BPC2001 for the Prevention of Acute Graft-Versus-Host Disease Following Haploidentical Stem Cell Transplantation

NCT07246031 | Recruiting Phase 2

• 1 other identifier: BPC2001-01
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 1

Brief Summary

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).

Conditions

Graft -Versus-host-disease; aGVHD; Haploidentical Stem Cell Transplantation; cGVHD

Keywords

aGvHD; Haplo-SCT; BPC2001; BioPhoenix; KRN-7000

Outcome Measures

Primary Outcomes (6)

• Grades II-IV aGVHD

  To assess the incidence of Grades II-IV aGvHD by Day 100 (D100) after the last infusion of stem cell (the Mount Sinai aGvHD International Consortium \[MAGIC\] criteria)

  Day 100 after the last infusion of stem cell

• AE of BPC2001 in addition to the graft-versus-host disease (GvHD) prophylaxis regimen

  Incidence, nature, and severity of treatment-emergent adverse events (AEs)

  1 year post-transplant

• SAE of BPC2001 in addition to the graft-versus-host disease (GvHD) prophylaxis regimen

  Incidence, nature, and severity of serious adverse events (SAEs)

  1 year post-transplant

• Lab test values of BPC2001 in addition to the graft-versus-host disease (GvHD) prophylaxis regimen

  Incidence, nature, and severity of laboratory test values (complete blood count, serum chemistry test, coagulation test and urinalysis)

  1 year post-transplant

• Vital sign of BPC2001 in addition to the graft-versus-host disease (GvHD) prophylaxis regimen

  Incidence, nature, and severity of vital sign measures including temperature, blood pressure (systolic/diastolic), pulse, and respiratory rate

  1 year post-transplant

• Graft failure of BPC2001 in addition to the graft-versus-host disease (GvHD) prophylaxis regimen

  Incidence, nature, and severity of graft failure

  1 year post-transplant

Secondary Outcomes (23)

• Grades II-IV aGVHD

  Day 180 post-transplant

• Total and moderate-severe cGvHD

  Day 180 and 1 year post-transplant

• Non-relapse Mortality (NRM) Rates

  Day 100, Day 180 and 1 year post-transplant

• Disease-free Survival (DFS)

  Day 180 and 1 year post-transplant

• GvHD-free, Relapse Free Survival (GRFS)

  Day 180 and 1 year post-transplant

Study Arms (1)

Safety run-in and Expansion

EXPERIMENTAL

Safety Run-in Phase: Up to 2 cohorts of at least 3 subjects will be enrolled in the Safety Run-in Phase. The Safety Run in Phase will enroll at least 6 subjects in total. Initially, 3 subjects will be enrolled, treated, and assessed for the dose-limiting toxicity (DLT). Expansion Phase: Once a dose/schedule with an acceptable safety/PK profile is determined by the SRC, enrollment will continue to the Expansion Phase. The Expansion Phase will enroll 44 subjects. The dose of BPC2001 will be tentatively 100 μg/kg on a weekly basis for 6 doses, and the specific dose and/or schedule will be determined by the SRC based on the data of the Safety Run-in Phase.

Drug: BPC-2001

Interventions

BPC-2001 DRUG

Subjects will receive 6 weekly doses of BPC2001, 100 μg/kg via IV administration after completion of Haplo-SCT.

Also known as: KRN-7000

Safety run-in and Expansion

Eligibility Criteria

• Age: 18 Years - 65 Years
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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;

You may not qualify if:

• Any subjects who meet any of the following criteria will be excluded from study entry:
• Has had any other prior organ transplantation.
• Has had received an investigational drug within 4 half-lives or within 14 days prior to HSCT, whichever is longer; or plans to participate in another clinical study prior to completion of all scheduled evaluations in this clinical study.
• Has other malignancies that are not controlled.
• Has evidence of active central nervous system (CNS) disease.
• Patients with uncontrolled active bacterial, viral, or fungal infections.
• Known history of human immunodeficiency virus (HIV) or positive HIV antibody test.
• Hepatitis B virus surface antigen (HBsAg) or hepatitis B virus core antibody (HBcAb) is positive, and the hepatitis B virus (HBV) DNA in peripheral blood is above the limit of quantification; or hepatitis C virus (HCV) antibody and peripheral HCV RNA are positive; or the syphilis TRUST test is positive.
• Pregnant or lactating females.
• Has undergone major surgery within 1 month prior to the first dose of investigational drug.
• In the opinion of the investigator, the subject has any other medical condition that renders the subject unsuitable for participation in the study.
• Has a history of uncontrolled autoimmune disease or on active treatment.
• Vaccinated with live or attenuated vaccine within 4 weeks prior to the first dose of investigational drug.
• History of myocardial infarction, unstable angina, acute coronary syndrome, congestive heart failure (New York Heart Society classification ≥ class Ⅲ), or clinically significant arrhythmia within 6 months prior to receiving the investigational drug.
• Plan to use prophylaxis donor lymphocyte infusion (DLI) therapy.

Sponsors & Collaborators

• BioPhoenix Co., Ltd.: lead

Study Sites (1)

Peking University People's Hospital

Beijing, Beijing Municipality, 100044, China

RECRUITING

Related Publications (7)

• 7.General Office of National Health Commission. Clinical Application Management Standards for Allogeneic Hematopoietic Stem Cell Transplantation Technology (2022 Edition).

  BACKGROUND

• Ciurea SO, Al Malki MM, Kongtim P, Fuchs EJ, Luznik L, Huang XJ, Ciceri F, Locatelli F, Aversa F, Castagna L, Bacigalupo A, Martelli M, Blaise D, Ben Soussan P, Arnault Y, Handgretinger R, Roy DC, O'Donnell PV, Bashey A, Solomon S, Romee R, Gayoso J, Lazarus HM, Ballen K, Savani BN, Mohty M, Nagler A. The European Society for Blood and Marrow Transplantation (EBMT) consensus recommendations for donor selection in haploidentical hematopoietic cell transplantation. Bone Marrow Transplant. 2020 Jan;55(1):12-24. doi: 10.1038/s41409-019-0499-z. Epub 2019 Mar 4.

  PMID: 30833742 BACKGROUND

• Wang Y, Chang YJ, Xu LP, Liu KY, Liu DH, Zhang XH, Chen H, Han W, Chen YH, Wang FR, Wang JZ, Chen Y, Yan CH, Huo MR, Li D, Huang XJ. Who is the best donor for a related HLA haplotype-mismatched transplant? Blood. 2014 Aug 7;124(6):843-50. doi: 10.1182/blood-2014-03-563130. Epub 2014 Jun 10.

  PMID: 24916508 BACKGROUND

• Duramad O, Laysang A, Li J, Ishii Y, Namikawa R. Pharmacologic expansion of donor-derived, naturally occurring CD4(+)Foxp3(+) regulatory T cells reduces acute graft-versus-host disease lethality without abrogating the graft-versus-leukemia effect in murine models. Biol Blood Marrow Transplant. 2011 Aug;17(8):1154-68. doi: 10.1016/j.bbmt.2010.11.022. Epub 2010 Dec 8.

  PMID: 21145405 BACKGROUND

• Socie G, Blazar BR. Acute graft-versus-host disease: from the bench to the bedside. Blood. 2009 Nov 12;114(20):4327-36. doi: 10.1182/blood-2009-06-204669. Epub 2009 Aug 27.

  PMID: 19713461 BACKGROUND

• 2. Hematology Branch Stem Cell Application Group of Chinese Medical Association. Expert Consensus on Diagnosis and Treatment of Acute Graft-versus-Host Disease After Allogeneic Hematopoietic Stem Cell Transplantation (2024 Edition). Chinese Journal of Hematology. 2024;45(6):525-533.

  BACKGROUND

• 1. Huang Xiaojun. Haploidentical Hematopoietic Stem Cell Transplantation Beijing Protocol, Chinese Journal of Organ Transplantation. 2017;38(2): 65-68.

  BACKGROUND

MeSH Terms

Conditions

Graft vs Host Disease

Interventions

KRN 7000

Condition Hierarchy (Ancestors)

Immune System Diseases

Study Officials

• Xiaodong Mo, PhD

  Peking University People's Hospital

  STUDY CHAIR

Central Study Contacts

Nicole Shih, MSC

CONTACT

+886-2-2542-6789; nicoleshih@biophoenixco.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 22, 2025
• First Posted: November 24, 2025
• Study Start: October 29, 2025
• Primary Completion (Estimated): November 2, 2027
• Study Completion (Estimated): February 28, 2028
• Last Updated: November 24, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)