Aplastic AnemiaPhase 2NCT06518044
NAC for Hematopoietic Recovery in SAA
A Phase 2 study testing N Acetyl L Cysteine in people with Aplastic Anemia, run by Peking University People's Hospital.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 50 years
- Conditions the study is for
- Aplastic Anemia
- What is being tested?
- N Acetyl L Cysteine
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- No sites listed yet
- Study dates
- 25 Jul 2024to 1 Jul 2025
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 18 to 50 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 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
Who cannot join
- 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)
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- 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)
Trial locations and contacts
No sites are listed yet.
About this study
Official 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
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.
Read the 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
- Being tested
- N Acetyl L Cysteine
- Keywords
- hematopoietic recovery, NAC, HSCT
- 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 Acetyl L CysteineDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| 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). Receives: Drug: N Acetyl L Cysteine |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: 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).
Measured: Two months post-HSCT.
Secondary outcomes (5)
The cumulative incidences of transplantation related mortality (TRM).
Number of participants suffered TRM will be calculated.
Measured: Two months post-HSCT.
The cumulative incidences of Thrombotic Microangiopathy (TMA).
Number of participants suffered TMA will be calculated.
Measured: Two months post-HSCT.
The cumulative incidences of graft versus host disease (GvHD).
Number of participants suffered GvHD will be calculated.
Measured: Two months post-HSCT.
The cumulative incidences of overall survival (OS).
Number of participants survived for 1 year post diagnosed will be calculated.
Measured: One year post-HSCT.
Adverse reactions
Liver function, renal function, respiratory syndrom assessed by CTCAE v4.0 during oral administration of NAC.
Measured: Two months post-HSCT.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jul 2024
- Study start25 Jul 2024 (estimated)
- Primary completion31 Dec 2024 (estimated)
- Study completion1 Jul 2025 (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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 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
- NCT06518044
- Sponsor's protocol ID
- NAC in SAA post haplo-HSCT
- Status last verified
- Jul 2024
- First submitted
- 29 Jun 2024
- Last updated
- 24 Jul 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT06518044
- Acronym / sponsor ID
- NAC in SAA post haplo-HSCT
- Conditions
- Aplastic Anemia
- Keywords
- hematopoietic recovery, NAC, HSCT
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Peking University People's HospitalUniversity, hospital or other
- Study start
- 25 Jul 2024 (estimated)
- Primary completion
- 31 Dec 2024 (estimated)
- Study completion
- 1 Jul 2025 (estimated)
- First posted
- 24 Jul 2024 (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 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| NAC group | Experimental | Drug: N Acetyl L CysteineAplastic 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). |
Who may qualify
- Ages
- 18 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (9 listed)
- 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)
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 Acetyl L CysteineFor 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. | Drug | NAC group |
Primary outcome
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).
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).
5 secondary outcomes
The cumulative incidences of transplantation related mortality (TRM).
Time frame: Two months post-HSCT.
The cumulative incidences of Thrombotic Microangiopathy (TMA).
Time frame: Two months post-HSCT.
The cumulative incidences of graft versus host disease (GvHD).
Time frame: Two months post-HSCT.
The cumulative incidences of overall survival (OS).
Time frame: One year post-HSCT.
Adverse reactions
Time frame: Two months post-HSCT.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jul 2024.
- Individual participant dataSharing planned: No
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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. This study is not recruiting at the moment.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing N Acetyl L Cysteine 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 5 inclusion and 9 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: status not confirmed, as of the sponsor's last update on 24 Jul 2024. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Xiaojun 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?
The incidence of prolonged thrombcytopenia (PT), which was assessed at +2M post-HSCT. (time frame: Two months post-HSCT.). The study lists 2 primary outcomes in all. 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 24 Jul 2024.