An interventional study of Cryotherapy in Neurofibroma and Neurofibroma, Plexiform, sponsored by Centre Leon Berard. Recruiting at 1 site in France. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2024-02-09.
Sponsored by Centre Leon Berard · Not applicable, Interventional, and Treatment
Neurofibromatosis type 1 (NF1) is an autosomal dominant disease affecting chromosome 17. It is manifested by a neurogenic tumor proliferation that forms cutaneous, subcutaneous or deep neurofibromas.
Neurofibromas can cause functional discomfort, neurogenic pain that is difficult to treat, and severe cosmetic disorders.
Treatment is essentially surgical. It is sometimes a heavy invasive surgery with complicated postoperative follow-up and significant scarring on the aesthetic level. Currently, no systemic treatment has proven its effectiveness in this pathology.
Percutaneous cryotherapy is a cold thermoablation procedure using fine 17 G needles introduced into the lesion after targeting by imaging. This technique is used in the treatment of soft tissue tumors and desmoid tumors.
The treatment of neurofibromas with percutaneous cryotherapy is not well known. Encouraging results (unpublished) have been observed in patients with NF1 treated with cryotherapy at the Léon Bérard Center. The beneficial effect was observed in terms of quality of life (in particular, pain) as well as a decrease in tumor size.
On the basis of this first experience, it appears important to corroborate these preliminary results by a prospective study allowing the use of this technique to treat patients with unresectable or resectable neurofibromas but with mutilating surgery in a NF1 context.
63 studies on the registry are indexed under Neurofibroma, Plexiform; 19 are open to participants now.
This study's planned enrollment of 30 is below the median of 36 across 43 interventional studies indexed under Neurofibroma, Plexiform.
Browse Neurofibroma, Plexiform studies →Centre Leon Berard is the lead sponsor of 210 studies on the registry; 62 are open to participants now.
Compared within Neurofibroma, Plexiform, this study's primary condition as ClinicalTrials.gg classifies it; the registry's first condition term is Neurofibroma. The counts are of registry records indexed under each term, refreshed daily. How we classify →
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Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cryoablation of the tumor
Device: Cryotherapy
The cryotherapy treatment lasts approximately 30 minutes during which two 10-minute freezing cycles are performed, separated by a reheating phase. During the freezing phases, the temperature is lowered below -40°C in the ablation zone, a temperature that is lethal for the cells.
To evaluate the impact of percutaneous cryotherapy on physical health-related quality of life (in patients with inoperable (or operable with mutilating surgery) neurofibroma.
Rate of patients with clinically significant improvement in physical health-related quality of life. A patient will be considered successful if an increase of at least 3 points on the global physical score from the SF-36 questionnaire is observed at least once between baseline and 12 months after cryotherapy.
Time frame: 12 months after cryotherapy
To assess each quality of life dimension of the Short Form-36 (SF-36) questionnaire;
The score for each dimension, the mental global score and the physical global score from the SF-36 questionnaire
Time frame: At baseline, at Month1 post cryotherapy ; Month 3 post cryotherapy; Month 6 post cryotherapy, Month 9 post cryotherapy, Month 12 post cryotherapy, Month 18 post cryotherapy and Month 24 post post cryotherapy ;
To evaluate the tumor response
Local control rate using REiNS and RECIST 1.1 criteria
Time frame: Month 3, Month 6, Month 9, Month 12, Month 18 , and Month 24 after cryotherapy
To evaluate the functional discomfort
Functional discomfort assessed by a Likert scale
Time frame: At baseline, Month 1; Month 3; Month 6, Month 9, Month12, Month18 and Month 24 post cryotherapy
To evaluate the patients' pain
Pain assessed by the Brief Pain Inventory-Short Form (BPI-SF) questionnaire
Time frame: At baseline, Month 1; Month 3; Month 6, Month 9, Month 12, Month 18 and Month 24 post cryotherapy
To evaluate the safety of percutaneous cryotherapy
Tolerance of cryotherapy treatment assessed according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) v5.0
Time frame: From cryotherapy to 24 months after cryotherapy
To evaluate patients' satisfaction
Patient satisfaction with their first cryotherapy treatment assessed by a Likert scale
Time frame: At Month 12 post-cryotherapy;
To evaluate patients self-esteem
Self-esteem assessed by a questionnaire consisting of items evaluated on a Likert scale
Time frame: at baseline, Month 1; Month 3; Month 6, Month 9, Month12, Month 18 and Month 24 post-cryotherapy
To evaluate the need for multiple percutaneous cryotherapy procedures in case of incomplete response on the target lesion;
Rate of patients for whom at least one additional percutaneous cryotherapy procedure is required
Time frame: From cryotherapy to 24 months after cryotherapy
To evaluate operating room occupancy time.
Median operating room occupancy time calculated as the difference between operating room exit time and operating room entry time.
Time frame: At Day 0
Plan to share: No
No publications or documents are linked to this record.
Started Feb 2022 (actual). Primary completion is estimated for Feb 2026, 47 months after the start.
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