Follow-up of phase I/II study of CaspaCide T cells from an HLA-partially matched family donor after negative selection of TCR aß+ T cells in pediatric patients affected by hematological disorders

Phase 1

• Conditions: Hematological disorders (ALL; AML; Non-Hodgkin lymphoma; Myelodysplasticsyndromes; Osteopetrosis; Selected cases of hemoglobinopathies); MedDRA version: 19.0Level: HLGTClassification code 10018849Term: Haematological disorders NECSystem Organ Class: 10005329 - Blood and lymphatic system disorders; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]; Congenital immune deficiencies; Severe aplastic anemia

• Registration Number: EUCTR2016-003226-16-IT
• Lead Sponsor: Bellicum Pharmaceuticals, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2016-11-17
• Last Update Posted: 12 March 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 175

Inclusion Criteria

1. Signed written informed consent by the patient or the patient’s guardian for children who are minors
2. enrolled on BP-004 protocol, received BPX-501 infusion and completed 6 months of active treatment
Are the trial subjects under 18? yes
Number of subjects for this age range: 170
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 5
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Lack of parents’/guardian’s informed consent for children who are minors.

Study & Design

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

Primary Outcome Measures

Name	Time	Method
Main Objective: To evaluate the long-term safety of the infusion of BPX-501 gene<br>modified T cells and to evaluate disease-free survival at 1 and 2 years;Secondary Objective: Long-term safety for a total of 15 years from BPX-501<br>administration;Primary end point(s): Incidence of disease-free survival in both malignant and non-malignant<br>patients at 1 and 2 years.;Timepoint(s) of evaluation of this end point: Up to 2 years post-transplant

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): Long-term safety for a total of 15 years from BPX-501<br>administration;Timepoint(s) of evaluation of this end point: 15 years