Clinical trials: Genetic Cancer Syndromes
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
Neurofibromatosis 1, Neurofibroma, Plexiform
Medication Adherence in Children, Adolescents and Adults With Neurofibromatosis Type 1(NF1) on Clinical Treatment Trials
Background: Neurofibromatosis type 1 (NF1) is a genetic disorder. It has a broad variety of effects on the body. Up to half of people with NF1 get plexiform neurofibromas (PNs). These are benign tumors.
- Trial name
- NCT03531814
- Testing
- Medication Event Monitoring System (MEMS^TM), Questionnaires
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- National Cancer Institute (NCI)
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
Epilepsy, Tuberous Sclerosis Complex
Everolimus (RAD001) Therapy for Epilepsy in Patients With Tuberous Sclerosis Complex (TSC)
The goal of this study is to learn if the study drug RAD001 can reduced the number of epileptic seizures, and can be taken safety by people who have epilepsy associated with Tuberous Sclerosis Complex.
- Trial name
- NCT01070316
- Testing
- Everolimus
- Where
- 2 sites in United States
- Volunteers
- 20 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Neurofibromatosis Type 1, Plexiform Neurofibroma
AZD2171 in Treating Patients With Neurofibromatosis Type 1 and Plexiform Neurofibroma and/or Neurofibroma Near the Spine
This phase II trial is studying how well AZD2171 works in treating patients with neurofibromatosis type 1 and plexiform neurofibroma and/or neurofibroma near the spine.
- Conditions
- Neurofibromatosis Type 1, Plexiform Neurofibroma, Spinal Cord Neurofibroma
- Trial name
- NCT00326872
- Testing
- Cediranib Maleate
- Where
- 9 sites in United States
- Volunteers
- 26 people
- Sponsor
- National Cancer Institute (NCI)
Pregnancy, Infertility
Microsurgical Varicocelectomy Versus No Surgery in Men With a Palpable Varicocele and an Abnormal Semen Analysis
The primary research hypothesis is that microsurgical varicocelectomy will result in an increase in live birth in infertile couples where the male partner has a palpable varicocele and an abnormal semen analysis in comparison to…
- Trial name
- VIA (NCT00767338)
- Testing
- Timed intercourse, Microsurgical varicocelectomy, Intrauterine insemination
- Where
- 9 sites in United States
- Volunteers
- 3 people
- Sponsor
- Yale University
Medullary Thyroid Carcinoma, Multiple Endocrine Neoplasia Type 2A
Vandetanib to Treat Children and Adolescents With Medullary Thyroid Cancer
Background: Medullary thyroid carcinoma (MTC) is common in people with a genetic disorder called multiple endocrine neoplasia (MEN).
- Conditions
- Medullary Thyroid Carcinoma, Multiple Endocrine Neoplasia Type 2A, Multiple Endocrine Neoplasia Type 2B
- Trial name
- NCT00514046
- Testing
- Vandetanib
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- National Cancer Institute (NCI)
Tuberous Sclerosis Complex, Epilepsy
Stopping TSC Onset and Progression 2: Epilepsy Prevention in TSC Infants
This phase I/II clinical trial is an open-label clinical trial design to verify safety and dosing for TAVT-18 (sirolimus) powder for oral solution in TSC infants (N=5).
- Trial name
- STOP2 (NCT04595513)
- Testing
- TAVT-18 (sirolimus)
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Human Immunodeficiency Virus Infection, Sexually Transmitted Diseases
Men Together Making a Difference: Reducing HIV/STD Risk Behavior Among South African Men
Sub-Saharan Africa has about 10% of the world's population, but was home to more than 60% of all people living with HIV in 2003.
- Trial name
- NCT01490359
- Testing
- Men Making a Difference HIV/STD Risk Reduction Intervention, Health Promotion Control
- Volunteers
- 1,181 people, healthy volunteers welcome
- Sponsor
- University of Pennsylvania
Type 2 Diabetes, Men
PowerUp for Health: A Diabetes Prevention Program for Men
The purpose of this two-year proposal is to: 1. develop and 2. complete a pilot test of an adaptation of the evidence-based National Diabetes Prevention Program (NDPP) tailored to engage men with prediabetes in disadvantaged…
- Trial name
- NCT02555631
- Testing
- lifestyle
- Where
- United States
- Volunteers
- 29 people, healthy volunteers welcome
- Sponsor
- Albert Einstein College of Medicine
Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Childhood Non-Hodgkin Lymphoma
Vemurafenib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With BRAF V600 Mutations (A Pediatric MATCH Treatment Trial)
This phase II Pediatric MATCH trial studies how well vemurafenib works in treating patients with solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with BRAF V600 mutations that have spread to other places in the body…
- Conditions
- Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Childhood Non-Hodgkin Lymphoma, Ann Arbor Stage IV Childhood Non-Hodgkin Lymphoma, Ependymoma
- Trial name
- NCT03220035
- Testing
- Laboratory Biomarker Analysis, Vemurafenib
- Where
- 116 sites in United States, Puerto Rico
- Volunteers
- 4 people
- Sponsor
- National Cancer Institute (NCI)
Childhood Acute Lymphoblastic Leukemia in Remission, Childhood Acute Myeloid Leukemia in Remission
Supersaturated Calcium Phosphate Rinse in Preventing Oral Mucositis in Young Patients Undergoing Autologous or Donor Stem Cell Transplant
This randomized phase III trial is studying how well Caphosol rinse works in preventing mucositis in young patients undergoing autologous or donor stem cell transplant.
- Conditions
- Childhood Acute Lymphoblastic Leukemia in Remission, Childhood Acute Myeloid Leukemia in Remission, Childhood Chronic Myelogenous Leukemia, Childhood Myelodysplastic Syndromes
- Trial name
- NCT01305200
- Testing
- supersaturated calcium phosphate rinse, questionnaire administration, quality-of-life assessment
- Where
- 35 sites in United States, Australia and 1 more
- Volunteers
- 226 people
- Sponsor
- Children's Oncology Group
Tuberous Sclerosis Complex (TSC), Lymphangioleiomyomatosis (LAM)
Efficacy and Safety of RAD001 in Patients Aged 18 and Over With Angiomyolipoma Associated With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM)
This study will evaluate the safety and efficacy of RAD001 in treating patients with Angiomyolipoma associated with Tuberous Sclerosis Complex or Sporadic Lymphangioleiomyomatosis.
- Trial name
- EXIST-2 (NCT00790400)
- Testing
- Everolimus (RAD001)
- Where
- 25 sites in United States, Canada and 9 more
- Volunteers
- 118 people
- Sponsor
- Novartis Pharmaceuticals
Pancreatic Cancer
Secretin (ChiRhoStim) Pancreas Perfusion for Pancreatic Adenocarcinoma
The purpose of this study is to test if secretin-enhanced CT is a useful noninvasive screening tool for pancreatic cancer in a high-risk population.
- Trial name
- NCT00587132
- Testing
- Synthetic Human Secretin
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Mayo Clinic
Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia in Remission
Haploidentical Hematopoietic Cell Transplantation Using TCR Alpha/Beta and CD19 Depletion
Patients with medical conditions requiring allogeneic hematopoietic cell transplantation (allo-HCT) are at risk of developing a condition called graft versus host disease (GvHD) which carries a high morbidity and mortality.
- Conditions
- Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia in Remission, Myelodysplastic Syndromes, Chronic Myeloid Leukemia
- Trial name
- HAPLOTAB (NCT05236764)
- Testing
- CliniMACS
- Where
- 2 sites in United States
- Volunteers
- 3 people
- Sponsor
- Baylor College of Medicine
Advanced Malignant Solid Neoplasm, Malignant Solid Neoplasm
Ensartinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With ALK or ROS1 Genomic Alterations (A Pediatric MATCH Treatment Trial)
This phase II Pediatric MATCH treatment trial studies how well ensartinib works in treating patients with solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with ALK or ROS1 genomic alterations that have come back…
- Conditions
- Advanced Malignant Solid Neoplasm, Malignant Solid Neoplasm, Recurrent Ependymoma, Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor
- Trial name
- NCT03213652
- Testing
- Biospecimen Collection, Bone Marrow Aspiration and Biopsy, Bone Scan
- Where
- 126 sites in United States, Australia and 2 more
- Volunteers
- 13 people
- Sponsor
- National Cancer Institute (NCI)
Basal Cell Nevus Syndrome
Blue vs Red Light During Levulan Based Photodynamic Therapy in Patients With Basal Cell Nevus Syndrome
The investigators will be testing whether aminolevulinate-based (Levulan™) Photodynamic Therapy (PDT) shows effectiveness in the treatment and prevention of cutaneous basal cell carcinoma (BCC) in Basal Cell Nevus Syndrome (BCNS)…
- Trial name
- NCT02157623
- Testing
- Levulan, Red Light PDT, Blue Light PDT
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Edward Maytin, MD, PhD
Basal Cell Nevus Syndrome
Study of Patidegib Topical Gel, 2%, for the Reduction of Disease Burden of Persistently Developing Basal Cell Carcinomas (BCCs) in Subjects With Basal Cell Nevus Syndrome (Gorlin Syndrome)
This is a global, multicenter, randomized, double-blind, stratified, vehicle-controlled study of the efficacy and safety of Patidegib Topical Gel, 2%, applied topically twice daily to the face of adult participants with Gorlin…
- Trial name
- NCT03703310
- Testing
- Patidegib Topical Gel, 2%, Patidegib Topical Gel, Vehicle
- Where
- 49 sites in United States, Belgium and 8 more
- Volunteers
- 174 people
- Sponsor
- Sol-Gel Technologies, Ltd.
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
Lymphangioleiomyomatosis, Tuberous Sclerosis Complex
Feasibility of [11C]Acetate-PET in LAM and TSC
This study aims to assess [11C]acetate positron emission tomography (PET)/computed tomography (CT) as a biomarker for renal angiomyolipomas and pulmonary lymphangioleiomyomatosis (LAM) and an early biomarker of response to…
- Trial name
- NCT05467397
- Testing
- [11C]acetate
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Brigham and Women's Hospital