Clinical trials: Congenital Anomaly
Rett Syndrome
Esketamine for the Treatment of Rett Syndrome
The goal of this interventional study is to learn about the efficacy and safety of Esketamine for treating children with Rett syndrome (RTT).
- Trial name
- NCT06199700
- Testing
- Esketamine hydrochloride
- Where
- China
- Volunteers
- 3 people
- Sponsor
- Children's Hospital of Fudan University
Congenital Heart Disease
Multi-micronutrient Supplementation During Peri-conception and Congenital Heart Disease
The purpose of this study is to determine whether daily oral supplements of vitamin B complex along with folic acid or supplements of iron plus folic acid given to women during peri-conception can reduce the risk of congenital…
- Trial name
- NCT02537392
- Testing
- Vitamin B Complex and Folic Acid, Iron and Folic Acid, Folic Acid
- Where
- China
- Volunteers
- 7,315 people
- Sponsor
- Xi'an Jiaotong University
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
Dermatitis, Dermatitis, Atopic
Study to Investigate Efficacy and Safety of PF-04965842 in Subjects Aged 12 Years and Over With Moderate to Severe Atopic Dermatitis With the Option of Rescue Treatment in Flaring Subjects
B7451014 is a Phase 3 study to investigate PF-04965842 in patients aged 12 years and over with a minimum body weight of 40 kg who have moderate to severe atopic dermatitis.
- Conditions
- Dermatitis, Dermatitis, Atopic, Eczema, Skin Diseases
- Trial name
- NCT03627767
- Testing
- PF-04965842 100 mg, PF-04965842 200 mg
- Where
- 235 sites in United States, Argentina and 19 more
- Volunteers
- 1,235 people
- Sponsor
- Pfizer
Haemophilia B
An Open Study to Investigate the Safety and Efficacy of Replenine®-VF in Severe Haemophilia B Patients
To compare the first and second recovery assessments on Replenine®-VF and to evaluate recovery of different batches if patients changed batches during the study.
- Trial name
- NCT02231944
- Testing
- Replenine®-VF (High Purity Factor IX)
- Where
- 7 sites in Poland, United Kingdom
- Sponsor
- Bio Products Laboratory
Macular Degeneration, Low Vision
The Effect of Duration Between Sessions on Microperimetric Biofeedback Training in Patients With Maculopathies
Patients who develop macular diseases have several clinical complications,such as central vision loss, the central scotoma of the visual field, the decrease of reading speed and fixation stability.
- Conditions
- Macular Degeneration, Low Vision, Stargardt Disease
- Trial name
- NCT05904444
- Testing
- microperimetric biofeedback training
- Where
- China
- Volunteers
- 29 people
- Sponsor
- Aier Eye Hospital, Guangzhou
Retinopathy of Prematurity
A 24-week Study Evaluating the Effectiveness and Safety of Lucentis® 0.2mg in Retinopathy of Prematurity Participants in China
This is a 24-week, multicenter, open-label, single-arm, observational, post approval commitment study, which is designed to collect effectiveness, safety and other clinical information of intravitreal ranibizumab 0.2 mg for the…
- Trial name
- NCT05576792
- Testing
- Ranibizumab
- Where
- China
- Volunteers
- 62 people
- Sponsor
- Novartis Pharmaceuticals
Long QT Syndrome
Low Cost QTc Meter for Long QT Syndrome Screening in Primary Care
The purpose of this study is to further develop and evaluate a diagnostic procedure suitable for use in an inexpensive diagnostic instrument suitable for screening for Long QT Syndrome (LQTS) in the primary care environment.
- Trial name
- NCT04441892
- Testing
- QTc Meter
- Where
- United States
- Volunteers
- 33 people
- Sponsor
- Mayo Clinic
Congenital Talipes Equino Varus
Gait Analysis in Idiopathic Clubfoot Patients Treated by the Ponseti Method
The purpose of this study is to assess the walking pattern of clubfoot patients using a pressure sensing walkway, and investigate whether any of the data produced can be used to detect the early signs of relapse in their foot…
- Trial name
- NCT05572411
- Testing
- GAITRite Electronic Walkway
- Where
- United Kingdom
- Volunteers
- 20 people
- Sponsor
- Great Ormond Street Hospital for Children NHS Foundation Trust
Thyroid Eye Disease
Treatment of Graves' Orbitopathy to Reduce Proptosis With Teprotumumab Infusions in an Open-Label Clinical Extension Study
The overall objective is to evaluate the safety and efficacy of teprotumumab in the treatment of thyroid eye disease (TED) in participants who participated in the lead-in study HZNP-TEP-301 (NCT03298867; OPTIC) and who were…
- Trial name
- OPTIC-X (NCT03461211)
- Testing
- Teprotumumab
- Where
- 13 sites in United States, Germany and 1 more
- Volunteers
- 51 people
- Sponsor
- Amgen
Dilated Cardiomyopathy
Bone Marrow Derived Adult Stem Cells for Dilated Cardiomyopathy
A randomised, double-blind, placebo-controlled trial to evaluate the role of intracoronary injection of progenitor cells compared to placebo injection in patients with Dilated Cardiomyopathy who have been pre-treated with G-CSF…
- Trial name
- REGEN-DCM (NCT01302171)
- Testing
- granulocyte colony stimulating factor (GCSF), bone marrow mononuclear cells
- Where
- United Kingdom
- Volunteers
- 60 people
- Sponsor
- Barts & The London NHS Trust
Craniosynostoses, Pain, Postoperative
Scalp Block Decreases Pain and Side Effects
Postoperative analgesia after corrective surgery of paediatric craniosynostosis is crucial in term of short and long-term outcomes.
- Trial name
- NCT04133467
- Testing
- scalp block
- Where
- Italy
- Volunteers
- 26 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Congenital Bleeding Disorder, Haemophilia A
Safety, Tolerability, and Pharmacokinetics Study of Turoctocog Alfa Pegol Injected Under the Skin in Patients With Haemophilia A
The trial is conducted in Asia, Europe and North America. The aim of the study is to evaluate the safety of administration under the skin of turoctocog alfa pegol (SC N8-GP) in patients with severe haemophilia A.
- Trial name
- alleviate 1 (NCT02994407)
- Testing
- turoctocog alfa pegol
- Where
- 19 sites in United States, Austria and 4 more
- Volunteers
- 50 people
- Sponsor
- Novo Nordisk A/S
Crohn Disease
Study to Evaluate the Efficacy and Safety of PF-06480605 in Adult Participants With Moderate to Severe Crohn's Disease
This Phase 2a, multicenter, randomized, double-blind, placebo-controlled study examines subcutaneous dose of PF-06480605 150 mg administered every 4 weeks in participants with moderate to severe active Crohn's Disease to…
- Trial name
- NCT05471492
- Testing
- Investigational Product
- Sponsor
- Telavant, Inc.
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
Ventricular Septal Defect, Closure; Interventricular Septum
The Effect of Transcatheter Ventricular Septal Defect Closure on Heart Rate Variability Parameters
In this study; we evaluated the heart rate variability parameters of pediatric patients whose VSDs were closed with the transcatheter method before and after, and compared with the control group.
- Conditions
- Ventricular Septal Defect, Closure; Interventricular Septum, Autonomic Nervous System Imbalance
- Trial name
- NCT04859036
- Testing
- 24 hours ambulatory Holter monitoring
- Where
- Turkey (Türkiye)
- Volunteers
- 37 people, healthy volunteers welcome
- Sponsor
- Kayseri City Hospital
Congenital Heart Disease
Endothelin Blockade in Patients With Single Ventricle Physiology
Recent studies have shown beneficial effects of sildenafil in patients with single ventricle congenital heart disease. The purpose of this study is to determine whether Bosentan, a drug with similar effects, will have similar…
- Trial name
- NCT00989911
- Testing
- Bosentan
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- University of California, Los Angeles
Bronchopulmonary Dysplasia, Encephalomalacia
Randomized Trial of Hydrocortisone in Very Preterm High-Risk Infants
The purpose of this study is to determine whether treatment of very preterm infants at high-risk for lung and brain injury with low dose hydrocortisone results in improved pulmonary and neurologic outcomes.
- Conditions
- Bronchopulmonary Dysplasia, Encephalomalacia, Premature Birth
- Trial name
- NCT00167544
- Testing
- Hydrocortisone
- Where
- United States
- Volunteers
- 64 people
- Sponsor
- Nationwide Children's Hospital
Pompe Disease
First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221
This is an international, multi-center, open-label study designed to evaluate if the co-administration of investigational new drugs ATB200 and AT2221 is safe in adults with Pompe disease.
- Trial name
- NCT02675465
- Testing
- ATB200, AT2221
- Where
- 19 sites in United States, Australia and 4 more
- Volunteers
- 29 people
- Sponsor
- Amicus Therapeutics
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