Clinical trials: Congenital Malformation
Patent Foramen Ovale
PFx Closure System in Subjects With Cryptogenic Stroke,Transient Ischemic Attack,Migraine or Decompression Illness(PFO)
The primary objective of this study is to demonstrate the safety and performance of the PFx Closure System when utilized for patients with PFOs suffering from cryptogenic stroke, transient ischemic attack, migraine or…
- Trial name
- NCT00335296
- Testing
- PFx catheter
- Where
- 4 sites in Belgium, France and 1 more
- Volunteers
- 100 people
- Sponsor
- Cierra
Primary Immunodeficiency
An Open-label, Multicenter Study to Assess the Pharmacokinetics (PK), Safety, and Tolerability of Subcutaneous IgPro20 in Immunoglobulin (IG) Treatment-naïve Participants With Primary Immunodeficiency (PID)
This is an open-label, multicenter, phase 4 study in IG treatment-naïve participants with PID, conducted in the United States (US), to assess the PK, safety, and tolerability of IgPro20.
- Trial name
- NCT07076446
- Testing
- IgPro
- Where
- 8 sites in United States
- Volunteers
- 8 people
- Sponsor
- CSL Behring
Obesity
Prevention of Abdominal Incisional Hernia After Laparoscopic Digestive Surgery in Obese Patients (BMI>30kg/m2)
The hypothesis of this study is to show that after laparoscopic digestive surgery in obese patients (BMI>30kg/m2), the wearing of an abdominal support belt is an effective treatment for the Incisional Hernia and feasible in terms…
- Trial name
- EVINOV (NCT04537546)
- Testing
- OBESINOV elasto-compressive belt
- Where
- France
- Volunteers
- 350 people
- Sponsor
- Institut Mutualiste Montsouris
Atopic Dermatitis
Improvement In Scratch Behavior And Sleep In Patients With Atopic Dermatitis
This single blind study is to primarily evaluate wearable devices and Observer Reported Itch Assessment in children to assess reduction of itch and night-time scratch in response to Crisaborole treatment vs.
- Trial name
- PAD (NCT05200403)
- Testing
- Crisaborole 2%, Vehicle treatment, Accelerometry device for children
- Where
- United States
- Volunteers
- 72 people
- Sponsor
- Boston University
Cirrhosis, Congenital Heart Disease
Role of Sildenafil for Fontan Associated Liver Disease (SiFALD) Study
The purpose of this study is to determine if the medication, sildenafil (also known as Revatio), can slow or stop the progression of liver disease in patients who previously had a Fontan operation.
- Trial name
- SiFALD (NCT03154476)
- Testing
- Sildenafil
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Mayo Clinic
Sickle Cell Anemia
Longitudinal Changes in Exercise Capacity in Children and Young Adults With Sickle Cell Anemia
The purpose of this study is to use comprehensive exercise testing to examine longitudinal changes in exercise capacity over a 2 year period in children and young adults with sickle cell anemia.
- Trial name
- NCT01558076
- Where
- United States
- Volunteers
- 35 people, healthy volunteers welcome
- Sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
Cystic Fibrosis
(Study: Vertex IIS) Does Ivacaftor Alter Wild Type CFTR-Open Probability In The Sweat Gland Secretory Coil?
Clinical studies of lumacaftor + ivacaftor (combo therapy) produced better FEV1 (forced expiratory volume in 1 second) improvements than ivacaftor alone, without further improvement in sweat chloride results.
- Trial name
- NCT02310789
- Testing
- Ivacaftor, β-Adrenergic cocktail, Pilocarpine Nitrate 5%
- Where
- United States
- Volunteers
- 8 people, healthy volunteers welcome
- Sponsor
- Richard Barry Moss
Oxidative Phosphorylation Deficiencies, Electron Transport Chain Disorders, Mitochondrial
The NIH MINI Study: Metabolism, Infection, and Immunity in Inborn Errors of Metabolism
The Metabolism, Infection and Immunity (MINI) Study is a longitudinal natural history study at the National Institutes of Health (NIH) that aims to define the relationship between infection, immunity and clinical decline in…
- Conditions
- Oxidative Phosphorylation Deficiencies, Electron Transport Chain Disorders, Mitochondrial, Mitochondrial Disorders, Leigh Disease
- Trial name
- NCT01780168
- Where
- United States
- Volunteers
- 500 people, healthy volunteers welcome
- Sponsor
- National Human Genome Research Institute (NHGRI)
Infections, Pneumonia
Natural History, Management, and Genetics of the Hyperimmunoglobulin E Recurrent Infection Syndrome (HIES)
The Hyper IgE Syndromes (HIES) are primary immunodeficiencies resulting in eczema and recurrent skin and lung infections. Autosomal dominant Hyper IgE syndrome (AD-HIIES; Job's syndrome) is caused by STAT3 mutations, and is a…
- Conditions
- Infections, Pneumonia, Immune System Diseases, STAT3 Transcription Factor
- Trial name
- NCT00006150
- Where
- United States
- Volunteers
- 600 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Birth Asphyxia
Sildenafil to Repair Brain Injury Secondary to Birth Asphyxia
The investigators will determine the maximum tolerable dose of sildenafil and establish the pharmacokinetic and pharmacodynamic profile of sildenafil in human asphyxiated neonates treated with hypothermia.
- Trial name
- SANE-02 (NCT04169191)
- Testing
- Sildenafil Citrate
- Where
- Canada
- Volunteers
- 20 people
- Sponsor
- McGill University Health Centre/Research Institute of the McGill University Health Centre
Dermatologic Disease, Epidermolysis Bullosa
The State of Sexual Development in Children With Inherited Epidermolysis Bullosa
The aim of the study is to determine the state of sexual development in patients with inherited epidermolysis bullosa; the study is planned to include boys and girls aged 8 to 18 years with a diagnosis of epidermolysis bullosa…
- Conditions
- Dermatologic Disease, Epidermolysis Bullosa, Epidermolysis Bullosa Simplex, Epidermolysis Bullosa Dystrophica
- Trial name
- NCT05033574
- Testing
- Biochemical study of hormone levels, Ultrasound examination, Radiography of the hands
- Where
- Russia
- Volunteers
- 50 people
- Sponsor
- National Medical Research Center for Children's Health, Russian Federation
Alpha 1-Antitrypsin Deficiency
Study of DCR-A1AT in Healthy Adult Volunteers
This is a research study to test an experimental study drug (belcesiran, also known as DCR-A1AT). This drug is being tested to see if it helps people with a rare condition known as Alpha-1 Antitrypsin Deficiency, or A1ATD.
- Trial name
- NCT04174118
- Testing
- belcesiran
- Where
- 2 sites in New Zealand, Sweden
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Muscular Dystrophies, Cerebral Palsy
Cycle Exercise in Wheelchair Users With Muscular Dystrophy or Cerebral Palsy
Wheelchair bound patients often have pain in lower back and glutes, constipation and reduced quality of life - symptoms that exercise might ameliorate.
- Trial name
- NCT04677010
- Testing
- Cycle exercise
- Where
- Denmark
- Volunteers
- 33 people
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
Patent Ductus Arteriosus
Ibuprofen vs. Continuous Indomethacin in the Treatment of PDA
The purpose of this study is to determine whether closure of the PDA in premature neonates using IV ibuprofen vs continuous IV indomethacin has different side effects, eg.
- Trial name
- NCT00485160
- Testing
- Continuous indomethacin, ibuprofen
- Where
- Israel
- Volunteers
- 70 people
- Sponsor
- Shaare Zedek Medical Center
Cystic Fibrosis
Treatment of Dysglycemia Using Sitagliptin in Adolescents With Cystic Fibrosis
This pilot study will be conducted in adolescents with cystic fibrosis (CF) without diabetes but with abnormal glucose tolerance, and will assess the effects of sitagliptin on glucose regulation.
- Trial name
- NCT01721382
- Testing
- Sitagliptin
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Nemours Children's Clinic
Hereditary Angioedema (HAE)
Efficacy and Safety Study of DX-88 to Treat Acute Attacks of Hereditary Angioedema (HAE)
The purpose of this study is to determine if a subcutaneous dose of DX-88 (ecallantide; an investigational product) is safe and relieves symptoms of HAE in patients suffering from moderate to severe acute attacks of HAE.
- Trial name
- NCT00262080
- Testing
- ecallantide, Phosphate Buffer Saline (PBS),
- Where
- United States
- Volunteers
- 91 people
- Sponsor
- Shire
Polyneuropathy, Charcot Marie Tooth Disease (CMT)
Effects of Resistance Exercises in Hereditary Sensory-Motor Neuropathy (Charcot-Marie-Tooth Disease)
The goal of this clinical trial is to compare the effects of an 8-week resistance exercise training program on skeletal muscle quality, functional capacity, and quality of life in young individuals aged 15 to 29 years, with and…
- Trial name
- PNP (NCT07152197)
- Testing
- Progressive Resistance Exercise Training
- Where
- 2 sites in Chile
- Volunteers
- 22 people, healthy volunteers welcome
- Sponsor
- Universidad de La Frontera
Leukocyte Adhesion Deficiency Syndrome
Gene Therapy for Patients With Leukocyte Adherence Deficiency (Follow-Up of Phase 1 Trial)
This study will provide long-term monitoring of two patients who received gene therapy for leukocyte adherence deficiency (LAD) under the Food and Drug Administration investigational new drug study BB-IND-7949.
- Trial name
- NCT00023010
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- National Cancer Institute (NCI)
Healthy
A Study to Learn How Different Amounts of the Study Medicine Called PF-08049820 Are Tolerated and Act in the Body in Healthy Adults
The purpose of this study is to learn about the safety of the study medicine called PF-08049820 in healthy adults. The study will also see: how the body processes the study medicine and if food affects the amount of study…
- Trial name
- NCT06686797
- Testing
- PF-08049820
- Where
- United States
- Volunteers
- 71 people, healthy volunteers welcome
- Sponsor
- Pfizer
Uncontrolled Gout
Pegloticase and Methotrexate Co-administered in Participants With Uncontrolled Gout Who Previously Failed Pegloticase Monotherapy
This is a Phase 4, multicenter, open-label trial of pegloticase with methotrexate (MTX) in adult participants with uncontrolled gout who were previously treated with pegloticase without a concomitant immunomodulator and stopped…
- Trial name
- NCT04772313
- Testing
- Pegloticase, Methotrexate (MTX)
- Where
- 15 sites in United States
- Volunteers
- 11 people
- Sponsor
- Amgen
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