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

NCT ID: NCT07246031

Last Updated: 2025-11-24

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 50 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-10-29
• Study Completion Date: 2028-02-28

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).

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.

Conditions

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

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: PREVENTION
• Blinding Strategy: NONE

Study Groups

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.

Group Type: EXPERIMENTAL

BPC-2001

Intervention Type: DRUG

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

Interventions

BPC-2001

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

Intervention Type: DRUG

Other Intervention Names

KRN-7000

Eligibility Criteria

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.

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.

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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

BioPhoenix Co., Ltd.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Xiaodong Mo, PhD

Role: STUDY_CHAIR

Peking University People's Hospital

Locations

Peking University People's Hospital

Beijing, Beijing Municipality, China

Site Status: RECRUITING

Countries

China

Central Contacts

Nicole Shih, MSC

Role: CONTACT

nicoleshih@biophoenixco.com

+886-2-2542-6789 ext. 503

Facility Contacts

Xiaodong Mo, PhD

Role: primary

mxd453@163.com

+86 13810096698

References

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

Reference Type: 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.

Reference Type: BACKGROUND

PMID: 30833742 (View on PubMed)

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.

Reference Type: BACKGROUND

PMID: 24916508 (View on PubMed)

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.

Reference Type: BACKGROUND

PMID: 21145405 (View on PubMed)

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.

Reference Type: BACKGROUND

PMID: 19713461 (View on PubMed)

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.

Reference Type: BACKGROUND

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

Reference Type: BACKGROUND

Other Identifiers

BPC2001-01

Identifier Type: -

Identifier Source: org_study_id