Prophylactic NAC to Improve Platelet Engraftment After Haploidentical Transplantation in Severe Aplastic Anemia Patients

NCT07737262 | Not Yet Recruiting Phase 3 DMC

• 1 other identifier: 2026PHD022-001
• Study Type: interventional
• Target: 142
• Locations: 0 countries
• Sites: N/A

Brief Summary

This study aims to evaluate the efficacy and safety of prophylactic oral N-acetylcysteine (NAC) for facilitating platelet engraftment in patients with severe aplastic anemia (SAA) receiving haploidentical hematopoietic stem cell transplantation (haplo-HSCT). This is a prospective, multicenter, randomized controlled trial enrolling a total of 142 patients with SAA scheduled for their first haplo-HSCT, who will be randomly assigned at a 1:1 ratio to the NAC prophylaxis group or the control group, with 71 subjects in each arm. Patients in the intervention group will receive oral NAC 400 mg three times daily from Day -14 before transplantation to Day +60 post-transplant, while the control group will receive no prophylactic NAC, with all other transplant-related treatments identical between the two groups. The primary endpoint is the cumulative platelet engraftment rate at 2 months after transplantation. Secondary endpoints cover neutrophil engraftment rate, incidence of poor hematopoietic reconstitution, cumulative blood product transfusion volume, graft-versus-host disease (GVHD), overall survival, GVHD-free and failure-free survival, and biomarkers reflecting bone marrow hematopoietic microenvironment reconstruction. Safety outcomes will be assessed via adverse events graded per NCI CTCAE Version 5.0. Statistical analyses will be performed using R 4.4.0 software, primarily adopting competing risk models and the Kaplan-Meier method based on full analysis set, per-protocol set and safety set. This trial will clarify intergroup differences in platelet recovery, other efficacy endpoints and safety profiles, verify the clinical benefits and safety of NAC, and generate high-quality clinical evidence for prophylactic intervention targeting platelet engraftment after haplo-HSCT in SAA patients to optimize clinical management strategies.

Conditions

Aplastic Anemia

Outcome Measures

Primary Outcomes (1)

• Cumulative incidence of platelet engraftment at 2 months after transplantation

  Platelet engraftment was characterized as the first day with a platelet count of at least 20 × 10\^9/L without transfusion support for seven consecutive days.

  2 months post-HSCT

Secondary Outcomes (7)

• Cumulative incidence of neutrophil engraftment at 2 months after transplantation

  2 months post-HSCT

• Incidence of poor hematopoietic reconstitution at 2 months post-transplant

  2 months post-HSCT

• Cumulative volume of blood product transfusions

  From pre-transplant Day -14 to post-transplant Day +60

• Cumulative incidence of acute and chronic graft-versus-host disease (GVHD)

  100 days for aGVHD; 1 year for cGVHD after transplantation

• 1-year overall survival (OS)

  1 year after transplantation

Study Arms (2)

Prophylactic NAC Group

EXPERIMENTAL

Patients receive oral N-acetylcysteine 400 mg three times daily from Day -14 before transplantation to Day +60 post-transplant, combined with standard haploidentical hematopoietic stem cell transplantation and routine supportive care.

Drug: N-acetylcysteine

Control Group

NO INTERVENTION

Patients receive standard haploidentical hematopoietic stem cell transplantation and identical supportive treatments without prophylactic oral N-acetylcysteine throughout the study period.

Interventions

N-acetylcysteine DRUG

Oral N-acetylcysteine capsules, 400 mg per dose, administered three times daily. Treatment starts at Day -14 before transplantation and continues until Day +60 after transplantation.

Prophylactic NAC Group

Eligibility Criteria

• Age: 14 Years - 50 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Diagnosed with aplastic anemia and scheduled to receive first haplo-HSCT
• Aged 14 to 50 years
• Hematopoietic Cell Transplant Comorbidity Index (HCT-CI) ≤ 2
• Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2
• Negative donor-specific HLA antibodies
• No uncontrolled active infection before transplantation
• No irreversible severe organ dysfunction
• Voluntarily sign written informed consent and agree to complete scheduled follow-up

You may not qualify if:

• Confirmed allergy or hypersensitivity to NAC
• Medical history of bronchial asthma
• Uncontrolled severe psychiatric disorders unable to cooperate with treatment and follow-up
• Active peptic ulcer, gastrointestinal bleeding, inflammatory bowel disease or severe gastrointestinal dysfunction
• Female patients who are pregnant or breastfeeding

Sponsors & Collaborators

• Peking University People's Hospital: lead

MeSH Terms

Conditions

Anemia, Aplastic

Interventions

Acetylcysteine

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases

Intervention Hierarchy (Ancestors)

Cysteine; Amino Acids, Sulfur; Sulfur Compounds; Organic Chemicals; Amino Acids; Amino Acids, Peptides, and Proteins

Central Study Contacts

Xiao-Jun Huang, M.D.

CONTACT

+861088326006; xjhrm@medmail.com.cn

Zheng-Li Xu, M.D.

CONTACT

+8601088326900; xuzhengli0202@163.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: July 27, 2026
• First Posted: July 30, 2026
• Study Start (Estimated): August 1, 2026
• Primary Completion (Estimated): July 31, 2028
• Study Completion (Estimated): July 31, 2029
• Last Updated: July 30, 2026

Record last verified: 2026-07