Clinical trials: Hereditary Cancer
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
Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor
Busulfan in Treating Children and Adolescents With Refractory CNS Cancer
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. PURPOSE: Phase I trial to study the safety of delivering intrathecal busulfan in children and adolescents who…
- Conditions
- Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor, Leukemia, Lymphoma
- Trial name
- NCT00006246
- Testing
- busulfan
- Where
- 8 sites in United States
- Volunteers
- 28 people
- Sponsor
- Pediatric Brain Tumor Consortium
Retinoblastoma
Efficacy Study of Lucentis in the Treatment of Retinoblastoma
This study will evaluate the clinical efficacy of intravitreal injections of Ranibizumab (Lucentis) together with chemotherapy in the treatment of Retinoblastoma as compared to chemotherapy alone.
- Trial name
- NCT01899066
- Testing
- Lucentis, chemotherapy, chemotherapy
- Where
- China
- Volunteers
- 20 people
- Sponsor
- Sun Yat-sen University
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
Inherited Hematological Diseases, Rare Diseases
Longitudinal Studies of Patient With FPDMM
Background: Genes tell the body and its cells how to work. Familial platelet disease (FPD) or FPD with associated malignancies (FPDMM) is caused by a variant in the gene RUNX1.
- Conditions
- Inherited Hematological Diseases, Rare Diseases, FPDMM
- Trial name
- NCT03854318
- Where
- United States
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- National Human Genome Research Institute (NHGRI)
Xeroderma Pigmentosum, Basal Cell Carcinoma
Cancer Risk in Carriers of the Gene for Xeroderma Pigmentosum
This study will determine if family members of patients with xeroderma pigmentosum (XP) have various abnormalities, including: skin abnormalities; nervous system abnormalities, such as hearing problems; skin, eye, or internal…
- Conditions
- Xeroderma Pigmentosum, Basal Cell Carcinoma, Squamous Cell Carcinoma, Melanoma
- Trial name
- NCT00046189
- Where
- United States
- Volunteers
- 301 people
- Sponsor
- National Cancer Institute (NCI)
Chronic Myelogenous Leukemia, Short Stature
Assessment Of Gh-Igf-1 Axis In Children With Chronic Myelogenous Leukemia (CML) In Remission
CML is a myeloproliferative disorder defined by the presence of the Philadelphia chromosome, which arises from the reciprocal translocation of genes on chromosomes 9 and 22.It is rare in childhood and accounts for 2-3% of all…
- Trial name
- NCT01901666
- Testing
- Growth Hormone
- Where
- India
- Volunteers
- 20 people
- Sponsor
- Post Graduate Institute of Medical Education and Research, Chandigarh
Leukemia
Combination Chemotherapy, Interleukin-2, and Peripheral Stem Cell Transplant in Treating Patients With Acute Myeloid Leukemia
RATIONALE: Giving combination chemotherapy before a peripheral blood stem cell transplant stops the growth of cancer cells by stopping them from dividing or killing them.
- Trial name
- NCT00004128
- Testing
- aldesleukin, filgrastim, busulfan
- Volunteers
- 2,000 people
- Sponsor
- European Organisation for Research and Treatment of Cancer - EORTC
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
Colorectal Cancer Control and Prevention, Colorectal Cancer Recurrent
[68Ga]Ga-GZP PET for Early Response Prediction in Colorectal Cancer During Neoadjuvant Therapy
This diagnostic study investigates the value of 68Ga-NOTA-GZP PET imaging in predicting and evaluating immunotherapy response in malignant tumors by enrolling pathologically confirmed patients scheduled for immunotherapy and…
- Conditions
- Colorectal Cancer Control and Prevention, Colorectal Cancer Recurrent, Colorectal Cancer Metastatic, Colorectal Cancer Screening
- Trial name
- NCT06982053
- Testing
- Received 68Ga-NOTA-GZP PET scanning
- Where
- China
- Volunteers
- 21 people
- Sponsor
- Union Hospital, Tongji Medical College, Huazhong University of Science and Technology
Paraganglioma, Pheochromocytoma
Hereditary Paraganglioma: Evaluation of Screening Methods to Detect Tumors in SDH Positive Carriers
Hereditary paraganglioma -due to SDH (SDHD, SDHB, SDHC) germline mutations- causes paragangliomas and pheochromocytomas. Presymptomatic genetic testing should be offered to all first-degree relatives if an SDH mutation is…
- Trial name
- NCT00188019
- Testing
- diagnosis methods
- Where
- France
- Volunteers
- 248 people
- Sponsor
- University Hospital, Angers
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)
Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor
ABT-751 in Treating Young Patients With Refractory Solid Tumors
RATIONALE: Drugs used in chemotherapy, such as ABT-751, work in different ways to stop tumor cells from dividing so they stop growing or die.
- Conditions
- Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor, Extragonadal Germ Cell Tumor, Kidney Cancer
- Trial name
- NCT00036959
- Testing
- ABT-751
- Where
- 3 sites in United States
- Volunteers
- 90 people
- Sponsor
- National Institutes of Health Clinical Center (CC)
Amyloidosis, Amyloid
Monitoring of Early Disease Progression in Hereditary Transthyretin Amyloidosis
This study measures circulating, misfolded ATTR oligomers in asymptomatic ATTRm amyloidosis genetic carriers longitudinally over five years.
- Conditions
- Amyloidosis, Amyloid, Amyloid Neuropathies, Familial, Amyloid Cardiomyopathy
- Trial name
- MED-hATTR (NCT03431896)
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- The Cleveland Clinic
Peutz-Jeghers Syndrome
Poorly Differentiated Adenocarcinoma of the Jejunum in a Patient With Peutz-Jeghers Syndrome: A Case Report
Our study is a case report of one of the rarest risk factor, Peutz-Jeghers syndrome, of small bowel malignancy detected in a patient with poorly differentiated adenocarcinoma of small bowel(jejunum)
- Trial name
- NCT06242457
- Where
- Ethiopia
- Volunteers
- 1 people, healthy volunteers welcome
- Sponsor
- Haramaya Unversity
Leukemia
Biomarkers in Samples of Bone Marrow From Patients With Acute Myeloid Leukemia
RATIONALE: Studying samples of bone marrow 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
- NCT01290107
- Testing
- DNA analysis, gene rearrangement analysis, microarray analysis
- Volunteers
- 9 people
- Sponsor
- Children's Oncology Group
Recurrent Solid Tumor, Recurrent Ewing Sarcoma
Study of Onivyde With Talazoparib or Temozolomide in Children With Recurrent Solid Tumors and Ewing Sarcoma
The phase I portion of this study is designed for children or adolescents and young adults (AYA) with a diagnosis of a solid tumor that has recurred (come back after treatment) or is refractory (never completely went away).
- Conditions
- Recurrent Solid Tumor, Recurrent Ewing Sarcoma, Recurrent Hepatoblastoma, Recurrent Malignant Germ Cell Tumor
- Trial name
- NCT04901702
- Testing
- Onivyde, Talazoparib, Temozolomide
- Where
- 10 sites in United States, Canada
- Volunteers
- 90 people
- Sponsor
- St. Jude Children's Research Hospital
Benign Breast Disease, Breast Cancer
Prolactin Receptor and Breast Diseases
Prolactin is known to play an important role in breast development and differentiation. Thus proliferative breast diseases are good models to unravel PRl / PRLR function in proliferative processes.
- Trial name
- Prolacsein (NCT00842465)
- Testing
- blood collection for hormonal status analysis, breast Biopsy or surgery, blood collection
- Where
- France
- Volunteers
- 735 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
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