NCT05457010

Phase I Study of Cell Therapies for the Treatment of Patients With Relapsed or Refractory AML or High-risk MDS

Master Protocol for the Phase 1 Study of Cell Therapies for the Treatment of Patients With Relapsed or Refractory Acute Myeloid Leukemia or High-risk Myelodysplastic Syndrome, Including Long-term Safety Follow-up

• Status: Unknown
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 24 (estimated)
• Sponsor: Arcellx, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to evaluate the safety and preliminary activity of ARC-T cells and SPRX002 in participants with relapsed or refractory acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS)

Detailed description

This is a Phase I open-label, safety, and dose-escalation study of ARC-T cells and SPRX002 in participants with relapsed or refractory AML or high-risk MDS. The study will have the following sequential phases: screening, enrollment, pretreatment with lymphodepletion (LD) chemotherapy, treatment with SPRX002 and ARCT cells, treatment extension with SPRX002, follow up, and long-term safety follow-up. Following a single infusion of SPRX002 and ARC T (Day 0) and followed by regular administration of SPRX002 at the assigned dose level, both safety and efficacy data will be assessed. Dose limiting toxicities (DLTs) will be assessed through Day 28 and safety data will be collected throughout the study. Long-term safety data will be collected for up to 15 years per health authority guidelines. Efficacy will be assessed monthly for the first 6 months, then quarterly up to 2 years, or upon subject relapse. ARC-T cells are a genetically modified autologous T-cell product. The T cell has been transduced using a third-generation lentiviral vector encoding a binding domain (referred to as AF101) chimeric antigen receptor (CAR), followed by CD8 spacer and transmembrane region that is fused to the intracellular signaling domains 4-1BB and CD3ζ. AF101 specifically binds to the "TAG" protein (referred to as Q26) of the soluble protein antigen-receptor x-linker (sparX; specifically, SPRX002).

Conditions

• Acute Myeloid Leukemia
• Myelodysplastic Syndromes

Interventions

Type	Name	Description
BIOLOGICAL	SPRX002	SPRX002 is a soluble protein with a "TAG" region to which ARC-T cells bind and a binding region targeting CD123
BIOLOGICAL	ARC-T Cells	ARC-T Cells is a genetically modified autologous T-cell product. The T cell has a binding domain chimeric antigen receptor (CAR), which specifically binds to the "TAG" protein of the soluble protein antigen-receptor x-linker (sparX; specifically, SPRX002). When ARC-T cells bind to a "TAG" and the SPRX protein is bound to its target (in this case CD123), ARC-T cells are capable of activation, expansion, and killing (based on preclinical experiments).

Timeline

• Start date: 2022-11-28
• Primary completion: 2025-09-30
• Completion: 2025-11-17
• First posted: 2022-07-13
• Last updated: 2024-06-12

Locations

5 sites across 1 country: United States

Regulatory

• FDA-regulated drug study