National Clinical-biological Prospective Cohort of Incident Cases of Aggressive Fibromatosis (ALTITUDES) (ALTITUDES)
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|ClinicalTrials.gov Identifier: NCT02867033|
Recruitment Status : Active, not recruiting
First Posted : August 15, 2016
Last Update Posted : November 25, 2020
|Condition or disease||Intervention/treatment||Phase|
|Aggressive Fibromatosis||Procedure: biopsy Other: biobank constitution Procedure: Coloscopy Procedure: Blood sampling (facultative) Other: Pain evaluation Procedure: Tumor biobank realization||Not Applicable|
Aggressive fibromatosis (AF) is a rare non-metastasizing connective tissue tumor (< 300 cases/year in France), associated with high risk of local relapse, functional impairment and pain. AF can occur at any age, but most commonly between 25 and 40 with a significant female predominance. AF is most frequently (about 85%) sporadic and then associated with a somatic mutation of the CTNNB1 gene. AF is associated with heredity condition, as complication of familial adenomatous polyposis (with germinal mutation of Adenomatous polyposis coli (APC) gene). Most of AF arises on lims or abdominal wall. Nevertheless, some particular locations are life-threatening (mesenteric or cervical locations). The natural course of AF is unpredictable. One third of tumors are spontaneously stable. One third of tumor spontaneously decreases. One third of tumor is progressive, with a non-linear tumor growth dynamic. As the consequence the decision making for starting curative intent treatment is difficult, since some treatment could be mutilating (large en bloc surgery) or associated with late and severe complications (radiotherapy) and since these treatments could fail to control this benign tumor. Therapeutic options are: wait-and-see policy, surgery (sometimes mutilating), radiotherapy or systemic treatment (non-steroidal anti-inflammatory drugs, hormonotherapy, imatinib, chemotherapy). Level of evidence associated these options is very low, based on retrospective studies and rare non-randomized phase II clinical trials.
Regarding these uncertainties, physicians can hardly answer to patient questions.
Prospective data provided by a large multi-center cohort is needed. The objective of the present study is to create a large cohort of incident cases of AF associated with tumor bank and collection of blood samples.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||630 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||National Clinical-biological Prospective Cohort of Incident Cases of Aggressive Fibromatosis|
|Actual Study Start Date :||March 22, 2016|
|Estimated Primary Completion Date :||March 2025|
|Estimated Study Completion Date :||March 2026|
Tumor biobank realization (biopsy...) and biobank constitution. coloscopy associated with colonic chromoscopy. Blood sampling (facultative). Pain evaluation
pre-therapeutic or post-therapeutic biopsy or resected tissues
Other: biobank constitution
Constitution of a biobank with pre-therapeutic or post-therapeutic biopsy or resected tissues
For adult patients, a coloscopy with chromoscopy of ascending and sigmoid colon will be performed
Procedure: Blood sampling (facultative)
Blood sample can be collected at diagnostic or after medically significant events (progressive disease, local or systemic treatment, pregnancy...)
Other: Pain evaluation
Pain evaluation (EVA scale), anxiety (HADS questionnaire), quality of life questionnaire (EORTC-QLQ-C30)
Procedure: Tumor biobank realization
Realization of a tumor biobank is part of classical procedure of participating centers
- Incident cases of aggressive fibromatosis [ Time Frame: through study completion, an average of 5 years ]To constitute, at a national level, the largest cohort of incident cases of desmoid tumours
- Number of Aggressive Fibromatosis associated with familial adenomatous polyposis [ Time Frame: through study completion, an average of 5 years ]To describe and analyse the link between Aggressive Fibromatosis and familial adenomatous polyposis
- Percentage of CTNNB1 mutation in non-selected cases of Aggressive Fibromatosis [ Time Frame: through study completion, an average of 5 years ]To describe the proportion of AF cases characterized by CTNNB1 somatic mutation
- Treatment used for management of AF [ Time Frame: through study completion, an average of 5 years ]To describe the impact of different therapeutic strategies on AF recurrence and progression free survival, in homogenous risk level sub-groups of patients
- Hospital Anxiety and Depression Scale (HADS) [ Time Frame: at baseline, one year ]To describe the psychological impact of the disease at diagnosis and a year after diagnosis. And to compare changes between the time of diagnosis and one year after the treatments used.
- Quality of Life Questionnaire (QLQC30) [ Time Frame: at baseline, one year ]To describe the consequences of the disease on the quality of life at diagnosis and a year after diagnosis. And to compare changes between the time of diagnosis and one year after the treatments used.
- Mutation rate of APC [ Time Frame: through study completion, an average of 5 years ]To determine the mutation rate of APC at constitutional and somatic levels
- Mutation rate of CTNNB1 [ Time Frame: through study completion, an average of 5 years ]To determine the mutation rate of CTNNB1 at constitutional and somatic levels
- Correlation between APC and CTNNB1 mutations rates [ Time Frame: through study completion, an average of 5 years ]To demonstrate that APC and CTNNB1 mutations are two mutually exclusive molecular alterations
- APC mutation rate [ Time Frame: through study completion, an average of 5 years ]To correlate mutational somatic and constitutional rate of APC gene
- Occurrence of other mutations [ Time Frame: through study completion, an average of 5 years ]To search other molecular anomalies for patients without APC or CTNNB1 mutations (10 % of cases)
- Cell free (circulating) nucleic acid extraction technics [ Time Frame: through study completion, an average of 5 years ]To determine sensibility and specificity of cell free (circulating) nucleic acid extraction techniques
- AF outcome [ Time Frame: through study completion, an average of 5 years ]To describe patient outcomes and identify prognostic factors
- Treatment response [ Time Frame: through study completion, an average of 5 years ]To search for factors involved in response treatment prediction
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): NCT02867033
|Principal Investigator:||Nicolas PENEL, PhD||Centre Oscar Lambret|
|Principal Investigator:||Sébastien SALAS, PhD||Hopital Timone adultes|