To determine the real world response to standard of care CAR T-cell therapy, in order to better understand the intrinsic and extrinsic factors related to treatment success or failure.
ID
Source
Brief title
Condition
- Leukaemias
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
The primary endpoint of the study is to characterize and quantify changes in
the immune response during CAR T-cell treatment.
Secondary outcome
Secondary endpoints will include the impact of fludarabine levels on the immune
profile, CAR T-cell expansion, CAR T-cell persistence and quality of life.
Background summary
The treatment of relapse and refractory B-cell precursor- acute lymphoblastic
leukaemia (BCP-ALL) in children is changing since the
introduction of immunotherapy. Chimeric antigen receptor T (CAR T) cell therapy
was rapidly introduced after EMA approval based
on the positive results of CTL019 in refractory or relapsed after stem cell
transplantation BCP-ALL.(Maude NEJM 2018) The recent
introduction of this viral transduced, ex vivo expanded product, warrants an
extensive long-term follow-up of patients of real-world
data. This includes efficacy data, and scientific research - specifically
immune monitoring, to improve the efficacy, quality of life
aspects and the documentation of side effects of this novel treatment. The aim
is to register these data in a prospective CAR T cell
registry.
Study objective
To determine the real world response to standard of care CAR T-cell therapy, in
order to better understand the intrinsic and extrinsic factors related to
treatment success or failure.
Study design
Prospective longitudinal observational study of patients treated with CAR T
cell therapy in the Princess Máxima Center for pediatric
oncology. Stratum A includes children treated with tisagenlecleucel in standard
of care setting for BCP-ALL. Additional strata will be
amended in case other CAR T cell products receive market approval.
Study burden and risks
This study aims to collect clinical data in combination with biological samples
prior to and after CAR-T cell infusion. After consent a
bone biopsy will be performed during the standard bone marrow aspiration and
therefore patients will not be exposed to additional
procedures. The volume of blood that is withdrawn for the study does not exceed
the recommended maximum.
Heidelberglaan 25
Utrecht 3584 CS
NL
Heidelberglaan 25
Utrecht 3584 CS
NL
Listed location countries
Age
Inclusion criteria
In order to be eligible to participate in this study, a subject must meet all
of the following criteria:
1. Age 0-26 years
2. Intention to start CAR T-cell treatment in Princess Máxima Center
3. Signed written informed consent
4. Stratum specific inclusion criteria
• Stratum A: tisagenlecleucel in standard of care treatment for BCP-ALL
• Other strata will be amended after market approval
• Strata for patients in clinical studies will be amended
Exclusion criteria
No informed consent obtained
Design
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| Other | ISRTCN wordt definitief na goedkeuring |
| CCMO | NL87682.041.24 |