Clinical trials: Congenital Diseases
Delirium - Postoperative, Sleep Problems
Preoperative Sleep Intervention on Postoperative Delirium in Infants and Toddlers Undergoing Congenital Heart Surgery
This is a multicenter, randomized, controlled clinical trial aimed to determine whether preoperative sleep interventions could reduce the incidence of adverse outcomes, such as postoperative delirium, in sleep-disordered infants…
- Conditions
- Delirium - Postoperative, Sleep Problems, Congenital Heart Disease (CHD)
- Trial name
- NCT06861998
- Testing
- Message-based bedtime routine, sleep hygiene education
- Where
- China
- Volunteers
- 452 people
- Sponsor
- Yan Fuxia
Genetic Diseases, Inborn, Genetic Predisposition to Disease
NYCKidSeq: Incorporating Genomics Into Clinical Care of Diverse NYC Children
The NYCKidSeq program will significantly advance the implementation of genomic medicine, particularly for children, young adults and their families in Harlem and the Bronx.
- Trial name
- NCT03738098
- Testing
- Standard of Care, GUÍA
- Where
- 2 sites in United States
- Volunteers
- 650 people
- Sponsor
- Icahn School of Medicine at Mount Sinai
Hemophilia
First-in-Human and Proof-of-Mechanism Study of ARC19499 Administered to Hemophilia Patients
The purpose of this study is to examine the safety, tolerability and the way the body handles various single and multiple doses of ARC19499 in patients with hemophilia.
- Trial name
- NCT01191372
- Testing
- ARC19499
- Where
- United Kingdom
- Volunteers
- 17 people
- Sponsor
- Baxalta now part of Shire
Birth Defects, Pregnancy Complications
UCB Antiepileptic Drugs (AED) Pregnancy Registry (Formerly the Keppra® Pregnancy Registry)
This is a prospective, observational, exposure-registration and follow-up study of women and their offspring exposed to Keppra® (levetiracetam) and Keppra XR® at the time of conception (i.e., any time from the first day of the…
- Conditions
- Birth Defects, Pregnancy Complications, Epilepsy, Seizures
- Trial name
- NCT00345475
- Where
- United States
- Volunteers
- 516 people
- Sponsor
- UCB Pharma
Peutz-Jeghers Syndrome
Poorly Differentiated Adenocarcinoma of the Jejunum in a Patient With Peutz-Jeghers Syndrome: A Case Report
Our study is a case report of one of the rarest risk factor, Peutz-Jeghers syndrome, of small bowel malignancy detected in a patient with poorly differentiated adenocarcinoma of small bowel(jejunum)
- Trial name
- NCT06242457
- Where
- Ethiopia
- Volunteers
- 1 people, healthy volunteers welcome
- Sponsor
- Haramaya Unversity
Dentofacial Deformities
Custom Titanium Plates vs. 3D Splints in LeFort I Osteotomy
This randomized comparative study evaluates the accuracy and feasibility of two techniques for maxillary repositioning in Le Fort I osteotomy: Customized titanium plates (splintless technique) Three-dimensional (3D)-printed…
- Trial name
- NCT06933628
- Testing
- Patient-Specific Titanium Plates, 3D-Printed Intermediate Splint
- Where
- Egypt
- Volunteers
- 12 people
- Sponsor
- Al-Azhar University
Mucopolysaccharidosis I, Mucopolysaccharidosis II
Registry of Patients Diagnosed With Lysosomal Storage Diseases
This is an international prospective and retrospective registry of patients with Lysosomal Storage Diseases (LSDs) to understand the natural history of the disease and the outcomes of fetal therapies, with the overall goal of…
- Conditions
- Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI
- Trial name
- LSD Registry (NCT05619900)
- Testing
- There is no intervention
- Where
- United States
- Volunteers
- 250 people
- Sponsor
- University of California, San Francisco
Pompe Disease
Safety and Efficacy of Albuterol in Individuals With Late-onset Pompe Disease
In this study the study team proposes to investigate the efficacy of albuterol on motor function of individuals with Late Onset Pompe Disease (LOPD) who are receiving enzyme replacement therapy, given albuterol was well-tolerated…
- Trial name
- NCT01885936
- Testing
- Albuterol
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Duke University
Respiratory Distress Syndrome in Premature Infant, Patent Ductus Arteriosus After Premature Birth
Combined Lung Ultrasounds and Transthoracic Electrical Bioimpedance in Preterm Infants With Respiratory Distress.
Respiratory distress syndrome (RDS) is among the most common complications of preterm birth, and typically becomes manifested soon after birth.
- Conditions
- Respiratory Distress Syndrome in Premature Infant, Patent Ductus Arteriosus After Premature Birth, Perinatal Morbidity
- Trial name
- NCT04359134
- Where
- 2 sites in Italy
- Volunteers
- 60 people
- Sponsor
- IRCCS Azienda Ospedaliero-Universitaria di Bologna
Respiratory Distress Syndrome, Newborn, Respiratory Failure
Cerebral and Circulatory Effects of Neonatal Volume Targeted Ventilation
The aim of the study was to assess the effect of volume targeted vs. pressure-controlled mechanical ventilation (MV) on circulatory parameters and cerebral oxygenation in the extremely preterm infants.
- Conditions
- Respiratory Distress Syndrome, Newborn, Respiratory Failure, Premature Birth
- Trial name
- NCT04391634
- Testing
- volume guarantee, pressure-controlled
- Where
- Poland
- Volunteers
- 25 people
- Sponsor
- Poznan University of Medical Sciences
Spinocerebellar Ataxia Type 3
Optimizing Parameters of Transcranial Temporal Interference Stimulation for Spinocerebellar Ataxia Type 3
The purpose of this research study is to identify the optimal biological dose for tTIS that targets DN in SCA3 while achieving the best possible balance between therapeutic efficacy and safety.
- Trial name
- NCT07743866
- Testing
- Transcranial Temporal Interference Stimulation (tTIS)
- Where
- China
- Volunteers
- 24 people
- Sponsor
- First Affiliated Hospital of Fujian Medical University
Gaucher Disease, Type 1
Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease
Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme glucocerebrosidase (GCB). Due to the deficiency of functional GCB, glucocerebroside accumulates within macrophages leading to cellular…
- Trial name
- NCT00553631
- Testing
- velaglucerase alfa, imiglucerase
- Where
- 11 sites in United States, Argentina and 7 more
- Volunteers
- 34 people
- Sponsor
- Shire
Cystic Fibrosis
18FDG- PET/CT Contribution to the Assessment of Lesion Severity in Cystic Fibrosis (CF)
Cystic fibrosis (CF) is an autosomal recessive disorder caused by mutations in the CF trans-membrane conductance regulator (CFTR) protein.
- Trial name
- NCT00363402
- Testing
- PET-CT
- Where
- Israel
- Volunteers
- 20 people
- Sponsor
- Hadassah Medical Organization
Cystic Fibrosis, Nontuberculous Mycobacterial Pulmonary Infection
Prospective Healthcare-Associated Links in Transmission of Nontuberculous Mycobacteria
Pulmonary NTM infection is recognized as one of the most challenging infections to treat among people with cystic fibrosis (PwCF), notable for prolonged treatment courses and often poor response to therapy.
- Trial name
- pHALT NTM (NCT05686837)
- Where
- 7 sites in United States
- Volunteers
- 100 people
- Sponsor
- University of North Carolina, Chapel Hill
IgA Nephropathy, CKD Associated With Type 1 Diabetes
A Phase 2 Trial of the Safety and Efficacy of Bardoxolone Methyl in Patients With Rare Chronic Kidney Diseases - PHOENIX
This multi-center, open-label Phase 2 trial will study the safety, tolerability, and efficacy of bardoxolone methyl in qualified patients with the following rare chronic kidney diseases (CKD): CKD associated with type 1 diabetes…
- Conditions
- IgA Nephropathy, CKD Associated With Type 1 Diabetes, Focal Segmental Glomerulosclerosis, Autosomal Dominant Polycystic Kidney
- Trial name
- PHOENIX (NCT03366337)
- Testing
- Bardoxolone methyl capsules
- Where
- 30 sites in United States
- Volunteers
- 103 people
- Sponsor
- Biogen
Eczema, Atopic Dermatitis
A Study to Evaluate CLn® BodyWash As Added Therapy in Eczema Patients
The purpose of this study is to determine if CLn BodyWash, a novel new gel cleanser formulated with surfactants and preserved with sodium hypochlorite, is effective at decreasing severity, body surface area and itching for…
- Trial name
- CLeaN (NCT01714245)
- Where
- 2 sites in United States
- Volunteers
- 50 people
- Sponsor
- TopMD Skin Care, Inc.
End Stage Renal Disease, AV Fistula
Percutaneous Less Invasive AV Fistula for Vascular Access in ESRD
A prospective single-arm well-controlled study to evaluate the safety and effectiveness of a less invasive means of establishing vascular access to facilitate dialysis in patients with end stage renal disease.
- Conditions
- End Stage Renal Disease, AV Fistula, Fistula, End Stage Kidney Disease
- Trial name
- NCT02363972
- Testing
- Ellipsys Vascular Access Catheter
- Where
- 5 sites in United States
- Volunteers
- 103 people
- Sponsor
- Medtronic Endovascular
Plagiocephaly, Nonsynostotic
Cranial Orthotic Device Versus Repositioning Techniques for the Management of Plagiocephaly: the CRANIO Randomized Trial
This study evaluates the effectiveness of repositioning techniques and neck stretching exercises in comparison to cranial orthotic devices (COD) in correcting plagiocephaly in infants.
- Trial name
- CRANIO (NCT02370901)
- Testing
- Helmet, home therapies
- Where
- United States
- Volunteers
- 226 people, healthy volunteers welcome
- Sponsor
- Pat Ricalde,DDS, MD, FACS
Metabolic Disease, Fabry Disease
Getting Global Rare Disease Insights Through Technology Study
This project is a randomized controlled trial to use a mobile health journal, called Zamplo (formerly known as MyHealthJournal or ZoeInsights), to record patient reported outcomes (PROM) in patients with metabolic disorders.
- Conditions
- Metabolic Disease, Fabry Disease, Gaucher Disease, Pompe Disease
- Trial name
- GRIT (NCT04758130)
- Testing
- Zamplo Digital Health Platform
- Where
- Canada
- Volunteers
- 150 people
- Sponsor
- M.A.G.I.C. Clinic LTD
Postoperative Pain
Prediction of Acute Postoperative Pain and Analgesic Consumption
Pain is an expected part of surgical recovery but effective pain management remains challenging. The high variability in postoperative pain experience and analgesic treatment response between patients is part of the challenge.
- Trial name
- MIRPEX-3 (NCT02230865)
- Where
- Denmark
- Volunteers
- 40 people
- Sponsor
- Kasper Grosen
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