Clinical trials: Neoplastic Syndromes, Hereditary
NF1, Neurofibromatosis
Cabozantinib for Plexiform Neurofibromas (PN) in Subjects With NF1 in Children and Adults
This study, "A Phase II Study of Cabozantinib (XL l84) for Plexiform Neurofibromas in Subjects with Neurofibromatosis Type I in Children and Adults diagnosed with Neurofibromatosis Type 1 (NF1) and have a type of tumor called a…
- Conditions
- NF1, Neurofibromatosis, Plexiform Neurofibromas
- Trial name
- NF105-CABO (NCT02101736)
- Testing
- Cabozantinib
- Where
- 12 sites in United States
- Volunteers
- 45 people
- Sponsor
- University of Alabama at Birmingham
MPNST, NF1
Trial of Selumetinib and Bromodomain Inhibitor With Durvalumab for Sarcomas
A multi-institutional open-label phase 1/2 trial of selumetinib in combination with ZEN-3694 and durvalumab in refractory/unresectable sarcomas including MPNST.
- Conditions
- MPNST, NF1, Sarcoma
- Trial name
- NCT05253131
- Testing
- Selumetinib + ZEN-3694 ± Durvalumab
- Where
- United States
- Volunteers
- 41 people
- Sponsor
- University of Alabama at Birmingham
Cervical Cancer, Endometrial Cancer
Specimen and Data Study for Ovarian Cancer Early Detection and Prevention
RATIONALE: To improve strategies for detection and prevention of early-stage disease. PURPOSE: This research study is collecting specimens and data to develop better methods for early detection and prevention of ovarian cancer…
- Conditions
- Cervical Cancer, Endometrial Cancer, Fallopian Tube Cancer, Hereditary Breast/Ovarian Cancer (brca1, brca2)
- Trial name
- NCT00005095
- Testing
- Ca-125, screening questionnaire administration, Surgery
- Where
- United States
- Volunteers
- 6,000 people, healthy volunteers welcome
- Sponsor
- Northwestern University
Multiple Endocrine Neoplasia
Study and Follow-up of Multiple Endocrine Neoplasia Type 1
Multiple Endocrine Neoplasia type I (MEN1) or Wermer syndrome is an autosomal dominant disease that predisposes patients to the development of endocrine tumours, principally parathyroid, pituitary or duodenal-pancreatic tumours.
- Trial name
- GENEM (NCT03348501)
- Testing
- survey for long-term follow-up of the disease
- Where
- France
- Volunteers
- 2,000 people
- Sponsor
- Centre Hospitalier Universitaire Dijon
Neurofibromatosis Type 2, Progressive Vestibular Schwannomas
Phase 2 Study of Bevacizumab in Children and Young Adults With NF 2 and Progressive Vestibular Schwannomas
To determine the hearing response rate at 24 weeks after treatment with bevacizumab for symptomatic vestibular schwannomas (VS) in children and young adults with Neurofibromatosis Type 2 (NF 2).
- Trial name
- NCT01767792
- Testing
- Bevacizumab
- Where
- 12 sites in United States
- Volunteers
- 22 people
- Sponsor
- University of Alabama at Birmingham
Tuberous Sclerosis Complex, TSC Related Cognitive Disability
Efficacy of RAD001/Everolimus in Autism and NeuroPsychological Deficits in Children With Tuberous Sclerosis Complex
Tuberous sclerosis complex (TSC) is a genetic disease that leads to mental retardation in over 50% of patients, and to learning problems, behavioral problems, autism and epilepsy in up to 90% of patients.
- Conditions
- Tuberous Sclerosis Complex, TSC Related Cognitive Disability, TSC Related Autism, TSC Related Learning Problems
- Trial name
- RAPIT (NCT01730209)
- Testing
- Everolimus
- Where
- Netherlands
- Volunteers
- 60 people
- Sponsor
- Erasmus Medical Center
Neurofibromatosis Type 2, Neuroma, Acoustic
Study of RAD001 for Treatment of NF2-related Vestibular Schwannoma
The purpose of the study is to determine if RAD001 treatment will shrink or slow the growth of the vestibular schwannoma(s) in Neurofibromatosis 2 (NF2) patients.
- Trial name
- NCT01345136
- Testing
- RAD001, everolimus
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Jonsson Comprehensive Cancer Center
Neurofibromatoses
Phase I Photodynamic Therapy (PDT) for Benign Dermal Neurofibromas (NF1)
GENERAL OBJECTIVE The general objective is to assess the safety and efficacy of photodynamic therapy (PDT) in the treatment of neurofibromatosis 1 (NF1) tumors in the skin.
- Trial name
- NCT01682811
- Testing
- Part 1 Levulan injection, Part 1 Levulan surface application, Part 1 Levulan surface application twice
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Harry T Whelan, MD
Mitochondrial Disorders, Li-Fraumeni Syndrome
Role of p53 Gene in Metabolism Regulation in Patients With Li-Fraumeni Syndrome
This study will examine metabolic and biological factors in people with Li-Fraumeni syndrome, a rare hereditary disorder that greatly increases a person's susceptibility to cancer.
- Conditions
- Mitochondrial Disorders, Li-Fraumeni Syndrome, Carriers of p53 Mutation
- Trial name
- NCT00406445
- Where
- United States
- Volunteers
- 82 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Musculoskeletal Complications
Levocarnitine in Treating Patients With Vismodegib-Associated Muscle Spasms
This randomized clinical trial studies levocarnitine in treating patients with vismodegib-associated muscle spasms. Levocarnitine may decrease muscle spasms caused by vismodegib.
- Trial name
- NCT01893892
- Testing
- levocarnitine, questionnaire administration
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Stanford University
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
Multiple Osteochondroma, Osteogenesis Imperfecta
NGS Strategy Effectiveness in Molecular Diagnosis
The coming out of Next Generation Sequencing (NGS) technologies, with documented advantages and reduced costs respect to Sanger sequencing, has provided new appealing approaches to diagnostic testing.
- Trial name
- NCT03557567
- Testing
- NGS molecular screening
- Volunteers
- 400 people
- Sponsor
- Istituto Ortopedico Rizzoli
Neurofibromatosis Type 1
Effects of Physical Training on Bone and Muscle Quality, Muscle Strength, and Motor Coordination in Children With NF1
A physical training program will improve quality of life, participation in physical activity, motor coordination, muscle strength, and bone and muscle strength in children with neurofibromatosis type 1.
- Trial name
- NCT01058330
- Testing
- Plyometric training program
- Where
- 2 sites in United States
- Volunteers
- 36 people
- Sponsor
- Shriners Hospitals for Children
Low Grade Glioma, Neurofibromatosis Type 1
A Study of the Drugs Selumetinib Versus Carboplatin/Vincristine in Patients With Neurofibromatosis and Low-Grade Glioma
This phase III trial studies if selumetinib works just as well as the standard treatment with carboplatin/vincristine (CV) for subjects with NF1-associated low grade glioma (LGG), and to see if selumetinib is better than CV in…
- Conditions
- Low Grade Glioma, Neurofibromatosis Type 1, Visual Pathway Glioma
- Trial name
- NCT03871257
- Testing
- Carboplatin, Magnetic Resonance Imaging, Quality-of-Life Assessment
- Where
- 132 sites in United States, Canada and 1 more
- Volunteers
- 165 people
- Sponsor
- National Cancer Institute (NCI)
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
Tuberous Sclerosis
Remote Assessment and Intervention for Behavior Problems in Kids With TSC
The RAINBOW study is a fully remote study focused on understanding and treating behavior problems in young children with tuberous sclerosis complex (TSC).
- Trial name
- RAINBOW (NCT06311474)
- Testing
- Parent-Child Interaction Therapy (PCIT)
- Where
- United States
- Volunteers
- 101 people
- Sponsor
- University of California, Los Angeles
Leukemia, Myelodysplastic Syndromes
VNP40101M and Cytarabine in Treating Patients With Hematologic Malignancies
RATIONALE: Drugs used in chemotherapy, such as VNP40101M and cytarabine, use different ways to stop cancer cells from dividing so they stop growing or die. Combining more than one drug may kill more cancer cells.
- Trial name
- NCT00070538
- Testing
- cytarabine, laromustine
- Where
- United States
- Sponsor
- Vion Pharmaceuticals
Neurofibromatosis Type 1
A Study to Evaluate the Feasibility of a Physiologic Biomarker to Assess Pain and Other Sensory Problems Using Pupillometry in Participants With Neurofibromatosis Type 1 (NF1)
Background: Neurofibromatosis type 1 (NF1) is a genetic condition that causes tumors to grow along the nerves in the skin, brain, and other parts of the body.
- Trial name
- NCT06507748
- Testing
- AlgometRx Nociometer(R)
- Where
- 2 sites in United States
- Volunteers
- 70 people
- Sponsor
- National Cancer Institute (NCI)
Human Immunodeficiency Virus, Acquired Immunodeficiency Syndrome
Self-Test Strategies and Linkage Incentives to Improve ART and PrEP Uptake in Men
This is a randomized controlled trial to test a combination behavioral and biomedical interventions to improve the HIV prevention and care cascades in a population of mobile men in a high priority setting (fishermen in Kenya).
- Conditions
- Human Immunodeficiency Virus, Acquired Immunodeficiency Syndrome, HIV Infections, Sexually Transmitted Diseases, Viral
- Trial name
- NCT04772469
- Testing
- Provision of multiple self-tests, Small monetary incentives, Use of SMS/text reminders to motivate adherence to ART or PrEP
- Where
- Kenya
- Volunteers
- 934 people, healthy volunteers welcome
- Sponsor
- University of California, San Francisco
Adenomatous Polyposis Coli
Sirolimus and Familial Adenomatous Polyposis (FAP)
The aim of the study is to investigate the effect of sirolimus on the progression of intestinal adenomas in patients with FAP and to assess the safety of this treatment.
- Trial name
- NCT03095703
- Testing
- Sirolimus
- Where
- Netherlands
- Volunteers
- 4 people
- Sponsor
- Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
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