- Trial name
- PKU (NCT03516487)
- Testing
- SYNB1618
- Where
- 4 sites in United States
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- Synlogic
Aneuploidy, Trisomy 21
Prenatal Test for Fetal Aneuploidy Detection
The purpose of this study is to develop and evaluate a blood test for pregnant women for detection of fetal aneuploidy.
- Trial name
- NCT01256606
- Where
- 6 sites in United States
- Volunteers
- 4,000 people, healthy volunteers welcome
- Sponsor
- Roche Sequencing Solutions
Atopic Dermatitis
Phase 1 Study of QX-4533 in Healthy Volunteers and Patients With Moderate-to-Severe Atopic Dermatitis
The goal of this clinical trial is to evaluate the safety and tolerability of QX-453, an investigational treatment, in healthy Chinese volunteers and patients with moderate-to-severe atopic dermatitis.
- Trial name
- NCT07800104
- Testing
- QX4533
- Where
- China
- Volunteers
- 84 people, healthy volunteers welcome
- Sponsor
- QuantX Biosciences, Inc.
Multiple Endocrine Neoplasia
Metabolomics and Genetic Diagnosing Pancreatic Neuroendocrine Tumors in MEN1 Patients
Objectives: The aim of the present study is to assess the significance of metabolomics and genetics in diagnosing and survival evaluation for pNET in the periodic follow-up of MEN1 patients.
- Trial name
- NCT03048266
- Where
- United States
- Volunteers
- 629 people, healthy volunteers welcome
- Sponsor
- M.D. Anderson Cancer Center
ADPKD (Autosomal Dominant Polycystic Kidney Disease)
A Study to Investigate the Effects of JMKX003142 on QTc Interval in Healthy Adults
The primary objective of this study is to assess the effect of a therapeutic and supratherapeutic oral dose of JMKX003142 on the corrected cardiac QT interval relative to a placebo in healthy participants
- Trial name
- NCT07470177
- Testing
- JMKX003142 tablets
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Jemincare
Primary Ciliary Dyskinesia (PCD)
Comparison of Individuals With Primary Ciliary Dyskinesia and Healthy Controls
To date, the literature has not yet elucidated the status of malnutrition and sarcopenia in children and adolescents with PCD compared to healthy controls.
- Trial name
- NCT07740551
- Testing
- Physical Evaluations in Children with Primary Ciliary Dyskinesia, Physical Evaluations in Healthy Children
- Where
- Turkey (Türkiye)
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Izmir Democracy University
Hyperuricemia, Gout
Study 102: Multiple Ascending Dose Study of ALLN-346 (Engineered Urate Oxidase)
The purpose of this Phase I study is to evaluate the safety and tolerability of ALLN-346 in in normal healthy volunteers, in an ascending dose design.
- Trial name
- MAD (NCT04829435)
- Testing
- ALLN-346
- Where
- United States
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- Allena Pharmaceuticals
Marfan Syndrome
Clinical Variability in Marfan Syndrome
Marfan syndrome is an autosomal dominant connective tissue disorder caused by mutations in the fibrillin-1 gene (FBN1). Penetrance of FBN1 mutations is complete but intra and inter familial clinical expressivity is extremely…
- Trial name
- Variarfan (NCT01707563)
- Testing
- skin punch biopsy and molecular biology, fibroblast culture and molecular biology
- Where
- France
- Volunteers
- 160 people, healthy volunteers welcome
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Eye Diseases Hereditary, Retinal Disease
Inherited Retinal Degenerative Disease Registry
The My Retina Tracker® Registry is sponsored by the Foundation Fighting Blindness and is for people affected by one of the rare inherited retinal degenerative diseases studied by the Foundation.
- Conditions
- Eye Diseases Hereditary, Retinal Disease, Achromatopsia, Bardet-Biedl Syndrome
- Trial name
- MRTR (NCT02435940)
- Where
- United States
- Volunteers
- 20,000 people, healthy volunteers welcome
- Sponsor
- Foundation Fighting Blindness
Marfan Syndrome, Loeys-Dietz Syndrome
Micro RNAs as a Marker of Aortic Aneurysm in Hereditary Aortopathy Syndromes
The primary objective of this study is to determine whether specific patterns of circulating micro-ribonucleic acids (miRNAs) are associated with aortic aneurysm and dissection in patients with hereditary aortopathy syndromes.
- Conditions
- Marfan Syndrome, Loeys-Dietz Syndrome, Thoracic Aortic Aneurysm and Dissection Syndromes, Ehlers-Danlos Type IV Syndrome
- Trial name
- NCT02213484
- Where
- 2 sites in United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- University of Colorado, Denver
Post-Acute COVID-19 Syndrome, ME/CFS
Unhide® Project: A Digital Health Platform to Collect Lifestyle Data for Brain Inflammation Research
The unhide® Project is a non-interventional, longitudinal research study designed to establish a secure data repository of demographic, health, and lifestyle information from individuals with brain inflammation and related…
- Conditions
- Post-Acute COVID-19 Syndrome, ME/CFS, Rheumatic Arthritis, Juvenile Rheumatoid Arthritis (JRA)
- Trial name
- NCT04806620
- Where
- United States
- Volunteers
- 10,000 people, healthy volunteers welcome
- Sponsor
- Brain Inflammation Collaborative
Atopic Dermatitis
Study of CM512 in Healthy Subjects and Patients With Atopic Dermatitis
This is a randomized, double-blind, single/multiple dose escalation, placebo-controlled phase I study evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and immunogenicity of CM512 in healthy subjects and…
- Trial name
- NCT06553209
- Testing
- CM512
- Where
- China
- Volunteers
- 110 people, healthy volunteers welcome
- Sponsor
- Keymed Biosciences Co.Ltd
Cystic Fibrosis (CF), Primary Ciliary Dyskinesia (PCD)
Exercise Capacity, Muscle Oxygenation, Respiratory Muscle Strength, and Physical Activity Level in Pediatric CF and PCD
Cystic fibrosis (CF) and primary ciliary dyskinesia (PCD) are genetic diseases characterized by chronic respiratory tract infections.
- Trial name
- NCT07157644
- Where
- Turkey (Türkiye)
- Volunteers
- 88 people, healthy volunteers welcome
- Sponsor
- Gazi 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
Usher Syndrome, Retinitis Pigmentosa
Study of Dietary N-3 Fatty Acids in Patients With Retinitis Pigmentosa and Usher Syndrome
OBJECTIVES: I. Examine the concentration of docosahexanoic acid (DHA) and other n-3 fatty acids in plasma, erythrocyte, and adipose tissue in patients with various forms of retinitis pigmentosa and Usher syndrome. II.
- Trial name
- NCT00004345
- Where
- United States
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- National Center for Research Resources (NCRR)