Safety Study of Gene Modified Donor T-cells Following TCRaß+ Depleted Stem Cell Transplant

• Register: ClinicalTrials.gov
• Last refreshed on: 3 October 2023
• Main ID: NCT02065869
• Date of registration: 13/02/2014
• Prospective Registration: Yes
• Primary sponsor: Bellicum Pharmaceuticals
• Public title: Safety Study of Gene Modified Donor T-cells Following TCRaß+ Depleted Stem Cell Transplant
• Scientific title: Phase I/II Study of CaspaCIDe T Cells (BPX-501; Rivogenlecleucel) From an HLA Partially Matched Family Donor After Negative Selection of TCRaß+ T Cells in Paediatric Patients Affected by Haematological Disorders
• Date of first enrolment: April 2014
• Target sample size: 187
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/ct2/show/NCT02065869
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

Countries of recruitment

Germany, Italy, Spain, United Kingdom, United States

Contacts

• Name: Bellicum Pharmaceuticals
• Affiliation: Bellicum Pharmaceuticals, Inc.

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Age < 18 years and > 1 month (< 1 month upon approval by Sponsor)

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 acute leukemia (high-risk ALL in 1st CR, ALL in 2nd CR,
high-risk AML in 1st CR, AML in 2nd CR.) or myelodysplastic syndromes. Morphological
CR must be documented and minimal residual disease measurement before transplantation
is recommended.

6. Non-malignant disorders deemed curable by allogeneic transplantation: (a) primary
immune deficiencies, (b) severe aplastic anemia not responding to immune suppressive
therapy, (c) osteopetrosis, (d) selected cases of erythroid disorders such as ß0 ß0
thalassemia major, sickle cell disease, Diamond-Blackfan anemia, (e)
congenital/hereditary cytopenia, including Fanconi Anemia before any clonal malignant
evolution (MDS, AML).

Note: Subjects will be eligible if they meet either item 5 OR item 6.

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, HLA-
DRB1 and HLA-DQB1.

10. Lansky/Karnofsky score > 50

11. Signed informed consent by the patient or the patient's parent or guardian for
patients who are minors

Exclusion Criteria:

1. Greater than active 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 clinically active infectious disease (including positive HIV serology or viral
RNA)

6. Serious concurrent uncontrolled medical disorder

7. Pregnant or breast feeding female patient

8. Lack of parents'/guardian's informed consent for children who are minors.



Age minimum: 1 Month
Age maximum: 18 Years
Gender: All

Health Condition(s) or Problem(s) studied

Anemia, Sickle Cell

Acute Lymphoblastic Leukemia

Diamond Blackfan Anemia

Lymphoma, Non-Hodgkin

Primary Immunodeficiency

Leukemia, Acute Myeloid (AML), Child

Fanconi Anemia

Osteopetrosis

Thalassemia

Cytopenia

Hemoglobinopathies

Anemia, Aplastic

Myelodysplastic Syndrome

Intervention(s)

Biological: BPX-501 T cells

Drug: Rimiducid

Primary Outcome(s)

Event-free Survival (EFS) at 180 Days After Transplant [Time Frame: 180 days after transplant]

Secondary ID(s)

BP-004

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 04/01/2023
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02065869