NAC for Hematopoietic Recovery in SAA

NCT06518044 | Not Yet Recruiting Phase 2

• 1 other identifier: NAC in SAA post haplo-HSCT
• Study Type: interventional
• Target: 30
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Aplastic Anemia

Keywords

hematopoietic recovery; NAC; HSCT

Outcome Measures

Primary Outcomes (2)

• The incidence of prolonged thrombcytopenia (PT), which was assessed at +2M post-HSCT.

  PT was defined as platelet count less than 20×109/L or a dependence on platelet transfusion with the engraftment of other cell lines(ANC\>0.5×109/L and hemoglobin\>70 g/L without transfusion support) beyond day +60 post-HSCT in the presence of complete donor chimerism (CDC).

  Two months post-HSCT.

• The incidence of poor graft function (PGF), which was assessed at +2M post-HSCT.

  PGF was defined as the presence of 2 or 3 cytopenic counts (ANC≤0.5×109/L, platelet≤20×109/L, or hemoglobin≤70 g/L) for at least 3 consecutive days beyond day +28 post-HSCT with a transfusion requirement, related with hypoplastic-aplastic BM, in the presence of complete donor chimerism (CDC).

  Two months post-HSCT.

Secondary Outcomes (5)

• The cumulative incidences of transplantation related mortality (TRM).

  Two months post-HSCT.

• The cumulative incidences of Thrombotic Microangiopathy (TMA).

  Two months post-HSCT.

• The cumulative incidences of graft versus host disease (GvHD).

  Two months post-HSCT.

• The cumulative incidences of overall survival (OS).

  One year post-HSCT.

• Adverse reactions

  Two months post-HSCT.

Study Arms (1)

NAC group

EXPERIMENTAL

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).

Drug: N Acetyl L Cysteine

Interventions

N Acetyl L Cysteine 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.

NAC group

Eligibility Criteria

• Age: 18 Years - 50 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Diagnosed with SAA or vSAA
• Aged 18-50
• No severe organ injury
• No uncontrolled active infections
• Sign informed consent form, have the ability to comply with study and follow-up procedures

You may not qualify if:

• Hypersensitivity to NAC or history of bronchial asthma
• Life expectancy less than 30 days post-transplantation
• Uncontrolled infections pre-transplantation
• Cardiac dysfunction (particularly congestive heart failure, unstable coronary artery disease and serious cardiac ventricular arrhythmias requiring antiarrhythmic therapy)
• Respiratory failure ( PaO2 ≤60mmHg)
• Hepatic abnormalities (total bilirubin ≥2 times the upper limit of normal \[ULN\], alanine aminotransferase or aspartate aminotransferase ≥2 times the ULN)
• Renal dysfunction (creatinine ≥1.5 times the ULN or creatinine clearance rate \< 30 mL/min)
• ECOG performance status ≥3
• With any conditions not suitable for the trial (investigators' decision)

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

Xiaojun Huang, M.D.

CONTACT

+861088326006; xjhrm@medmail.com.cn

Zhengli Xu, M.D.

CONTACT

+8601088326900; xuzhengli0202@163.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: June 29, 2024
• First Posted: July 24, 2024
• Study Start: July 25, 2024
• Primary Completion: December 31, 2024
• Study Completion: July 1, 2025
• Last Updated: July 24, 2024

Record last verified: 2024-07

Data Sharing

• IPD Sharing: Will not share