Clinical trials: Multiple Congenital Anomaly
Congenital Heart Disease
Multi-micronutrient Supplementation During Peri-conception and Congenital Heart Disease
The purpose of this study is to determine whether daily oral supplements of vitamin B complex along with folic acid or supplements of iron plus folic acid given to women during peri-conception can reduce the risk of congenital…
- Trial name
- NCT02537392
- Testing
- Vitamin B Complex and Folic Acid, Iron and Folic Acid, Folic Acid
- Where
- China
- Volunteers
- 7,315 people
- Sponsor
- Xi'an Jiaotong University
Dermatitis, Dermatitis, Atopic
Study to Investigate Efficacy and Safety of PF-04965842 in Subjects Aged 12 Years and Over With Moderate to Severe Atopic Dermatitis With the Option of Rescue Treatment in Flaring Subjects
B7451014 is a Phase 3 study to investigate PF-04965842 in patients aged 12 years and over with a minimum body weight of 40 kg who have moderate to severe atopic dermatitis.
- Conditions
- Dermatitis, Dermatitis, Atopic, Eczema, Skin Diseases
- Trial name
- NCT03627767
- Testing
- PF-04965842 100 mg, PF-04965842 200 mg
- Where
- 235 sites in United States, Argentina and 19 more
- Volunteers
- 1,235 people
- Sponsor
- Pfizer
Congenital Bleeding Disorder, Haemophilia A
Safety, Tolerability, and Pharmacokinetics Study of Turoctocog Alfa Pegol Injected Under the Skin in Patients With Haemophilia A
The trial is conducted in Asia, Europe and North America. The aim of the study is to evaluate the safety of administration under the skin of turoctocog alfa pegol (SC N8-GP) in patients with severe haemophilia A.
- Trial name
- alleviate 1 (NCT02994407)
- Testing
- turoctocog alfa pegol
- Where
- 19 sites in United States, Austria and 4 more
- Volunteers
- 50 people
- Sponsor
- Novo Nordisk A/S
Bloom Syndrome
Biological Significance of the Bloom's Syndrome Protein
Since 1960, persons with the very rare disorder Bloom's syndrome (BS) have been followed clinically, documenting clinical matters as obtained from their doctors.
- Trial name
- NCT00021437
- Where
- United States
- Sponsor
- National Center for Research Resources (NCRR)
Phenylketonuria
Pharmacodynamics, Safety, Tolerability and Pharmacokinetics of CDX-6114 in Patients With Phenylketonuria (PKU)
This study is an international, multi-center, randomized, double-blind, placebo-controlled, two-treatment, two-period cross-over study to evaluate the pharmacodynamics, safety, tolerability and pharmacokinetics of a single oral…
- Trial name
- NCT04085666
- Testing
- CDX 6114
- Where
- 2 sites in Australia, Germany
- Volunteers
- 18 people
- Sponsor
- Société des Produits Nestlé (SPN)
Multiple Sclerosis, Exercise Intervention
Regular Exercise Improves Physical Capacity and Promotes Neurotrophins in Patients With Multiple Sclerosis
ABSTRACT Objective: The investigators aimed to determine the effect of regular exercise on aerobic capacity, strength values, and plasma levels of nerve growth factor (NGF) and Neurotrophin-3 (NT-3) in patients with multiple…
- Conditions
- Multiple Sclerosis, Exercise Intervention, Neuro-Degenerative Disease
- Trial name
- NCT04944251
- Testing
- Regular Exercise
- Where
- Turkey (Türkiye)
- Volunteers
- 43 people
- Sponsor
- Ege University
Biotinidase Deficiency
Study of ORL-1B in Patients With Biotinidase Deficiency
An Open-label, Single-arm, Phase 2 Study of Biotin in Patients With Biotinidase Deficiency.
- Trial name
- NCT03269045
- Testing
- ORL-1B
- Volunteers
- 20 people
- Sponsor
- Orpha Labs
Cystic Fibrosis (CF)
MRI Assessment of Lung Airways in Cystic Fibrosis: Evaluate MRI's Ability to Detect Changes in Airway Structure .
This study is being done to determine whether MRI can produce high quality lung and airway images in healthy and CF patients and if MRI can be used to evaluate size and shape of the airways with computer assistance.
- Trial name
- UTE Airway MR (NCT07454681)
- Testing
- Advanced ultrashort echo time (UTE) MRI - Ultrashort echo time (UTE) imaging is a technique that can quickly capture the fast-decaying 1H lung signal., Multiple Breath Wash out (MBW)
- Where
- Canada
- Volunteers
- 76 people, healthy volunteers welcome
- Sponsor
- The Hospital for Sick Children
Charcot-Marie-Tooth Disease, Type 1A
A First in Human Study to Assess the Safety, Tolerability, and Pharmacokinetics of EDK060 in Adults With CMT1A.
The purpose of this study is to characterize the safety, tolerability, and pharmacokinetics of EDK060 as compared to placebo in adult patients with CMT1A.
- Trial name
- NCT07140614
- Testing
- EDK060, dose A, EDK060, dose B, EDK060, dose C
- Where
- 4 sites in Canada
- Volunteers
- 28 people
- Sponsor
- Novartis Pharmaceuticals
Leukemia, Lymphoma
Umbilical Cord Blood Stem Cell Transplant in Treating Patients With Hematologic Cancer or Other Disease
RATIONALE: Giving low doses of chemotherapy and total-body irradiation before a donor umbilical cord blood stem cell transplant helps stop the growth of cancer or abnormal cells.
- Conditions
- Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes
- Trial name
- NCT00423826
- Testing
- Busulfan, Cytarabine, Fludarabine phosphate
- Where
- United States
- Sponsor
- Barbara Ann Karmanos Cancer Institute
Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission
Palifermin in Preventing Chronic Graft-Versus-Host Disease in Patients Who Have Undergone Donor Stem Cell Transplant for Hematologic Cancer
RATIONALE: Growth factors, such as palifermin, may prevent chronic graft-versus-host disease caused by donor stem cell transplant.
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
- Trial name
- NCT01233921
- Testing
- palifermin, flow cytometry, laboratory biomarker analysis
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Martin, Paul
Overweight, Healthy
Specificities of the Eccentric Exercise: Energy Expenditure and Nature of Oxidized Substrates
Main objective of this clinical trial is to compare the oxidation rates of main carbohydrate and lipid substrates during exercises carried out in eccentric dynamic pedaling mode on a cycloergometer compared to concentric…
- Conditions
- Overweight, Healthy, Men
- Trial name
- Speed-Enso (NCT05319704)
- Testing
- Test of effort on cycle on concentric or excentric mode, Kinetics on concentric or excentric mode during test of effort
- Where
- France
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- University Hospital, Clermont-Ferrand
Costello Syndrome
Pathophysiology Analysis of "Costello Syndrome" on Cellular Models
The objective is to collect skin biopsies rom patients with Costello syndrome to analyze the molecular mechanisms responsible for this syndrome caused by a mutation in the HRAS gene and the effects of this mutation on energy…
- Trial name
- COSTELLO (NCT02812511)
- Testing
- Biopsy
- Where
- France
- Volunteers
- 9 people
- Sponsor
- University Hospital, Bordeaux
Hypohidrotic Ectodermal Dysplasia
Impact of Dysregulation of Core Body Temperature on Sleep in Patients With Hypohidrotic Ectodermal Dysplasia
The aim of the study is to compare sleep efficiency by means of actigraphy in patients with hypohidrotic ectodermal dysplasia with healthy controls.
- Trial name
- DEH-Somno (NCT05378932)
- Testing
- Actigraphy, Recording of proximal and distal skin temperature and temperature of the bedroom, Questionnaires assessing subjective sleep quality
- Where
- France
- Volunteers
- 22 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Sickle-Cell Disease
Sevuparin Infusion for the Management of Acute VOC in Subjects With SCD
A Multi-Centre, Phase II, Randomized, Double-Blind, Placebo-Controlled Study to investigate Efficacy and Safety of Sevuparin Infusion for the Management of Acute Vaso-Occlusive Crisis (VOC) in Subjects with Sickle-Cell Disease…
- Trial name
- NCT02515838
- Testing
- Sevuparin
- Where
- 20 sites in Bahrain, Jamaica and 5 more
- Volunteers
- 147 people
- Sponsor
- Modus Therapeutics AB
Autosomal Dominant Polycystic Kidney, Hypertension
Effect of Beetroot Juice on Reducing Hypertension in Autosomal Dominant Polycystic Kidney Disease
People with autosomal dominant polycystic kidney disease (ADPKD) develop high blood pressure and kidney disease. Previous studies have shown that a commonly occurring chemical, nitric oxide (NO), is reduced in ADPKD, and may…
- Conditions
- Autosomal Dominant Polycystic Kidney, Hypertension, Endothelial Dysfunction
- Trial name
- BEET-PKD (NCT05401409)
- Testing
- Beetroot juice, Nitrate-depleted beetroot juice
- Where
- 2 sites in Australia
- Volunteers
- 60 people
- Sponsor
- Western Sydney Local Health District
Von Hippel Lindau Disease
Data Collection Protocol for Patients With Von Hippel Lindau Disease
To collect information from patients with vHL disease. Information collected will include data on the status of the disease, any surgeries or therapies patients have received for vHL disease, and quality of life.
- Trial name
- NCT05955014
- Where
- United States
- Volunteers
- 125 people
- Sponsor
- M.D. Anderson Cancer Center
Left Ventricular Noncompaction, Isolated Noncompaction of the Ventricular Myocardium
Cardiac Computed Tomography: Characteristics of Isolated Left Ventricular Non-compaction
The diagnostic criteria for isolated left ventricular non-compaction cardiomyopathy (IVNC) have been well established for echocardiography and cardiac magnetic resonance, such validation is lacking for computed tomography (CT).
- Trial name
- NCT01470014
- Where
- Switzerland
- Volunteers
- 39 people
- Sponsor
- University of Zurich
Glycogen Storage Disease Type II
Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa Treatment
Primary Objective: To evaluate effect of 52-week treatment with Alglucosidase Alfa in the extension of survival and improvement of cardiomyopathy measured by Left Ventricular Mass Index in Chinese patients with infantile-onset…
- Trial name
- APOLLO-IOPD (NCT03687333)
- Testing
- ALGLUCOSIDASE ALFA (MYOZYME)
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Genzyme, a Sanofi Company
Hypoparathyroidism, DiGeorge Syndrome
Effects of PTH Replacement on Bone in Hypoparathyroidism
Hypoparathyroidism is a rare condition associated with a low level of parathyroid hormone (PTH) in the blood. Hypoparathyroidism can be genetic and show up in childhood, or it can occur later in life.
- Trial name
- NCT00395538
- Testing
- PTH 1-34
- Where
- United States
- Volunteers
- 46 people
- Sponsor
- National Institute of Dental and Craniofacial Research (NIDCR)
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