AVIATOR2020: French Long Term Registry With Longitudinal Follow up of PDGFRA D842V-GIST Patients Treated With Avapritinib
Study Details
Study Description
Brief 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 will be accompanied by a protocol of therapeutic use and collection of information (PUT) that describes the frequency of patient visits.
The objective of this real-life registry is to perform a long-term longitudinal follow up of PDGFRA D842V-mutated GIST patients treated with avapritinib and to collect effectiveness and safety data. It will be implemented in parallel to the ATUc until June 2023.
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
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Study Design
Outcome Measures
Primary Outcome Measures
- Survival [up to 48 months]
survival of patients treated with Avapritinib in real life according to overall survival.
Secondary Outcome Measures
- Progression Free Survival [after 12, 24 and 36 months]
according to RECIST criteria
- Incidence of long-term responders [up to 48 months]
(>24 months)
- Duration of treatment [up to 48 months]
duration in months
- Safety: Nature of AEs [up to 48 months]
AEs graded using Common Terminology Criteria for Adverse Events (CTCAE) v5.0
- Safety : Frequency of AEs [up to 48 months]
AEs graded using Common Terminology Criteria for Adverse Events (CTCAE) v5.0
- Safety : Severity of AEs [up to 48 months]
AEs graded using Common Terminology Criteria for Adverse Events (CTCAE) v5.0
- Cognitive impairment in real-life according to MoCA questionnaires [Day 1, Day 15, Month 2, Month 3, Month 6, Month 12, Month 18, Month 24, Month 36]
Montreal Cognitive Assessment (MoCA)
- Cognitive impairment in real-life according to FACT-Cog [Day 1, Day 15, Month 2, Month 3, Month 6, Month 12, Month 18, Month 24, Month 36]
Functional Assessment of Cancer Therapy - Cognitive Function
- Quality of life in real-life according to FACT-G questionnaire [Day 1, Day 15, Month 2, Month 3, Month 6, Month 12, Month 18, Month 24, Month 36]
Functional Assessment of Cancer Therapy - General
Eligibility Criteria
Criteria
Inclusion Criteria:
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I1. Adult (≥18 years old), male or female
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I2. Patient with a histologically or cytologically-confirmed diagnosis of unresectable or metastatic GIST harboring the D842V mutation in the PDGFRα gene
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I3. Patient treated with Avapritinib
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I4. Non opposition to the use of her/his data
Exclusion criteria :
- E1. Patient not meeting all the inclusion criteria
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | CHRU Besançon | Besançon | France | ||
| 2 | Insitut Bergonié | Bordeaux | France | ||
| 3 | Centre Geroges François Leclerc | Dijon | France | ||
| 4 | Centre Léon Bérard | Lyon | France | 69008 | |
| 5 | CHU Robert Debré | Reims | France | ||
| 6 | Institut Gustave Roussy | Villejuif | France |
Sponsors and Collaborators
- Centre Leon Berard
- Blueprint Medicines Corporation
Investigators
- Principal Investigator: MEHDI BRAHMI, Centre Leon Berard
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- AVIATOR2020