Clinical trials: Neonatal, open to volunteers
Huntington Disease
Optimizing Parameters of Low-Intensity Focused Ultrasound for Pallidal Modulation in Huntington's Disease
The purpose of this research study is to determine the optimal pulse repetition frequency of low-intensity focused ultrasound that is safe and effective in improving motor symptoms in patients with Huntington's disease.
- Trial name
- NCT07513844
- Testing
- Low-Intensity Focused Ultrasound Stimulation（LIFU）
- Where
- China
- Volunteers
- 24 people
- Sponsor
- First Affiliated Hospital of Fujian Medical University
Sickle Cell Disease, Cerebral Stroke
Cerebrovascular Reactivity Measurements With High-Density Diffuse Optical Tomography
The purpose of the study protocol is to identify imaging biomarkers for brain tissue under high metabolic stress at risk for permanent injury.
- Trial name
- HDDOT CVR (NCT07776444)
- Testing
- Carbon Dioxide
- Where
- United States
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Washington University School of Medicine
Sickle Cell Disease
School Readiness Intervention for Preschool Children With Sickle Cell Disease
The study participant is being asked to take part in this clinical trial, a type of research study, because the participant is a young child with sickle cell disease or the caregiver of a child with sickle cell disease.
- Trial name
- NCT06367192
- Testing
- The adapted Kids in Transition to School Intervention, Standard school resources
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- St. Jude Children's Research Hospital
Premature Infants, Very Premature Infants
Anakinra Pilot 2 - A Study to Optimise Dose and Route of Administration of Anakinra in Preterm Infants
A phase 2 randomised, three-arm, parallel-group, dose-ranging trial to determine safety, efficacy and optimal dosing of intravenous anakinra in premature neonates, with subcutaneous pharmacokinetic sub-study.
- Conditions
- Premature Infants, Very Premature Infants, Inflammation
- Trial name
- AP2 (NCT07254000)
- Testing
- Anakinra (Kineret®)
- Where
- 2 sites in Australia, New Zealand
- Volunteers
- 24 people
- Sponsor
- Monash Medical Centre
Sickle Cell Disease
Sickle Cell Disease Biofluid Chip Technology (SCD BioChip)
'Sickle-shaped' anemia was first clinically described in the US in 1910, and the mutated heritable sickle hemoglobin molecule was identified in 1949.
- Trial name
- NCT02824471
- Testing
- SCD Group
- Where
- United States
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- University Hospitals Cleveland Medical Center
Preterm Birth Complication, Preterm Birth
Personalized Care for Prenatal Stress Reduction & Prevention of Preterm Birth (PTB) Disparities
The goal of this clinical trial is to learn if a personalized prenatal support program [(Personalized Toolkit Building a Comprehensive Approach to Resource optimization and Empowerment in Pregnancy \& Beyond, (PTBCARE+)] works to…
- Conditions
- Preterm Birth Complication, Preterm Birth, Preterm Birth Recurrence, Preeclampsia
- Trial name
- PTBCARE+ (NCT06915428)
- Testing
- Care coordination, Electronic massage, support gift #1
- Where
- United States
- Volunteers
- 1,228 people
- Sponsor
- University of North Carolina, Chapel Hill
Duchenne Muscular Dystrophy (DMD)
DMD and Gamified Physiotherapy
Duchenne Muscular Dystrophy (DMD) is a progressive neuromuscular disease that limits children's physical function, mobility, and participation in daily life.
- Trial name
- Gamified DMD (NCT07368400)
- Testing
- Gamified Mobile Physiotherapy Exercise Application, Standard Home Exercise Instructions
- Volunteers
- 46 people
- Sponsor
- Başak Çağla Arslan
Sickle Cell Disease, Venous Thromboembolism
Comparison of Thrombin Generation, Platelet Activation and Aggregation Profiles in Patients With Sickle Cell Disease Depending on Their SS or SC Genotype (DrepAgreg)
Sickle cell disease (SCD) is characterized by chronic hemolytic anemia, painful crises called vaso-occlusive crises (VOC) and chronic inflammation.
- Trial name
- DrepAgreg (NCT07852442)
- Where
- 2 sites in France, Guadeloupe
- Volunteers
- 60 people
- Sponsor
- Centre Hospitalier Universitaire de la Guadeloupe
Newborn, Infant, Disease, Neurodevelopmental Disorders
Perinatal Covid-19 Infection, NO Pathway, and Minipuberty
Some evidence exists that SARS-COV-2 may infect pituitary axis, and therefore may alter hypothalamic function. Whether perinatal COVID-19 is associated with alterations in the maturation of the Hypothalamic-Pituitary-Gonadal…
- Trial name
- miniNO-COVID (NCT04952870)
- Testing
- Inhaled NO, routine care
- Where
- 2 sites in France, Greece
- Volunteers
- 180 people
- Sponsor
- University Hospital, Lille
Eczema Atopic Dermatitis, Eczema
A Double-Blind, Randomized, Vehicle-Controlled Phase 2 Study to Assess the Efficacy and Safety of GX-03 in Adult Subjects With Moderate to Severe Atopic Dermatitis (Eczema)
This is a Phase 2, two-stage, adaptive, 1:1 randomized, double-blind, vehicle-controlled clinical study evaluating the efficacy, safety, and tolerability of GX-03 topical ointment in adult subjects with atopic dermatitis…
- Conditions
- Eczema Atopic Dermatitis, Eczema, Atopic Dermatitis
- Trial name
- GX-03 in AD (NCT07355075)
- Testing
- GX-03, Vehicle
- Where
- United States
- Volunteers
- 190 people
- Sponsor
- Turn Therapeutics
Wilson Disease, D-Penicillamine
Wilson's Disease Treated With D-Penicillamine: Characterization of Skin Damage Secondary to Treatment by Measuring Skin Elasticity
Wilson's disease is a genetic disorder, resulting from an anomaly present on the ATP7B gene located on chromosome 13, causing a progressive accumulation of copper in various organs such as the liver, nervous system and cornea…
- Conditions
- Wilson Disease, D-Penicillamine, Effect of D-penicilline on Cutaneous Elastity of Wilson's Patient
- Trial name
- WILDERME (NCT06945081)
- Testing
- Preparation of forearm molds with SILFLO® silicone
- Where
- 2 sites in France
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Saint Etienne
Cystic Fibrosis (CF)
Pharmacokinetics of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis
The goal of this clinical trial is to support the development of personalized therapy tailored to each patient's individual characteristics.
- Trial name
- NCT07809867
- Testing
- Elexacaftor, Tezacaftor, Ivacaftor, Ivacaftor (VX-770)
- Where
- Italy
- Volunteers
- 50 people
- Sponsor
- Istituto Giannina Gaslini
Stargardt, Stargardt's Disease
An Observational Study in Subjects to Follow the Progression of Stargardt Disease Type 1 (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ABCA4 Gene
This is an Observational Study to Follow the Progression of Stargardt Disease Type 1 (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ABCA4 Gene This is a multicenter study which will enroll approximately 75…
- Conditions
- Stargardt, Stargardt's Disease, Stargardt Disease, STGD1
- Trial name
- POLARIS (NCT06435000)
- Where
- 20 sites in United States, Germany and 1 more
- Volunteers
- 75 people
- Sponsor
- Splice Bio
Spinal Muscular Atrophy Type I
Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1
GB221 is a gene therapy that delivers a working SMN1 gene to the motor neurons of people with spinal muscular atrophy (SMA) Type 1. This study will evaluate the safety, tolerability and efficacy of GB221 in two groups: 1.
- Trial name
- NCT07070999
- Testing
- GB221
- Where
- Brazil
- Volunteers
- 22 people
- Sponsor
- Gemma Biotherapeutics
Dermatitis, Atopic, Eczema
Comparative Study of Crisaborole vs. Tacrolimus in Atopic Dermatitis
This study will compare the efficacy and safety of crisaborole ointment with tacrolimus ointment, in the treatment of patients with mild-to-moderate atopic dermatitis.
- Trial name
- NCT07367932
- Testing
- Application of crisaborole twice daily, Tacrolimus ointment in atopic dermatitis
- Volunteers
- 30 people
- Sponsor
- Cairo University
Huntington Disease
Longitudinal Endpoint Assessment of Disease Burden in HD
LEAD-HD is intended to collect and analyze self-reported health information from individuals with Huntington Disease (HD) or prodromal HD participating in a 24-month longitudinal natural history study using remote technologies.
- Trial name
- LEAD-HD (NCT06475898)
- Where
- United States
- Volunteers
- 600 people
- Sponsor
- Huntington Study Group
Congenital Anomaly
Examination of Pregnant Women in Third Trimester by Ultrasound to Detect Any Congenital Anomalies
this study aims to help the physicians to evaluate the practice of routine Third trimestric fetal scan whether it should be routinely performed to all cases including those with low risk singleton pregnancies.
- Trial name
- NCT06628063
- Testing
- ultrasound evaluation
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Sohag University
Aneurysm Aortic, Aneurysm Aortic Root
Strength Tracking From Wall Tichness Over Aortic Biomechanics (STORM)
The aim of the study is to analyse the resected portion of aneurysmatic ascending aorta in order to assess its strength to rupture, standardise the various measurements performed within the lab and try to correlate these…
- Trial name
- STORM (NCT07453251)
- Where
- Italy
- Volunteers
- 40 people
- Sponsor
- IRCCS Policlinico S. Donato
Duchene Muscular Dystrophy, Becker Muscular Dystrophy
Multiparametric Ultrafast Ultrasound Biomarkers for Duchenne and Becker Muscular Dystrophies
The purpose of this research study is to determine the potential of a multiparametric ultrasound approach to non-invasively monitor disease progression and to serve as an objective outcome measure for future clinical trials in…
- Trial name
- INNOVAN (NCT07378553)
- Where
- France
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Nantes University Hospital
Retinitis Pigmentosa (RP)
Prospective, Randomized, Sham-controlled, Dose-finding I/II Trial of Safety and Efficacy of Modified Optogenetic Gene Therapy (ZM-02 Injection)
This is a Phase 1/2, multi-center, randomized, sham-controlled, dose-escalation study evaluating ZM-02 in patients with advanced retinitis pigmentosa (RP).
- Trial name
- PRISM (NCT07282457)
- Testing
- ZM-02 (low dose), ZM-02 (high dose), ZM-02 (selected dose)
- Volunteers
- 21 people
- Sponsor
- Zhongmou Therapeutics
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