A Preliminary Clinical Study on the Efficacy of Cyclophosphamide and Sirolimus Combined With VIC-1911 in Preventing Graft-versus-Host Disease After Haploidentical Stem Cell Transplantation in Children

NCT07350057 | Not Yet Recruiting Phase 1

• 1 other identifier: JSI-1911-103
• Study Type: interventional
• Target: 9
• Locations: 0 countries
• Sites: N/A

Brief Summary

This study is a single arm, open label Phase Ib clinical trial, consisting of two parts: the first part is a safety introduction trial, and the second part is a dose escalation trial. In the first part, the tolerability, safety, PK characteristics, PD characteristics, and preliminary efficacy of VIC-1911 tablets in combination with PTCy and sirolimus will be explored. The first part will conduct safety introduction tests at the same dose as those already tested abroad to determine the dosage required for VIC-1911 tablets to meet safety and effective biological activity. The dose limiting toxicity (DLT) in Phase I study will be evaluated from the first administration of VIC-1911 tablets to 28 days after administration. On this basis, the second part of the experiment will be conducted to further explore the effectiveness and safety of VIC-1911 tablets combined with PTCy and sirolimus under RP2D for preventing GVHD in haplo HSCT patients after myeloablative pretreatment

Conditions

GVHD (Acute or Chronic)

Outcome Measures

Primary Outcomes (1)

• 1.Determine the recommended phase 2 dose (RP2D) of VIC-1911 tablets combined with post transplant cyclophosphamide (PTCy) and sirolimus for the prevention of GVHD in haplo HSCT pediatric patients (Only evaluated in Part 1)

  1. The incidence of dose limiting toxicity (DLT) during observation period; 2. When used in combination, the RP2D of VIC-1911 tablets. The dose of RP2D should achieve the minimum expected biological efficacy, and the incidence of DLT in patients at this dose should be less than 30%. The minimum expected biological efficacy is 65% of patients with CD4+pH3ser10+T cells\<54% at baseline by day 21 (-3 to+7), based on the lower confidence interval of pre transplant CD4+pH3ser10+T cell frequency (note: pH3ser10 is the phosphoprotein target of Aurora kinase A)

  Expected 2 years

Study Arms (1)

Phase Ib Study of Post-Transplant Cyclophosphamide (PTCy) Combined with Sirolimus and VIC-1911 Table

EXPERIMENTAL

Drug: 1mg /kg VIC-1911

Interventions

1mg /kg VIC-1911 DRUG

VIC-1911 D5-D45 1mg/kg（maximum 75mg）Oral administration on an empty stomach Bid VIC-1911 D5-D45 1.25mg/kg（maximum 75mg）Oral administration on an empty stomach Bid VIC-1911 D5-D45 0.75mg/kg（maximum 75mg）Oral administration on an empty stomach Bid

Also known as: 1.25mg/kg VIC-1911

Phase Ib Study of Post-Transplant Cyclophosphamide (PTCy) Combined with Sirolimus and VIC-1911 Table

Eligibility Criteria

• Age: 8 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Participants must meet all of the following criteria in order to participate in this study:
• Children aged 8-18, regardless of gender;
• Prior to the start of the study, the subjects and their legal guardians shall sign a written informed consent form;
• Patients diagnosed with the following blood diseases and deciding to undergo haplo HSCT:
• Patients diagnosed with acute myeloid leukemia or high-risk acute lymphoblastic leukemia who have achieved complete remission (CR) through induction therapy;;
• Patients diagnosed with myelodysplastic syndrome (MDS) and with an International Prognostic Scoring System (IPSS, please refer to Appendix 1a) score of medium risk 2 or high risk, or with severe blood cell reduction in the IPSS low-risk group, who have undergone ineffective treatment or have poor prognosis due to genetic abnormalities (such as -7,3q26 rearrangement, TP53 gene mutation, complex karyotype, monomeric karyotype);
• There are peripheral blood stem cell donors who are 5/10 HLA haploidentical;
• Karnofsky (KPS) score (see Appendix 2) ≥ 80 points;
• Possess sufficient organ functions, including:
• Serum creatinine ≤ 1.5 x upper limit of normal (ULN) or calculated creatinine clearance rate (using Cockcroft Gault formula, please refer to Appendix 3) ≥ 50ml/min;
• Ferritin\<2000ng/ml;
• Adequate lung function, defined as FEV1, FVC, DLCO ≥ 50% of expected values; Pulmonary function test: FEV1, FVC, DLCO ≥ 50% of expected values;
• Adequate cardiac function, defined as a left ventricular ejection fraction (LVEF) of ≥ 45% evaluated through echocardiography or multiple uptake gated acquisition (MUGA) scans;

You may not qualify if:

• Subjects who meet any of the following criteria will be excluded from participating in this study:
• Hematopoietic stem cell transplantation comorbidity index (HCT-CI)\>4 (Sorror criteria, please refer to Appendix 5);
• Plan to start post transplant maintenance treatment within 75 days after transplantation;
• Those who are allergic to cyclophosphamide, VIC-1911 tablets, sirolimus, and sirolimus derivatives, or any excipient component of the above drugs;
• Those who are unable or unwilling to discontinue other immunosuppressive treatments before the start of the study;
• History of eye diseases, patients with central or branch retinal artery or vein occlusion, accompanied by significant visual impairment, or other retinal diseases determined by ophthalmologists leading to visual impairment;
• Have a serious history of cardiovascular and cerebrovascular diseases, including but not limited to:
• Hypertension that cannot be controlled after standard clinical treatment (systolic blood pressure\>160mmHg or diastolic blood pressure\>100mmHg for more than 4 weeks);
• Severe cardiac rhythm or conduction abnormalities, such as ventricular arrhythmia requiring clinical intervention, II-III degree atrioventricular block, etc; According to the standards of the New York Heart Association (NYHA) in the United States (please refer to Appendix 6), patients with III-IV grade heart failure;
• Any factors that increase the risk of QTc interval prolongation or arrhythmia, such as heart failure, hypokalemia, congenital long QT syndrome, or the use of any known concomitant medication that may prolong the QT interval (see Appendix 7), except for antibiotics used for the prevention or treatment of infections;
• Those who have undergone major surgical procedures within 28 days prior to administration, or those who are expected to undergo major surgery during the trial period;
• During screening, there are clinically significant gastrointestinal abnormalities that may affect the intake, transport, or absorption of drugs (such as swallowing difficulties, uncontrollable nausea and vomiting, active gastric ulcers, ulcerative colitis, Crohn's disease, chronic diarrhea, intestinal obstruction, etc.);
• Have received the drug used in clinical trials or are currently participating in clinical trials involving the drug within 28 days before administration or within 5 half lives of the drug (whichever is longer);
• People who have difficulty with venous blood collection;
• Individuals who are positive for hepatitis B surface antigen (HBsAg) and/or core antibodies and positive for hepatitis B virus deoxyribonucleic acid (HBV DNA), positive for hepatitis C virus (HCV) antibodies and positive for hepatitis C virus ribonucleotide (HCV RNA) testing, positive for Treponema pallidum antibodies, and positive for human immunodeficiency virus antibodies (HIV Ab) during screening;

Sponsors & Collaborators

• Shanghai Jiao Tong University School of Medicine: lead

MeSH Terms

Conditions

Graft vs Host Disease; Bronchiolitis Obliterans Syndrome

Condition Hierarchy (Ancestors)

Immune System Diseases; Organizing Pneumonia; Bronchiolitis Obliterans; Bronchiolitis; Bronchitis; Bronchial Diseases; Respiratory Tract Diseases; Lung Diseases, Obstructive; Lung Diseases

Central Study Contacts

wang xiaodong, PI

CONTACT

189 3869 1232; donlevin@live.cn

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: December 19, 2025
• First Posted: January 20, 2026
• Study Start: March 25, 2026
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): December 1, 2027
• Last Updated: January 20, 2026

Record last verified: 2025-07