Clinical trials: Inherited Cancer Syndrome
Wilms Tumour
Collaborative Wilms Tumour Africa Project
Significant progress has been made in the treatment of Wilms tumor in high income countries, where survival is now around 85% - 90%.
- Trial name
- NCT01991652
- Where
- 3 sites in Cameroon, Ghana and 1 more
- Volunteers
- 400 people
- Sponsor
- Amsterdam UMC, location VUmc
Neurofibromatosis 1
Systematically Assessing Changes in Plexiform Neurofibroma Related Disfigurement From Photographs of Subjects With Neurofibromatosis Type 1 on a Phase 2 Clinical Trial
Background: People with Neurofibromatosis type 1 (NF1) have an increased risk of developing plexiform neurofibromas (PNs). PNs are tumors that form in the tissue. They can form anywhere in the body.
- Trial name
- NCT04879160
- Where
- United States
- Volunteers
- 52 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
Urothelial Carcinoma, Lynch Syndrome
Urothelial Cancer Screening in Individuals With Lynch Syndrome Using a Urine Tumor DNA Panel (LS-URO Study)
Lynch syndrome (LS) is an inherited cancer predisposition syndrome caused by pathogenic germline variants in DNA mismatch repair (MMR) genes.
- Trial name
- NCT06218433
- Testing
- Urothelial cancer screening using urine tumor DNA test, Urothelial cancer screening using urine cytology (comparator)
- Where
- 2 sites in Canada, Finland
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Tampere University Hospital
Neurofibromatosis 2
An Implant for Hearing Loss Due to Removal of Neurofibromatosis 2 Tumors
This is a study to gather some information on the safety and efficacy of the penetrating auditory brainstem implant (PABI) in patients with neurofibromatosis type 2.
- Trial name
- NCT00030043
- Testing
- Penetrating auditory brainstem implant
- Where
- 3 sites in United States
- Volunteers
- 10 people
- Sponsor
- FDA Office of Orphan Products Development
Constipation, Childhood Acute Lymphoblastic Leukemia
Comparison of Gastrointestinal Motility in Healthy Children and Children With Constipation
To gain a better understanding of the underlying dysmotility in constipation in children, we will use a novel minimal invasive method called 3D Transit.
- Conditions
- Constipation, Childhood Acute Lymphoblastic Leukemia, Neurofibromatosis 1, Chronic Constipation With Overflow
- Trial name
- NCT03981510
- Testing
- 3D-Transit
- Where
- Denmark
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- University of Aarhus
Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor
Temozolomide and O6-benzylguanine in Treating Children With Solid Tumors
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining more than one drug may kill more tumor cells.
- Conditions
- Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor, Extragonadal Germ Cell Tumor, Kidney Cancer
- Trial name
- NCT00020150
- Testing
- O6-benzylguanine, temozolomide
- Where
- United States
- Sponsor
- National Cancer Institute (NCI)
Wilms Tumor, Neuroblastoma
Pharmacokinetic Study of Doxorubicin in Children With Cancer
Analyze pharmacokinetics of doxorubicin in children with cancer. Furthermore investigate the predictive role of troponin and natriuretic peptides for anthracycline-induced cardiotoxicity .
- Conditions
- Wilms Tumor, Neuroblastoma, Soft Tissue Sarcoma, Acute Lymphoblastic Leukemia
- Trial name
- Doxo (NCT01095926)
- Testing
- doxorubicin
- Where
- 23 sites in France, Germany and 2 more
- Volunteers
- 101 people
- Sponsor
- University Hospital Muenster
Obesity, Polycystic Ovary Syndrome
Treatment of Obesity With Topiramate in Patients With Polycystic Ovary Syndrome
Treatment of obesity related to Polycystic Ovary Syndrome with topiramate or placebo to assess improvement of clinical and laboratory parameters after 6 months of follow-up
- Conditions
- Obesity, Polycystic Ovary Syndrome, Hyperandrogenism
- Trial name
- NCT04453306
- Testing
- Topiramate
- Where
- Brazil
- Volunteers
- 80 people
- Sponsor
- Hospital de Clinicas de Porto Alegre
Breast Cancer, Chronic Myeloproliferative Disorders
Aprepitant, Granisetron, & Dexamethasone in Preventing Nausea & Vomiting in Pts. Receiving Cyclophosphamide Before a Stem Cell Transplant
RATIONALE: Antiemetic drugs, such as aprepitant, granisetron, and dexamethasone, may help lessen or prevent nausea and vomiting in patients treated with chemotherapy.
- Conditions
- Breast Cancer, Chronic Myeloproliferative Disorders, Gestational Trophoblastic Tumor, Leukemia
- Trial name
- NCT00293384
- Testing
- Aprepitant, Cyclophosphamide, Dexamethasone
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Barbara Ann Karmanos Cancer Institute
Hyper-Immunoglobulin E. Syndrome (HEIS), Chronic Granulomatous Disease (CGD)
Skin Immunity Sample Collection Involving Blisters and Biopsies
Background: The way the body heals and protects itself from getting sick is called the immune response. Some people with weak immune systems get sick often or get rashes and skin infections.
- Trial name
- NCT03921515
- Testing
- Blister induction, Skin Biopsies
- Where
- United States
- Volunteers
- healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Zollinger-Ellison Syndrome, Multiple Endocrine Neoplasia
Treatment of Zollinger-Ellison Syndrome With Prevacid
The purpose of this study is to study the safety and efficacy of high dose Prevacid in the long-term treatment of patients who secrete abnormally large amounts of gastric acid.
- Trial name
- NCT00204373
- Testing
- Lansoprazole (Prevacid)
- Volunteers
- 72 people
- Sponsor
- Charles Mel Wilcox, MD
Neurofibromatosis, Neurofibromas
Pilot Study of Gleevec/Imatinib Mesylate (STI-571, NSC 716051) in Neurofibromatosis (NF1) Patient With Plexiform Neurofibromas
This is a second Pilot Study to determine the efficacy of Gleevec® in neurofibromatosis (NF1) patients with plexiform neurofibromas using new response assessment modalities with the secondary goals of assessing Gleevec toxicity…
- Trial name
- 0908-09 (NCT01140360)
- Testing
- Gleevec
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Kent Robertson
Gorlin Syndrome
Efficacy and Safety of Patidegib Gel 2% for Preventing Basal Cell Carcinomas on the Face of Adults With Gorlin Syndrome
The aim of this clinical study is to find out how well Patidegib Gel 2% works in preventing new basal cell carcinomas (BCCs) developing on the face of adults with Gorlin syndrome, and how safe Patidegib Gel 2% is to use.
- Trial name
- NCT06050122
- Testing
- Patidegib Topical Gel, Patidegib Topical Gel with no active patidegib
- Where
- 40 sites in United States, France and 5 more
- Volunteers
- 113 people
- Sponsor
- Sol-Gel Technologies, Ltd.
Multiple Osteochondroma, Enchondromatosis
Survey on Multiple Osteochondromas and Enchondromatoses During the Transition From Paediatric Care to Adult Care
This prospective observational study aims to understand the transition experiences and unmet needs of patients living with multiple osteochondromas or enchondromatoses during the paediatric to adult care, in order to produce a…
- Trial name
- PJMOE (NCT07839637)
- Where
- Italy
- Volunteers
- 91 people, healthy volunteers welcome
- Sponsor
- Istituto Ortopedico Rizzoli
Tuberous Sclerosis Complex
Trial of RAD001 and Neurocognition in Tuberous Sclerosis Complex (TSC)
Tuberous Sclerosis Complex (TSC) is a multi-system disease, usually presenting with seizures, mental retardation and autism, and exhibiting a high variability in clinical findings both among and within families.
- Trial name
- TSC (NCT01289912)
- Testing
- RAD001
- Where
- 2 sites in United States
- Volunteers
- 52 people
- Sponsor
- Mustafa Sahin
Polycystic Ovarian Syndrome (PCOS)
This Study Aims to Evaluate Whether Milamend's Product Could Improve Hormone Balance in Women Who Suffer From Self-reported PCOS Symptoms.
The rationale for this study is to observe the impact of a consumer-grade commercially available product called Milamend Hormone Balance on quality of life and hormonal health in women with self-reported symptoms associated with…
- Trial name
- NCT07764328
- Testing
- A dietary supplement powder for women
- Where
- United States
- Volunteers
- 225 people
- Sponsor
- Mila Mend Inc.
Hemangioma, Cavernous, Central Nervous System
Study on Treatment Decision-Making and Prognostic Follow-Up for Untreated Cerebral Cavernous Malformations
The goal of this observational study is to evaluate and predict the risk associated with cerebral cavernous malformations (CCMs) using advanced artificial intelligence and radiomics analysis technology.
- Trial name
- NCT06214767
- Where
- China
- Volunteers
- 1,200 people
- Sponsor
- Beijing Tiantan Hospital
Chronic Myeloproliferative Disorders, Graft Versus Host Disease
Immunologic Diagnostic Blood Test in Predicting Side-Effects in Patients Undergoing a Donor Stem Cell Transplant for Hematologic Cancer or Other Diseases
RATIONALE: Studying a diagnostic biomarker test in blood samples from patients who have undergone a donor stem cell transplant for cancer may help doctors plan treatment.
- Conditions
- Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Infection, Leukemia
- Trial name
- NCT00813501
- Testing
- immunosuppressive therapy, immunological diagnostic method, allogeneic hematopoietic stem cell transplantation
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- City of Hope Medical Center
Leukemia, Lymphoma
New York Blood Center National Cord Blood Program
Umbilical cord blood is used as a source of hematopoietic stem cells for bone marrow reconstitution in patients who would be potential candidates for a bone marrow transplant from an unrelated marrow donor.
- Conditions
- Leukemia, Lymphoma, Genetic Disease, Severe Aplastic Anemia
- Trial name
- NCT00212407
- Testing
- Umbilical Cord Blood Transplantation
- Where
- United States
- Volunteers
- 4,476 people
- Sponsor
- New York Blood Center
Lymphoma, Sarcoma
A Phase I Study of Vorinostat and Bortezomib in Children With Refractory of Recurrent Solid Tumors, Including CNS Tumors and Lymphomas
Background: Vorinostat and bortezomib are anti-tumor drugs that have been approved by the Food and Drug Administration to treat different kinds of myeloma and lymphoma in adults.
- Conditions
- Lymphoma, Sarcoma, WilmsTumor, Neuroblastoma
- Trial name
- NCT01132911
- Testing
- Vorinostat (SAHA), Velcade (PS-341, Bortezomib)
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- National Cancer Institute (NCI)
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