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Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT04927260
Other study ID # AVIATOR2020
Secondary ID
Status Active, not recruiting
Phase
First received
Last updated
Start date March 31, 2021
Est. completion date December 31, 2024

Study information

Verified date December 2023
Source Centre Leon Berard
Contact n/a
Is FDA regulated No
Health authority
Study type Observational [Patient Registry]

Clinical Trial Summary

GIST are rare mesenchymal tumors of the gastrointestinal tract characterized by somatic mutations in the gene encoding the KIT (85%) or the PDGFRα (8%) protein. Treatment of localized forms relies on adequate surgery without tumor spillage and sometimes systemic treatment with imatinib according to risk of relapse defined by localization, tumor size and mitotic count, as well as mutational status. More than 40% of cases may recur and metastasize. Advanced and relapsing forms are currently treated with oral tyrosine-kinase inhibitors (TKI) of KIT and PDGFR such as imatinib (standard treatment), sunitinib (2nd line) and regorafenib (3rd line). Nevertheless, imatinib has little or no activity in patients harboring the D842V mutation in the exon 18 of PDGFRα (20% of gastric GIST, 6% of all GIST patients). Consequently, other therapeutic alternatives are needed. Results from the phase I single-arm NAVIGATOR study show that avapritinib has significant efficacy in GIST patients with PDGFRα D842V mutation (ORR = 86 %). In France, an authorization for temporary use (ATUc) starting on September 21st, 2020 has been granted by the National Agency for Safety of Medicines and Health Products (ANSM). It allows the early availability of avapritinib in France while waiting for Market Authorization Approval (AMM). This ATUc is now being followed by a post-ATU period. The objective of this real-life registry is to perform a long-term longitudinal follow up of PDGFRA D842V-mutated GIST patients and to collect effectiveness and safety data. It will be implemented in parallel to the post-ATUc period until June 2023. Moreover, this registry fulfills the HAS's ("Haute Autorité de Santé") request regarding the Establishment of an exhaustive registry of patients with GIST, harboring the D842V / PDGFRA mutation in France. This registry will specifically describe: - patient characteristics, in particular patient age, of the disease characteristics, previous treatments; - the clinical course; - the occurrence of adverse events / effects; - and the therapeutic strategy (endpoint of treatment or continuation). Data from the electronic health record (EHR) will be collected. Moreover, as per the ANSM's requirements, quality of life and cognitive function will be investigated using FACT-G, FACT-Cog and MoCA questionnaires. Undesirable effects will be collected as well. Follow-up is envisioned for a minimum of 2 years.


Recruitment information / eligibility

Status Active, not recruiting
Enrollment 25
Est. completion date December 31, 2024
Est. primary completion date April 30, 2024
Accepts healthy volunteers No
Gender All
Age group 18 Years and older
Eligibility Inclusion Criteria: - I1. Adult (=18 years old), male or female - I2. Patient with a histologically or cytologically-confirmed diagnosis of unresectable or metastatic GIST harboring the D842V mutation in the PDGFRa gene - I3. . Diagnosis date later than Jan 1st 2010 - I4. Non opposition to the use of her/his data Exclusion criteria : - E1. Patient not meeting all the inclusion criteria

Study Design


Intervention

Drug:
Patient treated by Avapritinib in real life
The patients included are treated with Avapritinib as part of their care. There is no change in treatment associated with this study. Patients must regularly complete various questionnaires: FACT-G, FACT-COG, and a neuropsychiatrist or a trained physician will give them the MoCA.

Locations

Country Name City State
France CHRU Besançon Besançon
France Insitut Bergonié Bordeaux
France CHU Clermont Ferrand Clermont-Ferrand
France Centre Léon Bérard Lyon
France Institut de Cancérologie de l'Ouest (ICO) Nantes
France CHU Robert Debré Reims
France Institut Gustave Roussy Villejuif

Sponsors (2)

Lead Sponsor Collaborator
Centre Leon Berard Blueprint Medicines Corporation

Country where clinical trial is conducted

France, 

Outcome

Type Measure Description Time frame Safety issue
Primary Survival survival of patients treated with Avapritinib in real life according to overall survival. up to 48 months
Secondary Progression Free Survival according to RECIST criteria after 12, 24 and 36 months
Secondary Incidence of long-term responders (>24 months) up to 48 months
Secondary Duration of treatment duration in months up to 48 months
Secondary Safety: Nature of AEs AEs graded using Common Terminology Criteria for Adverse Events (CTCAE) v5.0 up to 48 months
Secondary Safety : Frequency of AEs AEs graded using Common Terminology Criteria for Adverse Events (CTCAE) v5.0 up to 48 months
Secondary Safety : Severity of AEs AEs graded using Common Terminology Criteria for Adverse Events (CTCAE) v5.0 up to 48 months
Secondary Cognitive impairment in real-life according to MoCA questionnaires Montreal Cognitive Assessment (MoCA) Day 1, Day 15, Month 2, Month 3, Month 6, Month 12, Month 18, Month 24, Month 36
Secondary Cognitive impairment in real-life according to FACT-Cog Functional Assessment of Cancer Therapy - Cognitive Function Day 1, Day 15, Month 2, Month 3, Month 6, Month 12, Month 18, Month 24, Month 36
Secondary Quality of life in real-life according to FACT-G questionnaire Functional Assessment of Cancer Therapy - General Day 1, Day 15, Month 2, Month 3, Month 6, Month 12, Month 18, Month 24, Month 36
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