Modified Second Haplo-transplantation for Graft Failure

Second Haploidentical Transplantation with Modified Regimen for Graft Failure After the First Allogeneic Stem Cell Transplantation

Graft failure is a fatal complication following allogeneic stem cell transplantation where a second transplantation is usually required for salvage. We previously reported encouraging results with a novel Flu/Cy regimen. However, there are still around 20% patients developed delayed platelet recovery. We designed a modified regimen to further improve the hematopoietic reconstitution after second transplantation. This prospective, single-arm study aims to investigate the safety and efficacy of this modified regimen.

Study Overview

• Status: Recruiting
• Conditions: Graft Failure; Stem Cell Transplant Complications
• Intervention / Treatment: Other: second allogeneic stem cell transplantation

• Study Type: Interventional
• Enrollment (Estimated): 12
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Yu-qian Sun, M.D.; Phone Number: +86-10-88324577; Email: sunyuqian83@hotmail.com

Study Locations

• China
  • Beijing
    • Beijing, Beijing, China, 100044
      • Recruiting
      • Peking University People's Hospital
      • Contact: Yu-qian Sun, M.D.; Phone Number: +86-10-88324577; Email: sunyuqian83@hotmail.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• 1.Primary disease: hematological malignancies(AML, CML, MDS, lymphoma, etc.); 2.Graft failure after first allogeneic stem cell transplantation; 3.Time from the first transplantation to the second transplantation is less than 180 days; 4. Age≥14 years.

Exclusion Criteria:

• 1. Active infections; 2. Active GVHD; 3. Organ dysfunction: hepatic injury (Tbil≥2ULN), renal injury (Cr≥1.5ULN), heart injury (EF%<50% or symptomatic heart failure); 4. Eastern Cooperative Oncology Group (ECOG) score>2; 5. Expected life time<30 days; 5. Patients could not cooperate; 6. Other situations that are considered inappropriate for enrollment by the investigators,

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: intervention arm; second haploidentical stem cell transplantation

Other: second allogeneic stem cell transplantation; Change another donor if possible; Conditioning regimen: fludarabine (30mg/m2/day, days -6 to -2 )/cyclophosphamide (1g/m2/day, days -5 to -4)/rabbit antithymocyte globulin (1.5mg/kg/day, days -4 to -2); GVHD prophylaxis: cyclosporine A (concentration 150-250ng/ml), mycophenolate mofetil (0.5g bid -3d to neutrophil engraftment)+anti CD25 monoAb (20mg, +1d, +8d, +15d)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Engraftment of neutrophils	Engraftment of neutrophils was defined as the first of 3 consecutive days when the absolute neutrophil count achieved 500/uL.	28 days after second transplantation

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
TRM at 28 day	treatment-related mortality(TRM) was defined as death from any cause other than relapse.	28 days after second transplantation
Engraftment of Platelet	Platelet engraftment was defined as the first of 7 consecutive days that platelet counts ≥20 × 10^9/L in the absence of platelet transfusion	100 days after second transplantation
TRM at 100 day	treatment-related mortality (TRM) was defined as death from any cause other than relapse	100 days after second transplantation
GVHD	Both acute and chronic GVHD was diagnosed and graded according to international criteria.	Participants will be followed for an expected average of 1 year
OS	Overall survival (OS)	Participants will be followed for an expected average of 1 year
LFS	Leukemia-free survival (LFS) was defined as the survival period with continuous CR.	Participants will be followed for an expected average of 1 year
CIR	The cumulative incidence of relapse (CIR)	Participants will be followed for an expected average of 1 year

Collaborators and Investigators

• Sponsor: Peking University People's Hospital
• Investigators: Principal Investigator: Xiao-Jun Huang, M.D., Institute of Hematology, Peking University People's Hospital

Study record dates

Study Major Dates

• Study Start (Actual): July 1, 2024
• Primary Completion (Estimated): June 30, 2026
• Study Completion (Estimated): June 30, 2026

Study Registration Dates

• First Submitted: July 16, 2024
• First Submitted That Met QC Criteria: July 16, 2024
• First Posted (Actual): July 22, 2024

Study Record Updates

• Last Update Posted (Actual): November 29, 2024
• Last Update Submitted That Met QC Criteria: November 26, 2024
• Last Verified: November 1, 2024

More Information

Terms related to this study

• Other Study ID Numbers: 2024PHB181-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No