Phase II extension study of CaspaCIDe T cells (BPX-501) 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; Congenital immune deficiencies; Severe aplastic anemia; Fanconi anemia; Osteopetrosis; Selected cases of hemoglobinopathies); MedDRA version: 19.1Level: HLGTClassification code 10018849Term: Haematological disorders NECSystem Organ Class: 10005329 - Blood and lymphatic system disorders

• Registration Number: EUCTR2014-000584-41-ES
• Lead Sponsor: Bellicum Pharmaceuticals, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2017-03-21
• Last Update Posted: 10 September 2018

Recruitment & Eligibility

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

Inclusion Criteria

1.Age < 18 years and > 1 month (< 1 month upon approval by Applicant)
2.Life expectancy > 10 weeks
3.Patients deemed clinically eligible for allogeneic stem cell transplantation.
4.Patients may have failed prior allograft
5.Patients with life-threatening hematological malignancies (high-risk ALL in 1st CR, ALL in 2nd, high-risk AML in 1st CR, AML in 2nd CR;
OR
6.Non-malignant disorders deemed curable by allogeneic transplantation:
•primary immune deficiencies,
•severe aplastic anemia not responding to immune suppressive therapy,
•osteopetrosis,
•selected cases of hemoglobinopathies, for example, ß0 ß0 thalassemia major, sickle cell disease, Diamond- Blackfin
•congenital/hereditary cytopenia, including Fanconi Anemia before any clonal malignant evolution (MDS, AML).
7.Lack of suitable conventional donor (HLA identical sibling or HLA phenotypically identical relative or 10/10 unrelated donor evaluated using high resolution molecular typing) or presence of rapidly progressive disease not permitting time to identify an unrelated donor
8.A minimum genotypic identical match of 5/10 is required.
9.The donor and recipient must be identical, as determined by high resolution typing, on at least one allele of each of the following genetic loci: HLA-A, HLA-B, HLA-Cw, and HLA- DRB1.
10.Lansky/Karnofsky score > 50
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.Greater than ongoing Grade II acute GvHD or chronic extensive GvHD due to a previous allograft at the time of screening
2.Patient receiving an immunosuppressive treatment for GvHD treatment due to a previous allograft at the time of screening
3.Dysfunction of liver (ALT/AST > 5 times normal value, or bilirubin > 3 times normal value), or of renal function (creatinine clearance <30ml/min/1.73m2)
4.Severe cardiovascular disease (arrhythmias requiring chronic treatment, congestive heart failure or left ventricular ejection fraction < 40%)
5.Current active infectious disease (including positive HIV serology or viral RNA)
6.Serious concurrent uncontrolled medical disorder
7.Pregnant or breast feeding female patient

Study & Design

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