Clinical trials: Neoplastic Syndromes, Hereditary
Plexiform Neurofibromas (PN)
Low-dose Selumetinib for the Treatment of Plexiform Neurofibromas in Chinese Children
The goal of this study is to compare the efficacy and safety of low-dose and internationally recommended standard dose of selumetinib in the treatment of plexiform neurofibromas in Chinese children.
- Trial name
- LS-NF1PNs (NCT06763315)
- Testing
- Selumetinib
- Where
- China
- Volunteers
- 50 people
- Sponsor
- West China Hospital
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
Graft Versus Host Disease, Leukemia
Treatment of Single or Double Umbilical Cord Trans + Graft-versus-host Disease (GVHD) Prophylaxis w/ Tacrolimus & Mycophenolate Mofetil
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
- Graft Versus Host Disease, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00608517
- Testing
- anti-thymocyte globulin, cyclophosphamide, fludarabine phosphate
- Where
- 4 sites in United States
- Volunteers
- 6 people
- Sponsor
- Vanderbilt-Ingram Cancer Center
Breast Cancer
Study to Evaluate the Effect of Metformin in the Prevention of HG in HR[+]/HER2[-] PIK3CA-mut Advanced BC Patients
Men and post- or induced menopausal women with ER[+] and/or PgR[+], HER2[- ] advanced BC, with centrally-confirmed PI3KCAMut who progressed to an aromatase inhibitor (AI) regimen.
- Trial name
- METALLICA (NCT04300790)
- Testing
- Alpelisib, Metformin, Fulvestrant
- Where
- 19 sites in Spain
- Volunteers
- 69 people
- Sponsor
- MedSIR
Familial Adenomatous Polyposis
Molecular Targeting of 15-Lipoxygenase-1 (15-LOX-1) for Apoptosis Induction in Human Colorectal Cancers
Primary Objective: To determine whether celecoxib downregulates GATA-6 expression to upregulate 15-LOX-1 expression and induce apoptosis in human rectal tumors, researchers will measure GATA-6 and 15-LOX-1 expression, 13-S-HODE…
- Trial name
- NCT00503035
- Testing
- Celecoxib, Colonoscopy Biopsy
- Where
- United States
- Volunteers
- 51 people
- Sponsor
- M.D. Anderson Cancer Center
Anaplastic Kidney Wilms Tumor, Recurrent Kidney Wilms Tumor
A Study of Combination Chemotherapy for Patients With Newly Diagnosed DAWT and Relapsed FHWT
This phase II trial studies how well combination chemotherapy works in treating patients with newly diagnosed stage II-IV diffuse anaplastic Wilms tumors (DAWT) or favorable histology Wilms tumors (FHWT) that have come back…
- Conditions
- Anaplastic Kidney Wilms Tumor, Recurrent Kidney Wilms Tumor, Stage II Kidney Wilms Tumor, Stage III Kidney Wilms Tumor
- Trial name
- NCT04322318
- Testing
- Biopsy Procedure, Biospecimen Collection, Bone Scan
- Where
- 205 sites in United States, Australia and 4 more
- Volunteers
- 256 people
- Sponsor
- Children's Oncology Group
Inherited Epidermolysis Bullosa, Ichthyosis
FARD (RaDiCo Cohort) (RaDiCo-FARD)
The goal of this observational study is to conduct a prospective assessment of the individual Burden of 9 rare skin diseases to assess disability in the broadest sense of the term (psychological, social, economic and physical)…
- Conditions
- Inherited Epidermolysis Bullosa, Ichthyosis, Ectodermal Dysplasia, Incontinentia Pigmenti
- Trial name
- FARD (NCT05954416)
- Where
- 15 sites in France
- Volunteers
- 900 people
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
Lynch Syndrome, Small Bowel Neoplasia
Capsule Endoscopy to Screen for Small Bowel Neoplasia in Lynch Syndrome
Lynch syndrome (LS), or hereditary nonpolyposis colorectal cancer (HNPCC), is a hereditary disorder characterized by a very high risk of early-onset colorectal and endometrial cancer and an increased risk of other cancers…
- Trial name
- CELSIUS (NCT00898768)
- Testing
- Capsule endoscopy
- Where
- Netherlands
- Volunteers
- 200 people
- Sponsor
- University Medical Center Groningen
Neurofibromatosis Type 1, Tibial Dysplasia
Neurofibromatosis Type 1 (NF1) and Tibial Dysplasia
The study is a multicenter four-year outcome study of the natural history of tibial dysplasia in patients with NF1 and selected patients without NF1.
- Trial name
- NF1TD (NCT00303368)
- Where
- United States
- Volunteers
- 395 people
- Sponsor
- Shriners Hospitals for Children
Pancreas Cancer, Peutz-Jeghers Syndrome (PJS)
The Cancer of the Pancreas Screening-5 CAPS5)Study
Johns Hopkins clinical research office quality assurance group will monitor and audit this study at Johns Hopkins. The Sub Investigator at each site will be responsible for internal monitoring at their site.
- Conditions
- Pancreas Cancer, Peutz-Jeghers Syndrome (PJS), Gene Mutation, Germline Mutation Carrier
- Trial name
- CAPS5 (NCT02000089)
- Testing
- Secretin, MRI, Tumor marker gene test with CA19-9
- Where
- 9 sites in United States
- Volunteers
- 9,000 people
- Sponsor
- Johns Hopkins University
Hereditary Breast and Ovarian Cancer Syndrome, BRCA Mutations
Development and Validation of an Ovarian Cancer Risk Prediction Model for Family Members of Ovarian Cancer Probands
Ovarian cancer is the gynecological malignancy with the highest fatality rate, seriously threatening the life and health of women.
- Trial name
- NCT07039552
- Testing
- risk-reducing salpingo-oophorectomy (RRSO)
- Where
- China
- Volunteers
- 10,000 people
- Sponsor
- Peking University Third Hospital
Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor
Amifostine to Protect From Side Effects of PSCT in Treating Patients With Solid Tumors
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining chemotherapy with peripheral stem cell transplantation may allow the doctor to give higher doses of…
- Conditions
- Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor, Chordoma, Kidney Cancer
- Trial name
- NCT00003926
- Testing
- amifostine trihydrate, busulfan, filgrastim
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Rhabdomyosarcoma, Ewing Sarcoma
Selective Antigen Specific T Cells and CAR T Cells in Subjects With Relapsed/Refractory Embryonal Tumors (SABRE)
This is a phase I dose-escalation study to determine the safety and feasibility of autologous CAR-TA T cells (B7-H3 CAR+ T cells administered with DNR-PRAME Tumor Antigen-specific T cells) following lymphodepleting chemotherapy…
- Conditions
- Rhabdomyosarcoma, Ewing Sarcoma, Neuroblastoma, Wilms Tumor
- Trial name
- SABRE (NCT07172958)
- Testing
- Selective Antigen Specific dTβRII-expressing T cells combined with B7-H3 CAR T cells
- Where
- 2 sites in United States
- Volunteers
- 18 people
- Sponsor
- Children's National Research Institute
Tuberous Sclerosis Complex, TSC Related Cognitive Disability
Efficacy of RAD001/Everolimus in Autism and NeuroPsychological Deficits in Children With Tuberous Sclerosis Complex
Tuberous sclerosis complex (TSC) is a genetic disease that leads to mental retardation in over 50% of patients, and to learning problems, behavioral problems, autism and epilepsy in up to 90% of patients.
- Conditions
- Tuberous Sclerosis Complex, TSC Related Cognitive Disability, TSC Related Autism, TSC Related Learning Problems
- Trial name
- RAPIT (NCT01730209)
- Testing
- Everolimus
- Where
- Netherlands
- Volunteers
- 60 people
- Sponsor
- Erasmus Medical Center
Accelerated Phase Chronic Myelogenous Leukemia, Acute Undifferentiated Leukemia
Fludarabine and Total-Body Irradiation Followed By Donor Stem Cell Transplant and Cyclosporine and Mycophenolate Mofetil in Treating HIV-Positive Patients With or Without Cancer
This clinical trial studies the side effects and best dose of giving fludarabine and total-body irradiation (TBI) together followed by a donor stem cell transplant and cyclosporine and mycophenolate mofetil in treating human…
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Acute Undifferentiated Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission
- Trial name
- NCT00112593
- Testing
- fludarabine phosphate, total-body irradiation, peripheral blood stem cell transplantation
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Fred Hutchinson Cancer Center
Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, Including, Sickle Cell Disease
Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders
Cell-free fetal DNA (cffDNA) is present in the maternal blood from the early first trimester of gestation and makes up 5%-20% of the total circulating cell-free DNA (cfDNA) in maternal plasma.
- Conditions
- Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, Including, Sickle Cell Disease, Cystic Fibrosis, Fragile X Syndrome
- Trial name
- DANNIgene (NCT06147414)
- Testing
- Blood sample
- Where
- France
- Volunteers
- 550 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Other Acute Pain
An Efficacy and Safety Trial of Intranasal Ketorolac in Emergency Department Patients for the Treatment of Acute Pain
The purpose of this study is to determine the effectiveness, the safety, and the tolerability of intranasal ketorolac (Sprix) in relieving acute pain in adults ages 18-65 who come to the ED seeking care.
- Trial name
- Sprix (NCT01471639)
- Testing
- intranasal ketorolac
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- The Cleveland Clinic
Recurrent Wilms Tumor and Other Childhood Kidney Tumors, Stage I Wilms Tumor
Gene Expression Profiles to Categorize Wilms Tumors
This laboratory study is using gene expression profiling to identify different categories of Wilms tumors. Studying the genes expressed in samples of tumor tissue from patients with cancer may help doctors identify biomarkers…
- Conditions
- Recurrent Wilms Tumor and Other Childhood Kidney Tumors, Stage I Wilms Tumor, Stage II Wilms Tumor, Stage III Wilms Tumor
- Trial name
- NCT00897637
- Testing
- laboratory biomarker analysis
- Where
- 2 sites in United States
- Volunteers
- 600 people
- Sponsor
- Children's Oncology Group
Endometrial Cancer, Lynch Syndrome
Assessing Uterine Cancer Risk in Lynch Syndrome Carriers Using Vaginal Self-sampling and a Health Questionnaire
The goal of this study is to find out if self-collected vaginal swabs can be used to detect early signs of uterine cancer or related conditions in people with Lynch syndrome (LS) who still have their uterus.
- Trial name
- Lynch-SCAN (NCT07194551)
- Testing
- Vaginal DNA swab, microbiome swab and pH test
- Where
- Canada
- Volunteers
- 30 people
- Sponsor
- University of British Columbia
Neurofibromatosis 1 and 2 (NF1 and NF2)
A Single Center Pilot Study to Assess the Intra-observer Reliability of Measuring Muscle Strength Using a Hand Held Dynamometer in Children and Adults With Neurofibromatosis Type 1 (NF1) and Type 2 (NF2)
Patients with clinically confirmed neurofibromatosis type 1 (NF1) or neurofibromatosis type 2 (NF2) or a known neurofibromatosis (NF) mutation aged 5 years and above will be eligible to participate and will be recruited from the…
- Trial name
- NCT03672838
- Testing
- hand-held dynamometer
- Where
- United States
- Volunteers
- 33 people
- Sponsor
- NYU Langone Health
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