A Non-interventional Ambispective Real-world Cohort of rE... | NCT05193448

Status Module

Record Verification Date

May 2023

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jul 5, 2021Actual

Primary Completion Date

Oct 31, 2021Actual

Completion Date

Jan 31, 2022Actual

First Submitted Date

Dec 31, 2021

First Submission Date that Met QC Criteria

Dec 31, 2021

First Posted Date

Jan 14, 2022Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

May 23, 2023

Last Update Posted Date

May 24, 2023Actual

Name	Class
Acute Leukemia French Association	OTHER

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

Brief Summary

Gilteritinib is available in early access in France through Temporary Authorisation of Use (or ATU program) since March 2019. The ATU program reflects a real-life treatment situation and the related clinical data would help to better understand the benefit/risk profile of gilteritinib and to better document gilteritinib efficacy and safety in patients who received midostaurine in First Line (1L) setting.

The main objective is to describe gilteritinib effectiveness in FLT3 (Fms Related Tyrosine Kinase 3) -mutated AML patients in Refractory/Relapsed(R/R) situation treated in the context of early access program to gilteritinib in France through Temporary Authorisation of Use, the so-called ATU program, and the post ATU period from marketing authorisation to launch when reimbursement and price are published.

Detailed Description

Not provided

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Yes

Target Follow-Up Duration

6 Months

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

No data available

No data is available for this block.

Enrollment

177Actual

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Gilteritinib effectiveness in FLT3-mutated AML patients in R/R situation	best response obtained according to European Leukemia Net (ELN) 2017 recommendations and ADMIRAL definitions effectiveness will also described in the following subgroups : refractory after 1st line chemo, 1rst relapse =< 6 months after Complete Remission (CR) 1, 1st relapse > 6 months after CR1, refractory after 1 st relapse salvage treatment, beyond the first relapse (>= 2nd relapse), post Hematopoietic Stem Cell Transplantation (HSCT), post 1L midostaurine and by ELN 2017 risk groups	6 months

Secondary Outcomes

Not provided

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Adult patients ≥ 18 years at AML diagnosis
Patients that started gilteritinib during ATU and post-ATU period from 19th March 2019 to 30th March2021
Patients diagnosed with refractory or relapsed AML as defined by the World Health Organization (WHO) Classification
Patients with FLT3 genetic testing performed at diagnosis and/or at R/R (if available)
Gilteritinib with or without other drug (chemotherapy, hypomethylating agent, hydroxyurea, etc.)

Exclusion Criteria:

Newly diagnosed AML patients
Participant opposed to the collection and analysis of their medical data
Prescription of gilteritinib out of the scope of its marketing authorisation approval such as post HSCT maintenance in patients in first complete remission after intensive chemotherapy
persons placed in curatorship,guardianship or guardianship orders

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

Not provided

Standard Ages

AdultOlder Adult

Study Population

≥18 years of age FLT3-mutated R/R AML as defined by the WHO Classification between the periods of March 1st, 2019 to September 30, 2021 allowing 6-months follow-up for the last patient included (March, 19 2019 to September 18, 2019 for French "ATU nominative"; September 18, 2019 to January 08, 2020 for French "ATU de cohorte"; January 08, 2020 to March 30, 2021 for "post ATU" period) treated in virtually all AML center in France

Sampling Method

Probability Sample

IPD Sharing Statement Module

Plan to Share IPD

Description

Not provided

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

Not provided

Facility	Status	City	State	ZIP	Country	Contacts
Amiens CHU		Amiens			France
Angers CHU

ID	Term
D007951	Leukemia, Myeloid
D007938	Leukemia
D009370	Neoplasms by Histologic Type
D009369	Neoplasms
D006402	Hematologic Diseases
D006425	Hemic and Lymphatic Diseases