NAC for Hematopoietic Recovery in SAA

NCT ID: NCT06518044

Last Updated: 2024-07-24

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 30 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-07-25
• Study Completion Date: 2025-07-01

Brief Summary

This is a prospective single-arm clinical study to evaluate the role of NAC among patients with severe aplastic anemia (SAA) can promote hematopoietic recovery after haploidentical transplantation.

Detailed Description

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is an effective treatment of severe aplastic anemia (SAA). However, poor hematopoietic reconstitution including poor graft function (PGF) and prolonged isolated thrombocytopenia (PT), remains a life-threatening complication after allo-HSCT. Especially with the increasing use of haploidentical allo-HSCT (haplo-HSCT) in the past ten years, PGF and PT have become growing obstacles contributing to high morbidity and mortality after allo-HSCT. A previous clinical prospective cohort study showed that NAC could improve the function of bone marrow endothelial progenitor cells and promote hematopoietic recovery among leukemia patients after haploidentical transplantation. Therefore, we hypothesized that the prophylactic administration of NAC could facilitate the recovery of hematopoietic capacity by improving the bone marrow microenvironment of patients with SAA after haploidentical transplantation.

Conditions

Aplastic Anemia

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

NAC group

Aplastic anemia patients receiving haploidentical transplantation will be enrolled, and NAC (400mg tid) will be administered orally from day 14 before conditioning until day +60 post-HSCT. The initial dose of NAC was 400mg orally three times daily (TID).

Group Type: EXPERIMENTAL

N Acetyl L Cysteine

Intervention Type: DRUG

For subjects in the experimental intervention arm (NAC group), if the patients met the inclusion criteria on day 14 before conditioning, they received NAC from day 14 before conditioning until day +60 post-HSCT. The initial dose of NAC was 400mg orally three times daily (TID). In cases of grade 3 or worse AEs (not including hematologic recovery), dose modifications including dose reductions or interruptions were permitted at the physician's discretion. After the resolution of AEs, the dose was re-escalated from to 400mg TID.

Interventions

N Acetyl L Cysteine

For subjects in the experimental intervention arm (NAC group), if the patients met the inclusion criteria on day 14 before conditioning, they received NAC from day 14 before conditioning until day +60 post-HSCT. The initial dose of NAC was 400mg orally three times daily (TID). In cases of grade 3 or worse AEs (not including hematologic recovery), dose modifications including dose reductions or interruptions were permitted at the physician's discretion. After the resolution of AEs, the dose was re-escalated from to 400mg TID.

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

1. Diagnosed with SAA or vSAA

2. Aged 18-50

3. No severe organ injury

4. No uncontrolled active infections

5. Sign informed consent form, have the ability to comply with study and follow-up procedures

Exclusion Criteria

1. Hypersensitivity to NAC or history of bronchial asthma

2. Life expectancy less than 30 days post-transplantation

3. Uncontrolled infections pre-transplantation

4. Cardiac dysfunction (particularly congestive heart failure, unstable coronary artery disease and serious cardiac ventricular arrhythmias requiring antiarrhythmic therapy)

5. Respiratory failure ( PaO2 ≤60mmHg)

6. Hepatic abnormalities (total bilirubin ≥2 times the upper limit of normal [ULN], alanine aminotransferase or aspartate aminotransferase ≥2 times the ULN)

7. Renal dysfunction (creatinine ≥1.5 times the ULN or creatinine clearance rate < 30 mL/min)

8. ECOG performance status ≥3

9. With any conditions not suitable for the trial (investigators' decision)

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 50 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Peking University People's Hospital

OTHER

Sponsor Role: lead

Responsible Party

Xiao-Jun Huang

Professor

Responsibility Role: PRINCIPAL_INVESTIGATOR

Central Contacts

Xiaojun Huang, M.D.

Role: CONTACT

xjhrm@medmail.com.cn

+861088326006

Zhengli Xu, M.D.

Role: CONTACT

xuzhengli0202@163.com

+8601088326900

Other Identifiers

NAC in SAA post haplo-HSCT

Identifier Type: -

Identifier Source: org_study_id