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

• Register: EUCTR
• Last refreshed on: 12 March 2024
• Main ID: EUCTR2016-003226-16-IT
• Date of registration: 17/11/2016
• Prospective Registration: Yes
• Primary sponsor: Bellicum Pharmaceuticals, Inc.
• Public title: 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
• Scientific title: 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 - CaspaCide TCR aß haplo HSCT
• Date of first enrolment: 03/02/2017
• Target sample size: 175
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2016-003226-16
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no
• Phase: Human pharmacology (Phase I): yes Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Italy

Contacts

• Name: Martha French
• Affiliation: Bellicum Pharmaceuticals, Inc

• Name: Martha French
• Affiliation: Bellicum Pharmaceuticals, Inc

Key inclusion & exclusion criteria

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.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Hematological disorders (ALL;AML;Non-Hodgkin lymphoma;Myelodysplastic syndromes;Congenital immune deficiencies;Severe aplastic anemia;Fanconi anemia; Osteopetrosis;Selected cases of hemoglobinopathies)
MedDRA version: 19.0 Level: HLGT Classification code 10018849 Term: Haematological disorders NEC System Organ Class: 10005329 - Blood and lymphatic system disorders

Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Intervention(s)

Product Name: AP1903
Pharmaceutical Form: Solution for infusion
CAS Number: 195514-63-7
Current Sponsor code: AP1903
Other descriptive name: AP1903
Concentration unit: mg/kg milligram(s)/kilogram
Concentration type: equal
Concentration number: 0.4-

Primary Outcome(s)

Primary end point(s): Incidence of disease-free survival in both malignant and non-malignant
patients at 1 and 2 years.

Secondary Objective: Long-term safety for a total of 15 years from BPX-501
administration

Timepoint(s) of evaluation of this end point: Up to 2 years post-transplant

Main Objective: To evaluate the long-term safety of the infusion of BPX-501 gene
modified T cells and to evaluate disease-free survival at 1 and 2 years

Secondary Outcome(s)

Secondary end point(s): Long-term safety for a total of 15 years from BPX-501
administration

Timepoint(s) of evaluation of this end point: 15 years

Secondary ID(s)

BP-404

Source(s) of Monetary Support

Bellicum Pharmaceuticals

Ethics review

Status: Approved
Approval date: 18/01/2017
Contact: