Neurofibromatosis Clinical Trials

1 recruiting

Frequently Asked Questions

Common questions about Neurofibromatosis clinical trials

A clinical trial is a carefully designed research study that tests new medical treatments, drugs, devices, or approaches in human volunteers. Every approved medication and treatment available today was proven safe and effective through clinical trials.

All clinical trials are reviewed and approved by Institutional Review Boards (IRBs) — independent committees that evaluate patient safety. Trials follow strict protocols, and your health is monitored closely throughout. You can withdraw at any time.

Not necessarily. Many trials compare the new treatment against the current standard of care, meaning all participants receive active treatment. When placebos are used, they are typically combined with standard treatment, not given alone. The trial description will always specify the design.

Under the Affordable Care Act, most private insurers are required to cover routine patient care costs during a clinical trial. The sponsor typically covers the investigational treatment itself. Medicare also covers routine costs for qualifying trials.

Yes. Participation is completely voluntary. You can withdraw at any time, for any reason, without it affecting your access to standard medical care.

Each trial has specific eligibility criteria — including age, diagnosis, disease stage, prior treatments, and general health. Browse the trials listed above and check their eligibility sections. You can also contact the trial site directly to discuss your situation.

Showing 19 of 9 trials

Recruiting

Familial Investigations of Childhood Cancer Predisposition

Pancreatic CancerHereditary Breast and Ovarian CancerHodgkin Lymphoma+43 more
St. Jude Children's Research Hospital1,500 enrolled1 locationNCT03050268
Recruiting

Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders

Cystic FibrosisHuntington DiseaseSickle Cell Disease+12 more
Assistance Publique - Hôpitaux de Paris550 enrolled1 locationNCT06147414
Recruiting

Neurofibromatosis Type 1 Tumor Early Detection Study

Malignant Peripheral Nerve Sheath TumorNeurofibromatosis Type 1Neurofibromatosis 1+4 more
David Miller1,000 enrolled1 locationNCT06515860
Recruiting

RASopathy Biorepository

RAS MutationNeurofibromatosis 1Noonan Syndrome+11 more
Children's Hospital Medical Center, Cincinnati1,000 enrolled1 locationNCT04395495
Recruiting

Surveillance for Malignant Transformation of Neurofibromatosis Type 1 (NF1) Related Peripheral Nerve Sheath Tumors (PNST)

Neurofibromatosis 1Nerve Sheath Neoplasms
National Cancer Institute (NCI)225 enrolled1 locationNCT06222203
Recruiting
Phase 2

MT2021-08T Cell Receptor Alpha/Beta Depletion PBSC Transplantation for Heme Malignancies

Acute Lymphoblastic LeukemiaAcute Myeloid LeukemiaAcute Leukemia+19 more
Masonic Cancer Center, University of Minnesota70 enrolled1 locationNCT05735717
Recruiting
Phase 1Phase 2

A Phase I/II Study of Trametinib and Azacitidine for Patients With Newly Diagnosed Juvenile Myelomonocytic Leukemia

Neurofibromatosis 1Leukemia, Juvenile MyelomonocyticJMML+2 more
Therapeutic Advances in Childhood Leukemia Consortium58 enrolled4 locationsNCT05849662
Recruiting

Genetic Bases of Neuroendocrine Neoplasms in Mexican Patients

Neuroendocrine NeoplasmParagangliomaPheochromocytoma+23 more
Universidad Nacional Autonoma de Mexico750 enrolled3 locationsNCT06523582
Recruiting

GROWing Up With Rare GENEtic Syndromes

Cornelia De Lange SyndromeCongenital Adrenal HyperplasiaPrader-Willi Syndrome+30 more
dr. Laura C. G. de Graaff-Herder600 enrolled1 locationNCT04463316