Myelodysplastic SyndromesPhase 2NCT03412266
RIC Regimen for Low- and Intermediate-risk MDS Receiving Haplo-HSCT
A Phase 2 study testing Cytarabine in people with Myelodysplastic Syndromes, run by Peking University People's Hospital at 1 site in China.
Trial summary
This study aimed to evaluate the efficacy of reduced intensity conditioning (RIC) regimen in low- and intermediate-risk myelodysplastic syndrome (MDS) patients who receive haploidentical hematopoietic stem cell transplantation (haplo-HSCT). Read more about this study
- Who is it for?
- Ages 1 year to 70 years
- Conditions the study is for
- Myelodysplastic Syndromes
- What is being tested?
- Cytarabine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 1 siteChina
- Study dates
- 1 Feb 2018to 1 Mar 2023
- 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 1 year to 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients who had low- and intermediate-risk MDS without ISD nor URD receiving haploidentical hematopoietic stem cell transplantation
Who cannot join
- Patients having ISD or URD; patients having high-risk MDS; patients with active infection; patients with poor compliance; patients with organ failure
Trial locations and contacts
1 site in 1 country.
Xiao-Dong Mo contact
China1 site, 1 recruiting
- Recruiting
Peking University Institute of Hematology,Beijing
Beijing, Beijing Municipality, 100044
Xiao-Dong Mo, mxd453@163.com
Xiao-Jun Huang, MD
About this study
Official title: Reduced Intensity Conditioning Regimen for Low- and Intermediate-risk Myelodysplastic Syndrome Patients Receiving Haploidentical Hematopoietic Stem Cell Transplantation
This study aimed to evaluate the efficacy of reduced intensity conditioning (RIC) regimen in low- and intermediate-risk myelodysplastic syndrome (MDS) patients who receive haploidentical hematopoietic stem cell transplantation (haplo-HSCT). Haplo-HSCT is an effective treatment option for MDS patients who did not have identical sibling donor (ISD) or unrelated donor (URD). However, post-transplant transplant-related mortality (TRM) is one of the major causes for transplant failure in MDS patients, and the risk of TRM for haplo-HSCT recipients was higher than that of ISD recipients. RIC regimen can decrease the risk of TRM for haplo-HSCT recipients; however, the risk for relapse may increase in these patients. Thus, RIC regimen may be more appropriate for low- and intermediate-risk MDS patients receiving haplo-HSCT. The study hypothesis: Using RIC haplo-HSCT regimen in patients with low- and risk MDS can reduce TRM and improve survival.
Read the detailed description
RIC regimen was given for low and intermediate MDS patients who would receive haplo-HSCT. The primary end point was transplant-related mortality. The secondary end points were overall survival, disease-free survival, relapse, , graft-versus-host disease (GVHD), and infections. Following time is 1 years.
- Conditions
- Myelodysplastic Syndromes
- Being tested
- Cytarabine
- Keywords
- RIC regimen; MDS; haplo-HSCT; survival
- Medical subject headings
- Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CytarabineDrug
Also called busulfan, Cyclophosphamide, Fludarabine, Semustine
RIC preconditioning regimen consisted of cytarabine (2 g/m2/day, days -10 to -9), busulfan (3.2 mg/kg/day on days -8 to -6), cyclophosphamide (1.0 g/m2/day, days -5 to -4), fludarabine (30 mg/m-2/day, days -6 to -2), semustine (250 mg/m-2, day -3), and rabbit antithymocyte globulin (thymoglobulin, 2.5 mg/kg/d, days -5 to -2; Sanofi, France).
| Study group | Role | What they receive |
|---|---|---|
| RIC regimen | Other | Low- and intermediate MDS patients without identical sibling donor or unrelated donor would receive RIC haplo-HSCT.
RIC preconditioning regimen consisted of cytarabine (2 g/m2/day, days -10 to -9), busulfan (3.2 mg/kg/day on days -8 to -6), cyclophosphamide (1.0 g/m2/day, days -5 to -4), fludarabine (30 mg/m-2/day, days -6 to -2), semustine (250 mg/m-2, day -3), and rabbit antithymocyte globulin (thymoglobulin, 2.5 mg/kg/d, days -5 to -2; Sanofi, France). Receives: Drug: Cytarabine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Transplant-related mortality
Death without disease progression or relapse
Measured: Participants will be followed for an expected average of 1 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Jan 2018
- Study start1 Feb 2018
- Primary completion1 Mar 2023 (estimated)
- Study completion1 Mar 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myelodysplastic SyndromesStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 50 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
- Investigators
- Xiao-Jun Huang, Principal investigator, Peking University Institute of Hematology
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03412266
- Sponsor's protocol ID
- RIC Haplo-MDS
- Status last verified
- Apr 2022
- First submitted
- 21 Jan 2018
- Last updated
- 20 Apr 2022
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Apr 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Reduced Intensity Conditioning Regimen for Low- and Intermediate-risk Myelodysplastic Syndrome Patients Receiving Haploidentical Hematopoietic Stem Cell Transplantation
- ClinicalTrials.gov ID
- NCT03412266
- Acronym / sponsor ID
- RIC Haplo-MDS
- Conditions
- Myelodysplastic Syndromes
- Keywords
- RIC regimen; MDS; haplo-HSCT; survival
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Peking University People's HospitalUniversity, hospital or other
- Study start
- 1 Feb 2018 (actual)
- Primary completion
- 1 Mar 2023 (estimated)
- Study completion
- 1 Mar 2023 (estimated)
- First posted
- 26 Jan 2018 (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 aimed to evaluate the efficacy of reduced intensity conditioning (RIC) regimen in low- and intermediate-risk myelodysplastic syndrome (MDS) patients who receive haploidentical hematopoietic stem cell transplantation (haplo-HSCT). Haplo-HSCT is an effective treatment option for MDS patients who did not have identical sibling donor (ISD) or unrelated donor (URD). However, post-transplant transplant-related mortality (TRM) is one of the major causes for transplant failure in MDS patients, and the risk of TRM for haplo-HSCT recipients was higher than that of ISD recipients. RIC regimen can decrease the risk of TRM for haplo-HSCT recipients; however, the risk for relapse may increase in these patients. Thus, RIC regimen may be more appropriate for low- and intermediate-risk MDS patients receiving haplo-HSCT. The study hypothesis: Using RIC haplo-HSCT regimen in patients with low- and risk MDS can reduce TRM and improve survival.
Detailed description
RIC regimen was given for low and intermediate MDS patients who would receive haplo-HSCT. The primary end point was transplant-related mortality. The secondary end points were overall survival, disease-free survival, relapse, , graft-versus-host disease (GVHD), and infections. Following time is 1 years.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| RIC regimen | Other | Drug: CytarabineLow- and intermediate MDS patients without identical sibling donor or unrelated donor would receive RIC haplo-HSCT. RIC preconditioning regimen consisted of cytarabine (2 g/m2/day, days -10 to -9), busulfan (3.2 mg/kg/day on days -8 to -6), cyclophosphamide (1.0 g/m2/day, days -5 to -4), fludarabine (30 mg/m-2/day, days -6 to -2), semustine (250 mg/m-2, day -3), and rabbit antithymocyte globulin (thymoglobulin, 2.5 mg/kg/d, days -5 to -2; Sanofi, France). |
Who may qualify
- Ages
- 1 year to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (1 listed)
- Patients who had low- and intermediate-risk MDS without ISD nor URD receiving haploidentical hematopoietic stem cell transplantation
Key exclusion criteria (1 listed)
- Patients having ISD or URD; patients having high-risk MDS; patients with active infection; patients with poor compliance; patients with organ failure
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 |
|---|---|---|
| CytarabineAlso: busulfan, Cyclophosphamide, Fludarabine, SemustineRIC preconditioning regimen consisted of cytarabine (2 g/m2/day, days -10 to -9), busulfan (3.2 mg/kg/day on days -8 to -6), cyclophosphamide (1.0 g/m2/day, days -5 to -4), fludarabine (30 mg/m-2/day, days -6 to -2), semustine (250 mg/m-2, day -3), and rabbit antithymocyte globulin (thymoglobulin, 2.5 mg/kg/d, days -5 to -2; Sanofi, France). | Drug | RIC regimen |
Primary outcome
Transplant-related mortality
Time frame: Participants will be followed for an expected average of 1 years
Death without disease progression or relapse
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Apr 2022.
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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.
Xiao-Dong Mo contact
Xiao-Jun Huang principal investigator
Peking University Institute of Hematology
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Cytarabine in people with Myelodysplastic Syndromes, 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?
The sponsor lists 1 inclusion and 1 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 20 Apr 2022. 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.
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 Xiao-Dong Mo (8610-8832-6001, mxd453@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Transplant-related mortality (time frame: Participants will be followed for an expected average of 1 years). 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 20 Apr 2022.