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RecruitingNCT07246031Updated Aug 17, 2026

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

A Phase 2 interventional study of BPC-2001 in Graft -Versus-host-disease, aGVHD and Haploidentical Stem Cell Transplantation, sponsored by BioPhoenix Co., Ltd.. Recruiting at 2 sites in China. Open to participants aged 18 Years to 65 Years. Per ClinicalTrials.gov, last updated 2026-08-17.

Sponsored by BioPhoenix Co., Ltd. · Phase 2, Interventional, and Prevention

From the registry’s dates

  • Started Oct 2025; still recruiting 11 months later.
Phase
Phase 2
Study type
Interventional
Enrollment
50
Allocation
Not applicable
Ages
18 Years to 65 Years
Sex
All
01

Study 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).

Read the detailed description

This is an open-label, single center, single-arm study to evaluate six weekly doses of BPC2001 in combination with standard of care treatment (Beijing Protocol) for the prevention of aGvHD in subjects following Haplo-SCT. The study includes a Safety Run-in Phase to assess the safety and tolerability of 30 days DLT after the first dose of BPC2001 followed by an Expansion Phase in which the efficacy of 6 weekly doses of BPC2001 in addition to standard of care for GvHD prophylaxis will be assessed.

02

Conditions studied

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

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Keywords

  • aGvHD
  • Haplo-SCT
  • BPC2001
  • BioPhoenix
  • KRN-7000
03

In context

Graft vs Host Disease

806 studies on the registry are indexed under Graft vs Host Disease; 138 are open to participants now.

This study's planned enrollment of 50 is above the median of 35 across 637 interventional studies indexed under Graft vs Host Disease.

Browse Graft vs Host Disease studies →

Lead sponsor

This is the only study on the registry with BioPhoenix Co., Ltd. as lead sponsor.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years to 65 Years
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  1. Male or female ages ≥18 and ≤ 65 years.
  2. 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).

  3. Subjects have a hematologic malignancy as defined below and are considered candidates for haplo-SCT:

    1. Acute leukemia with morphologic complete remission (acute myelogenous leukemia [AML] or acute lymphoblastic leukemia [ALL]);
    2. Myelodysplastic syndrome (MDS), chronic myelomonocytic leukemia (CMML), or myeloproliferative neoplasm (MPN) with \< 10% blasts in the bone marrow.

  4. Organ function tolerated for transplantation:

    1. Cardiac function: Left ventricular ejection fraction at rest ≥ 45%;
    2. 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;
    3. Serum creatine \< 2 mg/dL or estimated creatinine clearance > 50 mL/min calculated using the Cockcroft-Gault equation；
    4. 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%.
  5. Subject is suitable for myeloablative haplotype related donor transplant.
  6. Subject is suitable for receiving first alloHSCT.

  7. The transplant donor must meet the following criteria:

    1. Donor ages > 30 years; If the donor ages is equal to or less than 30 years, the donor should be female for male subject;
    2. High-resolution typing of human leukocyte antigen (HLA)-A, -B, -C, DR, and DQ are matched at least 5/10;
    3. Meet the criteria for peripheral blood stem cell (PBSC) donation;
    4. Donor's specific antibodies are negative, \<2,000 MFI.
  8. Source of allografts: using G-CSF as the mobilizing agent to mobilize PBSC transplant; bone marrow or cord blood is not allowed.
  9. Karnofsky Performance Status (KPS) score ≥ 60 points.
  10. Is a Candidate for anti-GvHD prophylaxis, including ATG, calcineurin inhibitor (CsA or tacrolimus [FK 506]) in combination with MTX and MMF.
  11. 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.
  12. 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.

Exclusion criteria

Exclusion Criteria:

Any subjects who meet any of the following criteria will be excluded from study entry:

  1. Has had any other prior organ transplantation.
  2. Planned use of any additional or alternative drugs for GvHD prophylaxis than listed in the inclusion criteria.
  3. 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.
  4. Has other malignancies that are not controlled.
  5. Has evidence of active central nervous system (CNS) disease.
  6. Patients with uncontrolled active bacterial, viral, or fungal infections.
  7. Known history of human immunodeficiency virus (HIV) or positive HIV antibody test.
  8. 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.
  9. Pregnant or lactating females.
  10. Has undergone major surgery within 1 month prior to the first dose of investigational drug.
  11. In the opinion of the investigator, the subject has any other medical condition that renders the subject unsuitable for participation in the study.
  12. Has a history of uncontrolled autoimmune disease or on active treatment.
  13. Vaccinated with live or attenuated vaccine within 4 weeks prior to the first dose of investigational drug.
  14. 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.
  15. Plan to use prophylaxis donor lymphocyte infusion (DLI) therapy.
  16. The transplant donor is the subject's mother or collateral relative.
05

Study design

Phase
Phase 2
Primary purpose
Prevention
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
50 participants (estimated)

Study arms

  • Experimental
    Safety run-in and Expansion

    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

  • DrugBPC-2001

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

    Also known as: KRN-7000

06

What researchers measure

Primary outcomes

  1. 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)

    Time frame: Day 100 after the last infusion of stem cell

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

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

    Time frame: 1 year post-transplant

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

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

    Time frame: 1 year post-transplant

  4. 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)

    Time frame: 1 year post-transplant

  5. 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

    Time frame: 1 year post-transplant

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

    Incidence, nature, and severity of graft failure

    Time frame: 1 year post-transplant

Secondary outcomes

  1. Grades II-IV aGVHD

    Acute GVHD will be graded and assessed within 180 days post-transplant

    Time frame: Day 180 post-transplant

  2. Total and moderate-severe cGvHD

    Incidence of total and moderate-severe cGvHD assessment

    Time frame: Day 180 and 1 year post-transplant

  3. Non-relapse Mortality (NRM) Rates

    The probability of mortality not preceded by relapse of the underlying malignancy will be estimated

    Time frame: Day 100, Day 180 and 1 year post-transplant

  4. Disease-free Survival (DFS)

    The probability of survival without relapse of the underlying malignancy will be estimated

    Time frame: Day 180 and 1 year post-transplant

  5. GvHD-free, Relapse Free Survival (GRFS)

    The probability of survival without relapse of the underlying malignancy, without severe (grades 3-4) acute GVHD, and without chronic GVHD requiring systemic immunosuppression will be estimated

    Time frame: Day 180 and 1 year post-transplant

  6. Overall Survival (OS)

    The probability of survival will be estimated

    Time frame: Day 180 and 1 year post-transplant

  7. PK Profile_Cmax after a single dose of BPC2001

    To calculate the PK parameters of the total drug and free drug (if feasible) in the plasma sample, including but not limited to: maximum observed concentration (Cmax) after a single dose

    Time frame: Day 0 through Day 35

  8. PK Profile_Tmax after a single dose of BPC2001

    To calculate the PK parameters of the total drug and free drug (if feasible) in the plasma sample, including but not limited to: time to maximum concentration (Tmax) after a single dose

    Time frame: Day 0 through Day 35

  9. PK Profile_AUC0-t after a single dose of BPC2001

    To calculate the PK parameters of the total drug and free drug (if feasible) in the plasma sample, including but not limited to: area under the concentration versus time curve from time 0 to the time point of the last measurable concentration (AUC0-t) after a single dose

    Time frame: Day 0 through Day 35

  10. PK Profile_AUC0-inf after a single dose of BPC2001

    To calculate the PK parameters of the total drug and free drug (if feasible) in the plasma sample, including but not limited to: area under the concentration versus time curve from time 0 extrapolated to infinite (AUC0-inf) after a single dose

    Time frame: Day 0 through Day 35

  11. PK Profile_t1/2 after a single dose of BPC2001

    To calculate the PK parameters of the total drug and free drug (if feasible) in the plasma sample, including but not limited to: terminal half-life (t1/2) after a single dose

    Time frame: Day 0 through Day 35

  12. PK Profile_CL after a single dose of BPC2001

    To calculate the PK parameters of the total drug and free drug (if feasible) in the plasma sample, including but not limited to: clearance (CL) after a single dose

    Time frame: Day 0 through Day 35

  13. PK Profile_Vd after a single dose of BPC2001

    To calculate the PK parameters of the total drug and free drug (if feasible) in the plasma sample, including but not limited to: volume of distribution (Vd) after a single dose

    Time frame: Day 0 through Day 35

  14. PK Profile_Cmax after multiple doses of BPC2001

    To calculate the PK parameters of the total drug and free drug (if feasible) in the plasma sample, including but not limited to: Cmax after multiple doses

    Time frame: Day 0 through Day 35

  15. PK Profile_Tmax after multiple doses of BPC2001

    To calculate the PK parameters of the total drug and free drug (if feasible) in the plasma sample, including but not limited to: Tmax after multiple doses

    Time frame: Day 0 through Day 35

  16. PK Profile_AUC0-t after multiple doses of BPC2001

    To calculate the PK parameters of the total drug and free drug (if feasible) in the plasma sample, including but not limited to: AUC0-t after multiple doses

    Time frame: Day 0 through Day 35

  17. PK Profile_AUC0-inf after multiple doses of BPC2001

    To calculate the PK parameters of the total drug and free drug (if feasible) in the plasma sample, including but not limited to: AUC0-inf after multiple doses

    Time frame: Day 0 through Day 35

  18. PK Profile_t1/2 after multiple doses of BPC2001

    To calculate the PK parameters of the total drug and free drug (if feasible) in the plasma sample, including but not limited to: t1/2 after multiple doses

    Time frame: Day 0 through Day 35

  19. PK Profile_Ctrough after multiple doses of BPC2001

    To calculate the PK parameters of the total drug and free drug (if feasible) in the plasma sample, including but not limited to: trough concentration (Ctrough) after multiple doses

    Time frame: Day 0 through Day 35

  20. PK Profile_CLss after multiple doses of BPC2001

    To calculate the PK parameters of the total drug and free drug (if feasible) in the plasma sample, including but not limited to: clearance at steady state (CLss) after multiple doses

    Time frame: Day 0 through Day 35

  21. PK Profile_Vss after multiple doses of BPC2001

    To calculate the PK parameters of the total drug and free drug (if feasible) in the plasma sample, including but not limited to: volume of distribution at steady state (Vss) after multiple doses

    Time frame: Day 0 through Day 35

  22. PK Profile_ARCmax after multiple doses of BPC2001

    To calculate the PK parameters of the total drug and free drug (if feasible) in the plasma sample, including but not limited to: accumulation factors ARCmax after multiple doses

    Time frame: Day 0 through Day 35

  23. PK Profile_ARAUC after multiple doses of BPC2001

    To calculate the PK parameters of the total drug and free drug (if feasible) in the plasma sample, including but not limited to: ARAUC after multiple doses

    Time frame: Day 0 through Day 35

07

Study locations

2 of 2 sites recruiting
  • Peking University People's Hospital
    Beijing, Beijing Municipality 100044, China
    • Xiaodong Mo, PhD · Contact · mxd453@163.com · +86 13810096698
    • Xiaodong Mo, PhD · Principal investigator
    Recruiting
  • Tongji Medical College of Huazhong University of Science and Technology
    Wuhan, China
    Recruiting
08

References and documents

Publications

  • 7.General Office of National Health Commission. Clinical Application Management Standards for Allogeneic Hematopoietic Stem Cell Transplantation Technology (2022 Edition).
  • 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. PubMed 30833742 ↗
  • 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. PubMed 24916508 ↗
  • 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. PubMed 21145405 ↗
  • 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. PubMed 19713461 ↗
  • Hematopoietic Stem Cell Application Group, Chinese Society of Hematology, Chinese Medical Association. [Chinese expert consensus on the diagnosis and treatment of acute graft-versus-host disease after hematopoietic stem cell transplantation (2024)]. Zhonghua Xue Ye Xue Za Zhi. 2024 Jun 14;45(6):525-533. doi: 10.3760/cma.j.cn121090-20240608-00214. Chinese. PubMed 39134482 ↗
  • 1. Huang Xiaojun. Haploidentical Hematopoietic Stem Cell Transplantation Beijing Protocol, Chinese Journal of Organ Transplantation. 2017;38(2): 65-68.

Individual participant data

Plan to share: No

09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Aug 17, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT07246031
Lead sponsor
BioPhoenix Co., Ltd.
Responsible party
Sponsor
First posted
Nov 24, 2025
Start date
Oct 29, 2025
Primary completion
Apr 30, 2028 (estimated)
Completion
Dec 31, 2028 (estimated)
Last update
Aug 17, 2026

Study contacts

Nicole Shih, MSC
Contact
nicoleshih@biophoenixco.com
+886-2-8978 8901 ext. 103
Xiaodong Mo, PhD
study chair · Peking University People's Hospital

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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