PAZOPANIB Efficacy and Tolerance in Desmoids Tumors (DESMOPAZ)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT01876082|
Recruitment Status : Completed
First Posted : June 12, 2013
Results First Posted : March 5, 2021
Last Update Posted : March 5, 2021
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.
|Condition or disease||Intervention/treatment||Phase|
|Progressive Desmoids Tumors||Drug: PAZOPANIB treatment Drug: Active Comparator: Vinblastine and Methotrexate||Phase 2|
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 Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||72 participants|
|Intervention Model:||Crossover Assignment|
|Masking:||None (Open Label)|
|Official Title:||PAZOPANIB Efficacy and Tolerance in Desmoids Tumors : Phase 2 Clinical Trial|
|Actual Study Start Date :||September 18, 2012|
|Actual Primary Completion Date :||March 2018|
|Actual Study Completion Date :||September 2019|
Drug: PAZOPANIB treatment
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.
Drug: Active Comparator: Vinblastine and Methotrexate
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.
- 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.
- Percentage of Patients Remaining Alive With Best Overall Response as Per RECIST v1.1. [ Time Frame: 1 year ]
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).
- 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. ]
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.
- 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. ]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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01876082
|Bordeaux, Aquitaine, France, 33000|
|Study Chair:||ITALIANO Antoine, MD||Institut Bergonié|