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Graft -Versus-host-diseasePhase 2NCT07246031

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

A Phase 2 study testing BPC-2001 in people with Graft -Versus-host-disease and aGVHD, run by BioPhoenix Co., Ltd. at 2 sites in China.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial 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 more about this study

Who is it for?
Ages 18 to 65 years
What is being tested?
BPC-2001
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
50 participants
Where
2 sitesChina
Study dates
29 Oct 2025to 31 Dec 2028
Phase
Phase 2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 65 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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:
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  • 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.

Who cannot join

  • Any subjects who meet any of the following criteria will be excluded from study entry:
  • Has had any other prior organ transplantation.
  • Planned use of any additional or alternative drugs for GvHD prophylaxis than listed in the inclusion criteria.
  • 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.
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  • 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.
  • The transplant donor is the subject's mother or collateral relative.

Trial locations and contacts

2 sites in 1 country. Contact the study team or a site near you to ask about taking part.

China2 sites, 2 recruiting
  • Peking University People's Hospital

    Beijing, Beijing Municipality, 100044

    Xiaodong Mo, PhD, +86 13810096698, mxd453@163.com

    Xiaodong Mo, PhD

    Recruiting
  • Tongji Medical College of Huazhong University of Science and Technology

    Wuhan

    Nicole Shih, +886289788901 ext. 103, nicoleshih@biophoenixco.com

    Yang Cao, PhD

    Recruiting

Other trials in China

About this study

Official title: 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).

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.

Being tested
BPC-2001
Keywords
aGvHD, Haplo-SCT, BPC2001, BioPhoenix, KRN-7000
Medical subject headings
Graft vs Host Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • BPC-2001Drug

    Also called KRN-7000

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

Study groups in NCT07246031
Study groupRoleWhat they receive
Safety run-in and ExpansionExperimentalSafety 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.
Receives: Drug: BPC-2001

What is being measured

The primary outcome is the main question the study is designed to answer.

  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)

    Measured: 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)

    Measured: 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)

    Measured: 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)

    Measured: 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

    Measured: 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

    Measured: 1 year post-transplant

Secondary outcomes (23)
  1. Grades II-IV aGVHD

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

    Measured: Day 180 post-transplant

  2. Total and moderate-severe cGvHD

    Incidence of total and moderate-severe cGvHD assessment

    Measured: 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

    Measured: 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

    Measured: 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

    Measured: Day 180 and 1 year post-transplant

  6. Overall Survival (OS)

    The probability of survival will be estimated

    Measured: 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

    Measured: 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

    Measured: 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

    Measured: 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

    Measured: 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

    Measured: 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

    Measured: 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

    Measured: 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

    Measured: 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

    Measured: 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

    Measured: 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

    Measured: 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

    Measured: 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

    Measured: 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

    Measured: 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

    Measured: 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

    Measured: 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

    Measured: Day 0 through Day 35

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted24 Nov 2025
  2. Study start29 Oct 2025
  3. Primary completion30 Apr 2028 (estimated)
  4. Study completion31 Dec 2028 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Prevention
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
50 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07246031
Sponsor's protocol ID
BPC2001-01
Status last verified
Aug 2026
First submitted
22 Sep 2025
Last updated
17 Aug 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

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.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing BPC-2001 in people with Graft -Versus-host-disease and aGVHD, run by BioPhoenix Co., Ltd. at 2 sites in China.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 22 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 17 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Nicole Shih, MSC (+886-2-8978 8901 ext. 103, nicoleshih@biophoenixco.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Grades II-IV aGVHD (time frame: Day 100 after the last infusion of stem cell). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT07246031, which also lists 7 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Aug 2026.

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