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NCT Number: NCT06518044

NAC for Hematopoietic Recovery in SAA

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.

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Key information

Conditions

Aplastic Anemia Anemia Anemia, Aplastic Bone Marrow Diseases Bone Marrow Failure Disorders Hematologic Diseases Hemic and Lymphatic Diseases

Age range

18 year–50 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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

Exclusion criteria

  • 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)

Treatment and study plan

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.

Primary outcomes

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

    Time frame: Two months 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).

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

    Time frame: Two months 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).

Secondary outcomes

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

    Time frame: Two months post-HSCT.

    Number of participants suffered TRM will be calculated.

  2. The cumulative incidences of Thrombotic Microangiopathy (TMA).

    Time frame: Two months post-HSCT.

    Number of participants suffered TMA will be calculated.

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

    Time frame: Two months post-HSCT.

    Number of participants suffered GvHD will be calculated.

  4. The cumulative incidences of overall survival (OS).

    Time frame: One year post-HSCT.

    Number of participants survived for 1 year post diagnosed will be calculated.

  5. Adverse reactions

    Time frame: Two months post-HSCT.

    Liver function, renal function, respiratory syndrom assessed by CTCAE v4.0 during oral administration of NAC.

Study contacts

Contact information is provided by the study sponsor or research team.

Xiaojun Huang, M.D.

CONTACT

[email protected]

+861088326006

Zhengli Xu, M.D.

CONTACT

[email protected]

+8601088326900

Sponsors and collaborators

Lead sponsor

Peking University People's Hospital

Other

Registry information

Official study title

N-acetyl-L-cysteine for Promoting Hematopoietic Recovery in Patients With Severe Aplastic Anemia (SAA) After Haploidentical Transplantation -- a Prospective Single-arm Clinical Study

Important dates

Study start
2024
Primary completion
2024
Study completion
2025
First posted
Jul 24, 2024
Registry last updated
Jul 24, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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