Clinical trials: Newborn Diseases
Liver Diseases, Alagille Syndrome
Longitudinal Study of Genetic Causes of Intrahepatic Cholestasis (LOGIC)
Cholestasis is a condition in which bile is not properly transported from the liver to the small intestine. Cholestasis can be caused by an array of childhood diseases, including the genetic diseases Alagille syndrome (ALGS)…
- Conditions
- Liver Diseases, Alagille Syndrome, Alpha 1-Antitrypsin Deficiency
- Trial name
- LOGIC (NCT00571272)
- Where
- 17 sites in United States, Canada
- Volunteers
- 1,675 people
- Sponsor
- Arbor Research Collaborative for Health
Genetic Conditions, Genetic Disorders
RNA to the RESCUE: Evaluation to Assess the Clinical Utility of RNA Sequencing in Establishing a Genetic Diagnosis or Adjudicating a Previously Established Genetic Diagnosis.
The goal of this observational study is to see if ribonucleic acid (RNA) sequencing can improve the diagnostic yield and accuracy of genetic testing compared to gene sequencing alone.
- Trial name
- NCT07787975
- Testing
- RNA sequencing
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- University of California, San Francisco
Asphyxia Neonatorum, Birth Asphyxia
Augmented Infant Resuscitator (AIR): Transitioning a Novel Behavior Change Innovation to Drive Newborn Ventilation Skills Enhancement
Augmented Infant Resuscitator (AIR) is an inexpensive add-on, compatible with nearly every existing bag-valve mask and many types of ventilation equipment.
- Conditions
- Asphyxia Neonatorum, Birth Asphyxia, Perinatal Asphyxia
- Trial name
- NCT05349175
- Testing
- Augmented Infant Resuscitator (AIR)
- Where
- United States
- Volunteers
- 250 people, healthy volunteers welcome
- Sponsor
- Massachusetts General Hospital
Functional Gastrointestinal Disorders, Infant, Newborn, Diseases
Quantification of Fecal Calprotectin Concentration Difference Between Infants Aged Under 4 Months Old Suffering From Functional Gastrointestinal Disorders (FGID) and Infants Aged Under 4 Months Old Free From Disorders : a Transversal Case-control Monocentric Study
There is a high prevalence of FGID among infants : 30 % for regurgitations, 20 % for colitis , 15 % for functional constipation.This represents a frequent motive for pediatrics consultation : 23 to 28 % of medical consultations…
- Trial name
- Cal-IBS (NCT05936112)
- Testing
- Fecal calprotectin dosage, Questionnaire and interview with parents
- Volunteers
- 100 people
- Sponsor
- Centre Hospitalier Universitaire de Besancon
Cystic Fibrosis
Urinary and Anorectal Functional Disorders and Their Impact on CF Adults (PerineoMucoRMO)
This observational study evaluates prevalence of functional perinea disorders (stress urinary incontinence, overactive bladder syndrome, dysuria, anal incontinence) on CF adults patients of the North-West CF Network.
- Trial name
- PerineoMuco (NCT02894619)
- Where
- 6 sites in France
- Volunteers
- 180 people
- Sponsor
- Fondation Ildys
Cystic Fibrosis
Antibiotic Treatment Of Staphylococcus Aureus In Stable People With CF
This is a randomized, double-blinded study that aims to assess the effect of an oral antibiotic called Cephalexin (150 mg/kg/day) compared to placebo in clinically stable children with cystic fibrosis who have grown a bacteria…
- Trial name
- ASAP-CF (NCT04553419)
- Testing
- Cephalexin
- Where
- 2 sites in Canada
- Volunteers
- 86 people
- Sponsor
- University of British Columbia
Atopic Dermatitis, Eczema
Effects of an Emollient Containing Postbiotic Saccharomyces and Lactobacillus on Pediatric Atopic Dermatitis
The goal of this study is to investigate whether the use of emollients containing postbiotic Saccharomyces and Lactobacillus can significantly reduce the risk of atopic dermatitis (AD) recurrence in pediatric patients aged 0-6…
- Conditions
- Atopic Dermatitis, Eczema, Emollient
- Trial name
- NCT06474728
- Testing
- Treatment with an Emollient Containing Postbiotic Saccharomyces and Lactobacillus
- Where
- China
- Volunteers
- 98 people
- Sponsor
- Children's Hospital of Fudan University
Adrenal Insufficiency
Extension Study for Patients Entered Into Study Infacort 003
A Phase 3, open-label, single-group, non-randomised, observational study of the safety and biochemical disease control of Infacort® in neonates, infants and children with adrenal insufficiency and congenital adrenal hyperplasia…
- Trial name
- NCT02733367
- Testing
- Infacort®
- Where
- Germany
- Volunteers
- 18 people
- Sponsor
- Neurocrine UK Limited
Univentricular Heart, Lymphatic Abnormalities
Lymphatic Function in Patients With a Fontan Circulation From Brazil
It is suggested that the elevated central venous pressure in patients with a Fontan circulation can provoke manifestation of lymphatic dysfunction, met as plastic bronchitis, peripheral oedema and most feared protein-losing…
- Conditions
- Univentricular Heart, Lymphatic Abnormalities, Lymphatic Edema
- Trial name
- NCT04394507
- Testing
- Near-Infrared Fluorescence imaging, Strain Gauge Plethysmography
- Where
- 2 sites in Denmark
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- University of Aarhus
Sickle Cell Disease (SCD)
Phenotypic and Transcriptomic Description of Megakaryocytes in Sickle Cell Patient
Sickle cell disease is the most common inherited blood disorder in the world. Chronic hemolysis induces platelet activation and chronic inflammation.
- Trial name
- MEGADREP (NCT06887907)
- Where
- France
- Volunteers
- 40 people
- Sponsor
- University Hospital, Toulouse
Fanconi's Anemia, Myelodysplastic Syndromes
Study of Allogeneic Bone Marrow Transplantation Following Cyclophosphamide and Radiotherapy in Patients With Myelodysplastic Syndrome and Acute Leukemia Related to Fanconi's Anemia
OBJECTIVES: I. Determine the effectiveness of moderate dose cyclophosphamide and radiotherapy in terms of improving survival and reducing the morbidity following allogeneic bone marrow transplantation in patients with…
- Conditions
- Fanconi's Anemia, Myelodysplastic Syndromes, Leukemia, Nonlymphocytic, Acute, Leukemia, Lymphocytic, Acute
- Trial name
- NCT00005892
- Testing
- cyclophosphamide, Allogeneic Bone Marrow Transplantation
- Where
- United States
- Sponsor
- Fairview University Medical Center
Aortic Geometry, Vascular Remodeling
Impact of Aortic Geometry on Vascular Remodeling After Stent Implantation in Coarctation of the Aorta
This study aims to assess: 1. Aortic geometrical changes and their relationship to hypertension and cardiovascular events. 2. Aortic geometrical differences between healthy individuals and patients with repaired coarctation of…
- Conditions
- Aortic Geometry, Vascular Remodeling, Coarctation of Aorta, Blood Pressure
- Trial name
- NCT07131111
- Testing
- CT aortography, CMR
- Where
- Egypt
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Assiut University
Hypoxic Ischaemic Encephalopathy
Xenon and Cooling Therapy in Babies at High Risk of Brain Injury Following Poor Condition at Birth
This study examines the effect of inhaled xenon gas in the treatment of newborn infants with hypoxic-ischemic encephalopathy (HIE) in combination with cooling, which is the standard treatment for this condition.
- Trial name
- CoolXenon2 (NCT01545271)
- Testing
- xenon gas, Whole body cooling
- Where
- United Kingdom
- Volunteers
- 32 people
- Sponsor
- University Hospitals Bristol and Weston NHS Foundation Trust
Muscular Dystrophy, Duchenne
Safety Study of BLS-M22 in Healthy Volunteers
BLS-M22 is being developed as an anti-myostatin agent for the treatment of Duchenne Muscular Dystrophy (Muscular Dystrophy). A total of 37 subjects participated in this study to confirm the safety of BLS-M22.
- Trial name
- NCT03789734
- Testing
- BLS-M22
- Where
- South Korea
- Volunteers
- 37 people, healthy volunteers welcome
- Sponsor
- BioLeaders Corporation
Hereditary Angioedema
Efficacy and Safety of GNR-038 vs Berinert® in Patients With Hereditary Angioedema
It is a placebo-controlled randomized trial to evaluate the efficacy and safety of GNR-038 in comparison with Berinert® in patients with hereditary angioedema
- Trial name
- NCT04898309
- Testing
- GNR-038, 50 МЕ/ kg, GNR-038, 100 МЕ/ kg, Berinert®, 20 МЕ/ kg
- Where
- 5 sites in Russia
- Sponsor
- AO GENERIUM
Cardiomyopathy, Dilated
Immunoadsorption Therapy for Patients With Non-Ischemic Dilated Cardiomyopathy (DCM)
The purpose of this study is to evaluate the clinical safety and feasibility of Mysorba in patients with chronic non-ischemic dilated cardiomyopathy (DCM).
- Trial name
- NCT01478087
- Testing
- Mysorba
- Where
- 2 sites in United States
- Volunteers
- 2 people
- Sponsor
- Asahi Kasei Medical Co., Ltd.
Niemann-Pick Type C Disease, GM1 Gangliosidosis
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis
An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of Niemann-Pick type C disease and in…
- Conditions
- Niemann-Pick Type C Disease, GM1 Gangliosidosis, GM2 Gangliosidosis
- Trial name
- NCT07054515
- Testing
- AZ-3102
- Where
- 39 sites in United States, Argentina and 15 more
- Volunteers
- 147 people
- Sponsor
- Azafaros B.V.
Birt-Hogg-Dube Syndrome
Assessment of Safety of Air Travel in Patients With Birt-Hogg-Dube Syndrome
The aim of this study is to conduct survey-based assessments for the safety of air travel in patients with Birt-Hogg-Dube syndrome (BHD).
- Trial name
- NCT03040115
- Testing
- Non-Interventional Study
- Where
- United States
- Volunteers
- 104 people
- Sponsor
- University of Cincinnati
Critical Illness, Parenteral Nutrition
Protein Restriction In Parenteral Nutrition For Critically Ill Neonates During The Acute Phase: A Preliminary Study
This study aims to determine the differences in outcomes in groups with protein restriction in parenteral nutrition of critically ill infants, compared with protein regimens according to local guidelines.
- Trial name
- NCT07801040
- Testing
- Amino acid infusion
- Where
- Indonesia
- Volunteers
- 52 people
- Sponsor
- Universitas Indonesia
Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0)
Clofarabine, Cytarabine, and Filgrastim Followed by Infusion of Non-HLA Matched Ex Vivo Expanded Cord Blood Progenitors in Treating Patients With Acute Myeloid Leukemia
This phase I trial is studying the safety and potential efficacy of infusing non-human leukocyte antigen (HLA) matched ex vivo expanded cord blood progenitors following treatment with clofarabine and cytarabine for patients with…
- Conditions
- Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0), Adult Acute Monoblastic Leukemia (M5a), Adult Acute Monocytic Leukemia (M5b)
- Trial name
- NCT01031368
- Testing
- cytarabine, clofarabine, filgrastim
- Where
- United States
- Volunteers
- 29 people
- Sponsor
- Nohla Therapeutics, Inc.
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