Clinical trials: NEWBORN
Hereditary Hemorrhagic Telangiectasia
A Study to Evaluate ALN-6400 in Healthy Volunteers and Patients With Hereditary Hemorrhagic Telangiectasia (HHT)
The purpose of this study is to: evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of single ascending doses of ALN-6400 in healthy volunteers evaluate the efficacy, safety, tolerability and PD of…
- Trial name
- InsigHHT (NCT06659640)
- Testing
- ALN-6400
- Where
- 16 sites in United States, Australia and 4 more
- Volunteers
- 89 people, healthy volunteers welcome
- Sponsor
- Alnylam Pharmaceuticals
Hemophilia A
Pharmacokinetic Assessment of Standard Half-Life (SHL) FVIII, Extended Half-Life (EHL) FVIII, and Efanesoctocog Alfa (BIVV001) in Severe Hemophilia A
Primary objective To assess the half-life of BIVV001, Standard Half-Life (SHL) rFVIII and Extended Half-Life (EHL) rFVIII after a single intravenous (IV) injection Secondary objectives To characterize additional pharmacokinetic…
- Trial name
- NCT05042440
- Testing
- Efanesoctocog alfa, Octocog alfa, Rurioctocog alfa pegol
- Where
- Bulgaria
- Volunteers
- 13 people
- Sponsor
- Sanofi
Mesial Temporal Lobe Epilepsy
AMT-260 Gene Therapy Study in Adults With Unilateral Refractory Mesial Temporal Lobe Epilepsy
The main goals of this clinical study are to learn if AMT-260 is safe and tolerable and works to reduce the frequency of seizures in adults with unilateral mesial temporal lobe epilepsy (MTLE).
- Trial name
- GenTLE (NCT06063850)
- Testing
- AAV9-hSyn1-miGRIK2
- Where
- 18 sites in United States
- Volunteers
- 12 people
- Sponsor
- UniQure Biopharma B.V.
Human Papillomavirus Infection, HPV
Human Papillomavirus in Young People Epidemiological Research 4
Anal cancer is overrepresented among gay, bisexual and other men who have sex with men (MSM), particularly those living with HIV.
- Conditions
- Human Papillomavirus Infection, HPV, HPV Infection, Anal Cancer
- Trial name
- HYPER4 (NCT05587738)
- Where
- Australia
- Volunteers
- 500 people
- Sponsor
- Monash University
Atopic Dermatitis, Allergy
Bathing Babies and Allergy
Atopic dermatitis (AD) is a chronic allergic skin disease with onset in early childhood and increasing prevalence in Westernized countries. Current well newborn guidelines for washing babies with soap were adopted by U.S.
- Trial name
- BBA (NCT03050658)
- Where
- United States
- Volunteers
- 28 people, healthy volunteers welcome
- Sponsor
- University of Virginia
Embolic Stroke of Undetermined Source, Transient Ischemic Attack
RoBotic TCD Ultrasound BubbLe Study Compared to Transthoracic Echocardiography for Detection of Right to Left Shunt
This study is a multi-center, prospective, single-arm, non-significant risk (NSR) device study in which up to 150 evaluable subjects with suspicion of embolic stroke of undetermined source (ESUS) will be evaluated with NB-IS TCD…
- Conditions
- Embolic Stroke of Undetermined Source, Transient Ischemic Attack, Right-To-Left Atrial Shunt, Patent Foramen Ovale
- Trial name
- BUBL (NCT04604015)
- Testing
- NeuralBot Investigational System, Transthoracic Echocardiography (TTE)
- Where
- 7 sites in United States
- Volunteers
- 154 people
- Sponsor
- NeuraSignal Inc dba NovaSignal
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
Stillbirth, Neonatal Death
Accelerating Newborn Survival in Ghana Through a Low-dose, High-frequency Health Worker Training Approach
This study assesses the effect of a low-dose, high-frequency training approach on long-term evidence-based skill retention among skilled birth attendants and impact on adverse birth outcomes at hospitals in Ghana.
- Trial name
- NCT03290924
- Testing
- Low dose high frequency health worker training approach, Active Comparison
- Volunteers
- 40 people
- Sponsor
- Jhpiego
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
Shock
Dobutamine in the Treatment of Haemodynamic Insufficiency in the Immediate Postnatal Period
Haemodynamic insufficiency after birth is seen commonly in babies born prematurely and is associated with adverse outcomes. In current clinical practice, a combination of blood pressure and clinical signs is used to guide…
- Trial name
- NeoCirc-001 (NCT03311178)
- Testing
- Dobutamine
- Where
- Spain
- Volunteers
- 15 people
- Sponsor
- Fundacion para la Investigacion Biomedica del Hospital Universitario la Paz
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