Clinical trials: Newborn Diseases
Urinary Incontinence, Epispadias, Male
Autologous Muscle Stem Cell Therapy for Treatment of Congenital Urinary Incontinence in Epispadias Patients
The aim of this trial is to evaluate the safety and efficacy of an autologous muscle stem cell therapy in the treatment of congenital urinary incontinence in isolated epispadias.
- Trial name
- MUST (NCT04729582)
- Testing
- Primary human muscle stem cells (Satori-01)
- Where
- 2 sites in Germany
- Volunteers
- 21 people
- Sponsor
- Simone Spuler, MD
Rendu Osler Disease, Telangiectasia, Hereditary Hemorrhagic
The ELLIPSE Study: A Phase-1 Study Evaluating the Tolerance of Bevacizumab Nasal Spray to Treat Epistaxis in Hereditary Hemorrhagic Telangiectasia
Antiangiogenic drugs, such as bevacizumab, are a new treatment strategy in Hereditary Hemorrhagic Telangiectasia (HHT). Its systemic administration in patients with HHT improves liver damage-related symptoms and epistaxis (cases…
- Trial name
- ELLIPSE (NCT01507480)
- Testing
- Bevacizumab
- Volunteers
- 42 people
- Sponsor
- Hospices Civils de Lyon
Dwarfism
Efficacy and Safety of rhGH (Jintropin®) in Pediatric Participants With ISS
Phase 1: To evaluate the safety and efficacy of 0.05mg/kg/d of rhGH (Jintropin®) in the treatment of children with idiopathic short stature (ISS) in 52 weeks.
- Trial name
- NCT03635580
- Testing
- Negative control
- Where
- 9 sites in China
- Volunteers
- 480 people
- Sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.
Osteogenesis Imperfecta
Bisphosphonate Treatment of Osteogenesis Imperfecta
The primary purpose of this trial is to evaluate whether the investigational medication is safe, effective and has the ability to increase spine bone density in osteogenesis imperfecta (OI) patients.
- Trial name
- NCT00063479
- Testing
- Zoledronic Acid
- Where
- 10 sites in United States
- Volunteers
- 158 people
- Sponsor
- Novartis Pharmaceuticals
Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
A Phase II Study Of The Farnesyltransferase Inhibitor ZANESTRA (R115777, NSC #702818, IND #58,359) In Complete Remission Following Induction And/Or Consolidation Chemotherapy In Adults With Poor-Risk Acute Myelogenous Leukemia (AML) And High-Risk Myelodysplasia (MDS)
Tipifarnib may stop the growth of cancer cells by blocking the enzymes necessary for their growth. Phase II trial to study the effectiveness of tipifarnib in treating patients who have acute myeloid leukemia or myelodysplastic…
- Conditions
- Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22)
- Trial name
- NCT00045396
- Testing
- tipifarnib
- Where
- United States
- Volunteers
- 44 people
- Sponsor
- National Cancer Institute (NCI)
Dentinogenesis Imperfecta, Amelogenesis Imperfecta
Psycho-social Impact of Amelogenesis and Dentinogenesis Imperfecta
The purpose of this research is to study the impact of dentinogenesis (DI) and amelogenesis (AI) imperfecta on oral quality of life, exposure to bullying and dental anxiety in a population of adolescents.
- Trial name
- AiDiBull (NCT04927962)
- Testing
- Questionnaires
- Where
- 7 sites in France
- Volunteers
- 87 people
- Sponsor
- University Hospital, Toulouse
Osteoporosis, Turner's Syndrome
Hormone Replacement Therapy to Treat Turner Syndrome
This study will evaluate the effects of hormone replacement therapy on patients with Turner syndrome (TS)-a genetic disorder in females in which part or all of one X chromosome is missing.
- Trial name
- NCT00013546
- Testing
- TMTDS
- Where
- United States
- Volunteers
- 92 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Hereditary Spherocytosis, Idiopathic Thrombocytopenic Purpura
Single Incision Versus Standard Laparoscopic Splenectomy
This is a prospective trial of single incision versus standard 4-port laparoscopic splenectomy. The hypothesis is that there may be a difference in wound infection rates, operative time, doses of analgesics post-operatively, and…
- Trial name
- NCT01276561
- Testing
- Single Incision Splenectomy, Laparoscopic Splenectomy
- Where
- United States
- Sponsor
- Children's Mercy Hospital Kansas City
Thalassemia, Brain Function (Measured by ERP Level)
A Case Control Study to Evaluate the Cognitive and Brain Function of β-thalassemia Patients.
β-thalassemia syndromes are a group of hereditary disorders characterized by a genetic deficiency in the synthesis of beta-globin chains.
- Trial name
- NCT01935661
- Where
- Israel
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- HaEmek Medical Center, Israel
Respiratory Distress Syndrome (RDS), Infant, Premature, Diseases
FiO2 Delivered With Neonatal bCPAP
Continuous positive airway pressure (CPAP) is the mainstay of treatment recommended for preterm infants with respiratory distress syndrome in all resource contexts.
- Conditions
- Respiratory Distress Syndrome (RDS), Infant, Premature, Diseases, Infant, Newborn, Diseases
- Trial name
- NCT06615427
- Where
- Vietnam
- Volunteers
- 100 people
- Sponsor
- Karolinska Institutet
Cystic Fibrosis
Treatment of Anxiety/Depression and Pain Through Relaxation Yoga for Patient With Cystic Fibrosis (CF)
The investigators research question is whether or not yoga effectively alleviates symptoms of pain, sleep disturbance, anxiety and depression in children with cystic fibrosis.
- Trial name
- NCT01180842
- Testing
- Yoga treatment program
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Children's Hospitals and Clinics of Minnesota
Friedreich Ataxia
Transitional Life Events in Patients With Friedreich's Ataxia: Implications for Genetic Counseling
The purposes of this study are to learn about significant life changes for people with Friedreich's ataxia and about patients' experiences with health care providers.
- Trial name
- NCT00056186
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Pain, Fever
Pharmacokinetics, -Dynamics and Safety of Intravenous Paracetamol in Neonates
The purpose of this study is triple, i.e. document single dose pharmacokinetics and -dynamics of intravenous paracetamol in preterm and term neonates after a loading dose (20 mg/kg iv bolus paracetamol), document multiple dose…
- Trial name
- PARANEO (NCT00969176)
- Testing
- paracetamol
- Where
- Belgium
- Volunteers
- 60 people
- Sponsor
- Universitaire Ziekenhuizen KU Leuven
Periventricular Leukomalacia
Telerehabilitation of Working Memory in Children With Periventricular Leukomalacia and Bilateral Cerebral Palsy
Periventricular Leukomalacia (PVL) is a white matter lesion surrounding the lateral ventricles of the brain occurring in the prenatal period, associated with a disorder of movement and posture, known as bilateral cerebral palsy.
- Trial name
- NCT02342990
- Testing
- Cluster B CogMed: an home-based and computer-based solution to improve working memory deficit, Cluster A No training. Delay start of CogMed
- Where
- Italy
- Volunteers
- 20 people
- Sponsor
- IRCCS Fondazione Stella Maris
Fabry Disease
German Observational Multicenter Study of Patients With Fabry Disease Under Chaperone Therapy With Migalastat-HCl.
The objective of the study is to document long term data on treatment with Migalastat under "real world" conditions. The selection of patients is based on the SmPC/Fachinformation. The study duration/patient will be 2 years.
- Trial name
- NCT03135197
- Where
- Germany
- Volunteers
- 75 people
- Sponsor
- University Hospital Muenster
Hypohidrotic Ectodermal Dysplasia, X-Linked Hypohidrotic Ectodermal Dysplasia
X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED) Carrier Outlook Toward Reproduction Survey
In this survey of X-Lined Hypohidrotic Ectodermal Dysplasia (XLHED) carrier females, Edimer proposes to review responses to an XLHED Carrier Outlook toward Reproduction Survey in order to understand attitudes toward reproduction…
- Trial name
- X-CORS (NCT01398813)
- Where
- United States
- Volunteers
- 500 people
- Sponsor
- Edimer Pharmaceuticals
Atopic Dermatitis
Twice Daily Versus Twice Weekly Soak-and-Seal Baths in Pediatric Atopic Dermatitis
There are few studies evaluating best bathing practices in the management of pediatric atopic dermatitis (AD). Trans-epidermal water loss plays a key role in the pathophysiology of AD.
- Trial name
- NCT03397979
- Testing
- Infrequent versus Frequent Soaking Baths
- Where
- United States
- Volunteers
- 63 people, healthy volunteers welcome
- Sponsor
- MaineHealth
POEMS Syndrome
Treatment of POEMS Syndrome With Daratumumab
This trial investigates the use of Daratumumab (DARA), an antibody directed at the human cluster of differentiation 38 (CD38) molecule, for the treatment of patients with Polyneuropathy, Organomegaly, Endocrinopathy, m Protein…
- Trial name
- NCT04396496
- Testing
- Daratumumab Injection
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- University of Arkansas
Oesophageal Atresia With Tracheo-Oesophageal Fistula, Tracheomalacia
Primary Posterior Tracheopexy Prevents Tracheal Collapse
Introduction: Children born with a blind-ending gullet (oesophagus), or Oesophageal Atresia (OA), need to undergo surgical correction in the first week of life.
- Conditions
- Oesophageal Atresia With Tracheo-Oesophageal Fistula, Tracheomalacia, Oesophageal Atresia
- Trial name
- PORTRAIT (NCT06335862)
- Testing
- Randomisation between the PPT- and no-PPT-group, Primary Posterior Tracheopexy, Preoperative tracheobronchoscopy
- Where
- 3 sites in Netherlands, United Kingdom
- Volunteers
- 78 people
- Sponsor
- UMC Utrecht
Growth Hormone Disorder, Growth Hormone Deficiency in Children
Norditropin NordiFlex® Device Compared to the Device Previously Used by Patients or Parents
This study is conducted in Europe. The aim of this study is to compare the easiness of use of Norditropin NordiFlex® device to the device previously used by patients or parents.
- Conditions
- Growth Hormone Disorder, Growth Hormone Deficiency in Children, Foetal Growth Problem, Small for Gestational Age
- Trial name
- NCT01245374
- Testing
- Norditropin NordiFlex®
- Where
- France
- Volunteers
- 103 people
- Sponsor
- Novo Nordisk A/S
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