Clinical trials: Inherited Cancer Syndrome
Myelofibrosis, Gaucher Disease
Clinical and Pathophysiological Investigations Into Erdheim Chester Disease
Background: Erdheim Chester Disease (ECD) is a very rare disease in which abnormal white blood cells start growing and affect the bones, kidneys, skin, and brain.
- Conditions
- Myelofibrosis, Gaucher Disease, Pulmonary Fibrosis, Hermansky-Pudlak Syndrome (HPS)
- Trial name
- NCT01417520
- Where
- United States
- Volunteers
- 88 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q)
Iodine I 131 Monoclonal Antibody BC8, Fludarabine Phosphate, Total Body Irradiation, and Donor Stem Cell Transplant Followed by Cyclosporine and Mycophenolate Mofetil in Treating Patients With Advanced Acute Myeloid Leukemia or Myelodysplastic Syndrome
This phase II trial studies the side effects and best dose of iodine I 131 monoclonal antibody BC8 when given together with fludarabine phosphate, total-body irradiation, and donor stem cell transplant followed by cyclosporine…
- 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
- NCT00119366
- Testing
- iodine I 131 monoclonal antibody BC8, fludarabine phosphate, total-body irradiation
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Fred Hutchinson Cancer Center
Chronic Myeloproliferative Disorders, Leukemia
Donor Stem Cell Transplant in Treating Older or Frail Patients With Hematologic Cancer
RATIONALE: Giving low doses of chemotherapy, such as fludarabine and busulfan, before a donor bone marrow or peripheral blood stem cell transplant helps stop the growth of cancer cells.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00296023
- Testing
- anti-thymocyte globulin, filgrastim, therapeutic allogeneic lymphocytes
- Where
- 2 sites in United States
- Volunteers
- 25 people
- Sponsor
- University of California, San Francisco
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
Accelerated Phase Chronic Myelogenous Leukemia, Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome
Fludarabine Phosphate, Melphalan, Total-Body Irradiation, Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer or Bone Marrow Failure Disorders
This clinical trial is studying how well giving fludarabine phosphate and melphalan together with total-body irradiation followed by donor stem cell transplant works in treating patients with hematologic cancer or bone marrow…
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission
- Trial name
- NCT00856388
- Testing
- fludarabine phosphate, melphalan, total-body irradiation
- Where
- United States
- Volunteers
- 62 people
- Sponsor
- Roswell Park Cancer Institute
Cancer
High-Dose Combination Chemotherapy Plus Peripheral Stem Cell Transplantation in Treating Patients With Advanced Cancer
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Peripheral stem cell transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill…
- Trial name
- NCT00002854
- Testing
- filgrastim, carboplatin, cisplatin
- Where
- United States
- Volunteers
- 33 people
- Sponsor
- City of Hope Medical Center
Adenomatous Polyposis Coli
Molecular Fluorescence Endoscopy in Patients With Familial Adenomatous Polyposis, Using Bevacizumab-IRDye800CW
There is a need for better visualization of polyps during surveillance endoscopy in patients with hereditary colon cancer syndromes like Familial Adenomatous Polyposis (FAP) and Lynch Syndrome (LS), to improve the adenoma…
- Trial name
- FLUOFAP (NCT02113202)
- Testing
- Bevacizumab-IRDye800CW, Near infrared fluorescence endoscopy platform
- Where
- Netherlands
- Volunteers
- 17 people
- Sponsor
- University Medical Center Groningen
Cutaneous Neurofibroma, Neurofibromatosis Type 1
Selumetinib in Treating Patients With Neurofibromatosis Type 1 and Cutaneous Neurofibroma
This pilot phase II trial studies how well selumetinib works in treating patients with neurofibromatosis type 1 and cutaneous neurofibromas.
- Conditions
- Cutaneous Neurofibroma, Neurofibromatosis Type 1, Optic Nerve Glioma
- Trial name
- NCT02839720
- Testing
- Laboratory Biomarker Analysis, Selumetinib Sulfate
- Where
- 2 sites in United States
- Volunteers
- 11 people
- Sponsor
- National Cancer Institute (NCI)
Lynch Syndrome, Colonoscopy
Linked Color Imaging Versus High-definition White Light Endoscopy for the Detection of Polyps in Patients With Lynch Syndrome (LCI-LYNCH)
The aim of the present study is to compare polyp detection rates of LCI with high-definition white light endoscopy (HD-WLE) in patients with Lynch syndrome in a parallel, international, multicenter, randomized controlled…
- Trial name
- NCT03344289
- Testing
- Colonoscopy
- Where
- Netherlands
- Volunteers
- 357 people
- Sponsor
- Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Hereditary Breast and Ovarian Cancer Syndrome, Lynch Syndrome
Telemedicine vs. Face-to-Face Cancer Genetic Counseling
Cancer genetic counseling (CGC) has been found to have "substantial" benefits for individuals with breast cancer and their family members; it has been deemed by multiple organizations as "standard of care" for women with breast…
- Trial name
- NCT00609505
- Testing
- Telemedicine, Face-to-Face
- Where
- 4 sites in United States
- Volunteers
- 130 people, healthy volunteers welcome
- Sponsor
- Duke University
Neurofibromatosis 2, Meningioma
AZD2014 In NF2 Patients With Progressive or Symptomatic Meningiomas
The goal of this clinical research study is to learn if the study drug AZD2014 can shrink growing or symptomatic meningiomas.
- Trial name
- NCT02831257
- Testing
- AZD2014
- Where
- 2 sites in United States
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- Massachusetts General Hospital
Rectal Cancer, Ulcerative Colitis
Bioabsorbable Staple Line Reinforcement in Colorectal,Coloanal and Ileoanal Anastomoses
The primary purpose of this prospective, randomized multicenter center study is to evaluate and compare the outcomes of colorectal, coloanal and ileoanal anastomoses reinforced with a bioabsorbable staple line reinforcement…
- Conditions
- Rectal Cancer, Ulcerative Colitis, Familial Adenomatous Polyposis, Diverticulitis
- Trial name
- NCT00663819
- Testing
- GORE SEAMGUARD® Bioabsorbable Staple Line Reinforcement, Staple line without reinforcement
- Where
- 16 sites in United States
- Volunteers
- 258 people
- Sponsor
- W.L.Gore & Associates
Cancer
Donor Natural Killer Cell Infusion in Preventing Relapse or Graft Failure in Patients Who Have Undergone Donor Bone Marrow Transplant
RATIONALE: Giving donor natural killer cells to patients who have undergone donor bone marrow transplant may make the transplant work better and keep cancer cells from coming back.
- Trial name
- NCT00569283
- Testing
- therapeutic allogeneic lymphocytes
- Where
- 2 sites in South Korea
- Volunteers
- 18 people
- Sponsor
- Asan Medical Center
Testosterone, Androgens
Comparison Between Calculated Bioavailable Testosterone Level With Radio-immunoassay Result.
Currently, the gold standard to obtain bioavailable testosterone results is radioimmunoassay (RIA). However, this assay requires specific equipment.
- Trial name
- TESTOBIO (NCT04807231)
- Testing
- Blood withdrawal
- Where
- France
- Volunteers
- 270 people
- Sponsor
- Centre Hospitalier Universitaire, Amiens
Vasomotor Symptoms as a Sex Hormone-dependent Disorder in Women and Men
A Study to Learn What Happens to Elinzanetant in the Body When Taken With or Without Carbamazepine, and How Safe it is in Healthy Men and Women Aged 18 to 60 Years.
Researchers are looking for a better way to treat people who have symptoms caused by hormonal changes, such as hot flashes. Before a treatment can be approved for patients to take, researchers do clinical studies to better…
- Trial name
- NCT05028608
- Testing
- Elinzanetant (BAY3427080), Midazolam, Carbamazepine
- Where
- Germany
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Bayer
Breast Cancer, Chronic Myeloproliferative Disorders
T Cells in Predicting Acute Graft-Versus-Host Disease in Patients Undergoing Donor Stem Cell Transplant
RATIONALE: Studying samples of blood from patients with cancer in the laboratory may help doctors predict whether patients undergoing donor stem cell transplant will develop acute graft-versus-host disease.
- Conditions
- Breast Cancer, Chronic Myeloproliferative Disorders, Gestational Trophoblastic Tumor, Leukemia
- Trial name
- NCT00651716
- Testing
- flow cytometry, laboratory biomarker analysis, Data Collection
- Where
- United States
- Volunteers
- 150 people
- Sponsor
- Vanderbilt-Ingram Cancer Center
Spinal Cord Injury, Spinal Cord Injury No Sensory/Motor in S4-S5
The Aim of This Research is to Better Understand How Complete Spinal Cord Injury May Change Erogenous Zones in Adult Males
A complete spinal cord injury (SCI) in men is characterized by the total loss of motor skills and sensations below the injury. The loss of penile sensation, the erogenous zone primarily involved in sexual response, is involved in…
- Trial name
- QualiEroSCI (NCT06908850)
- Where
- France
- Volunteers
- 15 people
- Sponsor
- Fondation Garches
Congenital Myasthenic Syndrome, Lambert-Eaton Myasthenic Syndrome
Expanded Access Study Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS),Congenital Myasthenic Syndrome
Primary: The primary objective of this study under the original protocol was to provide neuromuscular specialists and neurologists access to amifampridine phosphate therapy for their patients with LEMS, CMS or downbeat nystagmus…
- Conditions
- Congenital Myasthenic Syndrome, Lambert-Eaton Myasthenic Syndrome, Nystagmus, Acquired
- Trial name
- EAP-001 (NCT02189720)
- Testing
- Amifampridine Phosphate
- Where
- United States
- Sponsor
- Catalyst Pharmaceuticals, Inc.
Diamond Blackfan Anemia, Dyskeratosis Congenita
Cancer in Inherited Bone Marrow Failure Syndromes
Background: A prospective cohort of Inherited Bone Marrow Failure Syndrome (IBMFS) will provide new information regarding cancer rates and types in these disorders.
- Conditions
- Diamond Blackfan Anemia, Dyskeratosis Congenita, Fanconi Anemia, Shwachman Diamond Syndrome
- Trial name
- NCT00027274
- Where
- 2 sites in United States
- Volunteers
- 4,000 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
Neuropathy, Diabetic, Diabetes Mellitus, Type 2
DECODING Study (Dermal Electrochemical Conductance in Diabetic Neuropathy)
Diabetes mellitus is the leading cause of polyneuropathy in the Western world. Diabetic neuropathy is a frequent complication of diabetes and may have great clinical transcendence due to pain and possible ulceration of the lower…
- Trial name
- DECODING (NCT03495089)
- Testing
- dermal electrochemical conductance (DEC), Monofilament testing
- Where
- 3 sites in Spain
- Volunteers
- 147 people
- Sponsor
- Fundacio d'Investigacio en Atencio Primaria Jordi Gol i Gurina
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