A Phase 3 interventional study of N-acetylcysteine in Aplastic Anemia, sponsored by Peking University People's Hospital. Not yet recruiting. Open to participants aged 14 Years to 50 Years. Per ClinicalTrials.gov, last updated 2026-09-16.
Sponsored by Peking University People's Hospital · Phase 3, Interventional, and Prevention
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 by 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 according to the National Cancer Institute Common Terminology Criteria for Adverse Events (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.
371 studies on the registry are indexed under Anemia, Aplastic; 98 are open to participants now.
This study's planned enrollment of 142 is above the median of 37 across 281 interventional studies indexed under Anemia, Aplastic.
Browse Anemia, Aplastic studies →Peking University People's Hospital is the lead sponsor of 609 studies on the registry; 247 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Allogeneic and autologous transplant
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECOG performance status
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Patients receive oral NAC 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
Patients receive standard haploidentical hematopoietic stem cell transplantation and identical supportive treatments without prophylactic oral NAC throughout the study period.
Oral NAC capsules, 400 mg per dose, administered three times daily. Treatment starts at Day -14 before transplantation and continues until Day +60 after transplantation.
Cumulative incidence of platelet engraftment by 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.
Time frame: 2 months post-HSCT
Cumulative incidence of neutrophil engraftment by 2 months after transplantation
Neutrophil engraftment was defined as the first of three consecutive days with a neutrophil count of at least 0.5 × 10\^9/L
Time frame: 2 months post-HSCT
Incidence of poor hematopoietic reconstitution by 2 months post-transplant
Poor hematopoietic reconstitution includes delayed platelet engraftment and poor graft function (PGF). Delayed platelet engraftment: platelet \<20×10\^9/L or persistent platelet transfusion dependence at Day +60. PGF refers to persistent cytopenia with complete donor chimerism and hypoplastic marrow.
Time frame: 2 months post-HSCT
Cumulative volume of blood product transfusions
Total units of platelet and red blood cell transfusions received by each patient. Transfusion standards follow institutional clinical guidelines for anemia and severe thrombocytopenia.
Time frame: From pre-transplant Day -14 to post-transplant Day +60
Cumulative incidence of acute and chronic graft-versus-host disease (aGVHD/cGVHD)
aGVHD was graded by modified Glucksberg criteria within 100 days; chronic GVHD was diagnosed by National Institutes of Health consensus criteria within 1 year post-transplant.
Time frame: 100 days for aGVHD; 1 year for cGVHD after transplantation
1-year overall survival (OS)
OS was defined as the duration from HSCT to death from any cause or the date of the last follow-up.
Time frame: 1 year after transplantation
1-year graft-versus-host disease-free, failure-free survival (GFFS)
GFFS was defined as the absence of grade III-IV acute GVHD, extensive chronic GVHD, graft failure, or death from any cause.
Time frame: 1 year after transplantation
Bone marrow endothelial progenitor cell (EPC) count and intracellular reactive oxygen species (ROS) level
Laboratory biomarkers reflecting bone marrow hematopoietic microenvironment reconstruction, including EPC quantity and intracellular ROS level detected at scheduled bone marrow puncture time points.
Time frame: Baseline (pre-transplant), post-transplant 1 months and 2 months
No study locations are listed for this record.
Start is estimated for Sep 2026; primary completion for Sep 2028, 24 months later.
How these figures are worked out, and which studies are left out of them.
This study is not yet recruiting, as verified in Sep 2026. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Studies of the drug tested here: Acetylcysteine
Peking University People's Hospital →