Clinical trials: Inherited Cancer Syndrome
Neurofibromatosis Type 1
Pharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥1 to <7 Years With NF1-related Symptomatic, Inoperable PN
This study is designed to define a dosing regimen and assess the pharmacokinetics(PK) and safety of the granule formulation; the study will also include descriptive analyses of exploratory efficacy endpoints.
- Trial name
- SPRINKLE (NCT05309668)
- Testing
- Selumetinib granule formulation, Selumetinib capsule formulation
- Where
- 16 sites in United States, Germany and 5 more
- Volunteers
- 36 people
- Sponsor
- AstraZeneca
Chronic Myeloproliferative Disorders, Leukemia
Donor Stem Cell Transplant or Donor White Blood Cell Infusions in Treating Patients With Hematologic Cancer
RATIONALE: A peripheral stem cell transplant or an umbilical cord blood transplant from a donor may be able to replace blood-forming cells that were destroyed by chemotherapy or radiation therapy.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00281879
- Testing
- anti-thymocyte globulin, filgrastim, busulfan
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- OHSU Knight Cancer Institute
Chronic Myeloproliferative Disorders, Fatigue
American Ginseng in Treating Patients With Fatigue Caused by Cancer
RATIONALE: American ginseng may reduce fatigue in patients with cancer. It is not yet known whether American ginseng is more effective than a placebo in treating cancer-related fatigue.
- Conditions
- Chronic Myeloproliferative Disorders, Fatigue, Leukemia, Lymphoma
- Trial name
- NCT00719563
- Testing
- American ginseng
- Where
- 326 sites in United States
- Volunteers
- 364 people
- Sponsor
- Alliance for Clinical Trials in Oncology
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
Tuberous Sclerosis Complex
Open-label Study of Adjunctive GNX Treatment in Children and Adults With TSC-related Epilepsy
This is a Phase 3, global, open-label extension (OLE) study of adjunctive GNX treatment in children and adults with TSC who previously participated in either Study 1042-TSC-3001 or Study 1042-TSC-2001
- Trial name
- NCT05604170
- Testing
- Ganaxolone
- Where
- 54 sites in United States, Australia and 8 more
- Volunteers
- 117 people
- Sponsor
- Marinus Pharmaceuticals
Leukemia
Prophylactic White Cell Transfusions Versus Therapeutic White Cell Transfusions in Patients With Leukemia
Patients with leukemia often have low white blood cell counts after chemotherapy, which puts them at greater risk for infection.
- Trial name
- NCT01204788
- Testing
- Prophylactic White Cell Transfusion, Therapeutic White Cell Transfusion
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- M.D. Anderson Cancer Center
Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders
Opioid Titration Order Sheet or Standard Care in Treating Patients With Cancer Pain
RATIONALE: An Opioid Titration Order Sheet that allows healthcare providers to adjust the dose and schedule of pain medication may help improve pain treatment for patients with cancer.
- Conditions
- Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders, Leukemia, Lymphoma
- Trial name
- NCT00666211
- Testing
- educational intervention, Titrated pain management, questionnaire administration
- Where
- 11 sites in United States
- Volunteers
- 98 people
- Sponsor
- Vanderbilt-Ingram Cancer Center
Adult Kidney Wilms Tumor, Beckwith-Wiedemann Syndrome
Combination Chemotherapy and Surgery in Treating Young Patients With Wilms Tumor
This phase III trial studies how well combination chemotherapy and surgery work in treating young patients with Wilms tumor. Drugs used in chemotherapy work in different ways to stop the growth of tumor cells, either by killing…
- Conditions
- Adult Kidney Wilms Tumor, Beckwith-Wiedemann Syndrome, Childhood Kidney Wilms Tumor, Diffuse Hyperplastic Perilobar Nephroblastomatosis
- Trial name
- NCT00945009
- Testing
- Dactinomycin, Doxorubicin Hydrochloride, Radiation Therapy
- Where
- 169 sites in United States, Australia and 4 more
- Volunteers
- 249 people
- Sponsor
- Children's Oncology Group
CCM, Cavernoma
CASH (Cavernous Angiomas With Symptomatic Hemorrhage) Trial Readiness
Brain Cavernous Angiomas with Symptomatic Hemorrhage (CASH) are rare, but they exact a heavy burden of neurologic disability from recurrent bleeding, for which there is no proven therapy.
- Conditions
- CCM, Cavernoma, Cerebral Cavernous Malformation, Cerebral Cavernous Malformations 1
- Trial name
- NCT03652181
- Where
- United States
- Volunteers
- 123 people
- Sponsor
- University of Chicago
Cancer
Aprepitant in Preventing Nausea and Vomiting in Patients Who Are Undergoing a Stem Cell Transplant
RATIONALE: Antiemetic drugs, such as aprepitant, ondansetron, and dexamethasone, may help lessen or prevent nausea and vomiting in patients undergoing a stem cell transplant.
- Trial name
- NCT00248547
- Testing
- aprepitant, dexamethasone, ondansetron
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- OHSU Knight Cancer Institute
Basal Cell Nevus Syndrome
Extension Study of Patidegib Topical Gel, 2% in Subjects With Gorlin Syndrome (Basal Cell Nevus Syndrome)
This is a multicenter, open label extension study evaluating the safety of Patidegib Topical Gel, 2%, applied topically twice daily to the face of adult subjects with Gorlin syndrome.
- Trial name
- NCT04308395
- Testing
- Patidegib Topical Gel, 2%
- Where
- 37 sites in United States, Belgium and 7 more
- Volunteers
- 108 people
- Sponsor
- Sol-Gel Technologies, Ltd.
Leukemia, Lymphoma
Donor Stem Cell Transplant in Treating Patients With High-Risk Hematologic Cancer
RATIONALE: Giving low doses of chemotherapy before a donor stem cell transplant helps stop the growth of cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells.
- Conditions
- Leukemia, Lymphoma, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases
- Trial name
- NCT00818961
- Testing
- alemtuzumab, graft-versus-tumor induction therapy, rituximab
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- Northside Hospital, Inc.
Sleep Disorders
Efficacy and Safety of Circadin® in the Treatment of Sleep Disturbances in Children With Neurodevelopment Disabilities
The purpose of this study is to establish the efficacy and safety of Circadin in children with neurodevelopmental disorders and to determine the dose, this randomized, placebo-controlled study is planned to evaluate the efficacy…
- Trial name
- NCT01906866
- Testing
- Circadin 2/5/10 mg
- Where
- 28 sites in United States, Finland and 3 more
- Volunteers
- 125 people
- Sponsor
- Neurim Pharmaceuticals Ltd.
Lymphangioleiomyomatosis, Tuberous Sclerosis Complex
Safety of Simvastatin in LAM and TSC
The purpose of this research study is to see if simvastatin can be taken safely in patients with either LAM or TSC, who are already being treated with everolimus or sirolimus.
- Trial name
- SOS (NCT02061397)
- Testing
- Simvastatin, Sirolimus Oral Product, Everolimus Oral Product
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- University of Pennsylvania
Advanced Breast Cancer
Study of Efficacy and Safety of LEE011 in Men and Postmenopausal Women With Advanced Breast Cancer.
The main aim of this study was to evaluate the efficacy and safety of adding ribociclib to fulvestrant in men and postmenopausal women with hormone receptor positive (HR+), HER2-negative advanced breast cancer.
- Trial name
- MONALEESA-3 (NCT02422615)
- Testing
- Ribociclib, Fulvestrant
- Where
- 174 sites in United States, Australia and 28 more
- Volunteers
- 726 people
- Sponsor
- Novartis Pharmaceuticals
Carcinoma, Basal Cell, Recurrent Skin Cancer
Pilot Study of Sonidegib and Buparlisib in Treating Patients With Advanced or Metastatic Basal Cell Carcinoma
This pilot trial studies how well sonidegib and buparlisib work in treating patients with basal cell carcinoma that has spread to other places in the body.
- Conditions
- Carcinoma, Basal Cell, Recurrent Skin Cancer, Skin Neoplasms, Basal Cell Nevus Syndrome
- Trial name
- NCT02303041
- Testing
- Buparlisib, Sonidegib
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Anne Chang
Basal Cell Nevus Syndrome, Gorlin Syndrome
To Determine The Efficacy and Safety of GDC-0449 in Patients With Basal Cell Nevus Syndrome (BCNS)
The purpose of this study is to reduce the number of new surgically eligible BCCs by 50% appearing during month 3-18 of medication ingestion.
- Trial name
- GDC-0449 (NCT00957229)
- Testing
- GDC-0449
- Where
- 2 sites in United States
- Volunteers
- 41 people
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q)
Sirolimus and Azacitidine in Treating Patients With High Risk Myelodysplastic Syndrome or Acute Myeloid Leukemia That is Recurrent or Not Eligible for Intensive Chemotherapy
This phase II trial studies how well sirolimus and azacitidine works in treating patients with high-risk myelodysplastic syndrome or recurrent acute myeloid leukemia.
- Conditions
- Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12)
- Trial name
- NCT01869114
- Testing
- Sirolimus, Azacitidine
- Where
- 5 sites in United States
- Volunteers
- 57 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University
Chronic Myeloproliferative Disorders, Leukemia
Combination Chemotherapy, Bone Marrow Transplant, and Post Transplant Cyclophosphamide for Hematologic Cancer
RATIONALE: Giving chemotherapy before a donor bone marrow transplant helps stop the growth of cancer and abnormal cells and helps stop the patient's immune system from rejecting the donor's stem cells.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00134017
- Testing
- Busulfan, Cyclophosphamide
- Where
- United States
- Volunteers
- 142 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Lynch Syndrome
Overcoming Barriers to the Uptake of Cascade Screening for Lynch Syndrome: Workbook Feasibility Study
The investigators will evaluate the feasibility of an intervention to improve Lynch syndrome cascade screening uptake. The investigators will conduct a pilot study among 15 patients diagnosed with Lynch Syndrome and 5 genetic…
- Trial name
- NCT04978350
- Testing
- Educational Workbook
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- UNC Lineberger Comprehensive Cancer Center