NCT03459170

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

A Phase I Study Of Safety, Pharmacokinetics, And Efficacy Of Donor BPX-501 Cells and Rimiducid Infusion For Children With Recurrent Hematologic Malignancies or Minimal Residual Disease After Allogeneic Transplant

• Status: Active Not Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 15 (estimated)
• Sponsor: Bellicum Pharmaceuticals · Industry
• Sex: All
• Age: 1 Year – 18 Years
• Healthy volunteers: Not accepted

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.

Detailed description

Main Study: Approximately 16 subjects will participate in the BPX-501 main study. The treatment consists of three courses of BPX-501 T cell infusions at 30 day intervals with 2 escalating dose levels (DL). DL1 on Day 0; DL2 on Days 30 and 60. Two doses of rimiducid (AP1903) will be investigated for the treatment of aGvHD after BPX-501 T cell infusion. A 0.1mg/kg initial dose of rimiducid which has demonstrated the ability to induce \>50% BPX-501 T cell eradication in preclinical animal models will first be administered in the event of uncontrollable aGvHD. If there is no response to this dose within 24hrs + 12hrs a second dose of 0.4 mg/kg (which has been reported to induce T cell eradication of \> 90%) will be administered. If there is no measurable GvHD response to the initial dose of 0.1 mg/kg rimiducid in 2 subjects, the starting dose of rimiducid will be 0.4 mg/kg for all subsequent subjects. Rimiducid (AP1903) Optional PK Sub-Study: Approximately 12 subjects will be recruited to participate in the optional Rimiducid (AP1903) PK sub-study. Subjects will be assigned to one of two arms and receive either 0.04mg/kg or 0.4mg/kg of Rimiducid (AP1903). Each arm will have a target enrollment of 6 subjects. * Arm 1: 0.04mg/kg Rimiducid (AP1903), 6 subjects; * Arm 2: 0.4mg/kg Rimiducid (AP1903), 6 subjects. Rimiducid PK samples and ECG data will be collected at Pre-dose (0 hour), 30 minutes, 2 hours and 8 hours following the initiation of rimiducid (AP1903) infusion. Efforts shall be made to enroll at least one subject from each age subset into the PK sub-study: infants and toddlers (12 months to 23 months); children (2-11 years); and adolescents (12-18 years).

Conditions

• Hematologic Malignancy

Interventions

Type	Name	Description
BIOLOGICAL	BPX-501 T cells	Biological: T cells transduced with CaspaCIDe® safety switch
DRUG	rimiducid	administered to eliminate BPX-501 cells in the event of GVHD

Timeline

• Start date: 2018-02-01
• Primary completion: 2022-09-01
• Completion: 2035-09-01
• First posted: 2018-03-08
• Last updated: 2022-07-12

Locations

3 sites across 1 country: Italy

Regulatory

• FDA-regulated drug study