A Preliminary Clinical Study on the Efficacy of Cyclophosphamide and Sirolimus Combined With VIC-… (NCT07350057) | Clinical Trial Compass
Not Yet RecruitingPhase 1
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
9 participantsStarted 2026-03-25
Plain-language 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
Who can participate
Age range
8 Years – 18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. 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);
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
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)
Timeframe: Expected 2 years
Trial details
NCT IDNCT07350057
SponsorShanghai Jiao Tong University School of Medicine
. 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:
Exclusion criteria
. 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;