Clinical trials: Syndrome
TSC, Tuberous Sclerosis Complex
Long-term, Prospective Study Evaluating Clinical and Molecular Biomarkers of Epileptogenesis in a Genetic Model of Epilepsy - Tuberous Sclerosis Complex
The primary objective of clinical part of EPISTOP project is to identify the clinical and molecular biomarkers of epileptogenesis in a prospective clinical study of patients with TSC.
- Conditions
- TSC, Tuberous Sclerosis Complex, Epilepsy
- Trial name
- EPISTOP (NCT02098759)
- Testing
- epilepsy early diagnosis protocol
- Where
- 9 sites in Austria, Belgium and 6 more
- Volunteers
- 100 people
- Sponsor
- Sergiusz Jozwiak
Wiskott-Aldrich Syndrome
Gene Therapy for WAS
This is a phase I/II study to evaluate the safety and efficacy of Hematopoietic Stem Cell genetherapy for the Wiskott-Aldrich Syndrome.
- Trial name
- NCT01347346
- Testing
- Autologous CD34 positive cells transduced with a lentiviral vector containing human WAS gene
- Where
- France
- Volunteers
- 5 people
- Sponsor
- Genethon
Prader-Willi Syndrome
Study of Tesomet With Open-label Extension in Subjects With Prader-Willi Syndrome
This study will evaluate the safety and efficacy of Tesomet (tesofensine + metoprolol) in subjects with PWS.
- Trial name
- PWS (NCT05198362)
- Testing
- Tesomet
- Where
- United States
- Sponsor
- Saniona
Cryptosporidiosis, HIV Infections
Safety and Effectiveness of Nitazoxanide for the Treatment of Cryptosporidiosis in AIDS Patients
The purpose of this study is to see if it is safe and effective to treat cryptosporidiosis in AIDS patients with nitazoxanide.
- Trial name
- NCT00004986
- Testing
- Nitazoxanide
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Romark Laboratories L.C.
Fanconi Anemia
FANCA Gene Transfer for Fanconi Anemia Using a High-safety, High-efficiency, Self-inactivating Lentiviral Vector
This is a Phase I/II clinical trial of gene therapy for treating Fanconi anemia using a self-inactivating lentiviral vector to functionally correct the defective gene.
- Trial name
- NCT03351868
- Testing
- Gene-modified autologous stem cells
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Shenzhen Geno-Immune Medical Institute
Chronic Pain, Chronic Pain Syndrome
Interoceptive Awareness and Function in Adolescents With Chronic Pain
The main objectives of this study are to compare the levels of interoceptive awareness and self-reported function pre and post completion of an occupational therapy treatment plan.
- Conditions
- Chronic Pain, Chronic Pain Syndrome, Pain, Chronic
- Trial name
- NCT07325110
- Testing
- Occupational Therapy
- Where
- United States
- Volunteers
- 49 people
- Sponsor
- Connecticut Children's Medical Center
Leukemia, Myelodysplastic Syndromes
S9910, Collecting and Storing Blood and Bone Marrow Samples From Patients With Hematologic Cancer
RATIONALE: Collecting and storing samples of blood and bone marrow from patients with cancer to study in the laboratory may help doctors learn more about diagnosing cancer and how well patients will respond to treatment.
- Conditions
- Leukemia, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms
- Trial name
- NCT00919425
- Testing
- biologic sample preservation procedure
- Where
- 292 sites in United States
- Volunteers
- 1,294 people
- Sponsor
- SWOG Cancer Research Network
HIV Infections
An Open-Label Safety Program for the Use of Zalcitabine (Dideoxycytidine; ddC) in Pediatric Patients With Symptomatic HIV Infection Who Have Failed or Are Intolerant to AZT Monotherapy, or Who Have Completed Other ddC Protocols, or Are Ineligible for Other Ongoing Clinical Studies
To allow, on a compassionate use basis, zalcitabine (ddC) for pediatric patients with symptomatic HIV disease who have failed treatment or who are intolerant to zidovudine (AZT), or who have completed other ddC protocols, or who…
- Trial name
- NCT00002118
- Testing
- Zalcitabine
- Where
- United States
- Volunteers
- 600 people
- Sponsor
- Hoffmann-La Roche
Parvovirus B19 Infection
Parvovirus B19 Infection in Hereditary Hemolytic Anemias Patients
Although many studies investigated the prevalence and manifestations of HPV-B19 infection in patients with sickle cell anemia (SCA), thalassemia, and hereditary spherocytosis (HS) separately, there is limited information about…
- Trial name
- NCT04822090
- Testing
- Supportive treatment with or without red cell transfusion/intravenous immune globulin (IVIG)
- Where
- Egypt
- Volunteers
- 244 people
- Sponsor
- Sohag University
Multiple Osteochondroma, Osteogenesis Imperfecta
Resilience and Coping in a Rare Skeletal Disease Population to Face Coronavirus (COVID-19) Outbreak Distress: a Longitudinal Study
In the COVID-19 outbreak context, people living with rare diseases have been highly troubled with anxiety, loneliness, and depression.
- Conditions
- Multiple Osteochondroma, Osteogenesis Imperfecta, Ollier Disease, Maffucci Syndrome
- Trial name
- RELOAD (NCT04844697)
- Where
- Italy
- Volunteers
- 15 people
- Sponsor
- Istituto Ortopedico Rizzoli
Celiac Disease, Eosinophilic Esophagitis
Prevalence of Eosinophilic Esophagitis in Children With Celiac Disease
Celiac disease (CD) and eosinophilic esophagitis (EE) are distinct diseases of the gastrointestinal tract with specific clinico-pathological characteristics.
- Trial name
- NCT02190513
- Volunteers
- 56 people
- Sponsor
- St. John Health System, Michigan
Severe Sepsis, Sepsis
Early Identification and Effective Management of Pediatric Sepsis
In patients diagnosed as sepsis on PICU admission, early and accurate identification of patients who will develop organ dysfunction (severe sepsis) is critical for effective management and positive outcome.
- Conditions
- Severe Sepsis, Sepsis, Septic Shock
- Trial name
- NCT03996720
- Where
- 4 sites in China
- Volunteers
- 175 people
- Sponsor
- Children's Hospital of Fudan University
Neurofibromatosis Type 1
A Randomized Placebo-Controlled Study of Lovastatin in Children With Neurofibromatosis Type 1
The specific aim of this study is to determine whether Lovastatin ™ significantly improves visual spatial learning and/or sustained attention in children with NF1.
- Trial name
- STARS (NCT00853580)
- Testing
- Lovastatin ™
- Where
- 12 sites in United States, Australia
- Volunteers
- 146 people, healthy volunteers welcome
- Sponsor
- University of Alabama at Birmingham
MPS IIIB (Sanfilippo B Syndrome)
Evaluation of Blood Brain Barrier Integrity and Structural Abnormalities in MPS IIIB Patients Using Multimodal Magnetic Resonance Imaging
The purpose of the study is to characterize structural abnormalities in the brain and the integrity of the blood brain barrier in patients with mucopolysaccharidosis type IIIB (MPS IIIB).
- Trial name
- NCT02090179
- Where
- United Kingdom
- Volunteers
- 5 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Metabolic Cardiovascular Syndrome
Health Improvement After Pregnancy (HIP) Program Randomized Control Trial
Recent Canadian studies, public opinion polls and the Canadian Heart Health Strategy and Action Plan state that women's heart health is a key national priority; it should be addressed through improving heart health awareness and…
- Trial name
- HIP (NCT02077179)
- Testing
- HIP Program
- Where
- Canada
- Volunteers
- 43 people
- Sponsor
- Queen's University
Neonatal Respiratory Distress Syndrome, PreTerm Neonate
MINImising Total Radiation EXposure in Preterm Infants
Being born too early (preterm birth) is the leading cause of death in children world-wide. In Australia, 97% of very preterm babies who are admitted to Neonatal Intensive Care Units need breathing support after birth to survive.
- Trial name
- MINI T-Rex (NCT06975189)
- Testing
- Lung ultrasound group, Standard Care
- Where
- Australia
- Volunteers
- 180 people
- Sponsor
- Western Health, Australia
Angelman Syndrome
Angelman Syndrome Natural History Study
The goal of this study is to conduct a prospective, longitudinal natural history study of children and adults with Angelman Syndrome using investigator-observed and parent-reported outcome measures to obtain data that will be…
- Trial name
- NCT04507997
- Where
- 11 sites in United States, Canada
- Volunteers
- 300 people
- Sponsor
- Boston Children's Hospital
Systemic Lupus, Sjogren's Syndrome
Evaluation of Influenza A/H3N2 Vaccine in Patients With Rheumatologic Diseases
Studies in the literature have shown reduced effectiveness of influenza A (H3N2) virus vaccine (20-40%) when compared to A (H1N1) and influenza B.
- Trial name
- NCT03540823
- Testing
- Inactivated and fragmented influenza vaccine (A / Michigan / 45/2015 (H1N1) pdm09-like virus, A / Singapore / INFIMH-16-0019 / 2016 (H3N2) -like virus; B / Phuket / 3073/2013-like virus)
- Where
- Brazil
- Volunteers
- 300 people, healthy volunteers welcome
- Sponsor
- University of Sao Paulo General Hospital
Kawasaki Disease
Different Doses of IVIG for Kawasaki Disease
The objective of this study is to investigate the effect of different doses of intravenous immunoglobulin (IVIG) (1g/kg once, 1g/kg twice, 2g/kg once) for Kawasaki disease (KD) in a multicentre, prospective,randomised trial.
- Trial name
- NCT02439996
- Testing
- IVIG (1g/kg,once), IVIG (1g/kg,twice), IVIG (2g/kg.once)
- Where
- China
- Volunteers
- 404 people
- Sponsor
- Children's Hospital of Fudan University
Congenital Adrenal Hyperplasia
Androgen Reduction in Congenital Adrenal Hyperplasia, Phase 1
Children with congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency tend to have elevated circulating levels of androgens, which can accelerate skeletal maturation and adversely impact adult height.
- Trial name
- NCT02574910
- Testing
- Abiraterone acetate
- Where
- 4 sites in United States
- Volunteers
- 4 people
- Sponsor
- University of Texas Southwestern Medical Center
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