Clinical trials: Neoplastic Syndromes, Hereditary
Developmental and Epileptic Encephalopathy, Dravet Syndrome
Study to Investigate LP352 in Subjects With Developmental and Epileptic Encephalopathies
The objective of this study is to assess the safety, tolerability, efficacy, and pharmacokinetics of adjunctive therapy of LP352 in adults and adolescents with developmental and epileptic encephalopathies.
- Conditions
- Developmental and Epileptic Encephalopathy, Dravet Syndrome, Lennox Gastaut Syndrome
- Trial name
- PACIFIC (NCT05364021)
- Testing
- LP352
- Where
- 34 sites in United States, Australia
- Volunteers
- 52 people
- Sponsor
- Longboard Pharmaceuticals
Myopathy
Maintenance of Muscle Mass in Older People: the Negative Impact of Statin Therapy
A major contributor to frailty and immobility in the elderly is the age related loss of muscle mass (sarcopenia). Cardiovascular disease (CVD) is the leading cause of mortality in the elderly, with high blood cholesterol and…
- Trial name
- NCT01047163
- Where
- United Kingdom
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- University of Nottingham
PTEN Hamartoma Syndrome
Natural History With Focus on Oncological Risk Evaluation in Pediatric Patients With PTEN Pathogenic Variants
This is an observational study in pediatric patientis carryng PTEN pathogenic variants aimed to define oncological risk in children and provide a deeper insight of the clinical course, establishing an updated follow-up protocol.
- Trial name
- PTEN_Ped (NCT06805734)
- Where
- Italy
- Volunteers
- 50 people
- Sponsor
- Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta
Neoplasms
A Rollover Study of Alectinib in Patients With Anaplastic Lymphoma Kinase (ALK)-Positive or Rearranged During Transfection (RET)-Positive Cancer
The purpose of this study is to provide continued treatment with alectinib or crizotinib as applicable to participants with ALK- or RET positive cancer who were previously enrolled in any Roche-sponsored alectinib study and who…
- Trial name
- NCT03194893
- Testing
- Alectinib, Crizotinib
- Where
- 29 sites in United States, France and 7 more
- Volunteers
- 50 people
- Sponsor
- Hoffmann-La Roche
Colonic Polyps, Polyposis Coli
Non-Invasive Prediction of Colorectal Neoplasia
Hypothesis:The Adnab-9 antigen is a predictive biomarker in individuals at risk of developing colorectal neoplasia. Study aim is to evaluate the potential of the Adnab-9 monoclonal antibody (MAb) as a marker of cancer risk in a…
- Conditions
- Colonic Polyps, Polyposis Coli, Colorectal Cancer
- Trial name
- NIPCON (NCT01815463)
- Where
- United States
- Volunteers
- 2,282 people
- Sponsor
- Corporal Michael J. Crescenz VA Medical Center
Hydration Status
The Impact of Self-assessment on Hydration
The studies objective is to assess the efficacy of hydration education and the use of a self-assessment worksheet vs. a no-intervention control on improving fluid intake and hydration status in underhydrated wildland firefighters…
- Trial name
- NCT06954909
- Testing
- Hydration education and self-assessment
- Where
- United States
- Volunteers
- 38 people, healthy volunteers welcome
- Sponsor
- Arizona State University
Colonic Neoplasms
Establishing Effective Screening Methods for Diagnosing Hereditary Nonpolypoisis Colorectal Cancer
Correct identification of Lynch syndrome at the time of colorectal cancer presentation is important. We aim to find best ways to screen patients with colorectal cancer in Korea.
- Trial name
- NCT00516230
- Where
- South Korea
- Volunteers
- 200 people
- Sponsor
- Samsung Medical Center
Metastatic Colorectal Carcinoma (mCRC), Colorectal Cancer (CRC)
Safety and Efficacy of Tegavivint in Patients With Metastatic Colorectal Carcinoma
This trial will evaluate the safety, tolerability, and preliminary efficacy of tegavivint as monotherapy (single) and in combination with standard therapies in patients with metastatic colorectal carcinoma (mCRC).
- Conditions
- Metastatic Colorectal Carcinoma (mCRC), Colorectal Cancer (CRC), Adenomatous Polyposis Coli (APC) Gene Mutation, Catenin Beta-1 (CTNNB1) Gene Mutation
- Trial name
- NCT07463599
- Testing
- Tegavivint
- Where
- United States
- Volunteers
- 126 people
- Sponsor
- HonorHealth Research Institute
Neurofibromatosis Type 1
Analysis of Peripheral Nerve Sheath Tumors (PNSTs) in Neurofibromatosis Type 1 (NF1) Patients
Neurofibromatosis type 1 (NF1) is a frequent, autosomal dominant disorder caused by heterozygous mutations (intragenic or microdeletion) of the NF1 tumor suppressor gene (chr.17q11.2).
- Trial name
- NCT01218139
- Where
- Belgium
- Volunteers
- 50 people
- Sponsor
- KU Leuven
Prader-Willi Syndrome, PWS-like Syndrome
GROWing Up With Rare GENEtic Syndromes
Introduction Rare complex syndromes Patients with complex genetic syndromes, by definition, have combined medical problems affecting multiple organ systems, and intellectual disability is often part of the syndrome.
- Conditions
- Prader-Willi Syndrome, PWS-like Syndrome, Silver Russel Syndrome, Congenital Hypopituitarism
- Trial name
- GROW UR GENES (NCT04463316)
- Testing
- Retrospective file studies
- Where
- Netherlands
- Volunteers
- 600 people
- Sponsor
- dr. Laura C. G. de Graaff-Herder
Breast Cancer, Chronic Myeloproliferative Disorders
Aprepitant, Granisetron, & Dexamethasone in Preventing Nausea & Vomiting in Pts. Receiving Cyclophosphamide Before a Stem Cell Transplant
RATIONALE: Antiemetic drugs, such as aprepitant, granisetron, and dexamethasone, may help lessen or prevent nausea and vomiting in patients treated with chemotherapy.
- Conditions
- Breast Cancer, Chronic Myeloproliferative Disorders, Gestational Trophoblastic Tumor, Leukemia
- Trial name
- NCT00293384
- Testing
- Aprepitant, Cyclophosphamide, Dexamethasone
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Barbara Ann Karmanos Cancer Institute
Wilms Tumour
Collaborative Wilms Tumour Africa Project
Significant progress has been made in the treatment of Wilms tumor in high income countries, where survival is now around 85% - 90%.
- Trial name
- NCT01991652
- Where
- 3 sites in Cameroon, Ghana and 1 more
- Volunteers
- 400 people
- Sponsor
- Amsterdam UMC, location VUmc
Hereditary Breast and Ovarian Cancer Syndrome, Lynch Syndrome
Pediatric Reporting of Adult-Onset Genomic Results
The Investigators will conduct a longitudinal, mixed-methods cohort study to assess primary and secondary psychosocial outcomes among MyCode adolescent participants and their parents, and health behaviors of children who received…
- Conditions
- Hereditary Breast and Ovarian Cancer Syndrome, Lynch Syndrome, Familial Hypercholesterolemia
- Trial name
- NCT03832985
- Testing
- Child(ren) receive an adult-onset result, Child(ren) received a pediatric-onset result, Control - Negative Result
- Where
- United States
- Volunteers
- 162 people, healthy volunteers welcome
- Sponsor
- Geisinger Clinic
Kidney Cancer
Studying Genes Using Cord Blood and Placenta Samples From Relatively Healthy Newborns and Samples From Younger Patients With Wilms Tumor
RATIONALE: Studying samples of blood and tissue from newborns and 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
- NCT01576211
- Testing
- DNA methylation analysis, RNA analysis, allele-specific oligonucleotide real-time quantitative polymerase chain reaction
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Children's Oncology Group
Carcinoid, Neuroendocrine
Safety Study of Seneca Valley Virus in Patients With Solid Tumors With Neuroendocrine Features
The primary purpose of the study is to determine if Seneca Valley Virus may be administered safely to patients with certain types of advanced cancer.
- Trial name
- NCT00314925
- Testing
- Seneca Valley Virus (biological agent)
- Where
- 10 sites in United States
- Volunteers
- 60 people
- Sponsor
- Neotropix
Plexiform Neurofibroma Associated With Neurofibromatosis Type 1
Efficacy and Safety of RAD001 in Treating Plexiform Neurofibromas (PN) Associated With Neurofibromatosis (NF1)
This study was to evaluate the antitumor activity and safety of RAD001 in patients with Plexiform neurofibromas (PN) associated with Neurofibromatosis Type 1 (NF1). The aim of the study was to : 1.
- Trial name
- NCT01365468
- Testing
- Everolimus (RAD001)
- Where
- 2 sites in Israel
- Volunteers
- 9 people
- Sponsor
- Novartis Pharmaceuticals
Familial Adenomatous Polyposis
Effect of EPA-FFA on Polypectomy in Familial Adenomatous Polyposis
2 Year randomised, double-blind, placebo-controlled, parallel group study to determine the safety and efficacy of EPA-FFA gastro resistant capsules in FAP.
- Trial name
- NCT03806426
- Testing
- Eicosapentaenoic acid free fatty acid (EPA-FFA)
- Where
- Italy
- Volunteers
- 204 people
- Sponsor
- S.L.A. Pharma AG
Chronic Myeloproliferative Disorders, Diamond-blackfan Anemia
Donor Umbilical Cord Blood Transplant in Treating Patients With Hematologic Cancer
RATIONALE: Giving chemotherapy and total-body irradiation before a donor umbilical cord blood transplant helps stop the growth of cancer and abnormal cells and helps stop the patient's immune system from rejecting the donor's…
- Conditions
- Chronic Myeloproliferative Disorders, Diamond-blackfan Anemia, Fanconi Anemia, Graft Versus Host Disease
- Trial name
- NCT00290628
- Testing
- anti-thymocyte globulin, busulfan, cyclophosphamide
- Where
- United States
- Volunteers
- 43 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
MutYH-associated Polyposis
MUTYH-associated Polyposis
This is single-center, prospective, non-randomized study
- Trial name
- MAP (NCT03847532)
- Testing
- Sanger sequencing method
- Where
- Russia
- Volunteers
- 25 people, healthy volunteers welcome
- Sponsor
- State Scientific Centre of Coloproctology, Russian Federation
Glioma, Neurofibromatosis Type 1
MicroRNAs in Patients With Neurofibromatosis Type 1
MicroRNAs are small molecules which have recently been discovered in cells. They are known to be responsible for the normal development of cells and when they are disrupted can contribute to the development of cancer.
- Trial name
- NCT01595139
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
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