Clinical trials: Diagnostic Errors
Wilson Disease
Efficacy and Safety Study of WTX101 (ALXN1840) in Adult Wilson Disease Patients
The main purpose of the study was to evaluate the efficacy of ALXN1840 (formerly WTX101) for 24 weeks on non-ceruloplasmin-bound copper (NCC) concentrations adjusted for molybdenum plasma concentration in participants newly…
- Trial name
- NCT02273596
- Testing
- ALXN1840
- Where
- 9 sites in United States, Austria and 3 more
- Volunteers
- 29 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Cerebrotendinous Xanthomatosis (CTX)
Cerebrotendinous Xanthomatosis (CTX) Prevalence Study
Iiopathic bilateral cataracts occur in many children with Cerebrotendinous Xanthomatosis (CTX) so the opportunity to use contact with health care providers in connection with pediatric cataract evaluation and treatment is a…
- Trial name
- NCT02638220
- Where
- 44 sites in United States
- Volunteers
- 442 people
- Sponsor
- Travere Therapeutics, Inc.
G6PD Deficiency
Evaluation of a Diagnostic to Identify Glucose-6-phosphate Dehydrogenase (G6PD) Deficiency in Brazil
Th objectives of this study are: To determine the performance of G6PD tests in detecting G6PD activity and hemoglobin (Hb) compared to a reference assay To assess the comprehension of the G6PD test packaging and labelling among…
- Trial name
- NCT04033640
- Testing
- SD Biosensor STANDARD G6PD Test, Pointe Scientific Test Kit, HemoCue System
- Where
- 2 sites in Brazil
- Volunteers
- 1,754 people, healthy volunteers welcome
- Sponsor
- PATH
Fabry Disease
Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-naïve Adult Male Patients With Fabry Disease
Primary Objective: To assess the safety, pharmacokinetics (PK), pharmacodynamics (PD), and exploratory efficacy of GZ/SAR402671 in enzyme replacement therapy treatment-naïve adult male participants diagnosed with Fabry disease.
- Trial name
- NCT02228460
- Testing
- GZ/SAR402671
- Where
- 8 sites in United States, France and 3 more
- Volunteers
- 11 people
- Sponsor
- Genzyme, a Sanofi Company
COPD Asthma
Reducing Diagnostic Error to Improve Patient Safety in COPD and Asthma (REDEFINE Study)
The purpose of this study is to evaluate the use of spirometry in identifying Diagnostic Error in COPD and Asthma patients.
- Trial name
- REDEFINE (NCT03137303)
- Testing
- Spirometry at initial visit, Albuterol, Spirometry at 12-month follow-up
- Where
- 2 sites in United States
- Volunteers
- 402 people
- Sponsor
- University of Illinois Chicago
G6PD Deficiency
Validation of a Diagnostic Test for Glucose-6-phosphate Dehydrogenase (G6PD) Deficiency in Anti-coagulated Blood
The primary objective of the study is to assess the accuracy of the SD Biosensor STANDARD™ point-of-care (POC) G6PD test in measuring G6PD activity and classifying results compared to a reference assay and across repeated…
- Trial name
- NCT04054661
- Testing
- SD Biosensor STANDARD G6PD Test, Pointe Scientific Test Kit, HemoCue System
- Where
- United States
- Volunteers
- 229 people, healthy volunteers welcome
- Sponsor
- PATH
Hunter Syndrome
A Safety and Dose Ranging Study of Idursulfase (Intrathecal) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Hunter Syndrome Who Have Central Nervous System Involvement and Are Receiving Treatment With Elaprase®
Elaprase (idursulfase), a large molecular protein, is not expected to cross the blood brain barrier at therapeutic levels when administered intravenously.
- Trial name
- NCT00920647
- Testing
- Control, Idursulfase IT (1 mg), Idursulfase IT (10 mg)
- Where
- 3 sites in United States, United Kingdom
- Volunteers
- 16 people
- Sponsor
- Shire
Medical Errors, Decision Support Systems, Clinical
Integrating Contextual Factors Into Clinical Decision Support
Preventing contextual errors requires heightening clinician responsiveness to clues that there are contextual factors during the clinical encounter, in real time.
- Conditions
- Medical Errors, Decision Support Systems, Clinical, Diagnostic Errors
- Trial name
- NCT03244033
- Testing
- Contextual clinical decision support, Contextual survey
- Where
- 2 sites in United States
- Volunteers
- 452 people
- Sponsor
- University of Illinois Chicago
Hunter Syndrome
An Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients
The objective of this study is to evaluate the effect of anti-idursulfase antibodies on idursulfase safety (measured by infusion related adverse events) between patients who develop anti-idursulfase antibodies and patients who do…
- Trial name
- NCT00882921
- Testing
- Idursulfase
- Where
- 6 sites in United States, Brazil and 1 more
- Volunteers
- 26 people
- Sponsor
- Shire
Education, Medical, Undergraduate, Medical History Taking
Measuring Quality of Medical Student Performance at Contextualizing Care
During the project, fourth-year medical students participating in a Medicine sub-internship will be randomized to an intervention group or a control group; the intervention group will receive additional training in the…
- Conditions
- Education, Medical, Undergraduate, Medical History Taking, Diagnosis
- Trial name
- NCT01088438
- Testing
- Contextualization workshop
- Where
- United States
- Volunteers
- 189 people
- Sponsor
- University of Illinois Chicago
Colon Cancer, Lung Cancer
Effectiveness of Electronic Health Record-Based Interventions for Improving Follow-Up in Primary Care
Diagnostic delays in ambulatory care are often due to breakdowns of related care processes. Electronic systems can improve follow-up and reduce delays by detecting missed appointments or incomplete procedures so that patients are…
- Conditions
- Colon Cancer, Lung Cancer, Prostate Cancer
- Trial name
- NCT01346839
- Testing
- Contact Intervention
- Where
- 2 sites in United States
- Volunteers
- 1,256 people
- Sponsor
- Baylor College of Medicine
Transthyretin (TTR) Amyloid Cardiomyopathy
Safety and Efficacy of Tafamidis in Patients With Transthyretin Cardiomyopathy
This Phase 3 study will investigate the efficacy, safety and tolerability of an oral daily dose of 20 mg or 80 mg tafamidis meglumine capsules compared to placebo in subjects with either transthyretin genetic variants or…
- Trial name
- ATTR-ACT (NCT01994889)
- Testing
- Tafamidis
- Where
- 80 sites in United States, Belgium and 11 more
- Volunteers
- 441 people
- Sponsor
- Pfizer
Transthyretin (TTR) Amyloid Cardiomyopathy
Long-term Safety of Tafamidis in Subjects With Transthyretin Cardiomyopathy
Open label study to evaluate tafamidis for the treatment of transthyretin cardiomyopathy
- Trial name
- NCT02791230
- Testing
- Tafamidis
- Where
- 80 sites in United States, Argentina and 15 more
- Volunteers
- 1,733 people
- Sponsor
- Pfizer
Fabry Disease
Evaluation of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-Naïve Adult Male Patients With Fabry Disease
Primary Objective: To assess the long-term safety of GZ/SAR402671 in adult male participants with Fabry disease who previously completed study ACT13739 (NCT02489344).
- Trial name
- NCT02489344
- Testing
- GZ/SAR402671
- Where
- 7 sites in United States, France and 3 more
- Volunteers
- 8 people
- Sponsor
- Genzyme, a Sanofi Company
Leukodystrophy, White Matter Disease
LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies
Leukodystrophies, and other heritable disorders of the white matter of the brain, were previously resistant to genetic characterization, largely due to the extreme genetic heterogeneity of molecular causes.
- Conditions
- Leukodystrophy, White Matter Disease, 4H Syndrome, Adrenoleukodystrophy
- Trial name
- NCT02699190
- Where
- United States
- Volunteers
- 236 people
- Sponsor
- Children's Hospital of Philadelphia
Niemann-Pick Disease, Type C
Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C
A prospective, randomized, double-blind, placebo controlled therapeutic study in participants with confirmed diagnosis of Niemann-Pick disease type C (NPC).
- Trial name
- NCT02612129
- Testing
- arimoclomol
- Where
- 14 sites in United States, Denmark and 7 more
- Volunteers
- 50 people
- Sponsor
- ZevraDenmark
Adrenal Insufficiency, Cirrhosis
Is Adrenal Insufficiency Under-diagnosed in Hospitalized Cirrhosis Patients?
The hepatoadrenal syndrome has been well described in the literature and is known to be associated with poorer outcomes in both stable and critically ill cirrhotic patients.
- Conditions
- Adrenal Insufficiency, Cirrhosis, Spur Cell Anemia, Lecithin Acyltransferase Deficiency
- Trial name
- NCT03368066
- Testing
- Cosyntropin
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- University of Virginia
Lung Cancer, Breast Cancer
Measuring and Improving the Safety of Test Result Follow-Up
Improving communication is foundational to improving patient safety. Electronic health records (EHRs) can improve communication, but also introduce unique vulnerabilities.
- Conditions
- Lung Cancer, Breast Cancer, Colon Cancer, Bladder Cancer
- Trial name
- NCT04166240
- Testing
- SAFER TRACKS Intervention
- Where
- 2 sites in United States
- Volunteers
- 46 people
- Sponsor
- VA Office of Research and Development
Hunter Syndrome, Mucopolysaccharidosis II (MPS II)
Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Idursulfase
Study TKT024EXT was a long-term, single-arm, open-label extension of Study TKT024, a one year Phase 2/Phase 3 registration study.
- Trial name
- NCT00630747
- Testing
- Idursulfase
- Where
- 52 sites in United States, Brazil and 8 more
- Volunteers
- 94 people
- Sponsor
- Shire
Hunter Syndrome
Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment
Study HGT-HIT-094 is a multicenter study designed to determine the effect on clinical parameters of neurodevelopmental status of monthly IT administration of idursulfase-IT 10 mg for 12 months in pediatric patients with Hunter…
- Trial name
- AIM-IT (NCT02055118)
- Testing
- idursulfase-IT, No IT treatment
- Where
- 9 sites in United States, Australia and 5 more
- Volunteers
- 58 people
- Sponsor
- Shire