GISTAVIATOR2020NCT04927260
French Long Term Registry With Longitudinal Follow up of PDGFRA D842V-GIST Patients
An observational study of GIST and GIST, Malignant, run by Centre Leon Berard at 7 sites in France.
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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- GIST, GIST, Malignant, PDGFR-Alpha D842V
- What is being tested?
- Patient treated by Avapritinib in real life
- How many people
- 25 participants
- Where
- 7 sitesFrance
- Study dates
- 31 Mar 2021to 31 Dec 2024
- How long will I take part?
- 2 Years
- Phase
- Observational
- Are there results?
- None posted in the registry
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
Study population: All patients with GIST harboring a PDGFRa D842V mutation diagnosed from 2010 and treated with Avapritnib or not can be included.
Who may be able to join
- 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 PDGFRα gene
- I3. . Diagnosis date later than Jan 1st 2010
- I4. Non opposition to the use of her/his data
Who cannot join
- E1. Patient not meeting all the inclusion criteria
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
France7 sites
CHRU Besançon
Besançon
Insitut Bergonié
Bordeaux
CHU Clermont Ferrand
Clermont-Ferrand
Centre Léon Bérard
Lyon, 69008
Institut de Cancérologie de l'Ouest (ICO)
Nantes
CHU Robert Debré
Reims
Institut Gustave Roussy
Villejuif
About this study
Official title: French Long Term Registry With Longitudinal Follow up of PDGFRA D842V-GIST Patients : AVapritinib Real-life observatTORy
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.
Read more
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.
- Conditions
- GIST, GIST, Malignant, PDGFR-Alpha D842V
- Being tested
- Patient treated by Avapritinib in real life
- Medical subject headings
- Gastrointestinal Stromal Tumors
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Patient treated by Avapritinib in real lifeDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| COHORTE PROSPECTIF | Not described Receives: Drug: Patient treated by Avapritinib in real life | |
| COHORTE RETROSPECTIF | Not described Receives: Drug: Patient treated by Avapritinib in real life |
What is being measured
The primary outcome is the main question the study is designed to answer.
Survival
survival of patients treated with Avapritinib in real life according to overall survival.
Measured: up to 48 months
Secondary outcomes (9)
Progression Free Survival
according to RECIST criteria
Measured: after 12, 24 and 36 months
Incidence of long-term responders
(>24 months)
Measured: up to 48 months
Duration of treatment
duration in months
Measured: up to 48 months
Safety: Nature of AEs
AEs graded using Common Terminology Criteria for Adverse Events (CTCAE) v5.0
Measured: up to 48 months
Safety : Frequency of AEs
AEs graded using Common Terminology Criteria for Adverse Events (CTCAE) v5.0
Measured: up to 48 months
Safety : Severity of AEs
AEs graded using Common Terminology Criteria for Adverse Events (CTCAE) v5.0
Measured: up to 48 months
Cognitive impairment in real-life according to MoCA questionnaires
Montreal Cognitive Assessment (MoCA)
Measured: Day 1, Day 15, Month 2, Month 3, Month 6, Month 12, Month 18, Month 24, Month 36
Cognitive impairment in real-life according to FACT-Cog
Functional Assessment of Cancer Therapy - Cognitive Function
Measured: Day 1, Day 15, Month 2, Month 3, Month 6, Month 12, Month 18, Month 24, Month 36
Quality of life in real-life according to FACT-G questionnaire
Functional Assessment of Cancer Therapy - General
Measured: Day 1, Day 15, Month 2, Month 3, Month 6, Month 12, Month 18, Month 24, Month 36
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jun 2021
- Study start31 Mar 2021
- Primary completion30 Apr 2024 (estimated)
- Study completion31 Dec 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for GISTStudies of the same condition, open and under way.
- More trials run by Centre Leon BerardThe sponsor's other registered studies.
- 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
- Observational
- Observational model
- Cohort
- Time perspective
- Other
- Enrolment
- 25 participants (actual)
- Target duration
- 2 Years
Sponsor and investigators
- Lead sponsor
- Centre Leon Berard
- Sponsor type
- University, hospital or other
- Collaborators
- Blueprint Medicines Corporation
- Responsible party
- Sponsor
- Investigators
- MEHDI BRAHMI, Principal investigator, Centre Leon Berard
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04927260
- Sponsor's protocol ID
- AVIATOR2020
- Acronym
- AVIATOR2020
- Status last verified
- Dec 2023
- First submitted
- 2 Jun 2021
- Last updated
- 26 Dec 2023
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Dec 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- French Long Term Registry With Longitudinal Follow up of PDGFRA D842V-GIST Patients : AVapritinib Real-life observatTORy
- ClinicalTrials.gov ID
- NCT04927260
- Acronym / sponsor ID
- AVIATOR2020 / AVIATOR2020
- Conditions
- GIST, GIST, Malignant, PDGFR-Alpha D842V
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 25 participants (actual)
- Lead sponsor
- Centre Leon BerardUniversity, hospital or other
- Collaborators
- Blueprint Medicines Corporation
- Study start
- 31 Mar 2021 (actual)
- Primary completion
- 30 Apr 2024 (estimated)
- Study completion
- 31 Dec 2024 (estimated)
- First posted
- 15 Jun 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Other
- Target follow-up
- 2 Years
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| COHORTE PROSPECTIF | – | Drug: Patient treated by Avapritinib in real life |
| COHORTE RETROSPECTIF | – | Drug: Patient treated by Avapritinib in real life |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- All patients with GIST harboring a PDGFRa D842V mutation diagnosed from 2010 and treated with Avapritnib or not can be included.
Key inclusion criteria (4 listed)
- 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 PDGFRα gene
- I3. . Diagnosis date later than Jan 1st 2010
- I4. Non opposition to the use of her/his data
Key exclusion criteria (1 listed)
- E1. Patient not meeting all the inclusion criteria
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 |
|---|---|---|
| Patient treated by Avapritinib in real lifeThe 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. | Drug | COHORTE PROSPECTIF, COHORTE RETROSPECTIF |
Primary outcome
Survival
Time frame: up to 48 months
survival of patients treated with Avapritinib in real life according to overall survival.
9 secondary outcomes
Progression Free Survival
Time frame: after 12, 24 and 36 months
Incidence of long-term responders
Time frame: up to 48 months
Duration of treatment
Time frame: up to 48 months
Safety: Nature of AEs
Time frame: up to 48 months
Safety : Frequency of AEs
Time frame: up to 48 months
Safety : Severity of AEs
Time frame: up to 48 months
Cognitive impairment in real-life according to MoCA questionnaires
Time frame: Day 1, Day 15, Month 2, Month 3, Month 6, Month 12, Month 18, Month 24, Month 36
Cognitive impairment in real-life according to FACT-Cog
Time frame: Day 1, Day 15, Month 2, Month 3, Month 6, Month 12, Month 18, Month 24, Month 36
Quality of life in real-life according to FACT-G questionnaire
Time frame: Day 1, Day 15, Month 2, Month 3, Month 6, Month 12, Month 18, Month 24, Month 36
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Dec 2023.
Related trials for GIST
RecruitingPhase 2
A Study to Learn More About How Well Treatment With Sevabertinib (BAY 2927088) Tablets Works and How Safe it is in Participants Who Have a Solid Tumor With Mutations of the Human Epidermal Growth Factor Receptor 2 (HER2)
Advanced Solid Tumors, HER2 Mutation
RecruitingPhase 2
Belzutifan/MK-6482 for the Treatment of Advanced Pheochromocytoma/Paraganglioma (PPGL), Pancreatic Neuroendocrine Tumor (pNET), Von Hippel-Lindau (VHL) Disease-Associated Tumors, Advanced Gastrointestinal Stromal Tumor (wt GIST), or Solid Tumors With HIF-2α Related Genetic Alterations (MK-6482-015)
Pheochromocytoma/Paraganglioma, Pancreatic Neuroendocrine Tumor
RecruitingObservational study
Surgery in Gastrointestinal Stromal Tumors (GISTs) for Treatment, Tumor Modeling, and Genomic Analysis
Gastric Cancer, Gastric Neoplasm
RecruitingPhase 1
A Study of Ziftomenib in Combination With Imatinib in Patients With Advanced Gastrointestinal Stromal Tumors (GIST)
Gastrointestinal Stromal Tumor (GIST), Gastrointestinal Stromal Cancer
RecruitingPhase 1
Phase I Trial to Determine the Dose and Evaluate the PK and Safety of Lutetium Lu 177 Edotreotide Therapy in Pediatric Participants With SSTR-positive Tumors
Somatostatin Receptor Positive, NETs
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.
MEHDI BRAHMI principal investigator
Centre Leon Berard
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of GIST and GIST, Malignant, run by Centre Leon Berard at 7 sites 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 4 inclusion and 1 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: status not confirmed, as of the sponsor's last update on 26 Dec 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites 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.
Are results available for this study?
Results are not available in the source record.
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?
Survival (time frame: up to 48 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 26 Dec 2023.