Clinical trials: Neurofibromatosis
Plexiform Neurofibroma Associated With Neurofibromatosis Type 1
Efficacy and Safety of RAD001 in Treating Plexiform Neurofibromas (PN) Associated With Neurofibromatosis (NF1)
This study was to evaluate the antitumor activity and safety of RAD001 in patients with Plexiform neurofibromas (PN) associated with Neurofibromatosis Type 1 (NF1). The aim of the study was to : 1.
- Trial name
- NCT01365468
- Testing
- Everolimus (RAD001)
- Where
- 2 sites in Israel
- Volunteers
- 9 people
- Sponsor
- Novartis Pharmaceuticals
CNS Cancer, Meningioma
Phase II Trial of Sunitinib (SU011248) in Patients With Recurrent or Inoperable Meningioma
The purpose of this study is to find out what effects, good and/or bad, sunitinib has on patients and their tumors. At this time, no drugs are routinely used to treat meningioma, hemangioblastoma or hemangiopericytoma.
- Conditions
- CNS Cancer, Meningioma, Intracranial Hemangiopericytoma, Hemangioblastoma
- Trial name
- NCT00589784
- Testing
- Sunitinib
- Where
- 6 sites in United States
- Volunteers
- 50 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Neurofibromatosis
Phase II Study of Gleevec/Imatinib Mesylate (STI-571, NCS 716051) in Neurofibromatosis (NF1) Patients With Plexiform Neurofibromas
THe primary objective is to estimate the response rate at 6 months to Gleevec® in patients with plexiform neurofibromas
- Trial name
- NCT01673009
- Testing
- Gleevec
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- Indiana University
Pediatric Low-grade Glioma
Phase 1/2 Study of Mirdametinib + Vinblastine for Newly Diagnosed/Previously Untreated PLGG + Activation of MAPK
This is a 3-part open-label study (feasibility phase, treatment phase and follow-up phase) of orally administered mirdametinib in combination with intravenous vinblastine chemotherapy in patients with PLGG with activation of MAPK…
- Trial name
- MIRV (NCT06666348)
- Testing
- Mirdametinib
- Where
- 10 sites in Canada
- Volunteers
- 50 people
- Sponsor
- St. Justine's Hospital
Neurofibromatosis 1
iCanCope With NF: Innovating an Efficacious Digital Self-management and Transitional Care Program for Adolescents With Neurofibromatosis
Neurofibromatosis Type 1 (NF1) often significantly impairs the mental health, social life, self-esteem, and quality of life of adolescent patients. These impacts can be made worse with the experience of NF1-related pain.
- Trial name
- NCT07077408
- Testing
- iCanCope-NF Youth app, iCanCope-NF Youth app + CM
- Volunteers
- 60 people
- Sponsor
- The Hospital for Sick Children
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
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
Neurofibromatosis
Relationship Between Psychosocial Factors, Health Literacy, Quality of Life and Satisfaction With Medical Visits in Adults With NF
This is a prospective study through the NF clinic at Massachusetts General Hospital that will examine the relationship between psychosocial factors, health literacy, and satisfaction with the medical visit in adults with NF1…
- Trial name
- NCT02435628
- Testing
- Questionnaires
- Where
- United States
- Volunteers
- 86 people, healthy volunteers welcome
- Sponsor
- Massachusetts General Hospital
Healthy
Bioequivalence Study of Two Formulations of Pregabalin CR (Controlled-release) Table 150 mg
The purpose of this clinical trial is to (1) evaluate the bioequivalence(BE) of GLA5PR GLARS-NF3 tablet 150mg administered regular diet relative to GLA5PR GLARS-NF1 tablet 150mg administered regular diet and (2) determine the…
- Trial name
- NCT02955472
- Testing
- GLA5PR GLARS-NF3 tablet 150mg, GLA5PR GLARS-NF1 tablet 150mg
- Where
- South Korea
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- GL Pharm Tech Corporation
Neurofibromatosis 1, NF1
Developing Biomarkers of Plexiform Tumor Burden in Patients With Neurofibromatosis-Type 1
The purpose of this study is to identify tumor biomarkers in individuals with Neurofibromatosis type 1 (NF1). Biomarkers are signals that the investigator can measure that tell us about a process such as progress of a disease or…
- Conditions
- Neurofibromatosis 1, NF1, Neurofibromatosis Type 1
- Trial name
- NCT05238909
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
Advanced Malignant Neoplasm, Castleman Disease
Bevacizumab and Temsirolimus Alone or in Combination With Valproic Acid or Cetuximab in Treating Patients With Advanced or Metastatic Malignancy or Other Benign Disease
This phase I trial studies the side effects and best dose of bevacizumab and temsirolimus alone or in combination with valproic acid or cetuximab in treating patients with a malignancy that has spread to other places in the body…
- Conditions
- Advanced Malignant Neoplasm, Castleman Disease, Digestive System Carcinoma, Erdheim-Chester Disease
- Trial name
- NCT01552434
- Testing
- Bevacizumab, Cetuximab, Laboratory Biomarker Analysis
- Where
- United States
- Volunteers
- 154 people
- Sponsor
- M.D. Anderson Cancer Center
Profound Bilateral Deafness Due to, Bilateral Cochlear Aplasia
A Safety Study of the Auditory Brainstem Implant for Pediatric Profoundly Deaf Patients
Current treatment options for bilateral profoundly deaf children, diagnosed with inner ear anatomical abnormalities, are limited and, in the case of absent cochleas, non-existent.
- Conditions
- Profound Bilateral Deafness Due to, Bilateral Cochlear Aplasia, Bilateral Cochlear Nerve Deficiency, Bilateral Cochlear Ossification Secondary to Meningitis
- Trial name
- NCT02102256
- Testing
- Auditory Brainstem Implant
- Where
- 2 sites in United States
- Volunteers
- 10 people
- Sponsor
- Laurie Eisenberg
MPNST (Malignant Peripheral Nerve Sheath Tumor)
Combination of Avutometinib and Defactinib for Treatment of Relapsed, Refractory, Metastatic, or Unresectable Malignant Peripheral Nerve Sheath Tumor
The purpose of this study is to learn whether the study drugs avutometinib and defactinib can help stop or shrink malignant peripheral nerve sheath tumors (MPNST) that have returned or cannot be removed with surgery.
- Trial name
- NF120 (NCT07743554)
- Testing
- avutometinib capsule + defactinib tablet
- Volunteers
- 25 people
- Sponsor
- Girish Dhall, MD
Advanced Malignant Solid Neoplasm, Metastatic Malignant Solid Neoplasm
Testing Whether Cancers With Specific Mutations Respond Better to Glutaminase Inhibitor, Telaglenastat Hydrochloride, Anti-Cancer Treatment, BeGIN Study
This phase II trial studies how well glutaminase inhibitor telaglenastat hydrochloride (CB-839 HCl) works in treating patients with specific genetic mutations and solid tumors or malignant peripheral nerve sheath tumors that have…
- Conditions
- Advanced Malignant Solid Neoplasm, Metastatic Malignant Solid Neoplasm, NF1 Mutation Positive Malignant Peripheral Nerve Sheath Tumor, Unresectable Malignant Solid Neoplasm
- Trial name
- NCT03872427
- Testing
- Biospecimen Collection, Computed Tomography, Magnetic Resonance Imaging
- Where
- 31 sites in United States
- Volunteers
- 54 people
- Sponsor
- National Cancer Institute (NCI)
Neurofibromatosis 1, Neurofibromatosis Type 1
AZD6244 Hydrogen Sulfate for Children With Nervous System Tumors
Background: Plexiform neurofibromas are tumors that grow in and around nerves. The only way to treat them is with surgery. Some of these tumors cannot be completely removed.
- Conditions
- Neurofibromatosis 1, Neurofibromatosis Type 1, NF1, Neurofibroma, Plexiform
- Trial name
- NCT01362803
- Testing
- AZD6244
- Where
- 4 sites in United States
- Volunteers
- 99 people
- Sponsor
- National Cancer Institute (NCI)
Leukemia, Juvenile Myelomonocytic, JMML
A Phase I/II Study of Trametinib and Azacitidine for Patients With Newly Diagnosed Juvenile Myelomonocytic Leukemia
This clinical trial will test the safety and efficacy of combining trametinib and azacitidine in patients with juvenile myelomonocytic leukemia (JMML).
- Conditions
- Leukemia, Juvenile Myelomonocytic, JMML, JCML, Neurofibromatosis 1
- Trial name
- NCT05849662
- Testing
- Trametinib, Azacitidine, Fludarabine
- Where
- 21 sites in United States
- Volunteers
- 58 people
- Sponsor
- Therapeutic Advances in Childhood Leukemia Consortium
Neurofibromatosis 1, Neurofibroma, Plexiform
Pirfenidone in Children and Young Adults With Neurofibromatosis Type I and Progressive Plexiform Neurofibromas
Background: Neurofibromatosis Type 1 (NF1) is an autosomal dominant, progressive genetic disorder characterized by diverse clinical manifestations.
- Trial name
- NCT00076102
- Testing
- Pirfenidone
- Where
- 15 sites in United States
- Volunteers
- 36 people
- Sponsor
- National Cancer Institute (NCI)
Tumor, Solid, Pancreatic Cancer
Ulixertinib/Palbociclib in Patients With Advanced Pancreatic and Other Solid Tumors
This phase I study is designed to establish the safety, maximally tolerated dose (MTD) and recommended phase II dose (RP2D) of the ERK inhibitor ulixertinib (BVD-523) when combined with the CDK4/6 inhibitor palbociclib.
- Conditions
- Tumor, Solid, Pancreatic Cancer, Melanoma
- Trial name
- NCT03454035
- Testing
- Ulixertinib, Palbociclib
- Where
- United States
- Volunteers
- 45 people
- Sponsor
- UNC Lineberger Comprehensive Cancer Center
Neurofibromatosis Type 1, Neurofibromatosis 1
Neurofibromatosis Type 1 Tumor Early Detection Study
The goal of this observational study is to determine if a liquid biopsy (i.e. blood test) is an effective clinical tool for monitoring the development of malignant peripheral nerve sheath tumor (MPNST) among adults (18 years and…
- Conditions
- Neurofibromatosis Type 1, Neurofibromatosis 1, Plexiform Neurofibroma, Plexiform Neurofibromas
- Trial name
- NF1-TED (NCT06515860)
- Testing
- Liquid biopsy for MPNST development
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- David Miller
Healthy
A PK Study GL2702 GLARS-NF1 and Omix Ocas® in Healthy Male Volunteers
Study to Investigate The Pharmacokinetics of GL2702 GLARS-NF1 and Omix Ocas® in Healthy Male Volunteers
- Trial name
- NCT02072213
- Testing
- GL2702 GLARS-NF1, Omix Ocas
- Where
- South Korea
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- GL Pharm Tech Corporation