NeurofibromaCryoNF1NCT05199376
Evaluation of Percutaneous Cryotherapy in the Treatment of Plexiform Neurofibromas and Unresectable Neurofibromas in Neurofibromatosis Type 1
A study testing Cryotherapy in people with Neurofibroma and Neurofibroma, Plexiform, run by Centre Leon Berard at 1 site in France.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Neurofibroma, Neurofibroma, Plexiform
- What is being tested?
- Cryotherapy
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteFrance
- Study dates
- 14 Feb 2022to 7 Feb 2027
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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;
Who cannot join
- 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;
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- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Amine BOUHAMAMA, MD contact
France1 site, 1 recruiting
- Recruiting
Centre Leon Berard
Lyon, 69373
Patrick COMBEMALE, +3304 78 78 59 96, patrick.combemale@lyon.unicancer.fr
Patrick COMBEMALE
About this study
Official title: Evaluation of Percutaneous Cryotherapy in the Treatment of Plexiform Neurofibromas and Unresectable Neurofibromas in Neurofibromatosis Type 1 (NF1)
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.
Read more
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.
- Conditions
- Neurofibroma, Neurofibroma, Plexiform
- Being tested
- Cryotherapy
- Medical subject headings
- Neurofibroma, Neurofibroma, Plexiform
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CryotherapyDevice
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.
| Study group | Role | What they receive |
|---|---|---|
| Percutaneous cryotherapy | Experimental | Cryoablation of the tumor Receives: Device: Cryotherapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 12 months after cryotherapy
Secondary outcomes (9)
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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: From cryotherapy to 24 months after cryotherapy
To evaluate patients' satisfaction
Patient satisfaction with their first cryotherapy treatment assessed by a Likert scale
Measured: At Month 12 post-cryotherapy;
To evaluate patients self-esteem
Self-esteem assessed by a questionnaire consisting of items evaluated on a Likert scale
Measured: 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
Measured: 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.
Measured: At Day 0
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Jan 2022
- Study start14 Feb 2022
- Primary completion7 Feb 2026 (estimated)
- Study completion7 Feb 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for NeurofibromaStudies of the same condition, open and under way.
- More trials run by Centre Leon BerardThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Centre Leon Berard
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05199376
- Sponsor's protocol ID
- ET21-198
- Acronym
- CryoNF1
- Status last verified
- Feb 2024
- First submitted
- 21 Dec 2021
- Last updated
- 9 Feb 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of Percutaneous Cryotherapy in the Treatment of Plexiform Neurofibromas and Unresectable Neurofibromas in Neurofibromatosis Type 1 (NF1)
- ClinicalTrials.gov ID
- NCT05199376
- Acronym / sponsor ID
- CryoNF1 / ET21-198
- Conditions
- Neurofibroma, Neurofibroma, Plexiform
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Centre Leon BerardUniversity, hospital or other
- Study start
- 14 Feb 2022 (actual)
- Primary completion
- 7 Feb 2026 (estimated)
- Study completion
- 7 Feb 2027 (estimated)
- First posted
- 20 Jan 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Percutaneous cryotherapy | Experimental | Device: CryotherapyCryoablation of the tumor |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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);
Key exclusion criteria (12 listed)
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CryotherapyThe 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. | Device | Percutaneous cryotherapy |
Primary outcome
To evaluate the impact of percutaneous cryotherapy on physical health-related quality of life (in patients with inoperable (or operable with mutilating surgery) neurofibroma.
Time frame: 12 months after cryotherapy
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.
9 secondary outcomes
To assess each quality of life dimension of the Short Form-36 (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
Time frame: Month 3, Month 6, Month 9, Month 12, Month 18 , and Month 24 after cryotherapy
To evaluate the functional discomfort
Time frame: At baseline, Month 1; Month 3; Month 6, Month 9, Month12, Month18 and Month 24 post cryotherapy
To evaluate the patients' pain
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
Time frame: From cryotherapy to 24 months after cryotherapy
To evaluate patients' satisfaction
Time frame: At Month 12 post-cryotherapy;
To evaluate patients self-esteem
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;
Time frame: From cryotherapy to 24 months after cryotherapy
To evaluate operating room occupancy time.
Time frame: At Day 0
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Feb 2024.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Amine BOUHAMAMA, MD contact
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Cryotherapy in people with Neurofibroma and Neurofibroma, Plexiform, run by Centre Leon Berard at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 9 Feb 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Patrick COMBEMALE, MD (+33478785996, patrick.combemale@lyon.unicancer.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To evaluate the impact of percutaneous cryotherapy on physical health-related quality of life (in patients with inoperable (or operable with mutilating surgery) neurofibroma. (time frame: 12 months after cryotherapy). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Feb 2024.