Aplastic AnemiaPhase 3NCT07737262
Prophylactic NAC to Improve Platelet Engraftment After Haploidentical Transplantation in Severe Aplastic Anemia Patients
A Phase 3 study testing N-acetylcysteine in people with Aplastic Anemia, run by Peking University People's Hospital.
Trial 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). Read more about this study
- Who is it for?
- Ages 14 to 50 years
- Conditions the study is for
- Aplastic Anemia
- What is being tested?
- N-acetylcysteine
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 142 participants
- Where
- No sites listed yet
- Study dates
- 30 Sep 2026to 30 Sep 2029
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 14 to 50 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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 human leukocyte antigen (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
Who cannot join
- 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
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: Prophylactic N-Acetylcysteine to Facilitate Platelet Engraftment Following Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Severe Aplastic Anemia: A Prospective Multicenter Randomized Controlled Trial
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.
- Conditions
- Aplastic Anemia
- Being tested
- N-acetylcysteine
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
N-acetylcysteineDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| Prophylactic N-acetylcysteine (NAC) Group | Experimental | 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. Receives: Drug: N-acetylcysteine |
| Control Group | No intervention | Patients receive standard haploidentical hematopoietic stem cell transplantation and identical supportive treatments without prophylactic oral NAC throughout the study period. |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 2 months post-HSCT
Secondary outcomes (7)
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
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Baseline (pre-transplant), post-transplant 1 months and 2 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jul 2026
- Study start30 Sep 2026 (estimated)
- Primary completion30 Sep 2028 (estimated)
- Study completion30 Sep 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Aplastic AnemiaStudies of the same condition, open and under way.
- More trials run by Peking University People's HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 142 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking University People's Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Xiao-Jun Huang, Peking University People's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07737262
- Sponsor's protocol ID
- 2026PHD022-001
- Status last verified
- Sep 2026
- First submitted
- 27 Jul 2026
- Last updated
- 16 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prophylactic N-Acetylcysteine to Facilitate Platelet Engraftment Following Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Severe Aplastic Anemia: A Prospective Multicenter Randomized Controlled Trial
- ClinicalTrials.gov ID
- NCT07737262
- Acronym / sponsor ID
- 2026PHD022-001
- Conditions
- Aplastic Anemia
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 142 participants (estimated)
- Lead sponsor
- Peking University People's HospitalUniversity, hospital or other
- Study start
- 30 Sep 2026 (estimated)
- Primary completion
- 30 Sep 2028 (estimated)
- Study completion
- 30 Sep 2029 (estimated)
- First posted
- 30 Jul 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Prophylactic N-acetylcysteine (NAC) Group | Experimental | Drug: N-acetylcysteinePatients 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. |
| Control Group | No intervention | –Patients receive standard haploidentical hematopoietic stem cell transplantation and identical supportive treatments without prophylactic oral NAC throughout the study period. |
Who may qualify
- Ages
- 14 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (8 listed)
- 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 human leukocyte antigen (HLA) antibodies
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| N-acetylcysteineOral NAC capsules, 400 mg per dose, administered three times daily. Treatment starts at Day -14 before transplantation and continues until Day +60 after transplantation. | Drug | Prophylactic N-acetylcysteine (NAC) Group |
Primary outcome
Cumulative incidence of platelet engraftment by 2 months after transplantation
Time frame: 2 months post-HSCT
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.
7 secondary outcomes
Cumulative incidence of neutrophil engraftment by 2 months after transplantation
Time frame: 2 months post-HSCT
Incidence of poor hematopoietic reconstitution by 2 months post-transplant
Time frame: 2 months post-HSCT
Cumulative volume of blood product transfusions
Time frame: From pre-transplant Day -14 to post-transplant Day +60
Cumulative incidence of acute and chronic graft-versus-host disease (aGVHD/cGVHD)
Time frame: 100 days for aGVHD; 1 year for cGVHD after transplantation
1-year overall survival (OS)
Time frame: 1 year after transplantation
1-year graft-versus-host disease-free, failure-free survival (GFFS)
Time frame: 1 year after transplantation
Bone marrow endothelial progenitor cell (EPC) count and intracellular reactive oxygen species (ROS) level
Time frame: Baseline (pre-transplant), post-transplant 1 months and 2 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Sep 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing N-acetylcysteine in people with Aplastic Anemia, run by Peking University People's Hospital.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 16 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Xiao-Jun Huang, M.D. (+861088326006, xjhrm@medmail.com.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Cumulative incidence of platelet engraftment by 2 months after transplantation (time frame: 2 months post-HSCT). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Sep 2026.