Severe Aplastic AnemiaNCT07819812
Thiotepa-Based Conditioning for Haploidentical Hematopoietic Stem Cell Transplantation in Older Patients With Severe Aplastic Anemia
An observational study of Severe Aplastic Anemia and Hematopoietic Cell Transplant, run by Peking University People's Hospital.
Trial summary
This prospective, single-center observational cohort study will follow participants aged 50 years or older with severe or very severe aplastic anemia who receive haploidentical hematopoietic stem cell transplantation with a thiotepa-based conditioning regimen as part of routine clinical care. Read more about this study
- Who is it for?
- Ages 50 years and older
- Conditions the study is for
- Severe Aplastic Anemia, Hematopoietic Cell Transplant
- What is being tested?
- Thiotepa
- How many people
- 20 participants
- Where
- No sites listed yet
- Study dates
- 20 Sep 2026to 31 Dec 2028
- Phase
- Observational
- 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 50 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: The study population will consist of consecutively identified participants aged 50 years or older with severe or very severe aplastic anemia who are scheduled to undergo haploidentical hematopoietic stem cell transplantation at Peking University People's Hospital. The treating clinical team will independently select a thiotepa-based conditioning regimen as part of routine clinical care. The study will prospectively observe participants with available baseline, transplantation, and routine follow-up information for 1 year after transplantation. Up to 10 participants are planned for enrollment.
Who may be able to join
- Age 50 years or older at transplantation.
- Diagnosis of severe aplastic anemia or very severe aplastic anemia.
- Planned to undergo haploidentical hematopoietic stem cell transplantation at Peking University People's Hospital.
- The treating clinical team has independently selected a thiotepa-based conditioning regimen as part of routine clinical care before study enrollment.
- Required baseline and transplantation information is available in the medical record.
Who cannot join
- Diagnosis or transplantation type does not meet the study eligibility criteria.
- The conditioning regimen does not include thiotepa.
- Essential baseline information is unavailable, such that study eligibility cannot be determined.
- For a participant with more than one transplantation episode, only the first episode meeting all eligibility criteria will be included in the analysis.
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: A Prospective, Single-Center, Observational Cohort Study of Thiotepa-Based Conditioning in Older Participants With Severe or Very Severe Aplastic Anemia Undergoing Haploidentical Hematopoietic Stem Cell Transplantation
This prospective, single-center observational cohort study will follow participants aged 50 years or older with severe or very severe aplastic anemia who receive haploidentical hematopoietic stem cell transplantation with a thiotepa-based conditioning regimen as part of routine clinical care. Treatment decisions, including conditioning, prevention of graft-versus-host disease, and supportive care, are made by the clinical care team and are not assigned or changed by this study. The study will collect routine clinical and follow-up information for 1 year after transplantation to describe transplant-related mortality, survival, engraftment, graft failure, graft-versus-host disease, infections, and serious adverse events.
Read the detailed description
Severe aplastic anemia is a bone marrow failure disorder characterized by reduced blood cell production. For participants who need transplantation and do not have a fully matched sibling donor, haploidentical hematopoietic stem cell transplantation is an important treatment option. Older participants may have reduced organ function, a higher risk of infection, and lower tolerance of intensive treatment.
Thiotepa-based conditioning regimens are used in routine clinical practice before transplantation. In this study, the treating clinical team independently determines the conditioning regimen, medication doses and schedules, prevention of graft-versus-host disease, and supportive care according to each participant's clinical condition. Study participation does not assign, require, or change any treatment.
This is a prospective, single-center observational cohort study. The study will consecutively include participants aged 50 years or older with severe or very severe aplastic anemia who receive haploidentical hematopoietic stem cell transplantation with thiotepa-based conditioning at Peking University People's Hospital. Routine clinical information, transplantation information, and routinely collected follow-up information will be recorded through 1 year after transplantation.
The primary outcome is transplant-related mortality within 1 year after transplantation. Secondary outcomes include overall survival, hematopoietic engraftment, graft failure, acute and chronic graft-versus-host disease, cytomegalovirus and Epstein-Barr virus reactivation, serious infections, and serious adverse events. The study will not add treatment, examinations, blood collection, or follow-up visits beyond routine care.
- Being tested
- Thiotepa
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ThiotepaDrug
The exposure of interest is a conditioning regimen containing thiotepa. Thiotepa and all accompanying treatment are selected by treating clinicians as part of routine care, independently of study participation. The study does not mandate drug dose, schedule, graft-versus-host disease prophylaxis, or supportive care.
| Study group | Role | What they receive |
|---|---|---|
| Thiotepa-Based Conditioning Cohort | Participants aged 50 years or older with severe aplastic anemia or very severe aplastic anemia who receive haploidentical hematopoietic stem cell transplantation with a thiotepa-based conditioning regimen. The conditioning regimen is selected by the treating clinical team before study enrollment. The study does not assign or alter treatment. Receives: Drug: Thiotepa |
What is being measured
The primary outcome is the main question the study is designed to answer.
1-year transplant-related mortality (TRM)
The proportion of participants who die from transplant-related causes, including regimen-related toxicity, infection, and graft-versus-host disease, as defined in the study protocol.
Measured: From Day 0 through 1 year after transplantation
Secondary outcomes (6)
Neutrophil Engraftment
Time to neutrophil engraftment, defined as the first of 3 consecutive days with an absolute neutrophil count ≥0.5 × 10^9/L；
Measured: From Day 0 through Day 28
Platelet Engraftment
Time to platelet engraftment, defined as the first of 7 consecutive days with platelet count ≥20 × 10^9/L without platelet transfusion.
Measured: From Day 0 through Day 100
Primary Graft Failure
Proportion of participants without neutrophil engraftment or with graft failure, according to the study protocol.
Measured: From Day 0 through Day 100
Overall Survival
Proportion of participants alive, irrespective of cause of death.
Measured: From Day 0 through 1 year after transplantation
Grade II-IV Acute graft-versus-host disease（ GVHD）
Incidence of grade II-IV acute graft-versus-host disease.
Measured: From Day 0 through Day 100
Moderate or Severe Chronic graft-versus-host disease（GVHD）
Incidence of moderate or severe chronic graft-versus-host disease.
Measured: From Day 0 through 1 year after transplantation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Sep 2026
- Study start20 Sep 2026 (estimated)
- Primary completion31 Dec 2027 (estimated)
- Study completion31 Dec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Severe 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 20 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
- NCT07819812
- Sponsor's protocol ID
- PUPH20260907-01
- Status last verified
- Sep 2026
- First submitted
- 9 Sep 2026
- Last updated
- 21 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective, Single-Center, Observational Cohort Study of Thiotepa-Based Conditioning in Older Participants With Severe or Very Severe Aplastic Anemia Undergoing Haploidentical Hematopoietic Stem Cell Transplantation
- ClinicalTrials.gov ID
- NCT07819812
- Acronym / sponsor ID
- PUPH20260907-01
- Study type
- Observational
- Recruitment status
- Not yet recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Peking University People's HospitalUniversity, hospital or other
- Study start
- 20 Sep 2026 (estimated)
- Primary completion
- 31 Dec 2027 (estimated)
- Study completion
- 31 Dec 2028 (estimated)
- First posted
- 15 Sep 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 prospective, single-center observational cohort study will follow participants aged 50 years or older with severe or very severe aplastic anemia who receive haploidentical hematopoietic stem cell transplantation with a thiotepa-based conditioning regimen as part of routine clinical care. Treatment decisions, including conditioning, prevention of graft-versus-host disease, and supportive care, are made by the clinical care team and are not assigned or changed by this study. The study will collect routine clinical and follow-up information for 1 year after transplantation to describe transplant-related mortality, survival, engraftment, graft failure, graft-versus-host disease, infections, and serious adverse events.
Detailed description
Severe aplastic anemia is a bone marrow failure disorder characterized by reduced blood cell production. For participants who need transplantation and do not have a fully matched sibling donor, haploidentical hematopoietic stem cell transplantation is an important treatment option. Older participants may have reduced organ function, a higher risk of infection, and lower tolerance of intensive treatment.
Thiotepa-based conditioning regimens are used in routine clinical practice before transplantation. In this study, the treating clinical team independently determines the conditioning regimen, medication doses and schedules, prevention of graft-versus-host disease, and supportive care according to each participant's clinical condition. Study participation does not assign, require, or change any treatment.
This is a prospective, single-center observational cohort study. The study will consecutively include participants aged 50 years or older with severe or very severe aplastic anemia who receive haploidentical hematopoietic stem cell transplantation with thiotepa-based conditioning at Peking University People's Hospital. Routine clinical information, transplantation information, and routinely collected follow-up information will be recorded through 1 year after transplantation.
The primary outcome is transplant-related mortality within 1 year after transplantation. Secondary outcomes include overall survival, hematopoietic engraftment, graft failure, acute and chronic graft-versus-host disease, cytomegalovirus and Epstein-Barr virus reactivation, serious infections, and serious adverse events. The study will not add treatment, examinations, blood collection, or follow-up visits beyond routine care.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Thiotepa-Based Conditioning Cohort | – | Drug: ThiotepaParticipants aged 50 years or older with severe aplastic anemia or very severe aplastic anemia who receive haploidentical hematopoietic stem cell transplantation with a thiotepa-based conditioning regimen. The conditioning regimen is selected by the treating clinical team before study enrollment. The study does not assign or alter treatment. |
Who may qualify
- Ages
- 50 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- The study population will consist of consecutively identified participants aged 50 years or older with severe or very severe aplastic anemia who are scheduled to undergo haploidentical hematopoietic stem cell transplantation at Peking University People's Hospital. The treating clinical team will independently select a thiotepa-based conditioning regimen as part of routine clinical care. The study will prospectively observe participants with available baseline, transplantation, and routine follow-up information for 1 year after transplantation. Up to 10 participants are planned for enrollment.
Key inclusion criteria (5 listed)
- Age 50 years or older at transplantation.
- Diagnosis of severe aplastic anemia or very severe aplastic anemia.
- Planned to undergo haploidentical hematopoietic stem cell transplantation at Peking University People's Hospital.
- The treating clinical team has independently selected a thiotepa-based conditioning regimen as part of routine clinical care before study enrollment.
- Required baseline and transplantation information is available in the medical record.
Key exclusion criteria (4 listed)
- Diagnosis or transplantation type does not meet the study eligibility criteria.
- The conditioning regimen does not include thiotepa.
- Essential baseline information is unavailable, such that study eligibility cannot be determined.
- For a participant with more than one transplantation episode, only the first episode meeting all eligibility criteria will be included in the analysis.
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 |
|---|---|---|
| ThiotepaThe exposure of interest is a conditioning regimen containing thiotepa. Thiotepa and all accompanying treatment are selected by treating clinicians as part of routine care, independently of study participation. The study does not mandate drug dose, schedule, graft-versus-host disease prophylaxis, or supportive care. | Drug | Thiotepa-Based Conditioning Cohort |
Primary outcome
1-year transplant-related mortality (TRM)
Time frame: From Day 0 through 1 year after transplantation
The proportion of participants who die from transplant-related causes, including regimen-related toxicity, infection, and graft-versus-host disease, as defined in the study protocol.
6 secondary outcomes
Neutrophil Engraftment
Time frame: From Day 0 through Day 28
Platelet Engraftment
Time frame: From Day 0 through Day 100
Primary Graft Failure
Time frame: From Day 0 through Day 100
Overall Survival
Time frame: From Day 0 through 1 year after transplantation
Grade II-IV Acute graft-versus-host disease（ GVHD）
Time frame: From Day 0 through Day 100
Moderate or Severe Chronic graft-versus-host disease（GVHD）
Time frame: From Day 0 through 1 year after transplantation
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 21 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?
An observational study of Severe Aplastic Anemia and Hematopoietic Cell Transplant, 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 4 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 21 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.
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 Xu zheng li (88324002, xuzhengli0202@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
1-year transplant-related mortality (TRM) (time frame: From Day 0 through 1 year after transplantation). 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 21 Sep 2026.