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; Congenital immune deficiencies; Severe aplastic anemia; Fanconi anemia; Osteopetrosis; Selected cases of hemoglobinopathies); MedDRA version: 16.1Level: HLGTClassification code 10018849Term: Haematological disorders NECSystem Organ Class: 10005329 - Blood and lymphatic system disorders

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

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2014-04-15
• Last Update Posted: 23 November 2020

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 30

Inclusion Criteria

Males or females;Age < 21 years and > 3 months; Life expectancy > 10 weeks;
Deemed eligible for allogeneic stem cell transplantation; Children with life-threatening hematological malignancies (high-risk ALL in 1st CR,ALL in 2nd or subsequent CR, high-risk AML in 1st CR, AML in 2nd or subsequentCR,myelodysplastic syndromes, non-Hodgkin lymphomas in 2nd or subsequent CR);Non-malignant disorders amenable to be cured by an allograft:
a. primary immune deficiencies,b. severe aplastic anemia not responding to immune suppressive therapy,c. osteopetrosis,d. selected cases of hemoglobinopathies ande. congenital/hereditary cytopenia, including Fanconi Anemia before any clonalmalignant evolution (MDS, AML);Lack of suitable conventional donor (HLA identical sibling or HLA phenotipically identical relative or 9-10/10 unrelated donor evaluated using high resolution molecular typing) or presence of rapidly progressive disease not permitting time to identify an unrelated donor; A minimum genotypic identical match of 5 / 1 0 is required.
The donor and recipient must be identical, as determined by high resolution typing, at least one allele of each of the following genetic loci: HLA-A, HLA-B, HLA-Cw, and HLADRB1;Lansky/Karnofsky score > 50, WHO > 4;Signed written informed consent
Are the trial subjects under 18? yes
Number of subjects for this age range: 30
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

Age < 3 months or >21 years;Greater than grade II acute GvHD or chronic extensive GvHD due to a previous allograft at the time of inclusion; Patient receiving an immunosuppressive treatment for GvHD treatment at the time of
inclusion;Dysfunction of liver (ALT/AST > 5 times normal value, or bilirubin > 3 times normal value), or of renal function (creatinine clearance < 30 ml / min); Severe cardiovascular disease (arrhythmias requiring chronic treatment, congestive heart failure or left ventricular ejection fraction <40%); Current active infectious disease (including positive HIV serology or viral RNA);Serious concurrent uncontrolled medical disorder;Pregnant or breast feeding female patient; Lack of parents’ informed consent. Patients who have received more than 1 x 105 ??T cells/Kg with the graft infusion will be excluded.

Study & Design

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