Safety/PK Study of Gene Modified Donor T Cell Infusion in Children With Recurrent Hem Malignancies After Allo Transplant

NCT03459170 | Active Not Recruiting Phase 1 FDA Drug

• 1 other identifier: BP-I-008
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 3

Brief Summary

Phase I, open-label, non-randomized study of safety, pharmacokinetics and efficacy of donor BPX-501 T cell infusion in children with recurrent or minimal residual disease (MRD) hematologic malignancies post-allogeneic transplant. The study will consist of the Main Study and an optional Pharmacokinetics (PK) Sub-Study.

Conditions

Hematologic Malignancy

Keywords

leukemia; myelodysplastic syndrome; lymphoma; minimal residual disease; BPX-501; AP1903; rimiducid

Outcome Measures

Primary Outcomes (2)

• BPX-501 Safety

  Incidence of treatment emergent adverse events of 2 stratified dose levels of BPX-501 T cell infusions based on patient-donor match in pediatric subjects with hematologic malignancies

  Month 24

• Mean plasma concentration

  Measure plasma concentrations of rimiducid (AP1903) at two doses (Arm 1: 0.04mg/kg; Arm 2: 0.4mg/kg) in pediatric subjects, during and after a 2-hour infusion

  pre-dose, 30 min, 2 hours and 8 hours after start of infusion

Secondary Outcomes (2)

• Overall survival

  Month 24

• Response Rate

  Month 24

Study Arms (1)

BPX-501 T cells and rimiducid

EXPERIMENTAL

All subjects will receive 3 courses of BPX-501 T cell infusions at escalating dose levels (DL). DL1 on Day 0, DL2 on Days 30 and 60. The first dose of BPX-501 T cells will occur ≥30 days after hematopoietic stem cell transplant (HSCT). Two doses of AP1903 ( 0.1 mg/kg and 0.4 mg/kg) will be investigated for the treatment of aGvHD after BPX-501 T cell infusion.

Biological: BPX-501 T cells; Drug: rimiducid

Interventions

BPX-501 T cells BIOLOGICAL

Biological: T cells transduced with CaspaCIDe® safety switch

Also known as: rivogenlecleucel

BPX-501 T cells and rimiducid

rimiducid DRUG

administered to eliminate BPX-501 cells in the event of GVHD

Also known as: AP1903

BPX-501 T cells and rimiducid

Eligibility Criteria

• Age: 1 Year - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Patients aged \< 18
• Clinical diagnosis of one of the following pediatric hematological malignancies:
• High-risk Acute Leukemia (Acute lymphoblastic leukemia \[ALL\] or acute myeloid leukemia \[AML\]) in any CR
• Acute Leukemia that is minimal residual disease (MRD) positive at \> 1copy per 1 x 10,000 reference copies pre-HSCT
• Myelodysplastic Syndrome (MDS)
• Hodgkin or Non-Hodgkin lymphomas
• Other high-risk hematological malignancy in CR eligible for stem cell transplantation per institutional standard
• Patients with a hematological malignancy who have received a prior allogeneic HSCT
• Patients with on-treatment relapse of AML within 6 months of initial CR
• Patients relapsing within 6 months of initial diagnosis of hematological malignancy.
• Planned or previous treatment of hematological malignancy with one of the following:
• Matched related HSCT
• Mismatched related HSCT
• For patients who have received a transplant, occurrence of one of the following \> 30 days post-HSCT:
• Minimal residual disease (MRD) positive at \> 1 copy per 1 x 10,000 reference copies post-HSCT

You may not qualify if:

• ≥ Grade II acute GVHD or moderate to severe chronic GVHD due to a previous allograft at the time of screening;
• Active CNS involvement by malignant cells (\< 2 months prior to time of consent);
• Current uncontrolled bacterial, viral or fungal infection (currently taking medication with evidence of progression of clinical symptoms or radiologic findings).
• Positive HIV serology or viral RNA;
• Pregnancy (positive serum βHCG test) or breast-feeding female;
• Patients of reproductive age unwilling to use effective forms of birth control or abstinence for a year after BPX-501 T cell infusion.

Sponsors & Collaborators

• Bellicum Pharmaceuticals: lead

Study Sites (3)

San Matteo Hospital

Pavia, 27100, Italy

Location

IRCCS Ospedale Pediatrico Bambino Gesù

Rome, 00161, Italy

Location

Ospedale Infantile Regina Margherita

Turin, 10126, Italy

Location

MeSH Terms

Conditions

Hematologic Neoplasms; Leukemia; Myelodysplastic Syndromes; Lymphoma; Neoplasm, Residual

Interventions

AP 1903 reagent

Condition Hierarchy (Ancestors)

Neoplasms by Site; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Neoplasms by Histologic Type; Bone Marrow Diseases; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases; Neoplastic Processes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Bellicum Pharmaceuticals

  Bellicum Pharmaceuticals, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 23, 2018
• First Posted: March 8, 2018
• Study Start: February 1, 2018
• Primary Completion: September 1, 2022
• Study Completion (Estimated): September 1, 2035
• Last Updated: July 12, 2022

Record last verified: 2022-07

Locations

IT (3)