Using cryotherapy to treat neurofibromas in patients with Neurofibromatosis Type 1

Evaluation of Percutaneous Cryotherapy in the Treatment of Plexiform Neurofibromas and Unresectable Neurofibromas in Neurofibromatosis Type 1 (NF1)

This study is testing if using extreme cold to treat tough tumors can help people with Neurofibromatosis Type 1 feel better without needing surgery.

Quick facts

• Phase: Not applicable
• Study type: Interventional
• Enrollment: 30 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Centre Leon Berard (Academic / other)
• Drugs / interventions: chemotherapy
• Locations: 1 site (Lyon)
• Trial ID: NCT05199376 on ClinicalTrials.gov

What this trial studies

This study evaluates the effectiveness of percutaneous cryotherapy as a treatment for plexiform and unresectable neurofibromas in patients with Neurofibromatosis Type 1 (NF1). The procedure involves using fine needles to apply extreme cold to the tumors, potentially reducing pain and discomfort without the need for invasive surgery. Previous unpublished results from the Léon Bérard Center suggest that cryotherapy may offer a promising alternative to traditional surgical methods. The study aims to gather more data on this approach and its outcomes for patients suffering from NF1-related neurofibromas.

Who should consider this trial

Good fit: Ideal candidates for this study are adults aged 18 and older with Neurofibromatosis Type 1 and painful, unresectable neurofibromas.

Not a fit: Patients with malignant neurofibromas or those currently undergoing chemotherapy or targeted therapies may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a less invasive option for managing painful neurofibromas, improving quality of life for patients with NF1.

How similar studies have performed: While the use of cryotherapy for neurofibromas is not widely established, encouraging unpublished results have been observed in prior cases at the Léon Bérard Center.

Eligibility criteria

Inclusion Criteria:

* Age ≥18 years;
* Patient with neurofibromatosis type 1 according to NIH criteria ;
* Patient with a benign neurofibromatous lesion that is painful and/or generates functional discomfort and is unresectable or with unacceptable scarring ;
* Neutrophils \> 1 G/l in the 14 days prior to inclusion;
* Adequate coagulation test with normals values (as judged by the investigator);
* Signed and dated informed consent document indicating that the patient has been informed of all aspects of the trial prior to enrolment
* Covered by a medical insurance;

Exclusion Criteria:

* Patient treated with concomitant chemotherapy and/or targeted therapies ;
* Any contraindication to a percutaneous cryotherapy procedure, including the need for ice formation within 1 cm of the spinal cord, brain or other critical nerve structures, bowel or bladder (unless active or passive thermal protective maneuvers are performed);
* Patient with malignant neurofibroma or MPNST ; CT scan without abnormality and if tumor presents a max SUV T/F\>1.5 ratio, targeted biopsy is required to confirm benign or malignant histology.
* Patient with neurofibroma in areas at risk for neurological sequelae;
* Patient with cold urticaria with history of angioedema;
* Any cognitive impairment or condition that may limit the use of numerical scales and quality of life questionnaires;
* Patient for whom follow-up does not seem feasible even in the short term;
* Participation in another clinical trial that may interfere with the evaluation of the primary endpoint;
* Patient under tutorshio, curatorship or deprived of liberty;
* Pregnant or breast-feeding woman;
* Any contraindication to the performance of an MRI
* Patient with dysplastic neurofibroma

Where this trial is running

Lyon

• Centre Leon Berard — Lyon, France (Recruiting)

Study contacts

• Study coordinator: Patrick COMBEMALE, MD

Conditions Neurofibroma; Neurofibroma, Plexiform