Clinical trials: Neoplastic Syndromes, Hereditary
Neurofibromatosis 2
Efficacy and Safety Study of RAD001 in the Growth of the Vestibular Schwannoma(s) in Neurofibromatosis 2 (NF2) Patients
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
- AFINF2 (NCT01490476)
- Testing
- RAD001
- Where
- France
- Volunteers
- 10 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Solid Tumor (Excluding CNS), Liver Cell Carcinoma
Immunotherapy for Solid Tumor Malignancies in Pediatrics Using Interleukin-15 and -21 Armored Glypican-3-specific Chimeric Antigen Receptor T Cells
This Phase 1, open-label, non-randomized study will enroll pediatric and young adult subjects with relapsed or refractory non-central nervous system (CNS) malignant solid tumors expressing glypican-3 (GPC3) to examine the safety…
- Conditions
- Solid Tumor (Excluding CNS), Liver Cell Carcinoma, Malignant Rhabdoid Tumor, Yolk Sac Tumor
- Trial name
- IMPACT (NCT07148050)
- Testing
- SC-CAR.GPC3xIL15.21 CAR T cells
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Seattle Children's Hospital
Vestibular Schwannoma, Meningioma
Exploratory Evaluation of AR-42 Histone Deacetylase Inhibitor in the Treatment of Vestibular Schwannoma and Meningioma
This will be a multi-center, proof of concept phase 0 study to assess the suppression of p-AKT in Vestibular Schwannoma (VS) and meningiomas by AR-42 in adult patients undergoing tumor resection.
- Conditions
- Vestibular Schwannoma, Meningioma, Acoustic Neuroma, Neurofibromatosis Type 2
- Trial name
- NCT02282917
- Testing
- AR-42
- Where
- 4 sites in United States
- Volunteers
- 7 people
- Sponsor
- Massachusetts Eye and Ear Infirmary
BCCs in Gorlin Syndrome Patients
CODY: A Study Evaluating The Safety And Efficacy Of QTORIN 3.9% Sirolimus Topical Gel For The Prevention Of Basal Cell Carcinomas (BCCs) In Patients With Gorlin Syndrome
A Phase 2b study looks at the safety and efficacy of a treatment that is being investigated for people with certain diseases. This study will be conducted at multiple centers in the United States where participants with Gorlin…
- Trial name
- NCT04893486
- Testing
- PTX-022, Vehicle comparator
- Where
- 16 sites in United States, United Kingdom
- Volunteers
- 73 people
- Sponsor
- Palvella Therapeutics, Inc.
Tuberous Sclerosis Complex, Epilepsy
Stopping TSC Onset and Progression 2: Epilepsy Prevention in TSC Infants
This phase I/II clinical trial is an open-label clinical trial design to verify safety and dosing for TAVT-18 (sirolimus) powder for oral solution in TSC infants (N=5).
- Trial name
- STOP2 (NCT04595513)
- Testing
- TAVT-18 (sirolimus)
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission
Internet-Based Program With or Without Telephone-Based Problem-Solving Training in Helping Long-Term Survivors of Hematopoietic Stem Cell Transplant Cope With Late Complications
RATIONALE: A personalized Internet-based program may help improve fatigue, depression, and quality of life in long-term survivors of stem cell transplant.
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
- Trial name
- NCT00799461
- Testing
- internet-based intervention, questionnaire administration, psychosocial assessment and care
- Where
- United States
- Volunteers
- 1,337 people
- Sponsor
- Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium
Neurofibromatosis, NF1
Study of Tasigna®/Nilotinib (AMN107) in Neurofibromatosis (NF1) Patients With Plexiform Neurofibromas
The purpose of this Pilot Study is to determine if NF1 patients with plexiform neurofibromas treated with Tasgina® respond to therapy.
- Conditions
- Neurofibromatosis, NF1, Neurofibromas
- Trial name
- NCT01275586
- Testing
- Tasigna
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Indiana University
Solid Tumors, Soft Tissue Sarcoma
A Pilot Study of Thermodox and MR-HIFU for Treatment of Relapsed Solid Tumors
This is a pilot study of LTLD with MR-HIFU hyperthermia followed by ablation in subjects with refractory/relapsed solid tumors.
- Conditions
- Solid Tumors, Soft Tissue Sarcoma, Ewing Sarcoma, Malignant Epithelial Neoplasm
- Trial name
- NCT04791228
- Testing
- Magnetic Resonance-Guided High Intensity Focused Ultrasound, Lyso-thermosensitive Liposomal Doxorubicin
- Where
- United States
- Sponsor
- Children's National Research Institute
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
Skin Carcinoma
Topical Tazarotene in Treating Patients With Basal Cell Skin Cancer and Basal Cell Nevus Syndrome on the Chest and Back
RATIONALE: Drugs used in chemotherapy, such as tazarotene, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing.
- Trial name
- NCT00783965
- Testing
- tazarotene
- Where
- 3 sites in United States
- Volunteers
- 34 people
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Breast Cancer
A Companion Sample Collection Protocol to Support the Discovery of Breast Cancer Aberrations With Treatment of CDK4/6 Therapy/LEE011/Ribociclib
This study was a companion study to CLEE011A2404 which provided the opportunity for the collection of tumor tissue samples to better understand relevant mutations and the mechanisms responsible for resistance to treatment.
- Trial name
- NCT03050398
- Testing
- Ribociclib, letrozole
- Where
- 6 sites in United States
- Volunteers
- 8 people
- Sponsor
- Novartis Pharmaceuticals
Chronic Myeloproliferative Disorders, Leukemia
Flu Vaccine in Preventing Influenza Infection in Healthy Volunteers and in Patients Who Have Undergone Stem Cell Transplant
RATIONALE: Studying immune response to flu vaccine in patients who have undergone a stem cell transplant may help doctors plan the best treatment.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00964821
- Testing
- trivalent influenza vaccine, Immunoenzyme technique, Laboratory biomarker analysis
- Where
- 2 sites in United States
- Volunteers
- 459 people, healthy volunteers welcome
- Sponsor
- City of Hope Medical Center
Lynch Syndrome
The Yield of Artificial Intelligence (GI Genius) in Lynch Syndrome - A Randomized Tandem-colonoscopy Trial
We aim to investigate the incremental detection rate of polyps using AI- assisted colonoscopy compared with high-definition white-light endoscopy (HD-WLE).
- Trial name
- NCT06654128
- Testing
- artificial intelligence
- Where
- Israel
- Volunteers
- 100 people
- Sponsor
- Sheba Medical Center
Tuberous Sclerosis
A Study of Everolimus in the Treatment of Neurocognitive Problems in Tuberous Sclerosis
This is a single centre, two-arm, individually randomised, Phase II, double- blind, placebo-controlled trial of RAD001 (Everolimus) versus placebo in the treatment of neurocognitive problems in patients with tuberous sclerosis…
- Trial name
- TRON (NCT01954693)
- Testing
- Everolimus (RAD001)
- Where
- United Kingdom
- Volunteers
- 48 people
- Sponsor
- Cardiff University
Tuberous Sclerosis
JASPER Early Intervention for Tuberous Sclerosis
The investigators are running an intervention study for young children with Tuberous Sclerosis Complex (TSC). The study will include free play-based behavioral intervention administered remotely that may improve social and…
- Trial name
- JETS (NCT03422367)
- Testing
- JASPER
- Where
- 2 sites in United States
- Volunteers
- 64 people
- Sponsor
- University of California, Los Angeles
Headache, Tumor
Dexmedetomidine and Propofol for Pediatric MRI Sedation
The purpose of this study is to compare the results of combining two anesthetic medications (dexmedetomidine and propofol) in low doses with a standard dose of a single drug that is commonly used to provide sedation/anesthesia…
- Conditions
- Headache, Tumor, Seizure Disorder, Neurofibromatoses
- Trial name
- NCT03513757
- Testing
- propofol, Dexmedetomidine, Glycopyrrolate
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Medical College of Wisconsin
Lynch Syndrome
Multi-Organ Screening Recommendations in Patients With Lynch Syndrome
The purpose of this study is to examine how people with a family history of colon cancer and other related cancers respond to recommendations for cancer screening after genetic counseling.
- Trial name
- NCT00582296
- Testing
- questionnaires, telephone interview, optional blood drawn, questionnaires, telephone interview and optional blood drawn
- Where
- 8 sites in United States
- Volunteers
- 1,530 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Tuberous Sclerosis Complex
Acceptance and Commitment Therapy in Tuberous Sclerosis Complex
The study will assess the acceptability and feasibility of a randomised controlled trial of 6-12 sessions of remotely-delivered Acceptance and Commitment Therapy (ACT) versus waitlist control.
- Trial name
- ACT in TSC (NCT05867576)
- Testing
- Acceptance and Commitment Therapy (ACT)
- Where
- United Kingdom
- Volunteers
- 15 people
- Sponsor
- University Hospitals Bristol and Weston NHS Foundation Trust
Tuberous Sclerosis, Lymphangioleiomyomatosis
Trial of Efficacy and Safety of Sirolimus in Tuberous Sclerosis and LAM
The purpose of this study is to determine the safety and efficacy of the mTOR inhibitor sirolimus as a treatment for renal angiomyolipomas in patients with tyberous sclerosis complex or sporadic lymphangioleiomyomatosis.
- Trial name
- TESSTAL (NCT00490789)
- Testing
- sirolimus
- Where
- 3 sites in United Kingdom
- Volunteers
- 14 people
- Sponsor
- Cardiff University
Cancer
Fentanyl Sublingual Spray in Treating Patients With Breakthrough Cancer Pain
This is a phase III, randomized, double-blind, placebo-controlled, multicenter study of the clinical response to fentanyl sublingual spray as a treatment for breakthrough cancer pain.
- Trial name
- NCT00538850
- Testing
- Fentanyl sublingual spray
- Where
- United States
- Volunteers
- 130 people
- Sponsor
- INSYS Therapeutics Inc
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