Clinical trials: Hereditary Neoplastic Syndromes
- Condition Hereditary Neoplastic Syndromes
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
Neurofibromatosis
A Long-term Study of NPC-12G Gel in Neurofibromatosis Type I
The purpose of this tria is to evaluate the safety and efficacy of long-term treatment with NPC-12G gel to patients with neurofibromatosis type I.
- Trial name
- NCT04461886
- Testing
- NPC-12G gel
- Where
- 4 sites in Japan
- Volunteers
- 100 people
- Sponsor
- Nobelpharma
Tuberous Sclerosis Complex (TSC)
Treatment With Full-spectrum Cannabis Extract of Refractory Epilepsy Associated With Tuberous Sclerosis Complex (TSC)
1. To assess treatment efficacy with YCJ-01 by changes in the number of epileptic seizures in patients with refractory epilepsy secondary to Tuberous Sclerosis Complex. 2.
- Trial name
- Spectrum (NCT07403266)
- Testing
- YCJ-01 Full Spectrum cannbis Extract
- Where
- Spain
- Volunteers
- 84 people
- Sponsor
- Oils4Cure
Tuberous Sclerosis Complex
Adjunctive GNX Treatment Compared With Placebo in Children and Adults With TSC-related Epilepsy
This is a Phase 3, global, double-blind, randomized, placebo-controlled study of adjunctive GNX treatment in children and adults with TSC-related epilepsy.
- Trial name
- NCT05323734
- Testing
- Ganaxolone
- Where
- 63 sites in United States, Australia and 8 more
- Volunteers
- 129 people
- Sponsor
- Marinus Pharmaceuticals
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
Nevi, Dysplastic
INTER-PATHOLOGIST READER STUDY
This is a post study histology analysis from previously obtained pathology slides.
- Trial name
- NCT06246903
- Where
- United States
- Volunteers
- 500 people
- Sponsor
- Orlucent, Inc
Solid Tumors, CNS Tumors
Study Of Entrectinib (Rxdx-101) in Children and Adolescents With Locally Advanced Or Metastatic Solid Or Primary CNS Tumors And/Or Who Have No Satisfactory Treatment Options
This is an open-label, Phase 1/2 multicenter dose escalation study in pediatric patients with relapsed or refractory extracranial solid tumors (Phase 1), with additional expansion cohorts (Phase 2) in patients with primary brain…
- Trial name
- STARTRK-NG (NCT02650401)
- Testing
- Entrectinib
- Where
- 26 sites in United States, Canada and 7 more
- Volunteers
- 69 people
- Sponsor
- Hoffmann-La Roche
Neuroblastoma, Rhabdomyosarcoma
Enoblituzumab (MGA271) in Children With B7-H3-expressing Solid Tumors
This study is a Phase 1, open-label, dose escalation and cohort expansion trial designed to characterize the safety, tolerability, PK, PD, immunogenicity and preliminary antitumor activity of enoblituzumab administered IV on a…
- Conditions
- Neuroblastoma, Rhabdomyosarcoma, Osteosarcoma, Ewing Sarcoma
- Trial name
- NCT02982941
- Testing
- Enoblituzumab
- Where
- 6 sites in United States
- Volunteers
- 25 people
- Sponsor
- MacroGenics
Tuberous Sclerosis Complex
Roll-over Study to Collect and Assess Long-term Safety of Everolimus in Patients With TSC and Refractory Seizures Who Have Completed the EXIST-3 Study [CRAD001M2304] and Who Are Benefitting From Continued Treatment
The purpose of this study is to evaluate the long-term safety in patients with TSC and refractory seizures who are currently receiving everolimus treatment in the Novartis-sponsored EXIST-3 study and who are determined to be…
- Trial name
- NCT02962414
- Testing
- everolimus
- Where
- 67 sites in United States, Australia and 16 more
- Volunteers
- 206 people
- Sponsor
- Novartis Pharmaceuticals
Tuberous Sclerosis Complex, Focal Cortical Dysplasia
Astroscape: A Study of Radiprodil on Safety, Tolerability, Pharmacokinetics, and Effect on Seizures and Behavioral Symptoms in Patients With TSC or FCD Type II
Study RAD-GRIN-201 is a phase 1B/2A trial to assess safety, tolerability, pharmacokinetics (PK), and potential efficacy of radiprodil in participants with Tuberous Sclerosis Complex (TSC) or Focal Cortical Dysplasia (FCD) type…
- Trial name
- NCT06392009
- Testing
- Radiprodil
- Where
- 18 sites in Australia, Belgium and 6 more
- Volunteers
- 30 people
- Sponsor
- GRIN Therapeutics, Inc.
Neuroblastoma, Ewing's Sarcoma
Use of Racotumomab in Patients With Pediatric Tumors Expressing N-glycolylated Gangliosides
This study will be carried out in children with diagnosis of cancer with tumors known to express N-glycolylated gangliosides. The disease must be resistant to conventional therapy.
- Conditions
- Neuroblastoma, Ewing's Sarcoma, Wilm's Tumor, Retinoblastoma
- Trial name
- NCT01598454
- Testing
- racotumomab
- Where
- Argentina
- Volunteers
- 15 people
- Sponsor
- Laboratorio Elea Phoenix S.A.
Neurofibromatosis Type 1
PASS of Paediatric Patients Initiating Selumetinib
Neurofibromatosis type 1 (NF1) is a rare, autosomal dominant genetic disorder that is caused by germline mutations in the NF1 tumour suppressor gene, which encodes the tumour suppressor protein neurofibromin 1.
- Trial name
- NCT05388370
- Where
- 46 sites in Austria, France and 8 more
- Volunteers
- 124 people
- Sponsor
- AstraZeneca
Tuberous Sclerosis
Adjunctive Ganaxolone Treatment (Part A) in TSC Followed by Long-term Treatment (Part B)
To assess preliminary safety and efficacy of ganaxolone as adjunctive therapy for the treatment of primary seizure types in patients with genetically- or clinically-confirmed TSC-related epilepsy through the end of the 12 week…
- Trial name
- TSC (NCT04285346)
- Testing
- Ganaxolone
- Where
- 7 sites in United States
- Volunteers
- 23 people
- Sponsor
- Marinus Pharmaceuticals
Pancreatic Ductal Adenocarcinoma, Familial Pancreatic Cancer
A Study of IMMray™ PanCan-d Test for Early Detection of Pancreatic Cancer in High-risk Groups
PanFAM-1 is a clinical study for early detection of pancreatic cancer in high-risk groups. The goals of the study are to assess the performance and diagnostic accuracy of the IMMray™ PanCan-d test compared to standard-of-care…
- Conditions
- Pancreatic Ductal Adenocarcinoma, Familial Pancreatic Cancer, FAMMM - Familial Atypical Mole Malignant Melanoma Syndrome
- Trial name
- NCT03693378
- Where
- 21 sites in United States, Canada and 3 more
- Volunteers
- 1,349 people, healthy volunteers welcome
- Sponsor
- Immunovia, Inc.
Tuberous Sclerosis, Subependymal Giant Cell Astrocytoma
Efficacy and Safety of Everolimus (RAD001) in Patients of All Ages With Subependymal Giant Cell Astrocytoma Associated With Tuberous Sclerosis Complex (TSC)(EXIST-1)
This study evaluated the efficacy and safety of Everolimus in treating patients with Subependymal Giant Cell Astrocytomas associated with Tuberous Sclerosis Complex.
- Trial name
- EXIST-1 (NCT00789828)
- Testing
- Everolimus
- Where
- 24 sites in United States, Australia and 8 more
- Volunteers
- 117 people
- Sponsor
- Novartis Pharmaceuticals
Neurofibromatosis
Clinical and Demographic Characteristics of Adult p Atients With NEurofibromatosis in RUSsia
Clinical and Demographic Characteristics of Adult Patients with NEurofibromatosis in RUSsia (NEREUS)
- Trial name
- NEREUS (NCT07088991)
- Where
- 16 sites in Russia
- Volunteers
- 200 people
- Sponsor
- AstraZeneca
Tuberous Sclerosis Complex, Seizures
An Open-label Extension Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6)
This trial consists of 2 parts: a double-blinded phase and an open-label extension phase. The open-label extension phase only will be described in this record. All participants will receive the same dose of GWP42003-P.
- Trial name
- NCT02544750
- Testing
- GWP42003-P
- Where
- United States
- Volunteers
- 199 people
- Sponsor
- Jazz Pharmaceuticals
Sleep Disorders
Efficacy and Safety of Circadin® in the Treatment of Sleep Disturbances in Children With Neurodevelopment Disabilities
The purpose of this study is to establish the efficacy and safety of Circadin in children with neurodevelopmental disorders and to determine the dose, this randomized, placebo-controlled study is planned to evaluate the efficacy…
- Trial name
- NCT01906866
- Testing
- Circadin 2/5/10 mg
- Where
- 28 sites in United States, Finland and 3 more
- Volunteers
- 125 people
- Sponsor
- Neurim Pharmaceuticals Ltd.
Tuberous Sclerosis Complex, Seizures
A Randomized Controlled Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6)
This trial consists of 2 parts: a double-blinded phase and an open-label extension phase. The blinded phase only will be described in this record. Participants will receive 1 of 2 doses of GWP42003-P or matching placebo.
- Trial name
- NCT02544763
- Testing
- GWP42003-P
- Where
- 44 sites in United States, Australia and 4 more
- Volunteers
- 224 people
- Sponsor
- Jazz Pharmaceuticals
Neurofibromatosis Type 1, Plexiform Neurofibromas
US Selumetinib Registry
The purpose of this study is to understand treatment patterns and assess long-term effectiveness and safety outcomes associated with selumetinib treatment as well as to explore clinical and non-clinical factors affecting those…
- Trial name
- NCT05683678
- Where
- 10 sites in United States
- Volunteers
- 37 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Tuberous Sclerosis, Angiofibroma
Phase III Trial of Topical Formulation of Sirolimus to Skin Lesions in Patients With Tuberous Sclerosis Complex (TSC)
The purpose of this trial is to evaluate the efficacy and safety of NPC-12G gel (topical formulation of sirolimus) versus placebo gel to facial angiofibroma and other skin lesions in patients with tuberous sclerosis complex (TSC)
- Conditions
- Tuberous Sclerosis, Angiofibroma, Hypomelanotic Macule, Plaque
- Trial name
- NCT02635789
- Testing
- NPC-12G gel
- Where
- Japan
- Volunteers
- 62 people
- Sponsor
- Nobelpharma