Clinical trials: Newborn Diseases
Phenylketonuria
A Study of JNT-517 in Participants With Phenylketonuria (PKU)
This Phase 3 study will evaluate the safety, effectiveness, tolerability, and how the body processes JNT-517 in children, adolescents, and adults with phenylketonuria.
- Trial name
- NCT06971731
- Testing
- JNT-517
- Where
- 26 sites in United States, Australia and 8 more
- Volunteers
- 120 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
Dravet Syndrome (DS), Lennox Gastaut Syndrome (LGS)
Clinical Utility of Reduced EEG Home Monitoring in Fenfluramine Titration for Dravet and LGS
The goal of this real world data study is to evaluate the clinical utility of remote patient monitoring solution (using Byteflies' EpiCare@Home with reduced EEG montage and other vital signs) with Dravet and LGS patients (ages 3…
- Trial name
- TETRIS (NCT07225231)
- Testing
- Byteflies Remote patient monitoring solution (with reduced EEG)
- Where
- 3 sites in United States
- Volunteers
- 20 people
- Sponsor
- Byteflies
Acute Renal Failure
AV Fistula Salvage in Advanced CKD Using Sodium Bicarbonate Prophylaxis
This is an observational study assessing the impact of conventional dose iodinated contrast on the renal function of advanced chronic kidney disease patients undergoing arteriovenous fistula evaluation using a standard sodium…
- Trial name
- NCT00621972
- Where
- United States
- Sponsor
- MaineHealth
Noonan Syndrome
Neuroradiological Features of Noonan Syndrome and Genotype-Phenotype Correlations
Retrospective and prospective collection of brain and spinal MRI and MR angiography images from patients of all ages with a genetically confirmed diagnosis of Noonan syndrome.
- Trial name
- NCT07881081
- Testing
- Brain MRI and MR angiography (MRA)
- Where
- 10 sites in Italy
- Volunteers
- 60 people
- Sponsor
- Istituto Giannina Gaslini
Atopic Dermatitis
Efficacy of the "Атопик Soothing Cream" in Children With Mild to Moderate Atopic Dermatitis
A Double-Blind Randomized Placebo-Controlled Trial to Evaluate the Efficacy of the "Атопик Soothing Cream" in Children with Mild to Moderate Atopic Dermatitis
- Trial name
- NCT02716181
- Testing
- "Атопик Soothing Cream"
- Where
- Russia
- Volunteers
- 74 people
- Sponsor
- Avanta Trading Ltd.
Physical Therapy Modalities
Effects of Physical Therapy Associated With Photobiomodulation on Myelomeningocele
Myelomeningocele is a severe type of spina bifida, resulting from improper closure of the neural tube. This condition drastically affects the structures of the spinal cord, as a result the spinal cord, nerve roots and meninges…
- Trial name
- NCT04425330
- Testing
- physiotherapy exercises, PBM
- Volunteers
- 30 people
- Sponsor
- University of Nove de Julho
Pregnant Women
Pertussis Maternal Immunization Study
The purpose of this study is to assess whether immunization against pertussis in the mid third trimester of pregnancy provides passive protection to the infant by transfer of IgG transplacentally and by transfer of secretory IgA…
- Trial name
- NCT00553228
- Testing
- Tdap, Td
- Where
- Canada
- Volunteers
- 320 people, healthy volunteers welcome
- Sponsor
- Scott Halperin
Anemia, Sickle Cell, Blood Disease
Indices of Severity and Prognosis for Sickle Cell Disease
To develop a clinical severity index that could prospectively identify sickle cell disease patients who were at high risk for a turbulent clinical course and a poor prognosis.
- Trial name
- NCT00005467
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Silver Russell Syndrome, Prader-Willi Syndrome
Aromatase Inhibitor in Bone Maturation, Children With Silver Russell or Prader-Willi Syndrome
There is currently no drug with pediatric marketing authorization capable of limiting the advance in bone maturation of children with aggressive adrenarche.
- Trial name
- ANASILPRA (NCT01520467)
- Testing
- Anastrozole
- Where
- France
- Volunteers
- 27 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Cystic Fibrosis
Bioequivalence and Safety of Vantobra and TOBI in Healthy Subjects
This study will investigate the bioequivalence and compare the safety profiles following inhalation of Vantobra to TOBI nebulizer solution in healthy subjects.
- Trial name
- NCT01953367
- Testing
- Vantobra (tobramycin), TOBI (tobramycin)
- Where
- Germany
- Volunteers
- 72 people, healthy volunteers welcome
- Sponsor
- Pari Pharma GmbH
Urea Cycle Disorders, Inborn
Comparative Efficacy of Phenylbutyrate (PBA) vs. Benzoate in Urea Cycle Disorders
The investigators will study and compare how effectively sodium phenylbutyrate, sodium benzoate, and a combination of the two, help excrete nitrogen in healthy volunteers.
- Trial name
- BPA/Benzoate (NCT02111200)
- Testing
- Sodium Benzoate, Sodium Phenylbutyrate
- Where
- United States
- Volunteers
- 7 people, healthy volunteers welcome
- Sponsor
- Baylor College of Medicine
Hemophilia A With Inhibitor
A Pharmacokinetic and Clotting Activity Study of FVIII-PEGLip
The purpose of this study is to demonstrate that PEGylated liposomes (PEGLip) can shield FVIII from the immune system and inhibitors, and therefore provide a prophylactic FVIII replacement therapy for patients with inhibitors to…
- Trial name
- NCT04592692
- Testing
- PEGylated Liposome (PEGLip)
- Where
- 5 sites in Russia
- Volunteers
- 20 people
- Sponsor
- Ascension Healthcare Development Limited
Congenital Metabolic Disorder, Acute Disease
Rapid Genetic Diagnosis Employing Next Generation Sequencing for Critical Illness in Infants and Children
Under the joint efforts of genetic and intensive expert, to establish the high-throughput whole exon sequencing(WES) and analysis all the possible pathogenic genes.
- Trial name
- NCT03175692
- Testing
- Whole Exome Sequencing
- Where
- Taiwan
- Volunteers
- 150 people
- Sponsor
- National Taiwan University Hospital
Fecal Incontinence, Anorectal Malformation
NASHA/Dx as a Perianal Implant for the Treatment of Persistent Fecal Incontience After Anorectal Malformation
Persistent fecal incontinence (FI) after anorectal malformations (ARM) is a common occurence. During the last two decades perianal injection therapy has emerged as an option for treating patients with FI due to other causes than…
- Trial name
- NCT03746834
- Testing
- NASHA/Dx (Solesta®)
- Volunteers
- 7 people
- Sponsor
- Uppsala University
Dystrophinopathy, Duchenne Muscular Dystrophy
Safety, Tolerability and Effects of L-Arginine in Boys With Dystrophinopathy on Corticosteroids
The purpose of the study is to assess the safety, tolerability, and effects of L-Arginine on muscles in boys with dystrophinopathy on corticosteroids.
- Conditions
- Dystrophinopathy, Duchenne Muscular Dystrophy, Becker's Muscular Dystrophy
- Trial name
- NCT01388764
- Testing
- L-arginine
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Massachusetts General Hospital
Von Hippel-Lindau Syndrome, Renal Cell Carcinoma
Evaluation of Sunitinib Malate in Patients With Von Hippel-Lindau Syndrome (VHL) Who Have VHL Lesions to Follow
The goal of this clinical research study is to learn if sunitinib malate (SU011248) can help to control VHL. The safety of this drug will also be studied.
- Conditions
- Von Hippel-Lindau Syndrome, Renal Cell Carcinoma, Hemangioblastoma
- Trial name
- NCT00330564
- Testing
- SU011248
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- M.D. Anderson Cancer Center
Phenylketonurias
PheCheck Feasibility Study
The goal of this feasibility study clinical trial is to compare the accuracy of PheCheck™ for the rapid quantitative detection of phenylalanine (Phe) with the gold standard (HPLC amino acid analyzer), in patients with PKU.
- Trial name
- NCT05998109
- Testing
- PheCheck™, HPLC Amino Acid Analyzer, Dried Blood Spot Cards
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Lumos Diagnostics
Cystic Fibrosis, Bronchiectasis
Dose Escalation Study of ALX-009 in Healthy Men and Cystic Fibrosis (CF) and Non-CF Bronchiectasis Patients
This is a Phase 1, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability and pharmacokinetics of a single ascending doses (SAD) and multiple ascending doses (MAD) of Hypothiocyanite (OSCN-)…
- Trial name
- NCT02598999
- Testing
- ALX-009, OSCN-, bLF
- Where
- France
- Volunteers
- 92 people, healthy volunteers welcome
- Sponsor
- Alaxia SAS
Low Vision, Retinitis Pigmentosa
Visual Performance Measures in a Virtual Reality Environment for Assessing Clinical Trial Outcomes in Those With Severely Reduced Vision
Purpose: To validate a newly developed battery of performance-based tests of visual function to be presented using virtual reality.
- Conditions
- Low Vision, Retinitis Pigmentosa, Stargardt Disease 1, Stargardt Disease 3
- Trial name
- NCT04281732
- Testing
- Virtual Reality Headset based tests
- Where
- United Kingdom
- Volunteers
- 18 people
- Sponsor
- Queen's University, Belfast
Pelizaeus-Merzbacher Disease, PMD
Long-Term Follow-Up Study of Human Stem Cells Transplanted in Subjects With Connatal Pelizaeus-Merzbacher Disease (PMD)
The purpose of this study is to determine the long term safety and preliminary effect of HuCNS-SC cells transplanted in subjects with Connatal Pelizaeus-Merzbacher Disease (PMD).
- Trial name
- NCT01391637
- Testing
- HuCNS-SC transplant in the lead-in phase
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- StemCells, Inc.
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