CRYODESMO-02: Cryoablation Versus Medical Therapy in Desmoid Tumors Progressing After Watchful Waiting

• Sponsor: University Hospital, Strasbourg, France (Other)
• Overall Status: Not yet recruiting
• CT.gov ID: NCT06081400
• Collaborator: Direction Générale de l'Offre des Soins (DGOS) (Other)
• Enrollment: 150
• Locations: 13
• Arms: 2
• Anticipated Duration (Months): 63
• Patients Per Site: 11.5
• Patients Per Site Per Month: 0.2

Study Details

Study Description

Brief Summary

"Wait & see" is currently the standard of care of recently diagnosed desmoid tumors (DT). In case of progression or symptomatic disease, medical therapy is nowadays widely used including chemotherapy.

Cryoablation has proven to be beneficial for the treatment of large, progressive and symptomatic DT.

This randomized phase II trial aims to compare cryoablation versus medical therapy in DT patients progressing after the "wait & see" period. Moreover, a cross-over design has been anticipated to allow all patients to undergo cryoablation if necessary.

Condition or Disease	Intervention/Treatment	Phase
Desmoid Tumor	Procedure: Cryoablation; Drug: Chemotherapy drug	N/A

Study Design

• Study Type: Interventional
• Anticipated Enrollment: 150 participants
• Allocation: Randomized
• Intervention Model: Crossover Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: A Phase II Multicentric, Randomized Trial Assessing Cryoablation Versus Medical Therapy in Desmoid Tumors Progressing After Watchful Waiting
• Anticipated Study Start Date: Nov 1, 2023
• Anticipated Primary Completion Date: Nov 1, 2026
• Anticipated Study Completion Date: Feb 1, 2029

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Cryoablation One single cryoablation of the desmoid tumor at Day 0	Procedure: Cryoablation; Percutaneous imaging-guided cryoablation
Active Comparator: Medical therapy Chemotherapy: at the investigator's discretion: either methotrexate 30mg/m² + vinblastine 6mg/m² (IV infusion) once/week for 6 months, then every other week from months 7 to 12, or vinorelbine 90mg/week (per os: 3x30mg, soft capsules) for 12 months.	Drug: Chemotherapy drug; Either : methotrexate 30mg/m² + vinblastine 6mg/m² (IV infusion) once/week for 6 months, then every other week from months 7 to 12, or vinorelbine 90mg/week (per os: 3x30mg, soft capsules) for 12 months.

Outcome Measures

Primary Outcome Measures

1. Rate of non-progressive disease [12 months after treatment initiation (Day 0+12months)]

   Rates of non-progressive disease (sum of complete response (CR), partial response (PR), and stable disease (SD) according to modified RECIST criteria)

Secondary Outcome Measures

1. Progression-free survival 1 [from treatment initiation to PD or 24 months whichever comes first]

   Progression-free survival 1 (PFS1: from the beginning of cryoablation or medical therapy to first disease progression) in cryoablation and medical therapy group

2. Rate of patients who cross over to the other arm [24 months after treatment initiation]

3. Rate of complete response [12 months after treatment initiation and 12 months after cross-over]

   Rate of complete response at 12 months in cryoablation and medical therapy group with and without a cross-over treatment

4. Secondary Progression-free survival 2 [Through study completion, up to 39 months]

   Secondary Progression-free survival 2 (PFS 2: from the cross-over to second disease progression) in cryoablation and medical therapy group

5. Incidence of Treatment-Emergent Adverse Events as assessed by NCI-CTCAE version 5.0 toxicity scale [Through study completion, up to 39 months]

   The occurrence of complications and adverse reactions according to the classification of the NCI-CTCAE version 5.0 toxicity scale

6. QUALITY OF LIFE assessed by EUROQOL EQ 5D before and after treatment [Screening visit, Month 1, Month 2, Month 3, Month 6, Month 9 and Month 12 after treatment initiation]

   EUROQOL EQ 5D will be used; score ranging from 1 (good quality of life) to 5 (poor quality of life)

7. PAIN assessed by brief pain inventory (BPI) before and after treatment [Screening visit, Day 0/Day 1, Month 1, Month 2, Month 3, Month 6, Month 9 and Month 12 after treatment initiation]

   BPI will be used; score ranging from 0 (no pain) to 10 (high pain)

8. Health economics assessment [Through study completion, up to 39 months]

   cost and incremental cost utility ratio

Eligibility Criteria

Criteria

• Ages Eligible for Study: 13 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Subject (male or female) with extra-peritoneal desmoid tumor (confirmed by prior biopsy by an experienced pathologist within the RRePS network)

• 13 years of age or older

• Measurable viable tumor (according to the mRECIST criteria using MRI. The baseline MRI imaging is mandatory in the 2 months prior to treatment initiation (D0).

• Progressive disease (according to the mRECIST criteria) after the watchful waiting period or significant increase in symptoms requiring an active therapy, as advised in a multidisciplinary sarcoma tumor board

• Tumor deemed accessible for cryoablation procedure by the operator in a type I center. (In pediatric cases, a careful site tumor analysis will be performed with interventional radiologist to ensure for limited consequence of cryoablation in pediatric patients especially regarding growth plates).

• 100 % of destruction of the tumor achievable in one procedure of cryoablation with 1cm security margin according to assessment by referral center (type 1) for cryoablation

• ECOG performance status 0-2 at inclusion visit

• Biological and hematological parameters (neutrophils ≥ 1,5.109/L ; platelet count ≥ 100.109/L ; no significant hemostatic abnormalities) in the 4 weeks prior to treatment initiation (D0)

• Subject able to understand the objectives and risks of the research and to give dated and signed informed consent. For minors, the consent of the 2 parents must be obtained.

• Subject affiliated to a social health insurance plan

• For a woman of childbearing age: negative blood pregnancy test at screening/inclusion visit

• Subject agreeing to use a contraceptive method:

Exclusion criteria:

• Intra-peritoneal desmoid tumor

• Known hypersensitivity to vinorelbine or other vinca-alkaloids, or other constituents to navelbine, to vinblastine, to methotrexate or any excipients, current or recent (within 2 weeks) severe infection, severe renal failure, severe hepatic injury, chronic respiratory failure, concomitant treatment with: yellow fever vaccine, prophylactic treatment with phenytoin, trimethoprim, probenecid, acetylsalicylic acid, phenylbutazone.

• Any contra-indication for the procedure as stated by the interventional radiologist in terms of tumor size, proximity to neural/vascular structures or adjacent organs at risk making the procedure at unacceptable risk

• Impaired hemostasis, that may interfere with the conduct of the cryoablation

• Concurrent participation in other experimental studies that could affect endpoints of the present study

• Concurrent use of any antitumor agent or NSAIDs, penicillins, PPIs, acitretin, ciprofloxacin, azote protoxide

• Contraindication to any form of sedation

• Hypersensitivity to gadoteric acid, meglumine or any drug containing gadolinium

• Others contra-indications to MRI

• Pregnancy or breastfeeding

• Impossibility to give the subject informed information (subject in an emergency situation, patient with comprehension difficulties ...)

• Psychiatric disorders

• Incompetent subject (subject to a legal protection measure: curatorship, guardianship, future protection mandate, family habilitation)

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Service d'Oncologie Médicale, Service de Radiologie Interventionnelle, Institut BERGONNIE	Bordeaux		France	33000
2	Service d'Oncologie Médicale, Service de Radiologie Interventionnelle-Centre François BACLESSE	Caen		France	14076
3	Service d'Oncologie Médicale -Centre Georges François LECLERC	Dijon		France	21079
4	Service d'Oncologie Médicale, Service de Radiologie Interventionnelle-Centre Léon BERARD	Lyon		France	69373
5	Service d'Oncologie Médicale, service de Radiologie Interventionnelle,CHU de Marseille, Hopital La Timone	Marseille		France	13385
6	Service de Radiologie-CHU de Nantes	Nantes		France	44000
7	Service d'Oncologie Médicale-Centre Antoine LACASSAGNE	Nice		France	06189
8	Service d'Oncologie Médicale-Centre Henri BECQUEREL	Rouen		France	76038
9	Service d'Oncologie Médicale	Saint-Herblain		France	44805
10	Institut de cancérologie Strasbourg Europe (ICANS)	Strasbourg		France	67033
11	Service d'Imagerie Interventionnelle, Hôpitaux Universitaires de Strasbourg , France, 67091	Strasbourg		France	67091
12	Service d'Oncologie Médicale-Institut Claudius Régaud et Service de Radiologie Interventionnelle, CHU de Toulouse	Toulouse		France	31059
13	Service d'Oncologie médicale, Service de Radiologie Interventionnelle- Institut Gustave ROUSSY	Villejuif		France	94800

Sponsors and Collaborators

• University Hospital, Strasbourg, France
• Direction Générale de l'Offre des Soins (DGOS)

Investigators

• Principal Investigator: Afshin GANGI, MD, PhD, Hôpitaux Universitaires de Strasbourg

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: University Hospital, Strasbourg, France
• ClinicalTrials.gov Identifier: NCT06081400
• Other Study ID Numbers: 7361
• First Posted: Oct 13, 2023
• Last Update Posted: Oct 13, 2023
• Last Verified: Aug 1, 2023

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No
• Keywords provided by University Hospital, Strasbourg, France: Desmoids; Cryoablation; Drug therapy; Cross-Over Studies
• Additional relevant MeSH terms: Fibromatosis, Aggressive

Study Results

No Results Posted as of Oct 13, 2023