Clinical trials: Congenital Malformation
Primary Hyperoxaluria
Primary Hyperoxaluria Mutation Genotyping/Phenotyping
Specific mutations relating to hyperoxaluria will be determined via DNA analysis by the Mayo RKSC research staff.
- Trial name
- NCT02340689
- Testing
- Genetic Analysis
- Where
- United States
- Volunteers
- 1,235 people
- Sponsor
- Mayo Clinic
Gastrointestinal, Colic
Safety and Effect of L. Reuteri on Biomarkers of Inflammation in Healthy Infants With Colic
This protocol is a study of the effects of Lactobacillus reuteri in 45 healthy infants with colic. The study is being conducted in order to prove treatment dose with probiotic (Lactobacillus reuteri) in a clinical setting is safe…
- Trial name
- NCT01849991
- Testing
- Lactobacillus reuteri
- Where
- United States
- Volunteers
- 21 people, healthy volunteers welcome
- Sponsor
- The University of Texas Health Science Center, Houston
Bradykinin-mediated Angioedema
Characterization of Rhythmicity Profiles of Bradykinin-mediated Angioedema Attacks Using a Tracking Smartphone Application.
Bradykinin-mediated angioedema is a rare and disabling disease, characterized by the occurrence of attacks marked by localized swelling of skin, but also of the airways, which can be life-threatening.
- Trial name
- ANGIOAPP (NCT05304091)
- Testing
- Questionnaire on a smartphone application
- Sponsor
- University Hospital, Lille
Behcet Disease
A Study to Evaluate Efficacy and Safety of Infliximab in Participant With Moderate-to-Severe Refractory Intestinal Behcet's Disease
The purpose of this study is to evaluate the efficacy of infliximab in induction regimen by assessing the mean decrease in Disease Activity Index for intestinal Behcet's disease (DAIBD) score of 20 or more in participants with…
- Trial name
- BEGIN (NCT02505568)
- Testing
- Infliximab
- Where
- 5 sites in South Korea
- Volunteers
- 33 people
- Sponsor
- Janssen Korea, Ltd., Korea
Phenylketonuria
A Trial to Examine if Repinatrabit is Processed Differently in Adults With Reduced Liver or Kidney Function Compared to Adults With Normal Liver and Kidney Function
This trial is intended to understand how the body processes repinatrabit in people with moderate liver impairment and severe kidney impairment and also to compare these results with people of similar age, weight, and sex who have…
- Trial name
- NCT07713758
- Testing
- Repinatrabit
- Where
- United States
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
Solid Organ Transplantation, Pregnancy
Long-term Follow-up of the Offspring Born to Mothers With a Solid Organ Transplant, Transplantlines Next Generation
Background Pregnancy after all types of solid organ transplantation (SOT) is possible, although these have higher risk of pregnancy complications for mother and child, such as preeclampsia and preterm birth.
- Conditions
- Solid Organ Transplantation, Pregnancy, Long-term Follow-up, Kidney Transplant
- Trial name
- NCT07291258
- Where
- Netherlands
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- University Medical Center Groningen
Genetic Disease
Newborn Genomic Sequencing Pilot Study
The goal of this clinical trial is to test a new method for newborn screening using whole genome sequencing, called BeginNGS. Newborns who are not suspected of having genetic diseases and who are admitted to the NICU at Rady…
- Trial name
- NCT06276348
- Testing
- Whole genome sequencing, BeginNGS test, WES
- Where
- United States
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Rady Pediatric Genomics & Systems Medicine Institute
Duchenne Muscular Dystrophy
An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort
This is an open label, long-term extension study in approximately 24 male DMD subjects consisting of children (ages 4-12, inclusive) and adolescents (ages 13-16, inclusive) who participated in the MP-104-CL-005 PK study.
- Trial name
- NCT02295748
- Testing
- Deflazacort
- Where
- 4 sites in United States
- Volunteers
- 24 people
- Sponsor
- PTC Therapeutics
Pulmonary Hypertension
Inhaled Iloprost for the Treatment of Persistent Pulmonary Hypertension in the Term and Near Term Infants.
Inhaled Iloprost will be administered to near term infants with Persistent Pulmonary Hypertension of the Newborn in two different doses in order to test safety and efficacy in reducing pulmonary artery pressure.
- Trial name
- NCT00409526
- Testing
- Inhaled Iloprost
- Where
- 2 sites in United States, Israel
- Volunteers
- 1 people
- Sponsor
- University of Chicago
Late Infantile Neuronal Ceroid Lipofuscinosis, Batten Disease
AAVRh.10 Administered to Children With Late Infantile Neuronal Ceroid Lipofuscinosis
The investigators propose to assess the safety and efficacy of a new administration method to deliver a biologic to children with a form of Batten disease using an experimental gene transfer procedure.
- Trial name
- NCT01414985
- Testing
- AAVrh.10CUCLN2
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Weill Medical College of Cornell University
Hemophilia
Genetic Susceptibility to Factor VIII Inhibitors
This international study will identify genetic factors that may influence the development of inhibitory antibodies in patients with hemophilia A after treatment with factor VIII.
- Trial name
- NCT00344435
- Where
- Sweden
- Volunteers
- 1,187 people
- Sponsor
- National Cancer Institute (NCI)
Bronchopulmonary Dysplasia, Lung Diseases
Risk Factors in Bronchopulmonary Dysplasia (Newborn Lung Project)
To investigate risk factors in bronchopulmonary dysplasia (BPD) and to elucidate the relationship between BPD, acute lung disease severity, respiration-related variables, water balance, nutrition, familial predisposition, and…
- Conditions
- Bronchopulmonary Dysplasia, Lung Diseases, Respiratory Distress Syndrome
- Trial name
- NCT00005289
- Volunteers
- 2,202 people
- Sponsor
- University of Wisconsin, Madison
Atopic Dermatitis
Observational Study to Evaluate the Actual Use of Elidel® in Chinese Patients With Mild to Moderate Atopic Dermatitis
The primary objective of the present multicentre, prospective, non-interventional study (NIS) is gathering knowledge on the actual use and effectiveness of Elidel® in Chinese patients with mild to moderate AD affecting sensitive…
- Trial name
- NCT04976868
- Testing
- Pimecrolimus 1% Top Cream
- Where
- 10 sites in China
- Volunteers
- 130 people
- Sponsor
- Mylan Inc.
Solid Tumor (Excluding CNS), Liver Cell Carcinoma
Immunotherapy for Solid Tumor Malignancies in Pediatrics Using Interleukin-15 and -21 Armored Glypican-3-specific Chimeric Antigen Receptor T Cells
This Phase 1, open-label, non-randomized study will enroll pediatric and young adult subjects with relapsed or refractory non-central nervous system (CNS) malignant solid tumors expressing glypican-3 (GPC3) to examine the safety…
- Conditions
- Solid Tumor (Excluding CNS), Liver Cell Carcinoma, Malignant Rhabdoid Tumor, Yolk Sac Tumor
- Trial name
- IMPACT (NCT07148050)
- Testing
- SC-CAR.GPC3xIL15.21 CAR T cells
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Seattle Children's Hospital
Aging, Skin Abnormalities
Efficacy of Polypodium Leucotomos Extract Decreasing UVA Premutagenic and Photoaging Markers
With this study, the investigators will like to determine if taking a dose of the study medication, called Polypodium leucotomos (PL), prevents some of the changes in the skin caused by the adverse effects of UVA, a type of…
- Trial name
- NCT00520910
- Testing
- Polypodium leucotomos
- Where
- United States
- Volunteers
- 10 people, healthy volunteers welcome
- Sponsor
- University of Miami
Transfusion Dependent Beta-Thalassemia
A Study Evaluating the Safety and Efficacy of LentiRed Drug Product in Transfusion-dependent β-Thalassemia [TDT]
This is a single-arm, open label, single-dose study in subjects with transfusion dependent β-thalassaemia. The study will evaluate the safety and efficacy of autologous CD34+ Human Hematopoietic Stem Cells that was transduced…
- Trial name
- NCT05762510
- Testing
- GMCN-508B (LentiRed)
- Where
- China
- Volunteers
- 5 people
- Sponsor
- First Affiliated Hospital of Guangxi Medical University
Bronchopulmonary Dysplasia, Vitamin D Overdose
25OH Vitamin D Overdoses and Risk of Bronchopulmonary Dysplasia or Death
Several studies have demonstrated that vitamin D deficiency at birth is a risk factor of bronchopulmonary dysplasia. However, in an animal model of bronchopulmonary dysplasia vitamin D overdose has also been associated with an…
- Trial name
- 25OHVDORBPDD (NCT05944055)
- Testing
- Is 25 OH vitamin D overdose a risk factor of bronchopulmonary dysplasia or death ?
- Where
- France
- Volunteers
- 173 people
- Sponsor
- Hospices Civils de Lyon
Hemoglobinopathies, Myelodysplastic Syndromes
Magnetic Resonance Imaging (MRI) Assessments of the Heart and Liver Iron Load in Patients With Transfusion Induced Iron Overload
This study will evaluate the change in cardiac iron load over a 53 week period measured by MRI in 2 cohorts of patients
- Conditions
- Hemoglobinopathies, Myelodysplastic Syndromes, Other Inherited or Acquired Anaemia, MPD Syndrome
- Trial name
- NCT00673608
- Testing
- deferasirox
- Where
- 4 sites in Australia
- Volunteers
- 118 people
- Sponsor
- Novartis
Vestibular Schwannoma, Meningioma
Exploratory Evaluation of AR-42 Histone Deacetylase Inhibitor in the Treatment of Vestibular Schwannoma and Meningioma
This will be a multi-center, proof of concept phase 0 study to assess the suppression of p-AKT in Vestibular Schwannoma (VS) and meningiomas by AR-42 in adult patients undergoing tumor resection.
- Conditions
- Vestibular Schwannoma, Meningioma, Acoustic Neuroma, Neurofibromatosis Type 2
- Trial name
- NCT02282917
- Testing
- AR-42
- Where
- 4 sites in United States
- Volunteers
- 7 people
- Sponsor
- Massachusetts Eye and Ear Infirmary
Maxillary Sinusitis
Study to Measure the Impact of Antibiotics on Bacterial Flora in Adults With Acute Sinusitis
The purpose of this study is to measure the speed of microbial eradication due to azithromycin or telithromycin in acute maxillary sinusitis (AMS).
- Trial name
- NCT00245440
- Testing
- Azithromycin, Telithromycin
- Where
- United States
- Volunteers
- 192 people
- Sponsor
- CPL Associates
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