Clinical trials: Newborn Diseases
Phenylketonuria
A Phase 3 Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302
A Phase 3 substudy to evaluate executive function in adults with phenylketonuria who are participating in the phase 3 Study, 165-302. Approximately 100 subjects, aged ≥ 18 to ≤ 70 years old, with PKU will be enrolled.
- Trial name
- PRISM303 (NCT02468570)
- Testing
- Administration of CANTAB and Subject Global Assessment
- Where
- 4 sites in United States
- Volunteers
- 9 people
- Sponsor
- BioMarin Pharmaceutical
Fetal Alcohol Spectrum Disorders
Choline Supplementation as a Neurodevelopmental Intervention in Fetal Alcohol Spectrum Disorders
This is a randomized, double-blind, placebo-controlled trial of choline supplementation in children with Fetal Alcohol Spectrum Disorders (FASD). The primary outcome measures are cognitive measures.
- Trial name
- NCT02735473
- Testing
- Choline bitartrate
- Where
- United States
- Volunteers
- 46 people
- Sponsor
- University of Minnesota
Angelman Syndrome
A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)
The main goal of the study is to evaluate the safety and efficacy of GTX-102 in participants with Angelman syndrome.
- Trial name
- Aurora (NCT07157254)
- Testing
- No intervention, GTX-102
- Where
- 21 sites in United States, Argentina and 6 more
- Volunteers
- 55 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Advanced Malignant Solid Neoplasm, Recurrent Ependymal Tumor
Ulixertinib in Treating Patients With Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With MAPK Pathway Mutations (A Pediatric MATCH Treatment Trial)
This phase II Pediatric MATCH trial studies how well ulixertinib works in treating patients with solid tumors that have spread to other places in the body (advanced), non-Hodgkin lymphoma, or histiocytic disorders that have a…
- Conditions
- Advanced Malignant Solid Neoplasm, Recurrent Ependymal Tumor, Recurrent Ewing Sarcoma, Recurrent Glioma
- Trial name
- NCT03698994
- Testing
- Pharmacokinetic Study, Ulixertinib
- Where
- 107 sites in United States, Puerto Rico
- Volunteers
- 20 people
- Sponsor
- National Cancer Institute (NCI)
Lymphatic Malformation
A Prospective Study on the Treatment of cLM Based on ICG Imaging
The goal of this prospective randomized controlled study is to explore the role of indocyanine green-fluorescence imaging in management of cystic lymphatic malformation..
- Trial name
- NCT06275022
- Testing
- Inflow occlusion with perforation of septation and sclerotherapy, Perforation of septation and sclerotherapy, Indocyanine green-guided partial resection and sclerotherapy
- Where
- China
- Volunteers
- 110 people
- Sponsor
- Nanjing Children's Hospital
Congenital Adrenal Hyperplasia
Open-label Comparison of Chronocort® Versus Standard Glucocorticoid Replacement Therapy
This study is an open-label, randomised, titration-blinded, parallel arm, multicenter study to compare twice daily Chronocort® with standard care in participants with Congenital Adrenal Hyperplasia (CAH).
- Trial name
- RESTORE (NCT03532022)
- Testing
- Chronocort®, Standard Care
- Where
- United States
- Sponsor
- Neurocrine UK Limited
Mucopolysaccharidosis Type 7
An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient
Emergency access granted to treat a single patient with advanced Mucopolysaccharidosis Type 7 with UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy via IV administration every other week (QOW) for up…
- Trial name
- NCT02097251
- Testing
- UX003
- Where
- United States
- Sponsor
- Joyce Fox
Atopic Dermatitis, Psoriasis
Meditation in Inflammatory Dermatosis
Psoriasis and atopic dermatitis are multifactorial inflammatory dermatoses, with a very high prevalence, reaching more than 120 million patients in the world.
- Conditions
- Atopic Dermatitis, Psoriasis, Pruritus
- Trial name
- MediDermIn (NCT05500794)
- Testing
- MBSR (Mindfulness-Based Stress Reduction)
- Where
- France
- Volunteers
- 72 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Gaucher Disease
A Study of Velaglucerase Alfa (VPRIV) Given as Standard Patient Care in Young Children With Gaucher Disease
The main aim of this study is to learn if velaglucerase alfa (VPRIV) improves growth and symptoms in participants up to 5 years of age with Gaucher disease. Symptoms will be checked with blood tests.
- Trial name
- PEDS (NCT04721366)
- Testing
- Standard of Care
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- Takeda
Amyloid Cardiomyopathy
Unmasking the Prevalence of AC in an Unselected Echocardiographic Population
This study will investigate the prevalence of echocardiographic red-flags of amyloid cardiomyopathy (AC) in patients undergoing clinically-indicated echocardiography (observational phase) and the prevalence of AC among…
- Trial name
- AC-TIVE (NCT04738266)
- Testing
- Amyloid Cardiomyopathy
- Where
- 16 sites in Italy
- Volunteers
- 381 people
- Sponsor
- University of Trieste
Hutchinson-Gilford Progeria Syndrome
A Study Observing Disease Progression in a Patient With Hutchinson-Gilford Progeria Syndrome (HGPS)
This study is a prospective, non-interventional, single-patient observational study following one patient with Hutchinson-Gilford Progeria Syndrome (HGPS), a rare disease that causes rapid, premature aging, over a 3-year period…
- Trial name
- progeria (NCT07839351)
- Where
- South Korea
- Volunteers
- 1 people
- Sponsor
- Bundang CHA Hospital
Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Inv(16)(p13;q22)
GTI-2040 and High-Dose Cytarabine in Treating Patients With Refractory or Relapsed Acute Myeloid Leukemia
This phase I trial is studying the side effects and best dose of GTI-2040 and high-dose cytarabine in treating patients with refractory or relapsed acute myeloid leukemia.
- Conditions
- Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22)
- Trial name
- NCT00070551
- Testing
- GTI-2040, cytarabine, pharmacological study
- Where
- United States
- Volunteers
- 51 people
- Sponsor
- National Cancer Institute (NCI)
Infant, Premature, Diseases
The Effect of Diaper Change Position on Preterm Infants
The purpose of this study is to determine the effect of diaper change position on neonatal comfort and heart rate, respiratory rate and oxygen saturation in preterm infants.
- Trial name
- NCT04528602
- Testing
- Control (Extended leg position) Group, Experimental (Legs are flexed toward abdomen) Group
- Where
- Turkey (Türkiye)
- Volunteers
- 60 people
- Sponsor
- Acibadem University
Molar-Incisor Hypomineralization (MIH)
Er:YAG Laser Versus Conventional Cavity Preparation in Children With Molar-Incisor Hypomineralization
This randomized clinical trial compares Er:YAG laser-assisted and conventional rotary cavity preparation in children aged 6-12 years with molar-incisor hypomineralization.
- Trial name
- NCT07759739
- Testing
- Er:YAG Laser-Assisted Cavity Preparation, Conventional Rotary Cavity Preparation
- Where
- Turkey (Türkiye)
- Volunteers
- 52 people
- Sponsor
- Ege University
Enteral Feeding Intolerance, Necrotizing Enterocolitis
Bovine Colostrum for Preterm Newborns
Feeding intolerance is a common problem in very preterm infants due to their immature digestive system. This intolerance extends the time to full enteral feeding and thereby also prolongs the time on parenteral nutrition (PN).
- Conditions
- Enteral Feeding Intolerance, Necrotizing Enterocolitis, Late-Onset Neonatal Sepsis
- Trial name
- PreColos-RCT (NCT03085277)
- Testing
- Bovine Colostrum, Preterm Formula
- Where
- 8 sites in China
- Volunteers
- 350 people
- Sponsor
- Per Torp Sangild
Raloxifene, Postmenopausal Women
Raloxifene on Coagulation System in the Postmenopausal Women
Objective: The aim of this study is to assess the effects of raloxifene HCl on coagulation parameters in healthy postmenopausal women.
- Trial name
- NCT04454775
- Testing
- coagulation system parameters; factor 8, von Willebrand factor, protein C, protein S, antithrombin III and fibrinogen were studied.
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Karadeniz Technical University
Cardiovascular Abnormalities, Vascular Diseases
Characterization of Slow Coronary Flow
Description of the phenomena "Slow Coronary Flow" (SCF), according to the data that has been collected from patients hospitalized with specific criteria.
- Trial name
- NCT02025101
- Where
- Israel
- Volunteers
- 90 people
- Sponsor
- Hillel Yaffe Medical Center
Friedreich's Ataxia
Effect of Pioglitazone Administered to Patients With Friedreich's Ataxia: Proof of Concept
Friedreich's ataxia (FA) is a rare progressive neurological disorder affecting approximately 1/30, 000 individuals. No treatment is presently available to counteract the neurodegeneration of this extremely severe disease.
- Trial name
- ACTFRIE (NCT00811681)
- Testing
- pioglitazone
- Where
- France
- Volunteers
- 40 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Hypersensitive, Molar Incisor Hypomineralization
Effect of Glass-Ionomer Sealing Technique on Hypersensitive Teeth With Molar Incisor Hypomineralisation
This single armed cohort study is aimed to assess the acceptance, durability and efficiency of glass-Ionomer sealing technique on hypersensitive MIH molars (TNI-3 \& 4a/b/c) clinically shortly (15 minutes) after the application…
- Conditions
- Hypersensitive, Molar Incisor Hypomineralization, Pit and Fissure Sealants
- Trial name
- MIH_GIC (NCT05945381)
- Testing
- GIC on MIH
- Where
- Germany
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- University Medicine Greifswald
Molar Incisor Hypomineralization
Effects of Different Remineralization Agents on Molar Incisor Hypomineralization Defects: a Randomized Clinical Study
The aim of this study was to present a comparative evaluation of the long-term efficacy of fluoride varnish( Flúor protector), Clinpro™ White Varnish and pastes containing CPP-ACP in the remineralization of creamy-white and…
- Trial name
- NCT06362681
- Testing
- Fluoride
- Where
- Mexico
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Universidad Nacional Autonoma de Mexico
The first 1,020 results can be paged through. Add a filter or change the sort order to reach the rest.