Aplastic AnemiaNCT03821987
Risk Stratification Directed Conditioning Regimen for Haploidentical HSCT in SAA
A study testing Fludarabine in people with Aplastic Anemia and Stem Cell Transplant Complications, run by Peking University People's Hospital at 1 site in China.
Trial summary
The haplotype HSCT system including Bu(0.8mg/kg Q6hx2d)CTX(50mg/kgx4d)rATG(2.5mg/kgx4d) , established in Institute of Hematology of Peking University ,has been evaluated to be effective for acquired SAA.But some patients with high risk factors may not tolerate CTX 200mg/kg,alternative conditioning regimen including… Read more about this study
- Who is it for?
- Ages 3 to 55 years
- Conditions the study is for
- Aplastic Anemia, Stem Cell Transplant Complications, Engraft Failure
- What is being tested?
- Fludarabine
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 55 participants
- Where
- 1 siteChina
- Study dates
- 17 Dec 2018to 30 Mar 2022
- Phase
- Interventional
- 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 3 to 55 years
- All sexes
- No healthy volunteers
- Child, Adult
A：inclusion criteria
- Patients diagnosed as acquired severe aplastic anemia(SAA) /very vSAA
- patients with age 3-55 years
- patients have no matched sibling donor
- Patients have no matched unrelated donor
- patients have no severe infection
- Patients have no severe organ dysfunction
- patients have risk factors of potential intolerance to previous condition regimen including BuCy(200mg/kg)and ATG
- Consent form signed
- Exclusion criteria ：
- patients with congenital SAA/vSAA
- patients with age< 3years or >55 years
- patients with matched sibling donor
- patients with matched URD
- patients with severe infection
- patients with severe organ dysfunction
- pregnancy women
- no Consent form signed
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Peking University Institute of Hematology,People's hospital Peking University
Beijing, Beijing Municipality, 100044
Lanping Xu, Prof., 8613641028627, lpxu_0415@sina.com
Yanru Ma, 8613641134402, lpxu_0415@vip.sina.com
About this study
The haplotype HSCT system including Bu(0.8mg/kg Q6hx2d)CTX(50mg/kgx4d)rATG(2.5mg/kgx4d) , established in Institute of Hematology of Peking University ,has been evaluated to be effective for acquired SAA.But some patients with high risk factors may not tolerate CTX 200mg/kg,alternative conditioning regimen including Bu/Fludarabine/dercreased CTX was studied in this trial.
Read the detailed description
Patients enrolled in this study would receive Bu (IV)0.8mg/kg Q6hx2d,Fludarabine 30mg/m2x5d ,CTX(cyclophosphamide) 25mg/kg/dx4d,rATG (thymoglobulin,Sang Stat,France) 2.5mg/kg/dx4d.
BM or Blood samples from patients were obtained to assess engraftment and chimerism after HSCT. The time point that investigators monitor BM or blood samples at 1 month,2 months, 3 months,6 months, 9 months and 1year,2years,3years 5 years after HSCT.
- Being tested
- Fludarabine
- Keywords
- haploidentical transplantation, aplastic anemia
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FludarabineDrug
Also called Busulfan, Cyclophosphamide, Thymoglobulin
Patients enrolled in this study would receive Bu (IV)0.8mg/kg Q6hx2d,Fludarabine 30mg/m2x5d ,CTX (cyclophosphamide) 25mg/kg/dx4d,rATG (thymoglobulin,Sang Stat,France) 2.5mg/kg/dx4d.
| Study group | Role | What they receive |
|---|---|---|
| BFCA regimen | Experimental | Detail: Patients enrolled in this study would receive Busulfan(B) (IV)0.8mg/kg Q6hx2d,Fludarabine(F) 30mg/m2x5d ,cyclophosphamide(C) 25mg/kg/dx4d,thymoglobulin (A :rATG ,Sang Stat,France) 2.5mg/kg/dx4d.
BM or Blood samples from patients were obtained to assess engraftment and chimerism after HSCT. The time point that we monitored BM or blood samples included at 1 month,2 months, 3 months,6 months, 9 months and 1year,2years,3years 5 years after HSCT. Receives: Drug: Fludarabine |
What is being measured
The primary outcome is the main question the study is designed to answer.
1 year cumulative incidence overall survival
Tne cumulative incidence of overall survival at 1 year post HSCT
Measured: 1 year post HSCT
Secondary outcomes (5)
one month Transplantation related mortality
Tne cumulative incidence of transplantation related motality at 1 month post HSCT
Measured: 1 month post HSCT
Engraftment
Tne cumulative incidence of engraftment at 1 moths post HSCT
Measured: 1 month post HSCT
0ne month regimen-related toxicity
Tne cumulative incidence of regimen related toxicity at 1 month post HSCT
Measured: 1 month post HSCT
aGVHD
Tne cumulative incidence of acute GVHD at Day 100 post HSCT
Measured: 100 days post HSCT
failure-free survival
The cumulative incidence of failure-free survival at 1year post HSCT
Measured: 1 year post HSCT
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jan 2019
- Study start17 Dec 2018
- Primary completion30 Dec 2021 (estimated)
- Study completion30 Mar 2022 (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.
- 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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Patients enrolled in this study would receive Bu (IV)0.8mg/kg Q6hx2d,Fludarabine 30mg/m2x5d ,CTX(cyclophosphamide) 25mg/kg/dx4d,rATG (thymoglobulin,Sang Stat,France) 2.5mg/kg/dx4d. BM or Blood samples from patients were obtained to assess engraftment and chimerism after HSCT. The time point that we monitored BM or blood samples included at 1 month,2 months, 3 months,6 months, 9 months and 1year,2years,3years 5 years after HSCT.
- Masking
- None (open label)
- Enrolment
- 55 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking University People's Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Xiaojun Huang,MD, Peking University People's Hospital
- Investigators
- Xiaojun Huang, Prof., Principal investigator, Peking Universiy Institute of Hematology,People's Hospital Pekiking Universiyy
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03821987
- Sponsor's protocol ID
- PUIH-SAA
- Status last verified
- Mar 2020
- First submitted
- 23 Jan 2019
- Last updated
- 10 Mar 2020
- 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 10 Mar 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Risk Stratification Directed Conditioning Regimen for Haploidentical HSCT in SAA
- ClinicalTrials.gov ID
- NCT03821987
- Acronym / sponsor ID
- PUIH-SAA
- Keywords
- haploidentical transplantation, aplastic anemia
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 55 participants (estimated)
- Lead sponsor
- Peking University People's HospitalUniversity, hospital or other
- Study start
- 17 Dec 2018 (actual)
- Primary completion
- 30 Dec 2021 (estimated)
- Study completion
- 30 Mar 2022 (estimated)
- First posted
- 30 Jan 2019 (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.
The haplotype HSCT system including Bu(0.8mg/kg Q6hx2d)CTX(50mg/kgx4d)rATG(2.5mg/kgx4d) , established in Institute of Hematology of Peking University ,has been evaluated to be effective for acquired SAA.But some patients with high risk factors may not tolerate CTX 200mg/kg,alternative conditioning regimen including Bu/Fludarabine/dercreased CTX was studied in this trial.
Detailed description
Patients enrolled in this study would receive Bu (IV)0.8mg/kg Q6hx2d,Fludarabine 30mg/m2x5d ,CTX(cyclophosphamide) 25mg/kg/dx4d,rATG (thymoglobulin,Sang Stat,France) 2.5mg/kg/dx4d.
BM or Blood samples from patients were obtained to assess engraftment and chimerism after HSCT. The time point that investigators monitor BM or blood samples at 1 month,2 months, 3 months,6 months, 9 months and 1year,2years,3years 5 years after HSCT.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Patients enrolled in this study would receive Bu (IV)0.8mg/kg Q6hx2d,Fludarabine 30mg/m2x5d ,CTX(cyclophosphamide) 25mg/kg/dx4d,rATG (thymoglobulin,Sang Stat,France) 2.5mg/kg/dx4d. BM or Blood samples from patients were obtained to assess engraftment and chimerism after HSCT. The time point that we monitored BM or blood samples included at 1 month,2 months, 3 months,6 months, 9 months and 1year,2years,3years 5 years after HSCT.
| Arm | Type | Receives |
|---|---|---|
| BFCA regimen | Experimental | Drug: FludarabineDetail: Patients enrolled in this study would receive Busulfan(B) (IV)0.8mg/kg Q6hx2d,Fludarabine(F) 30mg/m2x5d ,cyclophosphamide(C) 25mg/kg/dx4d,thymoglobulin (A :rATG ,Sang Stat,France) 2.5mg/kg/dx4d. BM or Blood samples from patients were obtained to assess engraftment and chimerism after HSCT. The time point that we monitored BM or blood samples included at 1 month,2 months, 3 months,6 months, 9 months and 1year,2years,3years 5 years after HSCT. |
Who may qualify
- Ages
- 3 to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
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 |
|---|---|---|
| FludarabineAlso: Busulfan, Cyclophosphamide, ThymoglobulinPatients enrolled in this study would receive Bu (IV)0.8mg/kg Q6hx2d,Fludarabine 30mg/m2x5d ,CTX (cyclophosphamide) 25mg/kg/dx4d,rATG (thymoglobulin,Sang Stat,France) 2.5mg/kg/dx4d. | Drug | BFCA regimen |
Primary outcome
1 year cumulative incidence overall survival
Time frame: 1 year post HSCT
Tne cumulative incidence of overall survival at 1 year post HSCT
5 secondary outcomes
one month Transplantation related mortality
Time frame: 1 month post HSCT
Engraftment
Time frame: 1 month post HSCT
0ne month regimen-related toxicity
Time frame: 1 month post HSCT
aGVHD
Time frame: 100 days post HSCT
failure-free survival
Time frame: 1 year post HSCT
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Mar 2020.
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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.
Xiaojun Huang, Prof. principal investigator
Peking Universiy Institute of Hematology,People's Hospital Pekiking Universiyy
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Fludarabine in people with Aplastic Anemia and Stem Cell Transplant Complications, run by Peking University People's Hospital at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
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 10 Mar 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Xiaojun Huang, Prof. (861088326006, lpxu_0415@sina.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
1 year cumulative incidence overall survival (time frame: 1 year 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 10 Mar 2020; AzTrials last refreshed its copy on 1 Nov 2026.