Progressive Desmoids TumorsPhase 2DESMOPAZNCT01876082
PAZOPANIB Efficacy and Tolerance in Desmoids Tumors
A Phase 2 study testing PAZOPANIB treatment and Active Comparator: Vinblastine and Methotrexate in people with Progressive Desmoids Tumors, run by Institut Bergonié at 1 site in France.
Trial summary
Desmoids tumors are benign soft tissues tumors characterized by aggressiveness and potential local recurrence. There is a female predominance with a sex ratio of 2/1 and median age at diagnosis is about 30 years. Only a complete surgical excision is recommended in desmoids tumors. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Progressive Desmoids Tumors
- What is being tested?
- PAZOPANIB treatment and Active Comparator: Vinblastine and Methotrexate
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 72 participants
- Where
- 1 siteFrance
- Study dates
- 18 Sep 2012to Sep 2019
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- Written consent;
- Age ≥ 18 years;
- ECOG ≤ 1;
- Histologically confirmed desmoid tumor;
- Disease progression before the patient's inclusion : completion of two similar imaging obtained within 6 months apart (a tolerance of 6 weeks is accepted)
- Measurable target lesion (RECIST criteria) ;
Show all 22Show fewer
- Left ventricular ejection fraction (MUGA or ECHO) within the normal;
- Normal hematological, renal and liver functions :
- Hemoglobin ≥ 9 g / dl; neutrophils ≥ 1.5 x 109 / l; platelets ≥ 100 x 109 / l; prothrombin time or INR1 ≤ 1.2 ULN or activated partial thromboplastin time ≤ 1.2 ULN;
- Amino alanine transferase and aspartate amino transferase ≤ 2.5 ULN;
- Total bilirubin ≤ 1.5 ULN;
- Creatinine ≤ 1.5 mg/dl or, if creatinine> 1.5 mg/dl, Creatinine clearance ≥ 50 m/min;
- Urinary protein / urinary creatinine (Pu / Cu) <1. If PU/Cu ≥ 1, patients must have a proteinuria below 1g/24 h
- Women are eligible provided they:
- Physiologically incapable of childbearing (hysterectomy, oophorectomy, bilateral tubal ligation, menopause).
- Of childbearing age if they have had a negative pregnancy test in the week before the first dose of treatment.
- Women of childbearing potential and men must agree to take an adequate method of contraception. Permitted contraceptive methods : IUD with a documented failure rate of 1% per year, Partner's vasectomy, complete sex abstinence (for 14 days before inclusion, the test period and after cessation of treatment according to the chemotherapy as described below), dual contraception, oral contraceptives.
- Effective contraception must be implemented:
- Up to 6 months after treatment with vinblastine
- Up to 5 months after treatment with Methotrexate for men and up to 3 months after treatment with Methotrexate for women
- For the duration of treatment with Pazopanib;
- Affiliated to a social security system
Who cannot join
- Personal history of malignancy except:
- Cervical intraepithelial neoplasia;
- Skin basal cell carcinoma;
- Treated localized prostate carcinoma with PSA <1;
- Neoplasia treated with curative intent, in remission for at least five years and considered at low risk of relapse.
- Pretreatement by Pazopanib or Methotrexate - vinblastine;
Show all 46Show fewer
- Known allergy to Pazopanib, Methotrexate or vinblastine;
- Histological sampling not available for review or biological study;
- Clinical abnormalities which may increase the risk of gastric bleeding (not exhaustive list);
- Gastric tumor with known risk of bleeding;
- Inflammatory bowel disease or other gastrointestinal disease may increase the risk of gastric perforation.
- Pathologies that can lead to impaired intestinal absorption (not exhaustive):
- Malabsorption;
- Major resection of small intestine or stomach.
- Active uncontrolled infectious disease;
- Corrected QT interval> 480 ms;
- History of cardiovascular disease in the last 6 months:
- Cardiac angioplasty;
- Myocardial infarction;
- Unstable angina;
- Bypass surgery;
- Symptomatic arterial disease.
- Congestive heart failure grade II, III or IV according to the New York Hearth Association (NYHA) classification;
- Uncontrolled arterial hypertension (systolic blood pressure ≥ 140 mmHg or diastolic blood pressure ≥ 90 mmHg);
- History of stroke or transient ischemic attack, pulmonary embolism or deep vein thrombosis not treated, within 6 months;
- History of major surgery or trauma within 28 days prior the first day of treatment, or presence of a wound, fracture or non-healed ulcer;
- Evidence of active bleeding or bleeding tendency;
- Known endobronchial lesions and / or infiltrative lesions of the large vessels lung;
- History of hemoptysis of more than 2.5 ml in the eight weeks preceding the first day of chemotherapy;
- Pulmonary dysfunction, asthma, emphysema, chronic obstructive pulmonary bronchitis, pneumonia, pneumothorax, pulmonary contusion, hemothorax, distress acute respiratory syndrome, pulmonary fibrosis;
- Severe renal dysfunction;
- Severe hepatic dysfunction;
- History of psoriasis, rheumatoid arthritis, alcoholism, illness or chronic liver dysfunction;
- Any pre-existing severe or unstable medical or psychiatric condition, or other condition that may interfere with patient safety, the collection of its informed consent or adherence to treatment;
- Patient who refused or could not stop taking banned drugs for at least 14 days (or 5 half-lives of the drug) before the first day of start of chemotherapy and for the duration of the study;
- During cancer treatment:
- Radiotherapy, surgery or tumor embolization within 14 days before the 1st dose of pazobanib (arm A) or chemotherapy methotrexate, vinblastine (arm B);
- Chemotherapy, immunotherapy, biological treatment, experimental or hormone therapy within 14 days or 5 half-lives of medication before the first day of the pazopanib (arm A) or chemotherapy with methotrexate, vinblastine (arm B).
- History of cancer treatment toxicity> grade 1 and / or whose the intensity increases, outside of alopecia.
- Pregnancy and lactation
- Concomitant treatment which can't be interrupted or replaced and which is not indicated with methotrexate:
- Probenecid (alone or associated with sulfamethoxazole),
- Trimethoprim,
- Acetylsalicylic acid (for methotrexate doses above 20 mg per week with the acetylsalicylic acid and used at doses analgesics or antipyretics (≥ 500 mg per dose and/or <3 g per day)or anti-inflammatory (≥ 1 g per dose and / or > 3 g per day),
- Phenylbutazone,
- Yellow fever vaccine.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: PAZOPANIB Efficacy and Tolerance in Desmoids Tumors : Phase 2 Clinical Trial
Desmoids tumors are benign soft tissues tumors characterized by aggressiveness and potential local recurrence. There is a female predominance with a sex ratio of 2/1 and median age at diagnosis is about 30 years.
Only a complete surgical excision is recommended in desmoids tumors. Some forms of desmoid tumors are recurrent and/or symptomatic and are not accessible to a conservative surgical treatment. In these clinical situations, only a medical treatment may achieve tumor control and quality of life maintenance. Place of systemic treatments in the management of desmoids tumors is poorly evaluated. Regarding chemotherapy, methotrexate and vinblastine protocol is actually the best evaluated combination, which allowed observing objective response rate between 40 and 75%. Toxicity was mainly marked by the risk of haematological toxicity.
Pazopanib is an inhibitor of multi-target tyrosine kinase, in oral form, with selective type receptors -1, -2 and -3 of VEGF receptors on the PDGFA and B, and c-Kit. It is currently under clinical development in humans in the treatment of several tumor types.
Read the detailed description
This is a Phase II, randomized, multicenter, open label trial, evaluating efficacy and safety of pazopanib versus a chemotherapy protocol combining methotrexate and vinblastine in progressive and symptomatic desmoid tumors.
This study will include 72 patients in 15 centers of the French Sarcoma Group.
Patients will be treated according to therapeutic strategy allocated by randomization until documented RECIST progression and for a maximum of 12 months :
- Arm A = experimental strategy: daily oral administration of pazopanib.
- Arm B = reference strategy: methotrexate-vinblastine.
In case of documented radiological progression (RECIST criteria):
- Patients initially included in arm A will have the opportunity, as determined by the investigator, to receive arm B treatment, or leave the study,
- Patients initially included in arm B will have the opportunity, as determined by the investigator, to receive arm A treatment, or leave the study.
- Conditions
- Progressive Desmoids Tumors
- Being tested
- PAZOPANIB treatment, Active Comparator: Vinblastine and Methotrexate
- Keywords
- Desmoids tumors, Pazopanib, Phase 2 clinical trial, Cross over
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PAZOPANIB treatmentDrug
Pazopanib
- 800 mg per day
- oral administration
- at least 1 hour before or 2 hours after a meal,
- until disease progression or for 12 months maximum
Active Comparator: Vinblastine and MethotrexateDrug
Active Comparator: Vinblastine and Methotrexate vinblastine 5 mg / m², methotrexate 30 mg / m (J1, J8, J15, J21, 6 months and then J1, J15) 28 days per cycle until disease progression or for 12 months.
| Study group | Role | What they receive |
|---|---|---|
| PAZOPANIB | Experimental | Pazopanib
* 800 mg per day
* oral administration
* at least 1 hour before or 2 hours after a meal,
* until disease progression or for 12 months maximum Receives: Drug: PAZOPANIB treatment |
| Vinblastine and Methotrexate | Active comparator | vinblastine 5 mg / m², methotrexate 30 mg / m (J1, J8, J15, J21, 6 months and then J1, J15) 28 days per cycle until disease progression or for 12 months. Receives: Drug: Active Comparator: Vinblastine and Methotrexate |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Patients Remaining Alive and Progression-free at 6 Months as Per RECIST 1.1 After the Day of Randomisation (6-month Non-progression Rate).
Percentage of patients remaining alive and progression-free at 6 months as per RECIST 1.1 after the day of randomisation.
Progression is defined using New Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1), as a 20% increase in the sum of diameters of target lesions (taking as reference the smallest sum on study), or a unequivocal progression of existing non-target lesions, or the appearance of one or more new lesions.
Measured: 6 months
Secondary outcomes (3)
Percentage of Patients Remaining Alive With Best Overall Response as Per RECIST v1.1.
Best overall response is defined as the best response across all time points (RECIST v1.1). The best overall response is determined once all the data for the participant is known.
Each patient has been assigned one of the following categories (RECIST 1.1): complete response (disappearance of all target lesions); partial response (>=30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters); progression (20% increase in the sum of diameters of target lesions (taking as reference the smallest sum on study) and stable disease (nor CR, PR or progression).
Measured: 1 year
Progression-free Survival
Progression-free survival (PFS) defined as the time from randomization to the first documented progression (as per RECIST v1.1) or death (due to any cause), whichever occurs first. Patients alive and progression free were censored at the date of last follow-up, death, or last patient contact. Progression is assessed as per RECIST v1.1. Progression-free survival is estimated as a function of time using Kaplan-Meier method.
1- and 2-year PFS rates were reported.
Measured: Randomization to disease progression, or death due to any cause, whichever occurs first; until 2 years after the last patient randomized.
Overall Survival
Overall survival (OS) defined as the time from randomization to death (due to any cause). Patients alive were censored at the date of last follow-up or last patient contact. Overall survival was estimated as a function of time using Kaplan-Meier method.1- and 2-Year OS rates were reported.
Measured: Randomization to disease progression, or death due to any cause, whichever occurs first; until 2 years after the last patient randomized.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jun 2013 (estimated)
- Study start18 Sep 2012
- Primary completionMar 2018
- Study completionSep 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Progressive Desmoids TumorsStudies of the same condition, open and under way.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Enrolment
- 72 participants (actual)
Sponsor and investigators
- Lead sponsor
- Institut Bergonié
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- ITALIANO Antoine, MD, Study chair, Institut Bergonié
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01876082
- Sponsor's protocol ID
- IB2011-03
- Acronym
- DESMOPAZ
- Status last verified
- Feb 2021
- First submitted
- 6 Nov 2012
- Last updated
- 4 Sep 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Toulmonde M, Pulido M, Ray-Coquard I, Andre T, Isambert N, Chevreau C, Penel N, Bompas E, Saada E, Bertucci F, Lebbe C, Le Cesne A, Soulie P, Piperno-Neumann S, Sweet S, Cecchi F, Hembrough T, Bellera C, Kind M, Crombe A, Lucchesi C, Le Loarer F, Blay JY, Italiano A. Pazopanib or methotrexate-vinblastine combination chemotherapy in adult patients with progressive desmoid tumours (DESMOPAZ): a non-comparative, randomised, open-label, multicentre, phase 2 study. Lancet Oncol. 2019 Sep;20(9):1263-1272. doi: 10.1016/S1470-2045(19)30276-1. Epub 2019 Jul 19.
PubMed 31331699
Study at a glance
From the registry record, last updated by the sponsor on 4 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- PAZOPANIB Efficacy and Tolerance in Desmoids Tumors : Phase 2 Clinical Trial
- ClinicalTrials.gov ID
- NCT01876082
- Acronym / sponsor ID
- DESMOPAZ / IB2011-03
- Conditions
- Progressive Desmoids Tumors
- Keywords
- Desmoids tumors, Pazopanib, Phase 2 clinical trial, Cross over
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 72 participants (actual)
- Lead sponsor
- Institut BergoniéUniversity, hospital or other
- Study start
- 18 Sep 2012 (actual)
- Primary completion
- Mar 2018 (actual)
- Study completion
- Sep 2019 (actual)
- First posted
- 12 Jun 2013 (estimated)
- Results
- Posted 5 Mar 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Desmoids tumors are benign soft tissues tumors characterized by aggressiveness and potential local recurrence. There is a female predominance with a sex ratio of 2/1 and median age at diagnosis is about 30 years.
Only a complete surgical excision is recommended in desmoids tumors. Some forms of desmoid tumors are recurrent and/or symptomatic and are not accessible to a conservative surgical treatment. In these clinical situations, only a medical treatment may achieve tumor control and quality of life maintenance. Place of systemic treatments in the management of desmoids tumors is poorly evaluated. Regarding chemotherapy, methotrexate and vinblastine protocol is actually the best evaluated combination, which allowed observing objective response rate between 40 and 75%. Toxicity was mainly marked by the risk of haematological toxicity.
Pazopanib is an inhibitor of multi-target tyrosine kinase, in oral form, with selective type receptors -1, -2 and -3 of VEGF receptors on the PDGFA and B, and c-Kit. It is currently under clinical development in humans in the treatment of several tumor types.
Detailed description
This is a Phase II, randomized, multicenter, open label trial, evaluating efficacy and safety of pazopanib versus a chemotherapy protocol combining methotrexate and vinblastine in progressive and symptomatic desmoid tumors.
This study will include 72 patients in 15 centers of the French Sarcoma Group.
Patients will be treated according to therapeutic strategy allocated by randomization until documented RECIST progression and for a maximum of 12 months :
- Arm A = experimental strategy: daily oral administration of pazopanib.
- Arm B = reference strategy: methotrexate-vinblastine.
In case of documented radiological progression (RECIST criteria):
- Patients initially included in arm A will have the opportunity, as determined by the investigator, to receive arm B treatment, or leave the study,
- Patients initially included in arm B will have the opportunity, as determined by the investigator, to receive arm A treatment, or leave the study.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| PAZOPANIB | Experimental | Drug: PAZOPANIB treatmentPazopanib * 800 mg per day * oral administration * at least 1 hour before or 2 hours after a meal, * until disease progression or for 12 months maximum |
| Vinblastine and Methotrexate | Active comparator | Drug: Active Comparator: Vinblastine and Methotrexatevinblastine 5 mg / m², methotrexate 30 mg / m (J1, J8, J15, J21, 6 months and then J1, J15) 28 days per cycle until disease progression or for 12 months. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (22 listed)
- Written consent;
- Age ≥ 18 years;
- ECOG ≤ 1;
- Histologically confirmed desmoid tumor;
- Disease progression before the patient's inclusion : completion of two similar imaging obtained within 6 months apart (a tolerance of 6 weeks is accepted)
Key exclusion criteria (46 listed)
- Personal history of malignancy except:
- Pretreatement by Pazopanib or Methotrexate - vinblastine;
- Known allergy to Pazopanib, Methotrexate or vinblastine;
- Histological sampling not available for review or biological study;
- Clinical abnormalities which may increase the risk of gastric bleeding (not exhaustive list);
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 |
|---|---|---|
| PAZOPANIB treatmentPazopanib * 800 mg per day * oral administration * at least 1 hour before or 2 hours after a meal, * until disease progression or for 12 months maximum | Drug | PAZOPANIB |
| Active Comparator: Vinblastine and MethotrexateActive Comparator: Vinblastine and Methotrexate vinblastine 5 mg / m², methotrexate 30 mg / m (J1, J8, J15, J21, 6 months and then J1, J15) 28 days per cycle until disease progression or for 12 months. | Drug | Vinblastine and Methotrexate |
Primary outcome
Percentage of Patients Remaining Alive and Progression-free at 6 Months as Per RECIST 1.1 After the Day of Randomisation (6-month Non-progression Rate).
Time frame: 6 months
Percentage of patients remaining alive and progression-free at 6 months as per RECIST 1.1 after the day of randomisation. Progression is defined using New Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1), as a 20% increase in the sum of diameters of target lesions (taking as reference the smallest sum on study), or a unequivocal progression of existing non-target lesions, or the appearance of one or more new lesions.
3 secondary outcomes
Percentage of Patients Remaining Alive With Best Overall Response as Per RECIST v1.1.
Time frame: 1 year
Progression-free Survival
Time frame: Randomization to disease progression, or death due to any cause, whichever occurs first; until 2 years after the last patient randomized.
Overall Survival
Time frame: Randomization to disease progression, or death due to any cause, whichever occurs first; until 2 years after the last patient randomized.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Sep 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 31331699Toulmonde M, Pulido M, Ray-Coquard I, Andre T, Isambert N, Chevreau C, Penel N, Bompas E, Saada E, Bertucci F, Lebbe C, Le Cesne A, Soulie P, Piperno-Neumann S, Sweet S, Cecchi F, Hembrough T, Bellera C, Kind M, Crombe A, Lucchesi C, Le Loarer F, Blay JY, Italiano A. Pazopanib or methotrexate-vinblastine combination chemotherapy in adult patients with progressive desmoid tumours (DESMOPAZ): a non-comparative, randomised, open-label, multicentre, phase 2 study. Lancet Oncol. 2019 Sep;20(9):1263-1272. doi: 10.1016/S1470-2045(19)30276-1. Epub 2019 Jul 19.
Related trials for Progressive Desmoids Tumors
Active, not recruitingPhase 2
A Study of a New Drug, Nirogacestat, for Treating Desmoid Tumors That Cannot be Removed by Surgery
Desmoid Fibromatosis, Recurrent Desmoid Fibromatosis
Active, not recruitingPhase 2
A Study of Nirogacestat in Japanese Adults With Desmoid Tumors/Aggressive Fibromatosis (DT/AF)
Desmoid Tumor, Aggressive Fibromatosis
Active, not recruitingPhase 2/3
A Study of AL102 in Patients With Progressing Desmoid Tumors
Desmoid, Desmoid Tumor
Recruiting
Cryoablation Versus Medical Therapy in Desmoid Tumors Progressing After Watchful Waiting
Desmoid Tumor
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. This study is not recruiting at the moment.
ITALIANO Antoine, MD study chair
Institut Bergonié
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing PAZOPANIB treatment and Active Comparator: Vinblastine and Methotrexate in people with Progressive Desmoids Tumors, run by Institut Bergonié 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 22 inclusion and 46 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: finished, as of the sponsor's last update on 4 Sep 2025. 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 5 Mar 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of Patients Remaining Alive and Progression-free at 6 Months as Per RECIST 1.1 After the Day of Randomisation (6-month Non-progression Rate). (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01876082, which also lists 1 linked publication.
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 4 Sep 2025.