Clinical trials: Tumor and Abnormalities of the Development
Leukemia
Combination Chemotherapy, Interleukin-2, and Peripheral Stem Cell Transplant in Treating Patients With Acute Myeloid Leukemia
RATIONALE: Giving combination chemotherapy before a peripheral blood stem cell transplant stops the growth of cancer cells by stopping them from dividing or killing them.
- Trial name
- NCT00004128
- Testing
- aldesleukin, filgrastim, busulfan
- Volunteers
- 2,000 people
- Sponsor
- European Organisation for Research and Treatment of Cancer - EORTC
Exostoses, Multiple Hereditary
The Health-Related Quality of Life in Patients With Hereditary Multiple Exostoses
The purpose of this study is to assess the health-related quality of life of subjects who have Hereditary Multiple Exostoses and to develop a disease specific quality of life survey.
- Trial name
- NCT00474448
- Where
- Canada
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- University of British Columbia
Tuberous Sclerosis Complex
Clinical Presentation and Renal Outcome of Patients With Tuberous Sclerosis Complex and/or Renal Angiomyolipoma in the Great West Region of France
This study aims to investigate the factors (clinical, care-related and genetic) affecting renal outcome in patients with TSC (Tuberous sclerosis complex)
- Trial name
- TSAR (NCT02887781)
- Where
- 2 sites in France
- Volunteers
- 150 people
- Sponsor
- University Hospital, Brest
Tuberous Sclerosis Complex, Seizures
An Open-label Extension Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6)
This trial consists of 2 parts: a double-blinded phase and an open-label extension phase. The open-label extension phase only will be described in this record. All participants will receive the same dose of GWP42003-P.
- Trial name
- NCT02544750
- Testing
- GWP42003-P
- Where
- United States
- Volunteers
- 199 people
- Sponsor
- Jazz Pharmaceuticals
ALS, Autism Spectrum Disorder
Pharmacokinetic (PK) and Pharmacodynamics (PD) Study of Ilera Specific Products
This is an observational study of medical marijuana manufactured and dispensed by Ilera and given as standard treatment for a variety of approved serious medical conditions as defined by individual state law.
- Conditions
- ALS, Autism Spectrum Disorder, Cancer, Spasticity, Muscle
- Trial name
- Ilera (NCT03886753)
- Testing
- Registry, PK microsampling of blood
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Children's Hospital of Philadelphia
Lennox Gastaut Syndrome, Dravet Syndrome
Assessment of Potential for Chronic Liver Injury in Participants Treated With Epidiolex (Cannabidiol) Oral Solution
This study will monitor for potential chronic liver injury and liver fibrosis, in participants treated with cannabidiol oral solution.
- Conditions
- Lennox Gastaut Syndrome, Dravet Syndrome, Tuberous Sclerosis Complex
- Trial name
- NCT05044819
- Testing
- Cannabidiol
- Where
- 26 sites in United States
- Volunteers
- 154 people
- Sponsor
- Jazz Pharmaceuticals
Chronic Kidney Disease, Proteinuria
A Smart Phone Application to Improve Adoption of the 2024 Kidney Disease Improving Global Outcomes (KDIGO) Chronic Kidney Disease (CKD) Guidelines
The goal of this study is to establish whether use of a digital intervention can improve adherence and alignment with the Kidney Disease: Improving Global Outcomes (KDIGO) Chronic Kidney Disease (CKD) 2024 Guidelines.
- Conditions
- Chronic Kidney Disease, Proteinuria, Blood Pressure Control, Congenital Anomalies of the Kidneys and Urinary Tract
- Trial name
- NCT07561957
- Testing
- Use of an electronic digital Application
- Where
- 2 sites in Ireland
- Volunteers
- 80 people
- Sponsor
- St. James's Hospital, Ireland
Facial Angiofibromas
Topical Everolimus in Patients With Tuberous Sclerosis Complex
Tuberous sclerosis complex (TSC) is a rare genetic multisystem disorder characterized by the development of hamartomas in several organs (e.g. brain, heart, kidney, liver, lung), and skin in more than 90% of cases.
- Trial name
- EVEROST (NCT02860494)
- Testing
- Everolimus
- Where
- France
- Sponsor
- Hospices Civils de Lyon
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)
Pancreatic Exocrine Insufficiency
The Development of a Metabolomic Test to Diagnose and Quantify Pancreatic Exocrine Insufficiency (The DETECTION Study)
DETECTION. The development of a metabolomic test to diagnose and quantify pancreatic exocrine insufficiency.
- Trial name
- DETECTION (NCT05980221)
- Testing
- Metabolomics, 13C MTGT
- Where
- United Kingdom
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- University Hospital Birmingham NHS Foundation Trust
Osteogenesis Imperfecta, Osteoporosis
REMS25: Study on the Use of REMS Technology in Diseases Commonly Associated With Reduced Bone Mineral Density (BMD)
This study evaluates bone mineral density (BMD) in pediatric patients aged 5-18 years with conditions negatively affecting bone health, using REMS (Radiofrequency Echographic Multi Spectrometry), a non-invasive and radiation-free…
- Conditions
- Osteogenesis Imperfecta, Osteoporosis, Hypogonadisms, Neoplasia
- Trial name
- NCT07412782
- Testing
- REMS
- Where
- Italy
- Volunteers
- 100 people
- Sponsor
- Meyer Children's Hospital IRCCS
Polycystic Ovary Syndrome, Hyperandrogenism
Effect of Metformin on Sensitivity of the GnRH Pulse Generator to Suppression by Estradiol and Progesterone
Many, but not all, girls with high levels of the male hormone testosterone go on to develop polycystic ovary syndrome (PCOS) as adults.
- Trial name
- NCT01427595
- Testing
- Metformin, Progesterone, estrace
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- University of Virginia
Tuberous Sclerosis Complex, Epilepsy
The Effectiveness and Safety of Resective Epilepsy Surgery for TRE
A prospective cohort studies to identify clinical seizure control, cognitive changes, and safety in resective epilepsy surgery in patients with TSC-related drug-resistant epilepsy.
- Trial name
- NCT04198181
- Testing
- Surgery
- Where
- China
- Volunteers
- 200 people
- Sponsor
- Beijing Children's Hospital
Sleep Problems
Assessment of the Pharmacokinetics of Circadin® in Children With Neurodevelopmental Disorders and Sleep Disturbances
There is increasing evidence that chronic sleep disorders in children with autism spectrum disorder (ASD), Angelman Syndrome (AS) and Smith-Magenis syndrome (SMS) are associated with disturbed melatonin secretion and melatonin…
- Trial name
- NCT01903681
- Testing
- Circadin 2 mg, Circadin 10 mg
- Where
- Netherlands
- Volunteers
- 16 people
- Sponsor
- Neurim Pharmaceuticals Ltd.
Hepatic Sarcoma
Undifferentiated Embryonal Sarcoma of the Liver: Evaluation of the Relapse Profile According to the Therapies Administered
Undifferentiated embryonal sarcoma of the liver is the 3rd most common malignant liver tumor after hepatoblastoma and hepatocellular carcinoma with a peak incidence between 6 and 10 years of age.
- Trial name
- SARCOM (NCT05640960)
- Where
- France
- Volunteers
- 30 people
- Sponsor
- University Hospital, Strasbourg, France
Gut Microbiome, Gastrointestinal Microbiome
A Non-Interventional Pilot Study to Explore the Role of Gut Flora in Disease
This study seeks to correlate microbiome sequencing data with information provided by patients and their medical records.
- Conditions
- Gut Microbiome, Gastrointestinal Microbiome, Autism, Autism Spectrum Disorder
- Trial name
- NCT04031469
- Testing
- There is no intervention in this study
- Where
- United States
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- ProgenaBiome
Desmoplastic/Nodular Medulloblastoma, Medulloblastoma
Combination Chemotherapy in Treating Younger Patients With Newly Diagnosed, Non-metastatic Desmoplastic Medulloblastoma
This phase II trial studies how well combination chemotherapy works in treating younger patients with newly diagnosed, non-metastatic desmoplastic medulloblastoma.
- Conditions
- Desmoplastic/Nodular Medulloblastoma, Medulloblastoma, Medulloblastoma With Extensive Nodularity, Nevoid Basal Cell Carcinoma Syndrome
- Trial name
- NCT02017964
- Testing
- Carboplatin, Cognitive Assessment, Cyclophosphamide
- Where
- 173 sites in United States, Australia and 2 more
- Volunteers
- 26 people
- Sponsor
- Children's Oncology Group
Lung Disease, Pneumothorax
Study of the Disease Process of Lymphangioleiomyomatosis
Pulmonary lymphangioleiomyomatosis (LAM) is a destructive lung disease typically affecting women of childbearing age. Currently, there is no effective therapy for the disease and the prognosis is poor.
- Conditions
- Lung Disease, Pneumothorax, Tuberous Sclerosis, Lymphangioleiomyomatosis
- Trial name
- NCT00001465
- Testing
- Toshibia Aquilion One CT
- Where
- United States
- Volunteers
- 2,000 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Tuberous Sclerosis Complex, Epilepsy
The Effectiveness and Safety of Vagus Nerve Stimulation for TRE
Prospective cohort studies to identify clinical epilepsy control, cognitive changes, and safety of VNS in patients with tuberous sclerosis complex-related epilepsy.
- Trial name
- NCT04198207
- Where
- China
- Volunteers
- 70 people
- Sponsor
- Beijing Children's Hospital
Exostoses, Multiple Hereditary
An Efficacy and Safety Study of Palovarotene for the Treatment of MO
This is a randomized, double-blind, placebo-controlled study comparing the safety and efficacy of 2 dosage regimens of palovarotene versus placebo in preventing disease progression in pediatric subjects with multiple…
- Trial name
- MO-Ped (NCT03442985)
- Testing
- Palovarotene 2.5 mg, Palovarotene 5.0 mg
- Where
- 31 sites in United States, Australia and 10 more
- Volunteers
- 193 people
- Sponsor
- Clementia Pharmaceuticals Inc.