Study of Haplo-HSCT + Rivogenlecleucel vs Haplo-HSCT + Post Transplant Cyclophosphamide in Patients With AML or MDS

• Status: Terminated
• Trial end date: 2019-07-23
• Target enrollment: 0
• Participant gender: All

Summary

This study compares the safety and effectiveness of giving rivogenlecleucel (BPX-501 T cells) to patients with AML or MDS post haploidentical hematopoietic stem cell transplant compared to post-transplant cyclophosphamide.

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Bellicum Pharmaceuticals
• Treatments: Cyclophosphamide

Criteria

Inclusion Criteria:

Signed informed consent

Meeting institutional criteria to undergo allogenic HSCT

Age 18-70 y/o (12-70 y/o in US only)

Patients with AML or MDS as defined below:

AML Patients Patients with intermediate to adverse AML as defined by ELN (Dohner, 2017).

• AML in first complete remission (CR1) with high-risk features defined as > 1 cycle of induction therapy required to achieve remission OR preceding MDS or myeloproliferative disease

• AML in CR1 with intermediate-risk features

• AML in second or subsequent complete response

• AML with myelodysplasia-related changes (AML-MRC)

• Therapy related AML in first or subsequent complete remission

• De novo AML in second or subsequent complete remission

MDS Patients

• High or very-high risk MDS by IPSS-R classification

• Intermediate risk or higher MDS patients who failed a hypomethylating agent

Lack of suitable conventional donor (i.e. HLA 10/10 related or unrelated donor)

At least a 5/10 genotypic identical haplotype match

The donor and recipient must be identical, at least one allele of each of the following genetic loci: HLA-A, HLA-B, HLA-C, HLA-DRB1, and HLA-DQB1

Patients with adequate organ function

Eastern Cooperative Oncology Group (ECOG) performance status: 0-2

Exclusion Criteria:

• HLA 10/10 allele matched (HLA-A,-B,-C,-DRBl, and DQB1) related donor or unrelated donor

• Autologous hematopoietic stem cell transplant ≤ 3 months before enrollment

• Prior allogeneic transplantation

• Active CNS involvement by malignant cells (less than 2 months from the conditioning)

• Current uncontrolled clinically active bacterial, viral or fungal infection

• Positive HIV serology or viral RNA

• Pregnancy (positive serum or urine βHCG test) or breast-feeding

• Fertile men or women unwilling to use effective forms of birth control or abstinence for a year after transplantation

• Radiographic, histologic, or known history of cirrhosis

• Overlapping MDS and myeloproliferative neoplasms (MPN) disease

• Patients with acute promyelocytic leukemia (APL)

• Known hypersensitivity to dimethyl sulfoxide (DMSO)