Clinical trials: Outcome Measure
Cystic Fibrosis
Improving Outcome Measures For Adult CF ACT Trials
This is a randomised controlled cross-over trial involving adult cystic fibrosis (CF) patients from the Royal Brompton Hospital, London investigating outcome measures used in airway clearance trials.
- Trial name
- NCT02721498
- Testing
- Airway clearance session using the Active Cycle of Breathing Techniques (ACBT), Rest period
- Where
- United Kingdom
- Volunteers
- 68 people
- Sponsor
- Royal Brompton & Harefield NHS Foundation Trust
CDKL5, CDKL5 Deficiency Disorder
International CDKL5 Clinical Research Network
Pathogenic variants in the Cyclin-dependent kinase like 5 (CDKL5) gene cause CDKL5 deficiency disorder (CDD, MIM 300672, 105830), a severe developmental and epileptic encephalopathy associated with cognitive and motor impairments…
- Conditions
- CDKL5, CDKL5 Deficiency Disorder, CDD
- Trial name
- ICCRN (NCT05558371)
- Testing
- No intervention.
- Where
- 9 sites in United States, Australia
- Volunteers
- 1,000 people
- Sponsor
- University of Colorado, Denver
MDC1A, SELENON-related Myopathy
The Natural History of Patients With Mutations in SEPN1 (SELENON) or LAMA2
SEPN1 (SELENON) is a rare congenital myopathy due to mutations in the SELENON gene. MDC1A is a rare congenital muscle dystrophy due to mutations in the LAMA2 gene.
- Trial name
- NCT04478981
- Testing
- No intervention
- Where
- Netherlands
- Volunteers
- 38 people
- Sponsor
- Radboud University Medical Center
Musculoskeletal System Disease
Clinical and Biomolecular Assessment for the Monitoring of Patients Undergoing Regenerative Medicine Treatments with Orthobiologics
the aim is to improve the evaluation of the outcomes of regenerative medicine treatments performed in the regenerative medicine centers involved in the study.
- Trial name
- NCT04711551
- Testing
- PRP and related devices to abtain platelet concentrate from whole blood, medullary and adipose tissue and related devices
- Where
- 2 sites in Italy
- Volunteers
- 2,000 people
- Sponsor
- I.R.C.C.S Ospedale Galeazzi-Sant'Ambrogio
Rheumatic Diseases, Rheumatic Disorder
Exploring and Measuring the Impact of Steroids in Children and Young People
Assessing the impact of glucocorticoids in children and young people with rheumatic conditions, to develop a patient reported outcome measure (PROM).
- Conditions
- Rheumatic Diseases, Rheumatic Disorder, Rheumatologic Conditions (JRA,Lupus)
- Trial name
- NCT07350395
- Where
- 3 sites in United Kingdom
- Volunteers
- 70 people
- Sponsor
- University of the West of England
Sarcoma, Liposarcoma
Patient Reported Outcome Measures (PROMs) With Trabectedin
Patient reported outcome measures (PROMs) are central to understanding the impact of cancer treatments on patients. Treatment may control cancer growth, and even sometimes modestly improve the length of life, but at the expense…
- Conditions
- Sarcoma, Liposarcoma, Leiomyosarcoma
- Trial name
- NCT03959033
- Where
- United Kingdom
- Volunteers
- 7 people
- Sponsor
- University of Leeds
Spinal Muscular Atrophy
Clinical Assessment of Spinal Muscular Atrophy Type II and III (SMA Europe)
The aim of this project is to establish a network of clinical teams including the major neuromuscular centers in Europe. We plan to work together to find the best common outcome measures for the following multicenter therapeutic…
- Trial name
- NCT01611610
- Testing
- Functional tests
- Volunteers
- 4 people
- Sponsor
- Institut de Myologie, France
Traumatic Brain Injury
Brain Injury Assessment Study at Hennepin County Medical Center
The goals of this study are to develop an objective, multi-modal classification scheme and outcome measures for traumatic brain injury based on several measures: (1) blood-based biomarkers (indicates which cell types are…
- Trial name
- BASH (NCT02706574)
- Volunteers
- 774 people, healthy volunteers welcome
- Sponsor
- CentraCare
Mitochondrial Diseases, Kearns-Sayre Syndrome
Global Registry and Natural History Study for Mitochondrial Disorders
The main goal of the project is provision of a global registry for mitochondrial disorders to harmonize previous national registries, enable world-wide participation and facilitate natural history studies, definition of outcome…
- Conditions
- Mitochondrial Diseases, Kearns-Sayre Syndrome, MIDD, SANDO
- Trial name
- GENOMIT (NCT05554835)
- Where
- 34 sites in Austria, Germany and 1 more
- Volunteers
- 6,000 people
- Sponsor
- LMU Klinikum
Hemophilia
Canadian CHO-KLAT/H-FIT Study - Quality of Life of, and Burden of Caring for, Persons With Hemophilia
The primary goal of this observational study is to determine if: health-related quality of life of boys with moderate/severe hemophilia A who are followed in Canadian pediatric hemophilia treatment centres is significantly…
- Trial name
- NCT06191068
- Where
- Canada
- Volunteers
- 190 people
- Sponsor
- The Hospital for Sick Children
Duchenne Muscular Dystrophy (DMD), Cardiomyopathy
Modeling Mortality in Duchenne Muscular Dystrophy Cardiomyopathy: Identification of Surrogate Outcome Measures for DMD Drug Trials
Dystrophin associated heart dysfunction is a leading cause of death in patients with Duchenne and Becker Muscular dystrophy (DMD/BMD) and Duchenne and Becker muscular dystrophy carriers (MDC); however, the evolution of heart…
- Conditions
- Duchenne Muscular Dystrophy (DMD), Cardiomyopathy, Becker Muscular Dystrophy, Carrier of Duchenne Muscular Dystrophy
- Trial name
- NCT07674758
- Where
- 9 sites in United States
- Volunteers
- 1,000 people
- Sponsor
- Vanderbilt University Medical Center
Sickle Cell Disease, Sickle Cell Hemoglobin C
Home-based Assessment of PRO Measures in SCD Using A Smartphone App Platform: A Feasibility Study
The overarching goal of this proposal is to identify modifiable behavioral strategies based on patient-reported outcomes (PROs) and health-related quality of life (HRQOL) that will improve hydroxyurea (HU) adherence among…
- Conditions
- Sickle Cell Disease, Sickle Cell Hemoglobin C, Sickle Beta Zero Thalassemia, Sickle B+ Thalassemia
- Trial name
- NCT04678037
- Testing
- PROs assessment using sickle cell disease mobile app (SCD-app)
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
Burns
Therapeutic Effect of the Wireless Micro Current Stimulation in Pediatric Deep Dermal Burns and Scald Injuries
This study will determine the effect of the wireless micro current stimulation in pediatric deep dermal burns and scald injuries.
- Trial name
- ELSTHER (NCT02712580)
- Testing
- First investigational device, Second investigational device
- Where
- Germany
- Volunteers
- 188 people
- Sponsor
- Kinderkrankenhaus auf der Bult
Neurofibromatosis Type 1
Reliability of Functional Outcome Measures in Neurofibromatosis 1: Test- Retest
To examine the test-retest reliability (how stable the results are when the same participants, whose symptoms have remained stable, are assessed on 2 different occasions, 14 days apart) of the 10 meter walk test, the timed up and…
- Trial name
- NCT05186870
- Testing
- Outcome measures
- Where
- United Kingdom
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Guy's and St Thomas' NHS Foundation Trust
Knee Osteoarthritis
Effectiveness of Contemporary Knee Arthroplasty in Working-age Patients
The aim of our study was to assess the effectiveness of contemporary knee arthroplasty in working-age patients (< 65 years) by conducting a prospective cohort study with 2-year follow up.
- Trial name
- NCT03233620
- Testing
- Knee arthroplasty
- Volunteers
- 250 people, healthy volunteers welcome
- Sponsor
- Coxa, Hospital for Joint Replacement
Journey II BCS Total Knee System
Safety and Performance of Journey II BCS Total Knee System Patient Reported Outcome Measures
Safety and Performance of Journey II BCS Total Knee System. A Retrospective, Multicenter Study. Patient Reported Outcome Measures.
- Trial name
- JIIPROMS (NCT03903731)
- Testing
- Total knee arthroplasty with Journey II BCS Total Knee System
- Where
- 5 sites in United States, Belgium and 1 more
- Volunteers
- 600 people
- Sponsor
- Smith & Nephew, Inc.
Psychometrics of Two Translated Measurement Tools
Evaluation of Gait Performance in Children With Neuromuscular Diagnoses
The aims of this project are to evaluate the psychometric properties of the German versions of the two performance questionnaires 'Functional Mobility Scale' (FMS) and 'Gillette Functional Assessment Questionnaire - walking…
- Trial name
- NCT02237222
- Testing
- Gait rehabilitation
- Where
- Switzerland
- Volunteers
- 72 people
- Sponsor
- University Children's Hospital, Zurich
Affective Disorders, Anxiety Disorders
Routine Outcome Monitoring in Mental Health Outpatient
Scientific Background: Inherent gaps exist between the worlds of research and clinical therapy, especially in mental-health systems.
- Conditions
- Affective Disorders, Anxiety Disorders, Psychotic Disorders, Personality Disorders
- Trial name
- ROM-Shalvata (NCT02095457)
- Testing
- Frequent monitoring and feedback
- Where
- Israel
- Volunteers
- 900 people
- Sponsor
- Shalvata Mental Health Center
Chronic Kidney Diseases
Clinical Evaluation of PROMIS in CKD
The purpose of this study is to evaluate the clinical validity of a set of PROMIS pediatric person-reported outcome measures in patients with chronic kidney disease.
- Trial name
- NCT03842293
- Where
- 16 sites in United States, Canada
- Volunteers
- 213 people
- Sponsor
- Children's Hospital of Philadelphia
Cerebral Palsy, Spastic
The Efficacy of Distortion Banding on Outcome Measures in Children With Cerebral Palsy
The aim of the study is to compare the conventional rehabilitation program and the distortion taping applied with Kinesio Tex tape in addition to conventional rehabilitation in children with hemiplegic or monoplegic cerebral…
- Trial name
- NCT05251532
- Testing
- Kinesio Taping, Conventional Rehabilitation
- Where
- Turkey (Türkiye)
- Volunteers
- 32 people
- Sponsor
- Muğla Sıtkı Koçman University