Clinical trials: Newborn Diseases
Cystic Fibrosis
Natural History Study of Exocrine Pancreatic Function in Infants With Cystic Fibrosis (CF)
The purpose of the study is to evaluate the natural history of exocrine pancreatic function by assessing Fecal elastase-1 (FE-1) in infants with CF during their first year of life.
- Trial name
- NCT06506773
- Where
- 50 sites in United States, Australia and 9 more
- Volunteers
- 79 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Congenital Adrenal Hyperplasia
Comparison of Chronocort Versus Standard Hydrocortisone Replacement Therapy in Participants Aged 16 Years and Over With Congenital Adrenal Hyperplasia
This study is a randomized, double-blind, active-controlled, phase III study of Chronocort® compared with immediate-release hydrocortisone replacement therapy in participants aged 16 years and over with Congenital Adrenal…
- Trial name
- CONnECT (NCT05063994)
- Testing
- Chronocort, Cortef
- Where
- 21 sites in United States, France and 1 more
- Volunteers
- 55 people
- Sponsor
- Neurocrine UK Limited
Postural Kyphosis, Neck Pain
Comparison Between Effect of Posterior Cervical Weighting and Deep Cervical Flexion Exercise on Forward Head Posture
It has been reported that the head protrudes forward relative to the body from the sagittal plane associated with the anterior shifting of the line of gravity (LOG) relative to the base of support (BOS).
- Conditions
- Postural Kyphosis, Neck Pain, Deformity of Spine, Deformity
- Trial name
- FHP (NCT04796051)
- Testing
- Neck orthosis - with posterior cervical weight, Deep cervical flexors exercise
- Where
- Egypt
- Volunteers
- 61 people, healthy volunteers welcome
- Sponsor
- Alexandria University
Alpha1-antitrypsin Deficiency, Healthy Volunteers
GLASSIA Infusion Rate Study
The purpose of this study was to generate sufficient safety and tolerability information in support of an increase in the infusion rate of intravenous GLASSIA in the prescribing information from 0.04 to 0.2 mL/kg/min.
- Trial name
- NCT01651351
- Testing
- Alpha1-proteinase inhibitor
- Where
- United States
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Baxalta now part of Shire
Alagille Syndrome
Characterization of Pulmonary Artery Stenoses in Alagille Syndrome - a Medical Record Review
Patients who have Alagille Syndrome (AGS) also frequently have blockages (or "stenoses") of their pulmonary arteries. Little is known about the degree or variability of these stenoses, or the effect of this disease on the right…
- Trial name
- NCT01515631
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Stanford University
Behavioral Manifestations of Lesch-Nyhan Disease
Trial of Kuvan in Lesch-Nyhan Disease
To assess the possibility that treatment with Kuvan (a form of tetrahydrobiopterin) will lessen the abnormal behavior and/or neurology commonly found in Lesch-Nyhan disease (LND); to assess biochemical changes as measured in…
- Trial name
- NCT00935753
- Testing
- sapropterin
- Where
- United States
- Sponsor
- University of California, San Diego
Pain, Acute, Congenital Heart Disease
Transverse Thoracic Plane Block in the Pediatric Cardiac Patient
Postoperative pain after cardiothoracic surgery can be a significant problem interfering with recovery, yet difficult to manage due to the sedating effects of opiates.
- Trial name
- TTPPCT (NCT04138745)
- Testing
- Transverse Thoracic Plane Block
- Where
- United States
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Ochsner Health System
Diabetic Polyneuropathy
The Effect of Rosuvastatin in Diabetic Polyneuropathy: A Phase IIa Randomized Double-blind Placebo-controlled Study
To evaluate the impact of oral rosuvastatin in diabetic polyneuropathy, and the role of lipid peroxidation and nerve growth factor.
- Trial name
- NCT01622777
- Testing
- Rosuvastatin
- Where
- Mexico
- Volunteers
- 34 people
- Sponsor
- Alejandra Guillermina Miranda Diaz
Neurodevelopmental Disorders, Congenital Heart Disease
Altered Cerebral Growth and Development in Infants With Congenital Heart Disease
Background: Congenital heart disease (CHD) is the most frequent inborn defect with an incidence of 1 in 100 newborns per year, i.e. 800 children born in Switzerland per year.
- Conditions
- Neurodevelopmental Disorders, Congenital Heart Disease, Brain Injuries, Fetal Growth Retardation
- Trial name
- NCT04233775
- Where
- Switzerland
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- University Children's Hospital, Zurich
ATTR-CM (Transthyretin Amyloid Cardiomyopathy), Heart Failure
Right Ventricular Septal Biopsy to Detect Cardiac Amyloidosis
People eligible for this study have had the suggestion from their doctor to receive a cardiac pacemaker or defibrillator to treat a cardiac condition as part of their routine care.
- Trial name
- RV-CAM (NCT07784712)
- Volunteers
- 70 people
- Sponsor
- Geisinger Clinic
Primary Immune Deficiency Disorder
Identification of New Inborn Errors of Immunity
Analysis of DNA samples of patients with molecularly undetermined PID by whole exome/genome sequencing. Transcriptome analysis of patients with molecularly undetermined PID.
- Trial name
- NCT03414528
- Testing
- Diagnostic Test
- Where
- Switzerland
- Volunteers
- 9 people, healthy volunteers welcome
- Sponsor
- University of Zurich
Lynch Syndrome
Predictive Factor Study of the Occurrence of Endometrial Cancer in Patients With Lynch Syndrome
Lynch Syndrome increases Endometrial Cancer Occurrence in women due to failure in MisMatch Repair System. Lifetime Cumulative risk of developing endometrial Cancer is approximatively 40% in women with Lynch Syndrome.
- Trial name
- PrediLynch (NCT04452266)
- Testing
- questionnaire assesment
- Where
- France
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- University Hospital, Lille
Dry Eye Syndrome, Ptosis
The Effect of Eyelid Surgery on Dry Eye - a Prospective Study
The investigators aim to assess the effect of blepharoplasty with or without muller muscle resection on the symptoms of dry eye syndrome.
- Conditions
- Dry Eye Syndrome, Ptosis, Dermatochalasis
- Trial name
- NCT02376556
- Where
- Israel
- Volunteers
- 54 people
- Sponsor
- Sheba Medical Center
Elasticum, Incomplete Pseudoxanthoma
The Characteristics of Backscattering With Depth in the Progression of Keratoconus
To investigate the variation in backscattering with depth between forme fruste keratoconus (FFKC), keratoconus, and normal eyes; to determine the backscattering changes in the characteristics of keratoconus progression; and to…
- Trial name
- NCT06050629
- Where
- China
- Volunteers
- 223 people, healthy volunteers welcome
- Sponsor
- Tianjin Eye Hospital
Hemophilia A, Factor VIII Deficiency
A Study Comparing Factor Level and Inhibitor Titer Testing Results Drawn From Central Venous Lines and Venipuncture
The purpose of this research study is to see if factor levels and inhibitor levels in Hemophilia A and B subjects are accurate when they are drawn from a central venous line (CVL) instead of from a peripheral stick.
- Conditions
- Hemophilia A, Factor VIII Deficiency, Hemophilia, Hemophilia B
- Trial name
- CVL#2 (NCT02402829)
- Testing
- Peripheral Vein Blood draw
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Children's Mercy Hospital Kansas City
G6PD Deficiency
Validation of Diagnostics to Identify Glucose-6-phosphate Dehydrogenase Activity in the US
The primary objective of this study is to assess the accuracy of the SD Biosensor STANDARD G6PD Analyzer in measuring G6PD activity when used by trained health care workers.
- Trial name
- NCT04010695
- Testing
- SD Biosensor G6PD Analyzer, Pointe Scientific Test Kit, HemoCue System
- Where
- United States
- Volunteers
- 250 people, healthy volunteers welcome
- Sponsor
- PATH
Behçet Disease
Plasma Soluble Urokinase Plasminogen Activator Receptor and Behçet's Disease .
The purpose of the study is to determine whether plasma levels of the soluble urokinase plasminogen activator(suPAR) can serve as a blood-based biomarker for diagnosis of Behçet's disease and its correlation with disease activity.
- Trial name
- NCT04105439
- Volunteers
- 90 people
- Sponsor
- Assiut University
Healthy, Head and Neck Cancer
Rehabilitation Assessment and Progress Tracking System Based on Video Images for Dysphagic Patients
The current cervical range of motion (CROM) and tongue movement measurement methods are not reliable between observers, which is not practical in real-world usage.
- Trial name
- NCT05141539
- Testing
- Rehabilitation assessment software, VFSS and FOIS
- Where
- Thailand
- Volunteers
- 30 people
- Sponsor
- Mahidol University
Gaucher Disease
Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial
Gaucher disease, the most prevalent lysosomal storage disorder, is caused by mutations in the human glucocerebrosidase gene (GCD) leading to reduced activity of the lysosomal enzyme glucocerebrosidase and thereby to the…
- Trial name
- NCT00705939
- Testing
- Taliglucerase alfa
- Where
- 11 sites in United States, Australia and 6 more
- Volunteers
- 45 people
- Sponsor
- Pfizer
Hereditary Breast and Ovarian Cancer Syndrome
Enhance the Use of Genetic Counseling and Testing in Latinas
The investigators will conduct a two-arm RCT to evaluate the preliminary effectiveness of a culturally-adapted video developed by the team vs.
- Trial name
- NCT04544501
- Testing
- Culturally-Targeted Video, FORCE Fact Sheet
- Where
- 4 sites in United States
- Volunteers
- 61 people, healthy volunteers welcome
- Sponsor
- Georgetown University
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