Reduced ATG Plus Mini PTCy for GVHD Prophylaxis in Haplo-SCT

NCT06984536 | Recruiting Phase 2 DMC

• 1 other identifier: Lower ATG plus miniPTCy
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

Brief Summary

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is regarded as a curative therapy for a variety of hematological malignancies and nonmalignant diseases. However, donor limitations have restricted the widespread use of allo-HSCT for a long period. The development and success of haploidentical allografts worldwide makes "everyone has a donor" a reality. In the past two decades, researchers have established several haploidentical HSCT (haplo-HSCT) protocols based on different approaches to induce immune tolerance. The representative approaches for haplo-HSCT without in vitro. T cell depletion include granulocyte colony-stimulating factor (G-CSF) plus Anti-human Thymocyte Immunoglobulin (ATG) based (Beijing Protocol) and post-transplantation cyclophosphamide based (PT-Cy, Baltimore Protocol) protocols. Both of two protocols have common problems that need to be solved, including infection transplantation related mortality and disease relapse. The main aim of this study is to explore whether the combined protocol can improve the efficacy of haploidentical transplantation further.

Conditions

Myelodysplastic Syndrome; Acute Leukemia

Outcome Measures

Primary Outcomes (1)

• Non-relapse mortality

  None-relapse mortality within 100 days post transplantation

  100 days

Secondary Outcomes (4)

• Regimen related toxicity

  30 days post transplantation

• Engraftment

  Within 30 days post transplant. Myeloid engraftment was defined as the first of three consecutive days with an ANC 0.5×109 /L, and platelet engraftment was defined as the day the platelet count met or exceeded 20×10^9 /L without transfusion for a week.

• Disease relapse

  1 year post transplantation

• Disease free survival

  1 year post transplantation

Study Arms (1)

Reduced ATG puls mini PTCy

EXPERIMENTAL

Patients recieved ATG 7.5mg/kg plus PTCy 14.5mg/kg on day +3 and +4 for GVHD prophylaxis in haplo-SCT

Drug: Reduced ATG plus mini PTCy

Interventions

Reduced ATG plus mini PTCy DRUG

The conditioning protocol comprises cytarabine (Ara-C) (4 g/m2/day, days -9), busulfan (Bu) (3.2 mg/kg/day, days -8 to -6), cyclophosphamide (Cy) (1.8 g/m2/kg, days -5 and -4), simustine (250 mg/m2, day -3) and r-ATG (total 7.5mg/kg ,from days -5 to -2). Mini PTCy 14.5mg/kg/day will be given on day +3 and +4.

Reduced ATG puls mini PTCy

Eligibility Criteria

• Age: 12 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients with AL - CR and/or myelodysplastic syndromes undergoing allogeneic hematopoietic stem cell transplantation for the first time;
• No gender limit, aged 12 - 65 years;
• Planned haploidentical donor transplantation, excluding transplantation from maternal and collateral donors;
• Eastern Cooperative Oncology Group (ECOG) performance status score≤3 points;
• Baseline organ function tests meet the following criteria:
• (1) Left ventricular ejection fraction (LVEF) \> 55%; (2) Serum creatinine ≤ 1.5 × upper limit of normal (ULN).

You may not qualify if:

• Patients with severe dysfunction of brain, heart, kidney or liver;
• Those in refractory malignant status;
• Patients with other malignancies requiring treatment;
• Presence of uncontrolled severe active infection clinically;
• Expected survival period of less than 3 months;
• History of severe allergic reactions;
• Pregnant or breastfeeding women; (8)Presence of any condition deemed by the investigator as unsuitable for study enrollment.

Sponsors & Collaborators

• Peking University People's Hospital: lead

Study Sites (1)

Peking University People'S Hospital

Beijing, China

RECRUITING

MeSH Terms

Conditions

Myelodysplastic Syndromes

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Central Study Contacts

Yu Wang, M.D.

CONTACT

86-010-8832-6000; ywyw3172@sina.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Peking University People's Hospital

Study Record Dates

• First Submitted: May 3, 2025
• First Posted: May 22, 2025
• Study Start: May 21, 2025
• Primary Completion: July 31, 2025
• Study Completion (Estimated): July 31, 2026
• Last Updated: July 25, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)