Clinical trials: Neonatal, open to volunteers
Friedreich Ataxia, Cardiomyopathies
A Study to Investigate the Safety, Tolerability, and Pharmacokinetics of ATI-105 in Healthy Adults
The goal of this clinical trial is to learn how ATI-105 administered to healthy participants at works in the body at different doses and with or without food.
- Conditions
- Friedreich Ataxia, Cardiomyopathies, Central Nervous System Diseases, Neuromuscular Diseases
- Trial name
- NCT07869654
- Testing
- ATI-105
- Where
- United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Adjuvia Therapeutics, Inc.
Cystic Fibrosis (CF), Bronchiectasis Adult
Novel Sensors and Artificial Intelligence for Detection of Acute Pulmonary Exacerbations in Cystic Fibrosis and Bronchiectasis
The goal of this observational study is to learn about new sensors that measure changes in the body that happen during a chest infection, such as breathing rate and heart rate.
- Trial name
- NOVEL (NCT07289100)
- Testing
- Non-Interventional Study
- Where
- United Kingdom
- Volunteers
- 50 people
- Sponsor
- Papworth Hospital NHS Foundation Trust
Beta-Thalassemia
A Multiple Ascending Dose Study of 9MW3011 in Patients With Non-transfusion-dependent β-thalassemia
This is a phase Ib, randomized, double-blind, placebo-controlled, multiple ascending dose study . The objectives of the study are to evaluate the safety , tolerability, pharmacokinetics(PK), pharmacodynamics(PD), and…
- Trial name
- NCT06772766
- Testing
- 9MW3011
- Where
- 2 sites in China
- Volunteers
- 40 people
- Sponsor
- Mabwell (Shanghai) Bioscience Co., Ltd.
Brow Ptosis, Dermatochalasis
Enhancing the Durability of Thread-Assisted Brow Suspension Through Upper Blepharoplasty-Assisted Ligament Release
This prospective clinical study aims to investigate whether combining upper blepharoplasty with limited release of the lateral brow-retaining ligaments improves the long-term effectiveness of barbed thread brow suspension.
- Conditions
- Brow Ptosis, Dermatochalasis, Periorbital Aging
- Trial name
- NCT07879456
- Testing
- Upper Blepharoplasty, Barbed Thread Brow Suspension, Lateral Brow-Retaining Ligament Release
- Where
- Turkey (Türkiye)
- Volunteers
- 90 people
- Sponsor
- Muhammed Mustafa Demirok
Epstein-Barr Virus Infections, Primary Immune Deficiency Disorder
EBV-specific Cytotoxic T-lymphocytes (CTLs) for Refractory EBV Infection
Related donor Epstein-Barr Virus (EBV) specific cytotoxic T cells (CTLs) manufactured with the Miltenyi CliniMACS Prodigy Cytokine Capture System will be administered in children, adolescents and young adults with refractory EBV…
- Trial name
- NCT03266653
- Testing
- cytotoxic t-lymphocytes
- Where
- 8 sites in United States
- Volunteers
- 20 people
- Sponsor
- New York Medical College
Myelomeningocele
In Utero Surgery for Fetal Myelomeningocele: Decision-making Mechanisms and Psychological Impact of Prenatal Therapy
Myelomeningocele is a malformation of the spine and spinal cord, generally diagnosed prenatally, and responsible for a complex disability for the unborn child.
- Trial name
- PriumPsy (NCT06796972)
- Testing
- Semi-structured interviews with a psychologist
- Where
- France
- Volunteers
- 44 people, healthy volunteers welcome
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia
Palliative Care Needs of Children With Rare Diseases and Their Families
The palliative care needs of family caregivers of children with rare diseases and their children are largely unmet, including the need for support to prepare for future medical decision making.
- Conditions
- Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia, Asparagine Synthetase Deficiency, CHARGE Syndrome
- Trial name
- FACE-Rare (NCT06938542)
- Testing
- Family Centered pediatric palliative care for family caregivers of children with rare diseases.
- Where
- United States
- Volunteers
- 480 people, healthy volunteers welcome
- Sponsor
- Children's National Research Institute
Premature Birth, Premature Infant
Growing Little PEAPODS Study
The goal of this clinical trial is to learn more about how the food and nutrition babies receive while in the Neonatal Intensive Care Unit (NICU) influences their ability to gain weight and fat-free mass, and their future growth…
- Conditions
- Premature Birth, Premature Infant, Premature, Intrauterine Growth Restriction
- Trial name
- NCT06226051
- Testing
- PEAPOD Infant Body Composition measuring device
- Where
- United States
- Volunteers
- 120 people
- Sponsor
- University of Wisconsin, Madison
Refractory Hypercholesterolemia, Familial Hypercholesterolemia
Safety and Efficacy Study of NGGT006 in Refractory Hypercholesterolemia Patients
This is an early phase 1, open-label, single-center, dose-escalation pilot trial to evaluate the safety and efficacy of an intravenous infusion of NGGT006 in patients with refractory Hypercholesterolemia diagnosed by gene testing…
- Trial name
- NCT06293729
- Testing
- NGGT006
- Volunteers
- 9 people
- Sponsor
- Suzhou Municipal Hospital
Neurofibromas, Cutaneous, Neurofibromatosis 1
Assessing the Efficacy of Repeat, Monthly Treatments of Deoxycholate for NF1 Associated Cutaneous Neurofibromas (cNFs)
The goal of this clinical trial is to evaluate the tolerability and effectiveness of multiple treatments of an FDA-approved drug in those with Neurofibromatosis Type 1 (NF1) Cutaneous Neurofibromas (cNFs).
- Trial name
- NCT06300502
- Testing
- Kybella
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Massachusetts General Hospital
Shwachman-Diamond Syndrome, SDS
Shwachman-Diamond Syndrome Global Patient Survey and Partnering Platform
The Shwachman-Diamond Syndrome Global Patient Survey and Collaboration Program (SDS-GPS) is an opportunity for patients and their families - from anywhere in the world - to share their experience living with SDS via a safe…
- Conditions
- Shwachman-Diamond Syndrome, SDS, IBMF, Congenital Neutropenia
- Trial name
- SDS-GPS (NCT06999954)
- Where
- United States
- Volunteers
- 8,000 people
- Sponsor
- Shwachman-Diamond Syndrome Alliance Inc
Preeclampsia, Intrauterine Growth Restriction
sFlt1/PlGF and Planned Delivery to Prevent Preeclampsia at Term.
Preeclampsia (PE) affects ~5% of pregnancies. Although improved obstetrical care has significantly diminished associated maternal mortality, PE remains a leading cause of maternal morbidity and mortality in the world.
- Conditions
- Preeclampsia, Intrauterine Growth Restriction, Maternal Hypertension, Neonatal Outcome
- Trial name
- PE37 (NCT04766866)
- Testing
- sFlt1/PlGF screening in maternal blood at 35 to 36.6 weeks of gestation
- Where
- 21 sites in Belgium, Colombia and 7 more
- Volunteers
- 9,132 people, healthy volunteers welcome
- Sponsor
- Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau
Viral Infection, Hematopoietic Stem Cell Transplantation (HSCT)
Adoptive T Lymphocyte Administration for Chronic Norovirus Treatment in Immunocompromised Hosts
This is a Phase I dose-escalation study to evaluate the safety of norovirus -specific T-cell (NST) therapy for chronic norovirus infection in participants following hematopoietic stem cell transplantation (HSCT) or who are…
- Conditions
- Viral Infection, Hematopoietic Stem Cell Transplantation (HSCT), Primary Immunodeficiency Disorders (PID)
- Trial name
- ATLANTIC (NCT04691622)
- Testing
- Norovirus -specific T-cell (NST) therapy
- Where
- 3 sites in United States
- Volunteers
- 48 people
- Sponsor
- Children's National Research Institute
Atopic Dermatitis (Eczema), Atopic Dermatitis Eczema
Phase 2 Randomized Double-blind Study to Assess Topical Zabalafin Hydrogel Vs Vehicle in Mild to Moderate AD.
A Phase 2b study investigating the efficacy of zabalafin in people with mild to moderate atopic dermatitis (eczema).
- Conditions
- Atopic Dermatitis (Eczema), Atopic Dermatitis Eczema, Atopic Dermatitis, Atopic Dermatitis (AD)
- Trial name
- CLEAR-AD1 (NCT06855745)
- Testing
- zabalafin
- Where
- 12 sites in Australia
- Volunteers
- 72 people
- Sponsor
- Alphyn Biologics
Down Syndrome
Rebound Therapy on Ventilatory Functions in Down Syndrome
The purpose of this study is to examine the effects of rebound therapy on ventilatory functions in children with Down Syndrome.
- Trial name
- NCT07425353
- Testing
- Chest Physical Therapy Protocol, Rebound Therapy Protocol
- Volunteers
- 40 people
- Sponsor
- Beni-Suef University
Glucose Metabolism Disorders in Cystic Fibrosis
Reproducibility of Continuous Glucose Monitoring (CGM) in the Assessment of Glucose Metabolism Disorders in Children With Cystic Fibrosis
Glucose metabolism disorders in cystic fibrosis (CF-GMD) are common and have a negative clinical impact. Screening currently relies on the oral glucose tolerance test (OGTT); however, this procedure is burdensome and complex for…
- Trial name
- CGMUCODIAB (NCT07851831)
- Testing
- Continuous Glucose Monitoring, Eating Habits Questionnaire, Use of an actigraphy watch
- Where
- France
- Volunteers
- 30 people
- Sponsor
- Hospices Civils de Lyon
ADPKD (Autosomal Dominant Polycystic Kidney Disease)
A Study to Investigate the Effects of JMKX003142 on QTc Interval in Healthy Adults
The primary objective of this study is to assess the effect of a therapeutic and supratherapeutic oral dose of JMKX003142 on the corrected cardiac QT interval relative to a placebo in healthy participants
- Trial name
- NCT07470177
- Testing
- JMKX003142 tablets
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Jemincare
Huntington Disease
A Randomized Study of SPK-10001 Gene Therapy in Participants With Huntington's Disease
The main goal of this study is to evaluate the safety, tolerability, and preliminary efficacy of SPK-10001 in participants with Huntington's Disease.
- Trial name
- NCT06826612
- Testing
- SPK-10001
- Where
- 5 sites in United States
- Volunteers
- 53 people
- Sponsor
- Hoffmann-La Roche
Atopic Dermatitis
Changes in Skin Myco- and Microbiome After Lebrikizumab Treatment
The goal of this observational study is to characterize the skin myco- and microbiome of patients with atopic dermatitis before and after initiation of treatment with lebrikizumab.
- Trial name
- NCT07807228
- Where
- Denmark
- Volunteers
- 60 people
- Sponsor
- University Hospital Bispebjerg and Frederiksberg
Eczema/Atopic Dermatitis, Otitis Externa
Nigella Sativa Oil for Eczematous Otitis Externa
Eczematous otitis externa is a common inflammatory condition of the external auditory canal characterized by itching, discomfort, discharge, and pain.
- Trial name
- NCT07525505
- Testing
- Mometasone Furoate (MF), Nigella sativa oil
- Where
- Turkey (Türkiye)
- Volunteers
- 85 people
- Sponsor
- Bezmialem Vakif University
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