Clinical trials: Congenital Anomaly
Fetal Conditions, Maternal; Procedure
A Prospective Study on the Role of Karl Storz Curved and Straight Fetoscopes (11508AAK and 11506AAK) for Fetoscopic Intrauterine Procedures
In this research study, the investigators want to learn more about the role of new innovative surgical devices, the Karl Storz Curved and Straight Fetoscopes for in-utero surgery.
- Conditions
- Fetal Conditions, Maternal; Procedure, Pregnancy Related, Twin to Twin Transfusion Syndrome
- Trial name
- NCT06056635
- Testing
- Karl Storz Curved Scope, Karl Storz Straight Scope
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Boston Children's Hospital
Orofacial Clefts, Cleft Lip and Palate
Analgosedation With Ketamine, Nalbuphine, or Dexmedetomidine for Suture Removal in Children After Cleft Surgery
This prospective, randomized study is designed to compare the efficacy and safety of three sedative-analgesic agents-intramuscular ketamine, intranasal nalbuphine, and intranasal dexmedetomidine-for procedural sedation in…
- Conditions
- Orofacial Clefts, Cleft Lip and Palate, Alveolar Ridge Defect
- Trial name
- KID-CLEFT (NCT07340008)
- Testing
- ketamine, Nalbuphine, Dexmedetomidine
- Where
- Slovenia
- Volunteers
- 60 people
- Sponsor
- University Medical Centre Ljubljana
Hemophilia a
A Prospective Assessment of Bone Health in Patients With Severe Hemophilia A on Factor VIII vs Factor Mimetic Prophylaxis (Efa Emi Bone Health Study)
This study will compare bone mineral density in patients with severe hemophilia A receiving prophylaxis with emicizumab or efanesoctocog alfa.
- Trial name
- NCT07582276
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Arkansas Children's Hospital Research Institute
Marfan Syndrome, Marfan Syndrome Cardiovascular Manifestations
Living With Marfan Syndrome and Your Aorta
Marfan Syndrome (MFS) is a genetic disease affecting the eyes, skeleton, heart and arteries. Despite MFS affecting multiple organ systems, cardiovascular manifestations are the most serious and life threatening.
- Trial name
- LIMA I (NCT04776668)
- Where
- United Kingdom
- Volunteers
- 75 people
- Sponsor
- Barts & The London NHS Trust
Sickle Cell Disease, Pain
Integrative Medicine in Pain Management in Sickle Cell Disease, 2.0
The proposed research is to determine the clinical efficacy and neurobiological mechanisms of acupuncture analgesia in patients with sickle cell disease.
- Conditions
- Sickle Cell Disease, Pain, Acupuncture, Quantitative Sensory Testing
- Trial name
- NCT06725550
- Testing
- needling acupuncture, laser acupuncture
- Where
- United States
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- University of Cincinnati
Phenylketonurias
The Effect of Nursing Empowerment Program
The study has shown that the strengthening program applied to the families of children diagnosed with phenylketonuria (PKU) and the education given; It will be conducted in order to examine the effect of the family's coping…
- Trial name
- NCT05497050
- Testing
- Family education
- Where
- Turkey (Türkiye)
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Cukurova University
Sickle Cell Disease
Oxygen Therapy and Pregnancy in Sickle Cell Disease
The purpose of this study is to assess the efficiency of the preventive oxygen therapy on the occurrence of vaso-occlusive complications, which last more than 24 hours and require hospitalisation, in women with sickle cell…
- Trial name
- DRO2G (NCT02813850)
- Testing
- oxygen therapy
- Where
- France
- Volunteers
- 178 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Stroke, Cardiac Disease
Multifactorial Risk Stratification in Stroke Patients With Cardiac Disease
Prospective registry for multifactorial risk factor assessment, enrolling consecutive ischemic stroke/transient ischemic attack patients with structural, inflammatory, or arrhythmogenic cardiac disease, who are admitted to our…
- Trial name
- NCT04352790
- Volunteers
- 878 people
- Sponsor
- University Hospital Tuebingen
Atopic Dermatitis (AD), Cholesterol
A Study on the Efficacy of 2% Cholesterol Cream in Atopic Dermatitis
Atopic dermatitis is a common chronic inflammatory skin condition characterized by impaired skin barrier function, leading to increased transepidermal water loss (TEWL), dry skin, and itching.
- Conditions
- Atopic Dermatitis (AD), Cholesterol, Skin Barrier to Water Loss, Skin Allergy
- Trial name
- NCT07503197
- Testing
- Topical 2% cholesterol cream
- Where
- Thailand
- Volunteers
- 30 people
- Sponsor
- Institute of Dermatology, Thailand
Prader Willi Syndrome, Syndromic Obesity
Prader-Willi Syndrome Macronutrient Study
The overall objective is to explore the mechanisms by which macronutrients regulate food intake and weight gain in Prader Willi Syndrome (PWS).
- Conditions
- Prader Willi Syndrome, Syndromic Obesity, Childhood Obesity
- Trial name
- NCT02011360
- Testing
- Low Carbohydrate diet, Low Fat diet
- Where
- 2 sites in United States, Canada
- Volunteers
- 10 people
- Sponsor
- Duke University
Hereditary Sensory Neuropathy Type I
Hereditary Sensory Neuropathy Serine Trial
This is a randomised double-blind placebo-controlled trial of L-serine in Hereditary Sensory Neuropathy type 1 (HSN1) due to variants in SPTLC1/2 gene.
- Trial name
- SENSE (NCT06113055)
- Testing
- L-serine
- Where
- United Kingdom
- Volunteers
- 50 people
- Sponsor
- University College, London
Pneumonia, Bronchoalveolar Lavage
HCAPPED I: HCAP-Pcr in Emergency Department
Prospective Observational Study (on Diagnostic Procedure) - Single-Center, Non-Profit. A pilot study will be conducted on a prospective cohort of patients with HCAP (Healthcare-Associated Pneumonia).
- Conditions
- Pneumonia, Bronchoalveolar Lavage, Antimicrobial Treatment, Sputum
- Trial name
- HCAPPED-I (NCT06506617)
- Where
- Italy
- Volunteers
- 93 people
- Sponsor
- Azienda Ospedaliero-Universitaria Careggi
Foramen Ovale, Patent
The Paradigm II Trial: PFX Closure System in Subjects With Cryptogenic Stroke, Transient Ischemic Attack, Migraine or Decompression Illness
The primary objective of this study is to demonstrate the safety and efficacy of the PFX Closure System when utilized for patent foramen ovale (PFO) in patients suffering from cryptogenic stroke (undetermined cause of stroke)…
- Trial name
- NCT00196040
- Testing
- PFX Closure System
- Where
- Germany
- Volunteers
- 60 people
- Sponsor
- Cierra
Congenital Diaphragmatic Hernia
Diaphragmatic Hernia Research & Exploration, Advancing Molecular Science
The goal of this study is to identify genes that convey susceptibility to congenital diaphragmatic hernia in humans. The identification of such genes, and examination of their structure and function, will enable a delineation of…
- Trial name
- DHREAMS (NCT00950118)
- Where
- 14 sites in United States, Egypt
- Volunteers
- 3,000 people, healthy volunteers welcome
- Sponsor
- Columbia University
Methylmalonic Aciduria and Homocystinuria,Cblc Type, Genetic Disease
EPI-743 in Cobalamin C Defect: Effects on Visual and Neurological Impairment
The aim of the research is to investigate the safety and efficacy of EPI-743 treatment in patients with Cbl-C defect and related visual and neurological impairment.
- Conditions
- Methylmalonic Aciduria and Homocystinuria,Cblc Type, Genetic Disease, Retinopathy
- Trial name
- NCT01793090
- Testing
- Epi-743
- Where
- Italy
- Volunteers
- 30 people
- Sponsor
- Bambino Gesù Hospital and Research Institute
Fabry Disease
German Observational Multicenter Study of Patients With Fabry Disease Under Chaperone Therapy With Migalastat-HCl.
The objective of the study is to document long term data on treatment with Migalastat under "real world" conditions. The selection of patients is based on the SmPC/Fachinformation. The study duration/patient will be 2 years.
- Trial name
- NCT03135197
- Where
- Germany
- Volunteers
- 75 people
- Sponsor
- University Hospital Muenster
Mixed Hyperlipidemia
A Study of SYH2070 Injection in Participants With Mixed Hyperlipidemia
This is a randomized, double-blind, placebo-parallel, multicenter phase II clinical trial to evaluate the efficacy and safety of SYH2070 injection in participants with mixed hyperlipidemia.
- Trial name
- NCT07695597
- Testing
- SYH2070
- Where
- China
- Volunteers
- 240 people
- Sponsor
- CSPC Zhongnuo Pharmaceutical (Shijiazhuang) Co., Ltd.
Benign Breast Disease
Nuclear Morphology of Breast Cells in Ductal Lavage
Breast nipple aspirate fluids (NAF) are useful for non-invasive monitoring of the breast. NAF has been shown to exhibit large inter-individual differences in lipid peroxidation.
- Trial name
- NCT00259597
- Testing
- breast ductal lavage
- Where
- United States
- Volunteers
- 57 people, healthy volunteers welcome
- Sponsor
- University of Michigan
Phenylketonuria
GenSci144 Tablets Phase I Clinical Trial
This study is a Phase I single ascending dose clinical trial in which healthy adult volunteers were administered single oral doses of GenSci144 tablets at different levels.
- Trial name
- NCT07685210
- Testing
- GenSci144 tablets
- Where
- China
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.
Glycogen Storage Disease
Lingual Muscle Training in Late-Onset Pompe Disease (LOPD)
This study is being done to test the effects of Lingual Muscle Therapy (LMT) in patients with Late-Onset Pompe Disease (LOPD) who have tongue weakness.
- Trial name
- NCT03255213
- Testing
- Lingual Muscle Training
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Duke University