A study to evaluate the safety and efficacy of UCART19 in children with B cell lymphoblastic leukaemia that has relapsed or not responded to other treatments

Phase 1

• Conditions: Paediatric relapsed or refractory CD19-positive B-cell acute lymphoblastic leukemia; MedDRA version: 20.0Level: LLTClassification code 10060390Term: Leukaemia lymphoblastic acuteSystem Organ Class: 100000004864; Therapeutic area: Diseases [C] - Cancer [C04]

• Registration Number: EUCTR2015-004293-15-FR
• Lead Sponsor: Institut de Recherches Internationales Servier (I.R.I.S)

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2017-10-20
• Last Update Posted: 16 November 2020

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 10

Inclusion Criteria

1. Male or female patients

2. Age ranging between 6 months and <18years

3. Patients with relapsed or refractory CD19-positive B-acute lymphoblastic leukemia (B-ALL) (National Comprehensive Cancer Network (NCCN), 2015),
- Morphologically confirmed
- or presenting a quantifiable MRD load of 1x10-3 (by multiparameter flow cytometry and/or quantitative polymerase chain reaction) at the end of the last induction treatment
- Who have exhausted available treatment options
- Eligible for allogeneic hematopoietic stem cells transplantation with suitable donor available

4. Estimated life expectancy = 12 weeks (according to investigator’s judgement)

5. Considered medically fit for allo-HSCT

6. Eastern Cooperative Oncology Group ECOG performance status < 2

7. Written informed consent from parent(s) or legal representative and or written assent from patient when applicable obtained prior any study-specific procedure of the protocol
Are the trial subjects under 18? yes
Number of subjects for this age range: 10
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

9. Foreseeable poor compliance to the study procedures

10. Previous treatment with investigational gene or cell therapy medicine products. Prior treatment with blinatumomab is allowed

11. Use of other investigational products or previous chemotherapy including biologic/targeted therapy or immunological agents within 5 half-lives or within 14 days prior to UCART 19 administration, whichever has a
shorter duration

12. CD19-negative B-cell leukaemia

13. Absence of suitable HLA matched or mismatched donor

14. Weight< 8.8 kgs

15. Burkitt cell acute leukaemia (L3 ALL)

16. Autologous HSCT within 6 weeks or allogeneic HSCT within 3 months prior Inclusion

17. Uncontrolled CNS leukemia

18. Use of rituximab and other anti CD20 antibodies known to have the same epitope as rituximab or anti CD20 for which the epitope is unknown within 3 months prior to UCART19 infusion

19. Presence of donor-specific anti-HLA antibodies directed against UCART19

20. Active, acute or chronic GvHD requiring therapy

21. Patients currently treated with immunosuppressive agents that cannot be stopped (e.g., GvHD or autoimmune disease)

22. A known hypersensitivity to any of the test materials or related compounds including murine and bovine products

23. Unstable cardiovascular disease,

24. Active bacterial, fungal, protozoal or viral infection not controlled by adequate treatment, and presence of positive blood cultures within 7 days before Inclusion

25. Abnormal findings during the screening period, any other medical condition(s) or laboratory findings that in the opinion of the investigator renders the patient unsuitable for subsequent allo-HSCT.

26. Any planned medical/surgical treatment that might interfere with the ability to comply with the study requirements

27. Risk of pregnancy or non compliance with contraception (if applicable) .Girls of childbearing potential must have been tested negative in a pregnancy test within 7 days prior to inclusion. Within the frame of this study, female participants of childbearing potential and male participants with partners of childbearing potential must use an effective method of birth control for a duration of 12 months after Investigational Medicinal Product (IMP) administration.

32. Evidence of other malignancy within 2 years prior to Inclusion (except in situ basal or squamous cervix or skin cell carcinoma)

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified