Clinical trials: Newborn Diseases
Neurofibromatosis Type 1, Precancerous Condition
Fludeoxyglucose F 18 Positron Emission Tomography and Magnetic Resonance Perfusion Imaging in Patients With Neurofibromatosis 1 and Plexiform Neurofibroma
RATIONALE: New imaging procedures such as fludeoxyglucose F 18 positron emission tomography (FDG-PET) and magnetic resonance (MR) perfusion imaging may improve the ability to detect disease progression, help doctors predict a…
- Trial name
- NCT00060008
- Testing
- fludeoxyglucose F 18, gadopentetate dimeglumine
- Where
- 2 sites in United States
- Volunteers
- 18 people
- Sponsor
- Children's Hospital of Philadelphia
Patent Foramen Ovale
Evaluation of the Efficacy and Safety of a Simplified Procedure for the Percutaneous Closure of the Patent Foramen Ovale
Patent Foramen Ovale, is an embryonic remnant, formed by apposition of the septum primum and septum secondum composing the interatrial septum.
- Trial name
- IDFFOP (NCT03828825)
- Testing
- Patent Foramen Ovale closure
- Where
- 3 sites in France
- Volunteers
- 279 people
- Sponsor
- Fondation Hôpital Saint-Joseph
Major Sickle Cell Syndrome of Type SS or Sβ Thalassemia
Assessment of Tolerance of Mobilizing Peripheral Hematopoietic Stem Cells by Plerixafor in Sickle Cell Patients
The purpose of this study is to assess the tolerance and efficacy of mobilizing hematopoietic stem cells after a single injection of plerixafor (0.24mg/kg) in 3 adult patients (or 5, if results of the first 3 patients are not…
- Trial name
- DrepaMob (NCT02212535)
- Testing
- Plerixafor
- Where
- France
- Volunteers
- 3 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Foramen Ovale, Patent
Risk of Ischaemic Stroke After Pulmonary Embolism in Patients With and Without Patent Foramen Ovale
stroke's risk is increased in patients with pulmonary embolism and PFO compared to patients without PFO. Does this increased risk persist years after pulmonary embolism ?
- Trial name
- EPIC-FOPCohort (NCT04987008)
- Where
- 3 sites in France
- Volunteers
- 53 people
- Sponsor
- University Hospital, Brest
Mitochondrial Disorders, Mitochondrial Genetic Disorders
North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)
The North American Mitochondrial Disease Consortium (NAMDC) maintains a patient contact registry and tissue biorepository for patients with mitochondrial disorders.
- Conditions
- Mitochondrial Disorders, Mitochondrial Genetic Disorders, Mitochondrial Diseases, Disorder of Mitochondrial Respiratory Chain Complexes
- Trial name
- NAMDC (NCT01694940)
- Where
- 17 sites in United States, Canada
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- Columbia University
Phenylketonuria
A Study of PTC923 in Participants With Phenylketonuria
The main purpose of this trial is to evaluate the efficacy of PTC923 in reducing blood phenylalanine (Phe) levels in participants with phenylketonuria as measured by mean change in blood Phe levels from baseline to Weeks 5 and 6…
- Trial name
- NCT05099640
- Testing
- PTC923
- Where
- 43 sites in United States, Australia and 13 more
- Volunteers
- 157 people
- Sponsor
- PTC Therapeutics
Anemia, Sickle Cell
Antioxidant Therapy to Reduce Inflammation in Sickle Cell Disease
The purpose of this study is to determine whether alpha-lipoic acid and acetyl-L-carnitine will lower systemic inflammation in patients with Sickle Cell Disease by reducing oxidative stress, which will result in a decrease in the…
- Trial name
- NCT01054768
- Testing
- alpha-lipoic acid and acetyl-L-carnitine, Control
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Cystinosis, Native Kidney
Cystinosis and Mitochondrial Metabolism
Cystinosis is a monogenic autosomal recessive lysosomal storage disease with complete penetrance, caused by a biallelic mutation in the CTNS gene (17p13.2) encoding cystinosin, a ubiquitous membrane protein whose role is to clear…
- Trial name
- CYSTI-MITO (NCT07319091)
- Testing
- Mitochondrial metabolism
- Where
- 9 sites in France
- Volunteers
- 25 people
- Sponsor
- Hospices Civils de Lyon
Rhesus Isoimmunization
Delayed Cord Clamping in Rhesus Disease of the Newborn
The objective of this clinical trial is to investigate the effects of delayed cord clamping on hyperbilirubinemia, the need for phototherapy, neonatal intensive care (NICU) admission
- Trial name
- NCT04600674
- Testing
- Delayed cord clamping, Early cord clamping
- Where
- Egypt
- Volunteers
- 30 people
- Sponsor
- Benha University
Flexible Flatfoot
Foot-Core Training With/Without Visual Feedback in Pediatric FPP
This study investigates flexible pes planus (flat feet) in children, a condition where the medial arch collapses during weight-bearing, affecting gait, balance, and foot function.
- Trial name
- NCT07578974
- Testing
- Foot Core Training, Foot Core Training with Visual Feedback
- Where
- Pakistan
- Volunteers
- 24 people
- Sponsor
- Riphah International University
Atopic Dermatitis
Study to Evaluate the Efficacy, Safety and Pharmacokinetics of ASB17061 Capsules in Adult Subjects With Atopic Dermatitis
The purpose of this research study is to gather scientific information about the effectiveness of the study drug, ASB17061 capsules, when compared to placebo in adult subjects with atopic dermatitis.
- Trial name
- NCT01756898
- Testing
- 5 mg ASB17061, 10 mg ASB17061, 20 mg ASB17061
- Where
- 40 sites in United States
- Volunteers
- 370 people
- Sponsor
- Daiichi Sankyo
Ischemic-Hypoxic Encephalopathy
Long Term Prognostic of Neonatal Hypoxic Ischemic Encephalopathy With Hypothermia Treatment
The primary objective is to evaluate neonatal characteristics, and biological and clinical investigations as predictive factors of death, or of severe and moderate neurodevelopmental disability at 3 years, in a large…
- Trial name
- LyTONEPAL (NCT02676063)
- Where
- 22 sites in France
- Volunteers
- 800 people
- Sponsor
- University Hospital, Grenoble
Behçet's Syndrome, Behçet's Syndrome (BS)
Corticotropin for Behçet's Disease: A Multicenter Randomized Controlled TrialAcronym.
Adrenocorticotropic hormone (ACTH) is a polypeptide hormone secreted by the anterior pituitary gland. It can stimulate adrenal cortical hyperplasia and the secretion of glucocorticoids, thereby exerting anti-inflammatory…
- Trial name
- NCT07833163
- Testing
- Repository Corticotropin Injection (ACTH)
- Where
- China
- Volunteers
- 60 people
- Sponsor
- Peking University People's Hospital
Gastric Adenocarcinoma
Derazantinib Alone or in Combination With Paclitaxel, Ramucirumab or Atezolizumab in Gastric Adenocarcinoma
The purpose of this study was to evaluate the efficacy of derazantinib monotherapy or derazantinib in combination with paclitaxel and ramucirumab in patients with gastric adenocarcinoma (GAC) i.e.
- Trial name
- FIDES-03 (NCT04604132)
- Testing
- Derazantinib, Derazantinib-paclitaxel-ramucirumab combination
- Where
- 81 sites in United States, Argentina and 13 more
- Volunteers
- 47 people
- Sponsor
- Basilea Pharmaceutica
Infertility, Hereditary Diseases
Embryo Health Study
This study intends to determine the patients' perception and motivation to obtain additional information on their preimplantation embryos' risks of polygenic disorders.
- Trial name
- EHS (NCT04528498)
- Testing
- Preimplantation Genetic Testing
- Where
- United States
- Volunteers
- 500 people, healthy volunteers welcome
- Sponsor
- Genomic Prediction Inc.
Cystic Fibrosis, Pulmonary Fibrosis
Role of Genetic Factors in the Development of Lung Disease
This study is designed to evaluate the genetics involved in the development of lung disease by surveying genes involved in the process of breathing and examining the genes in lung cells of patients with lung disease.
- Conditions
- Cystic Fibrosis, Pulmonary Fibrosis, Tuberous Sclerosis, Asthma
- Trial name
- NCT00001532
- Where
- 2 sites in United States
- Volunteers
- 3,500 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
22Q11.2 Deletion Syndrome
Open-Label Study of ZYN002 Administered as a Transdermal Gel to Children and Adolescents With 22q11.2 Deletion Syndrome (INSPIRE)
To evaluate the safety and tolerability of ZYN002 administered as a transdermal gel formulation, for up to 38 weeks, in participants ages 4 to <18 years, in the treatment of 22q.11.2 Deletion syndrome (22qDS).
- Trial name
- INSPIRE (NCT05149898)
- Testing
- ZYN002
- Where
- 3 sites in United States, Australia
- Volunteers
- 20 people
- Sponsor
- Harmony Biosciences Management, Inc.
Prader-Willi Syndrome
Follow-up of Prader Willi Syndrome Infants Treated by Oxytocin and Comparison With Not-treated Infants.
The objective of this study is to collect data on tolerance and effects of early treatment with oxytocin in children with Prader Willi Syndrome aged from 3 to 4 years and to compare these infants with not treated age-matched…
- Trial name
- OT2SUITE (NCT03081832)
- Testing
- Oxytocin, Control
- Where
- France
- Volunteers
- 34 people
- Sponsor
- University Hospital, Toulouse
Thrombocytopenia, Neonatal Alloimmune
A Study of Nipocalimab or Intravenous Immunoglobulin (IVIG) in Pregnancies At Risk of Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT)
The purpose of this study is to assess the efficacy and safety of nipocalimab in reducing the risk of severe fetal and neonatal alloimmune thrombocytopenia (FNAIT).
- Trial name
- FREESIA-3 (NCT06533098)
- Testing
- Nipocalimab, Intravenous immunoglobulins (IVIG), Prednisone
- Where
- 38 sites in United States, Austria and 9 more
- Volunteers
- 50 people
- Sponsor
- Janssen Research & Development, LLC
Hemophilia A
A Study to Evaluate the Safety and Tolerability of Prophylactic Emicizumab in Hemophilia A Patients With Inhibitors
This is a phase IIIb, single arm, open-label, multi-center study to evaluate the safety and tolerability of emicizumab in participants with congenital hemophilia A who have documented inhibitors against Factor VIII (FVIII) at…
- Trial name
- STASEY (NCT03191799)
- Testing
- Emicizumab
- Where
- 72 sites in Australia, Belgium and 22 more
- Volunteers
- 195 people
- Sponsor
- Hoffmann-La Roche
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