A Phase I study of BPX-501 T cells and AP1903 in children with recurrent hematological cancer after allogeneic transplant.

Phase 1

• Conditions: recurrent or minimal residual disease (MRD) hematologic malignancies post-allogeneic transplant; MedDRA version: 20.0Level: HLGTClassification code 10018849Term: Haematological disorders NECSystem Organ Class: 10005329 - Blood and lymphatic system disorders; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2015-005176-17-IT
• Lead Sponsor: Bellicum Pharmaceuticals, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2016-07-13
• Last Update Posted: 12 March 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 15

Inclusion Criteria

Recipient inclusion criteria:
1.Subjects aged >1yrs and < 18yrs
2.Clinical diagnosis of one of the following pediatric hematological malignancies:
a)Leukemia
b)Myelodysplastic Syndromes
c)Lymphomas
d)Other high-risk hematological malignancy eligible for stem cell transplantation per institutional standard
3.Recurrent disease that presents >100 days after, or minimal residual disease (MRD) that presents > 30 days after either:
a)Matched related HSCT
b)Mismatched related HSCT
4.Life expectancy >10 weeks;
5.Signed donor and patient/guardian informed consent;
6.A minimum genotypic identical match of 4/8 is required, as determined by high resolution typing for at least one allele of each of the following genetic loci: HLA-A, HLA-B, HLA-C, and HLA- DRB1.
7. Performance status: Karnofsky/Lanksy score > 50%.
8.Subjects with adequate organ function as measured by:
a.Bone marrow:
· >25% donor T-cell chimerism
· ANC>1 x 109/L.
b.Cardiac: LVEF at rest >45%.
c.Pulmonary: FEV 1, FVC, DLCO (diffusion capacity) > 50% predicted (corrected for hemoglobin); for children who are unable to perform pulmonary function tests due to age or developmental ability, there must be no evidence of dyspnea or no need for supplemental oxygen as evidenced by 02 saturation = 92% on room air.
d.Hepatic: direct bilirubin = 3x upper limit of normal (ULN), or AST/ALT = 5x ULN.
e.Renal: creatinine clearance = 2x of ULN for age
DONOR
1. Donor must have been the donor of the prior allogeneic peripheral
blood stem cell (PBSC) or bone marrow (BMSC) transplant:
a. Matched related HSCT
b. Mismatched related HSCT
2. Donor age must be = 18 and = 60 years.
a. The donor should be sufficiently healthy not to be at increased risk
from the leukapheresis procedure.
3. Donors must meet the selection criteria as defined by the European
Directive 2006/17/CE and per the local regulations for donor selection.
4. The donor must have been informed of the investigational nature of
the BPX-501 product and have signed an informed consent form.
5. Donor must have adequate peripheral venous access for leukapheresis or must agree to placement of a central venous catheter.
Are the trial subjects under 18? yes
Number of subjects for this age range: 15
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 3
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Recipient
1.= Grade II acute GvHD or chronic extensive GvHD due to a previous
allograft at the time of screening;
2.Active CNS involvement by malignant cells (less than 2 months from
the conditioning);
3.Current uncontrolled bacterial, viral or fungal infection (currently
taking medication with evidence of progression of clinical symptoms or
radiologic findings).
4.Positive HIV serology or viral RNA (= Grade III per CTCAE criteria);
5.Pregnancy (positive serum ßHCG test) or breast-feeding female;
6.Fertile men or women unwilling to use effective forms of birth control
or abstinence for a year after BPX-501 T cell infusion;
7.Bovine product allergy.
Donor Exclusion Criteria:
1. Evidence of active infection (including urinary tract infection, or upper respiratory tract infection) or viral hepatitis exposure (on screening), unless HBs Ab+ and HBV DNA negative.
2. Factors which place the donor at increased risk for complications from leukapheresis (e.g., autoimmune disease, symptomatic coronary artery disease requiring therapy, previous thrombotic events).
3. Pregnancy (positive serum or urine ßHCG) or breastfeeding female

Study & Design

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