Clinical trials: Tumor and Abnormalities of the Development
Leukemia
S9333A Study of Blood and Bone Marrow Samples From Patients With Previously Untreated Primary Acute Myeloid Leukemia
RATIONALE: Studying samples of blood and bone marrow from patients with cancer in the laboratory may help doctors learn more about changes that occur in DNA and identify biomarkers related to cancer.
- Trial name
- NCT00899743
- Testing
- polyacrylamide gel electrophoresis, western blotting, immunologic technique
- Volunteers
- 132 people
- Sponsor
- SWOG Cancer Research Network
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
Hepatoblastoma, Hepatocellular Carcinoma
Yttrium-90 (TARE-Y90) in Children, Adolescents, and Young Adults With Liver Tumors
This study will be performed to evaluate the Clinical Outcomes and Quality of Life after Transarterial Radioembolization with Yttrium-90 (TARE-Y90) in Children, Adolescents, and Young Adults with Liver Tumors.
- Conditions
- Hepatoblastoma, Hepatocellular Carcinoma, Rhabdoid Tumor of Liver, Undifferentiated (Embryonal) Sarcoma of the Liver
- Trial name
- NCT04315883
- Testing
- Quality of Life Assessment, Transarterial Radioembolization
- Where
- 2 sites in United States
- Volunteers
- 3 people
- Sponsor
- Nemours Children's Clinic
Tuberous Sclerosis Complex, Focal Cortical Dysplasia
Astroscape: A Study of Radiprodil on Safety, Tolerability, Pharmacokinetics, and Effect on Seizures and Behavioral Symptoms in Patients With TSC or FCD Type II
Study RAD-GRIN-201 is a phase 1B/2A trial to assess safety, tolerability, pharmacokinetics (PK), and potential efficacy of radiprodil in participants with Tuberous Sclerosis Complex (TSC) or Focal Cortical Dysplasia (FCD) type…
- Trial name
- NCT06392009
- Testing
- Radiprodil
- Where
- 18 sites in Australia, Belgium and 6 more
- Volunteers
- 30 people
- Sponsor
- GRIN Therapeutics, Inc.
Seizure in Participants With Tuberous Sclerosis Complex, Seizure in Participants With Dravet Syndrome
Assessment of Adjunctive Cannabidiol Oral Solution (GWP42003-P) in Children With Tuberous Sclerosis Complex (TSC), Dravet Syndrome (DS), or Lennox-Gastaut Syndrome (LGS) Who Experience Inadequately-controlled Seizures
This study will be conducted to evaluate the safety, pharmacokinetics (PK), and efficacy of adjunctive GWP42003-P in participants < 2 years of age with tuberous sclerosis complex (TSC), Lennox-Gastaut syndrome (LGS), or Dravet…
- Conditions
- Seizure in Participants With Tuberous Sclerosis Complex, Seizure in Participants With Dravet Syndrome, Seizure in Participants With Lennox-Gastaut Syndrome
- Trial name
- NCT04485104
- Testing
- GWP42003-P
- Where
- 11 sites in United States, Italy and 1 more
- Volunteers
- 3 people
- Sponsor
- Jazz Pharmaceuticals
Epilepsy, Dravet Syndrome
A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy, Safety, and Tolerability of TAK-935 (OV935) as an Adjunctive Therapy in Pediatric Participants With Developmental and/or Epileptic Encephalopathies
The purpose of this study is to investigate the effect on the frequency of all seizures (convulsive and drop) in participants treated with TAK-935 compared to placebo.
- Conditions
- Epilepsy, Dravet Syndrome, Lennox-Gastaut Syndrome
- Trial name
- ELEKTRA (NCT03650452)
- Testing
- TAK-935
- Where
- 44 sites in United States, Australia and 6 more
- Volunteers
- 141 people
- Sponsor
- Takeda
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
Tumor and Abnormalities of the Development
Tumor and Development (TED)
The overall project goal is to build a database of childhood cancers associated with developmental anomalies; it aims at identifying new syndromes of genetic predisposition and at enabling the further study of their molecular…
- Trial name
- TED (NCT01915797)
- Testing
- blood and tumor samples
- Where
- France
- Volunteers
- 998 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Neurofibromatosis Type 1, Tumors of the Central Nervous System
Stem Cells in NF1 Patients With Tumors of the Central Nervous System
Objectives 1. Establish an induced pluripotent stem cell (iPSC) bank for phenotypically well-characterized patients with NF1. 2.
- Trial name
- NCT03332030
- Testing
- Collection of Stem Cells
- Where
- United States
- Volunteers
- healthy volunteers welcome
- Sponsor
- Roger Packer
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.
Focal Cortical Dysplasia, Tuberous Sclerosis Complex (TSC)
Safety, Tolerability, and Pharmacokinetics of SVG103 (Paxalisib) in Focal Cortical Dysplasia Type II (FCD-II), Tuberous Sclerosis Complex (TSC) or Hemimegalencephaly (HME)
This is a multinational, open-label, single-arm trial of adjunctive SVG103 (paxalisib) treatment in adults with FCD-II, TSC, and HME.
- Conditions
- Focal Cortical Dysplasia, Tuberous Sclerosis Complex (TSC), Hemimegalencephaly
- Trial name
- NCT07287202
- Testing
- SVG103
- Where
- 2 sites in Australia
- Volunteers
- 12 people
- Sponsor
- Sovargen
Epilepsy, Tuberous Sclerosis Complex
A Pilot Study To Evaluate The Effects of Everolimus on Brain mTOR Activity and Cortical Hyperexcitability in TSC and FCD
The purpose of this study is to measure if the drug called Everolimus effects mTOR signaling (an electrical activity signal in the brain) in patients with Tuberous Sclerosis Complex (TSC) and Focal Cortical Dysplasia (FCD) with…
- Conditions
- Epilepsy, Tuberous Sclerosis Complex, Focal Cortical Dysplasia
- Trial name
- NCT02451696
- Testing
- Everolimus
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- NYU Langone Health
Tuberous Sclerosis Complex
Basimglurant (NOE-101) in Children, Adolescents, and Young Adults With TSC
The study intends to show that basimglurant (NOE-101) provides effective seizure control in children, adolescents and young adults with Tuberous Sclerosis Complex (TSC).
- Trial name
- NCT05059327
- Where
- 27 sites in United States, India and 6 more
- Volunteers
- 61 people
- Sponsor
- Noema Pharma AG
Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1
Baby Detect : Genomic Newborn Screening
Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions.
- Conditions
- Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1, Phosphoglucomutase 1 Deficiency, Maturity Onset Diabetes of the Young
- Trial name
- BabyDetect (NCT05687474)
- Where
- Belgium
- Volunteers
- 6,824 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Liege
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
Leukemia
Tipifarnib, Cytarabine, and Daunorubicin in Treating Older Patients With Acute Myeloid Leukemia
RATIONALE: Tipifarnib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as cytarabine and daunorubicin, work in different ways to stop the growth of…
- Trial name
- NCT00101153
- Testing
- cytarabine, daunorubicin hydrochloride, tipifarnib
- Where
- 2 sites in Canada
- Volunteers
- 24 people
- Sponsor
- University Health Network, Toronto
Exostoses, Multiple Hereditary
Height, Ulnar Length and Forearm Function in Multiple Hereditary Exostoses
the purpose of the present registry is to describe the epidemiology of forearm deformities in patients with Hereditary Multiple Exostoses and to identify, independent predictors of severity of the disease and potential…
- Trial name
- NCT05914298
- Testing
- blood and buccal swab genetic test, PUL, Range of motion
- Where
- Italy
- Volunteers
- 408 people, healthy volunteers welcome
- Sponsor
- Istituto Ortopedico Rizzoli
Leukemia
Everolimus, Cytarabine, and Daunorubicin in Treating Patients With Relapsed Acute Myeloid Leukemia
RATIONALE: Drugs used in chemotherapy, such as cytarabine and daunorubicin, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing.
- Trial name
- NCT00544999
- Testing
- cytarabine, daunorubicin hydrochloride, everolimus
- Where
- France
- Volunteers
- 21 people
- Sponsor
- Institut de Recherche Clinique sur les Cancers et le Sang
Solid Tumor Malignancy
Efficacy and Safety of Pemigatinib in Previously Treated Locally Advanced/Metastatic or Surgically Unresectable Solid Tumor Malignancies Harboring Activating FGFR Mutations or Translocations (FIGHT-207)
The purpose of this study is to evaluate the efficacy and safety of pemigatinib in participants with previously treated locally advanced/metastatic or surgically unresectable solid tumor malignancies harboring activating FGFR…
- Trial name
- NCT03822117
- Testing
- Pemigatinib
- Where
- 90 sites in United States, Denmark and 9 more
- Volunteers
- 111 people
- Sponsor
- Incyte Corporation
Adult Acute Basophilic Leukemia, Adult Acute Eosinophilic Leukemia
Entinostat and Sorafenib Tosylate in Treating Patients With Advanced or Metastatic Solid Tumors or Refractory or Relapsed Acute Myeloid Leukemia
This phase I trial is studying the side effects and the best dose of entinostat when given together with sorafenib tosylate in treating patients with advanced or metastatic solid tumors or refractory or relapsed acute myeloid…
- Conditions
- Adult Acute Basophilic Leukemia, Adult Acute Eosinophilic Leukemia, Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0)
- Trial name
- NCT01159301
- Testing
- entinostat, sorafenib tosylate, pharmacological study
- Where
- United States
- Volunteers
- 44 people
- Sponsor
- National Cancer Institute (NCI)