Clinical trials: Infant, Newborn Diseases
Fabry Disease
Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease
Study to compare the efficacy and safety of migalastat and enzyme replacement therapy (ERT) in male and female participants with Fabry disease who are currently receiving ERT and who have an alpha galactosidase-A (α Gal-A)…
- Trial name
- NCT01218659
- Testing
- migalastat hydrochloride, agalsidase
- Where
- 25 sites in United States, Australia and 8 more
- Volunteers
- 68 people
- Sponsor
- Amicus Therapeutics
Chronic Kidney Diseases, Pain, Procedural
Effect of 4-7-8 Breathing on AV Fistula Needle Pain in Dialysis Patients
For many hemodialysis patients, needle insertion into the vascular access site (fistula) is a significant source of pain and anxiety.
- Conditions
- Chronic Kidney Diseases, Pain, Procedural, Arteriovenous Fistula
- Trial name
- NCT07700680
- Testing
- Breathing technique
- Volunteers
- 80 people
- Sponsor
- Izmir Kavram Vocational School
Cleft Lip and Palate
Test-Retest Reliability, Responsiveness and Interpretability of CLEFT-Q
The goal of this observational study is to test the questionnaire CLEFT-Q in a population of patients with cleft lip and/or palate. The main questions it aims to answer are: 1. Are CLEFT-Q scores reliable over time? 2.
- Trial name
- CLEFT-QSwePsyc (NCT05775679)
- Where
- Sweden
- Volunteers
- 260 people, healthy volunteers welcome
- Sponsor
- Department of Plastic and Reconstructive Surgery
Velocardiofacial Syndrome
Computerized Cognitive Skills Training for Adolescents With Velocardiofacial Syndrome
This study will test a computer-based treatment for youth with the genetic disorder velocardiofacial syndrome (VCFS) to help them improve skills in memory, attention, and executive functioning.
- Trial name
- NCT00917189
- Testing
- Challenging Our Minds Program
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- State University of New York - Upstate Medical University
Amyloidosis, Hereditary
Prospective Evaluation of NfL as a Biomarker in ATTRv
ATTR amyloidosis is a rare and progressively disabling disease caused by the deposition of misfolded TTR protein in multiple tissues including the nerves, heart, and gastrointestinal tract.
- Trial name
- NfLInATTRv (NCT05879874)
- Where
- Italy
- Volunteers
- 40 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Dilated Cardiomyopathy
Use of Ixmyelocel-T (Formerly Cardiac Repair Cell [CRC] Treatment) in Patients With Heart Failure Due to Dilated Cardiomyopathy (IMPACT-DCM)
This study is designed to assess the safety and tolerability of Cardiac Repair Cells (CRCs) compared to standard-of-care in patients with dilated cardiomyopathy (DCM).
- Trial name
- NCT00765518
- Testing
- Ixmyelocel-T, Standard of Care
- Where
- 5 sites in United States
- Volunteers
- 40 people
- Sponsor
- Vericel Corporation
Hereditary Angioedema
Pharmacokinetics of C1 Esterase Inhibitor in Hereditary Angioedema Subjects
The study objective was to describe the pharmacokinetics (PK) of one or two doses of C1 esterase inhibitor (C1INH-nf) in hereditary angioedema (HAE) subjects who were not experiencing an HAE attack.
- Trial name
- NCT00432510
- Testing
- C1 esterase inhibitor [human] (C1INH-nf)
- Where
- 6 sites in United States
- Volunteers
- 27 people
- Sponsor
- Shire
Dermatitis, Atopic
Safety and Efficacy of SCM-AGH in Subjects With Moderate to Severe Atopic Dermatitis
This study consists of two phases (Phase I and Phase II). Phase II will be conducted sequentially after the safety of SCM-AGH is secured in Phase I.
- Trial name
- NCT04179760
- Testing
- SCM-AGH
- Where
- South Korea
- Volunteers
- 92 people
- Sponsor
- SCM Lifescience Co., LTD.
Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders
Collecting and Storing Blood Samples From Patients With Cancer
RATIONALE: Collecting and storing samples of blood from patients with cancer to test in the laboratory may help the study of cancer in the future.
- Conditions
- Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders, Leukemia, Lymphoma
- Trial name
- NCT00900120
- Sponsor
- University of Medicine and Dentistry of New Jersey
Larynx Stenosis, Larynx Disease
Laryngeal Allograft Transplantation
The purpose of this proposed study is to obtain safety and efficacy data on human laryngeal allograft transplantation in an effort to safely use these procedures as a viable reconstructive option for patients with severe…
- Conditions
- Larynx Stenosis, Larynx Disease, Laryngectomy, Larynx
- Trial name
- NCT03269396
- Testing
- Larynx Allograft Transplantation
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Mayo Clinic
Neurofibromatosis Type 1, Malignant Peripheral Nerve Sheath Tumor
Natural History Study of Patients With Neurofibromatosis Type I
Background: Neurofibromatosis Type 1 (NF1) is a genetic disorder in which patients are at increased risk of developing tumors (usually non-cancerous) of the central and peripheral nervous system.
- Conditions
- Neurofibromatosis Type 1, Malignant Peripheral Nerve Sheath Tumor, Plexiform Neurofibroma, Optic Glioma
- Trial name
- NCT00924196
- Where
- United States
- Volunteers
- 259 people
- Sponsor
- National Cancer Institute (NCI)
Tracheobronchomalacia
The Effect of Bethanechol on Tracheobronchomalacia
The primary aim of this study is to determine if work of breathing estimated using swing Edi will be improved following initiation of bethanechol in infants with tracheobronchomalacia.
- Trial name
- NCT05299008
- Testing
- Bethanechol
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Arkansas Children's Hospital Research Institute
Newborns
Newborn Sequencing Screening in China
The project will carry out the genetic testing of 10000 neonates. The aim of the project is to assess the application of targeted sequencing technology in the screening of neonatal diseases.
- Trial name
- NCT05476640
- Testing
- NGS
- Where
- China
- Volunteers
- 3,348 people
- Sponsor
- Shanghai Children's Hospital
Neurofibromatosis 1, Peripheral Nerve Neoplasms, Malignant
Incidence of Malignant Peripheral Nerve Sheath Tumor (MPNST) Development in Participants With Neurofibromatosis Type 1 (NF1) Receiving and Not Receiving Medical Therapies Directed at Plexiform Neurofibromas (PN)
Background: NF1 is a genetic syndrome. Tumors appear early in life. Many people with NF1 develop PN. These tumors can become an aggressive cancer called MPNST.
- Trial name
- NCT04774289
- Where
- United States
- Volunteers
- 296 people
- Sponsor
- National Cancer Institute (NCI)
Duchenne Muscular Dystrophy
A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX)
The LYNX study is a 2-part, multicenter, Phase 2 study of safety, pharmacokinetics and biomarkers in children with Duchenne muscular dystrophy including a randomized, double-blind, placebo-controlled part A, followed by an…
- Trial name
- NCT05540860
- Testing
- Sevasemten Dose 1, Sevasemten Dose 2, Sevasemten Dose 3
- Where
- 14 sites in United States
- Volunteers
- 76 people
- Sponsor
- Edgewise Therapeutics, Inc.
Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Inv(16)(p13;q22)
Biological Therapy in Treating Patients With Advanced Myelodysplastic Syndrome, Acute or Chronic Myeloid Leukemia, or Acute Lymphoblastic Leukemia Who Are Undergoing Stem Cell Transplantation
This phase I/II trial is studying the side effects of biological therapy and to see how well it works in treating patients with advanced myelodysplastic syndrome, chronic myeloid leukemia, acute myeloid leukemia, or acute…
- 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
- NCT00052520
- Testing
- therapeutic allogeneic lymphocytes, aldesleukin, peripheral blood stem cell transplantation
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- Fred Hutchinson Cancer Center
Primary Hyperoxaluria, Nephrocalcinosis
International Registry for Primary Hyperoxaluria
The purpose of this study is to collect medical information from a large number of patients in many areas of the world with primary hyperoxaluria.
- Conditions
- Primary Hyperoxaluria, Nephrocalcinosis, Kidney Stones
- Trial name
- NCT00875823
- Where
- 4 sites in United States
- Sponsor
- Mayo Clinic
Congenital Anomaly, Pediatric Cancer
Genetic Overlap Between Anomalies and Cancer in Kids in the Children's Oncology Group: The COG GOBACK Study
One of the strongest risk factors for cancer in children and adolescents is being born with a congenital anomaly. In fact, data from registry linkage studies imply that 10-15% of childhood cancer risk could be attributable to…
- Trial name
- NCT05071859
- Testing
- Whole Genome Sequencing, Questionnaire Administration, Biospecimen collection
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- Children's Oncology Group
Pregnant Women Requiring Amniocentesis
Prenatal Screening for Imprinting Anomalies Implicated in Beckwith Wiedemann and Silver Russell Syndromes
Losses of imprinting are involved in various syndromes. Those occurring in the 11p15 region lead to Beckwith-Wiedemann and Silver-Russell Syndromes.
- Trial name
- DASIRUWIBE (NCT01842659)
- Testing
- Methylation Index
- Where
- France
- Volunteers
- 67 people, healthy volunteers welcome
- Sponsor
- Assistance Publique - Hôpitaux de Paris
glp1 Agonist, Gene Expression
Ellacor® Micro-Coring Technology® in Fitzpatrick Skin Types I-VI, Including Patients Receiving Glucagon-Like Peptide-1 Receptor Agonists
The main purpose of the study is to collect information about how the device functions in a variety of treatment areas and skin types and to find out what changes, if any, occur in the skin treated with the study device compared…
- Conditions
- glp1 Agonist, Gene Expression, Skin Laxity
- Trial name
- NCT07224880
- Testing
- ellacor
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Cytrellis Biosystems, Inc.