Clinical trials: NF1
Gastrointenstinal Stromal Tumor (GIST), GIST - Gastrointestinal Stromal Tumor
A Comprehensive, Multinational GIST Registry
Patients diagnosed with a gastrointestinal stromal tumor (GIST), a rare type of tumor in the digestive tractparticipate in this scientific research study, known as a registry study, to help improve understanding and treatment of…
- Trial name
- reGISTTry (NCT07273045)
- Where
- Germany
- Volunteers
- 1,000 people
- Sponsor
- Universität Duisburg-Essen
NF1, Congenital Pseudarthrosis of Tibia
A Study of INFUSE Bone Graft (BMP-2) in the Treatment of Tibial Pseudarthrosis in Neurofibromatosis Type 1 (NF1)
The current study proposes adding BMP-2 (INFUSE), an anabolic agent, at the surgical site of TPA (tibial pseudarthrosis) repair in children with NF1, compared to a control group of patients treated surgically without BMP-2.
- Trial name
- NF107-BMP2 (NCT02718131)
- Testing
- INFUSE Bone Graft (BMP-2), Control Group
- Where
- 16 sites in United States, Australia
- Volunteers
- 5 people
- Sponsor
- University of Alabama at Birmingham
Scoliosis; Idiopathic, Infantile, Scoliosis; Juvenile
Physical Activity, Motor Competence, Pulmonary Function, and Quality of Life in Children With Severe Spinal Disease
Observation study about physical activity, motor competence, pulmonary function, and health related quality of life in children, surgically treated for early onset scoliosis
- Conditions
- Scoliosis; Idiopathic, Infantile, Scoliosis; Juvenile, Scoliosis Neuromuscular, Scoliosis in Neurofibromatosis
- Trial name
- NCT03494829
- Where
- Norway
- Volunteers
- 50 people
- Sponsor
- Oslo University Hospital
Neurofibromatosis 1
Everolimus for Treatment of Disfiguring Cutaneous Lesions in Neurofibromatosis1 CRAD001CUS232T
This trial is evaluating the use of oral Everolimus to determine if there is a reduction in the size of the disfiguring cutaneous lesions in patients with Neurofibromatosis 1 over a 6 month period.
- Trial name
- DCLNF1 (NCT02332902)
- Testing
- Everolimus
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- The University of Texas Health Science Center, Houston
Cutaneous Neurofibroma
HIFU Treatment of Cutaneous Neurofibromas in Neurofibromatosis Type 1: Safety and Efficacy
The overall objective is to demonstrate safety and efficacy of HIFU treatment of cutaneous neurofibromas located close to the surface of the skin in patients with the genetic disease Neurofibromatosis Type 1.
- Trial name
- cNF-HFU2101 (NCT05119582)
- Testing
- TOOsonix System ONE-M
- Where
- 2 sites in Denmark, Sweden
- Volunteers
- 20 people
- Sponsor
- Joergen Serup
Neurofibromatosis 1 (NF1)
Efficacy of Mirdametinib Alone or Combination With Radiotherapy for Germline and Sporadic NF1-Altered High-Grade Glioma
This is a phase 2, open label, parallel multi-arm study of mirdametinib in combination with radiation in participants with recurrent sporadic glioblastoma (GBM) harboring NF1 alterations (Cohort 1); participants with NF1 with a…
- Trial name
- NF118 (NCT07521657)
- Testing
- mirdametinib with radiation, mirdametinib
- Where
- United States
- Volunteers
- 55 people
- Sponsor
- University of Alabama at Birmingham
Neurofibromatosis Type 1 (NF1), NF2-related Schwannomatosis
How Can the Experience of Hospital Outpatient Departments be Improved?
This project aims to improve outpatient departments by working with patients and staff to make these spaces more inclusive for people with sensory challenges (it focuses on people with nerve tumour conditions as they are more…
- Trial name
- NCT07818213
- Testing
- Qualitative data collection activities
- Where
- United Kingdom
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- King's College London