An Open-Label, Single-Arm, Phase Ⅱb Clinical Study of BPC2001 for the Prevention of Acute Graft-Versus-Host Disease Following Haploidentical Stem Cell Transplantation
A Phase IIb open label study evaluates the safety and efficacy of repeat doses of BPC2001 in combination with standard of care treatment for the prevention of acute graft-vs-host-disease (aGvHD) in subjects following Haploidentical Stem Cell Transplantation (Haplo-SCT).
• Male or female ages ≥18 and ≤ 65 years.
• Before the start of the trial, the subject or his/her guardian is sufficient to understand and voluntarily sign the written informed consent form (ICF).
• Subjects have a hematologic malignancy as defined below and are considered candidates for haplo-SCT:
∙ Acute leukemia with morphologic complete remission (acute myelogenous leukemia \[AML\] or acute lymphoblastic leukemia \[ALL\]);
‣ Myelodysplastic syndrome (MDS), chronic myelomonocytic leukemia (CMML), or myeloproliferative neoplasm (MPN) with \< 10% blasts in the bone marrow.
• Organ function tolerated for transplantation:
∙ Cardiac function: Left ventricular ejection fraction at rest ≥ 45%;
‣ Liver function: Total bilirubin \< 1.5 × upper limit of normal (ULN); alanine aminotransferase (ALT) and aspartate aminotransferase (AST) \< 2.5 × ULN. Subjects who have been diagnosed with Gilbert's syndrome or malignant disease involvement are allowed to have a total bilirubin value \> 1.5 × ULN;
‣ Serum creatine \< 2 mg/dL or estimated creatinine clearance \> 50 mL/min calculated using the Cockcroft-Gault equation；
‣ Pulmonary function tests (PFTs): diffusing capacity of the lung for carbon monoxide (DLCO) (corrected for hemoglobin) and/or forced expiratory volume in 1 second (FEV1) ≥ 50%.
• Subject is suitable for myeloablative haplotype related donor transplant.
• Subject is suitable for receiving first alloHSCT.
• The transplant donor must meet the following criteria:
∙ Donor ages \> 30 years; If the donor ages is equal to or less than 30 years, the donor should be female for male subject;
‣ High-resolution typing of human leukocyte antigen (HLA)-A, -B, -C, DR, and DQ are matched at least 5/10;
‣ Meet the criteria for peripheral blood stem cell (PBSC) donation;
‣ Donor's specific antibodies are negative, \<2,000 MFI.
• Source of allografts: using G-CSF as the mobilizing agent to mobilize PBSC transplant; bone marrow or cord blood is not allowed.
• Karnofsky Performance Status (KPS) score ≥ 60 points.
⁃ Is a Candidate for anti-GvHD prophylaxis, including ATG, calcineurin inhibitor (CsA or tacrolimus \[FK 506\]) in combination with MTX and MMF.
⁃ Female subjects of childbearing potential must have a negative serum pregnancy test prior to enrollment and must have agreed to use a double barrier method of contraception from the time of signing the ICF to 90 days after the last dose of investigational drug.
⁃ Male subjects must agree to use effective contraception from the time of signing the ICF to 90 days after the last dose of investigational drug.