Poor Graft FunctionPhase 3NCT05669079
Decitabine and Umbilical Cord Blood for Poor Graft Function Post Allo-HSCT
A Phase 3 study testing decitabine, umbilical cord blood and Granulocyte-colony stimulating factor in people with Poor Graft Function, run by The First Affiliated Hospital of Soochow University.
Trial summary
This randomized trial aimed at validating the efficacy and safety of low-dose decitabine, together with umbilical cord blood in PGF post allo-HSCT patients. Read more about this study
- Who is it for?
- Ages 16 to 65 years
- Conditions the study is for
- Poor Graft Function
- What is being tested?
- decitabine, umbilical cord blood and Granulocyte-colony stimulating factor
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 100 participants
- Where
- No sites listed yet
- Study dates
- 1 Aug 2023to 1 Nov 2026
- 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 16 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Diagnosed as PGF at day 28 post-HSCT or later. PGF was defined as two or three cytopenias, absolute neutrophil count ≤ 1.5 × 109/L, platelet count ≤ 30 × 109/L, hemoglobin ≤ 85g/L, lasting for more than 2 consecutive weeks;
- Full donor chimerism;
- Primary disease in remission;
- No severe GVHD and relapse.
Who cannot join
- Allergic to decitabine or any components of frozen preservation of umbilical cord blood;
- Active infections;
- Uncontrolled GVHD;
- Severe organ dysfunction;
- Relapse of underlying malignancies;
- Graft failure;
- Received decitabine or participated in other clinical trials within one month before screening.
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: Decitabine and Umbilical Cord Blood for Poor Graft Function Post Allogenic Hematopoietic Stem Cell Transplantation
This randomized trial aimed at validating the efficacy and safety of low-dose decitabine, together with umbilical cord blood in PGF post allo-HSCT patients.
Read the detailed description
Poor graft function (PGF), defined by the presence of multilineage cytopenias in the presence of 100% donor chimerism, is a serious complication of allogeneic stem cell transplant (allo-HSCT). Emerging evidence demonstrates that the inadequate stem cells infusion, bone marrow microenvironment and immune dysregulation play a crucial role in maintaining and regulating hematopoiesis. Current therapies remain debatable, including selected CD34+ cells infusion, mesenchymal stromal cells infusion, prophylactic N-acetyl cysteine administration, etc. Thereafter, the investigators conduct a randomized trial aiming at validating the efficacy and safety of low-dose decitabine, together with umbilical cord blood in PGF post allo-HSCT patients.
Patients were eligible if they were diagnosed as PGF at day 28 post-HSCT or later. PGF was defined as two or three cytopenias, absolute neutrophil count ≤ 1.5 × 109/L, platelet count ≤ 30 × 109/L, hemoglobin ≤ 85g/L, lasting for more than 14 consecutive weeks, in the presence of full donor chimerism and primary disease in remission without severe graft-versus- host disease (GVHD) and relapse.
Patients with the following conditions or diagnoses were excluded: allergic to decitabine or any components of frozen preservation of umbilical cord blood; active infections; uncontrolled GVHD; severe organ dysfunction; relapse of underlying malignancies; graft failure. Patients were also excluded if they had received decitabine or participated in other clinical trials within one month before screening.
Hematological improvement is defined as recovery of two or three blood lineages: absolute neutrophil count>1.5 × 109/L, platelet count>30 × 109/L, hemoglobin>85g/L, without G-CSF, red blood cell or platelet infusion.
Hematological response is defined as recovery of three blood lineages: absolute neutrophil count>2.5 × 109/L, platelet count>60 × 109/L, hemoglobin>100g/L, without G-CSF, red blood cell or platelet infusion.
No response: failed to achieve hematological improvement or response.
- Conditions
- Poor Graft Function
- Being tested
- decitabine, umbilical cord blood, Granulocyte-colony stimulating factor, Recombinant human thrombopoietin / thrombopoietin receptor agonist, Recombinant human erythropoietin
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
decitabineDrug
Also called Dec
15 mg/m2 daily intravenously for consecutive 3 days
umbilical cord bloodBiological
Also called UCB
MNC ≥ 3*108 cells; HLA compatibility ≥ 5/6
Granulocyte-colony stimulating factorDrug
Also called G-CSF
Granulocyte-colony stimulating factor will be used when absolute neutrophil count ≤ 1.5 × 109/L
Recombinant human thrombopoietin / thrombopoietin receptor agonistDrug
Also called rhTPO/TPO-RA
Recombinant human thrombopoietin or thrombopoietin receptor agonist will be used when platelet count ≤ 30 × 109/L
Recombinant human erythropoietinDrug
Also called EPO
Recombinant human erythropoietin will be used when hemoglobin ≤ 85 g/L
| Study group | Role | What they receive |
|---|---|---|
| Arm A | Experimental | decitabine (Chia Tai Tianqing Pharma) 15 mg/m2 daily intravenously for consecutive 3 days (day 1 to day 3), combined with umbilical cord blood infusion (day 8) Receives: Drug: decitabine; Biological: umbilical cord blood; Drug: Granulocyte-colony stimulating factor; Drug: Recombinant human thrombopoietin / thrombopoietin receptor agonist; Drug: Recombinant human erythropoietin |
| Arm B | Active comparator | Supportive therapy: G-CSF for patients with absolute neutrophil count ≤ 1.5 × 109/L, rhTPO/TPO-R with platelet count ≤ 30 × 109/L, EPO with hemoglobin ≤ 85g/L. Receives: Drug: Granulocyte-colony stimulating factor; Drug: Recombinant human thrombopoietin / thrombopoietin receptor agonist; Drug: Recombinant human erythropoietin |
What is being measured
The primary outcome is the main question the study is designed to answer.
The treatment response
The rate of hematological improvement and hematological response of 2 arms
Measured: day +28
Survival
The rate of overall survival
Measured: 1 year
Secondary outcomes (3)
Bone marrow recovery
Number of participants with granulopoiesis, erythropoiesis and megakaryopoiesis recovery of bone marrow
Measured: day +28
relapse and GVHD
The rate of relapse and GVHD
Measured: 3-month
Event free survival
The rate of event free survival
Measured: 1-year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Dec 2022
- Study start1 Aug 2023 (estimated)
- Primary completion1 Aug 2026 (estimated)
- Study completion1 Nov 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for Poor Graft FunctionStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- The First Affiliated Hospital of Soochow University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Yue Han, Principal investigator, The First Affiliated Hospital of Soochow University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05669079
- Sponsor's protocol ID
- SOOCHOW-HY-2022-12-15
- Status last verified
- Dec 2022
- First submitted
- 19 Dec 2022
- Last updated
- 9 Jun 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Alchalby H, Yunus DR, Zabelina T, Ayuk F, Kroger N. Incidence and risk factors of poor graft function after allogeneic stem cell transplantation for myelofibrosis. Bone Marrow Transplant. 2016 Sep;51(9):1223-7. doi: 10.1038/bmt.2016.98. Epub 2016 Apr 18.
PubMed 27088376Larocca A, Piaggio G, Podesta M, Pitto A, Bruno B, Di Grazia C, Gualandi F, Occhini D, Raiola AM, Dominietto A, Bregante S, Lamparelli T, Tedone E, Oneto R, Frassoni F, Van Lint MT, Pogliani E, Bacigalupo A. Boost of CD34+-selected peripheral blood cells without further conditioning in patients with poor graft function following allogeneic stem cell transplantation. Haematologica. 2006 Jul;91(7):935-40.
PubMed 16818281Prabahran A, Koldej R, Chee L, Ritchie D. Clinical features, pathophysiology, and therapy of poor graft function post-allogeneic stem cell transplantation. Blood Adv. 2022 Mar 22;6(6):1947-1959. doi: 10.1182/bloodadvances.2021004537.
PubMed 34492685Tang Y, Chen J, Liu Q, Chu T, Pan T, Liang J, He XF, Chen F, Yang T, Ma X, Wu X, Hu S, Cao X, Hu X, Hu J, Liu Y, Qi J, Shen Y, Ruan C, Han Y, Wu D. Low-dose decitabine for refractory prolonged isolated thrombocytopenia after HCT: a randomized multicenter trial. Blood Adv. 2021 Mar 9;5(5):1250-1258. doi: 10.1182/bloodadvances.2020002790.
PubMed 33646303Han Y, Tang Y, Chen J, Liang J, Ye C, Ruan C, Wu D. Low-Dose Decitabine for Patients With Thrombocytopenia Following Allogeneic Hematopoietic Stem Cell Transplantation: A Pilot Therapeutic Study. JAMA Oncol. 2015 May;1(2):249-51. doi: 10.1001/jamaoncol.2014.316. No abstract available.
PubMed 26181032
Study at a glance
From the registry record, last updated by the sponsor on 9 Jun 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Decitabine and Umbilical Cord Blood for Poor Graft Function Post Allogenic Hematopoietic Stem Cell Transplantation
- ClinicalTrials.gov ID
- NCT05669079
- Acronym / sponsor ID
- SOOCHOW-HY-2022-12-15
- Conditions
- Poor Graft Function
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- The First Affiliated Hospital of Soochow UniversityUniversity, hospital or other
- Study start
- 1 Aug 2023 (estimated)
- Primary completion
- 1 Aug 2026 (estimated)
- Study completion
- 1 Nov 2026 (estimated)
- First posted
- 30 Dec 2022 (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 randomized trial aimed at validating the efficacy and safety of low-dose decitabine, together with umbilical cord blood in PGF post allo-HSCT patients.
Detailed description
Poor graft function (PGF), defined by the presence of multilineage cytopenias in the presence of 100% donor chimerism, is a serious complication of allogeneic stem cell transplant (allo-HSCT). Emerging evidence demonstrates that the inadequate stem cells infusion, bone marrow microenvironment and immune dysregulation play a crucial role in maintaining and regulating hematopoiesis. Current therapies remain debatable, including selected CD34+ cells infusion, mesenchymal stromal cells infusion, prophylactic N-acetyl cysteine administration, etc. Thereafter, the investigators conduct a randomized trial aiming at validating the efficacy and safety of low-dose decitabine, together with umbilical cord blood in PGF post allo-HSCT patients.
Patients were eligible if they were diagnosed as PGF at day 28 post-HSCT or later. PGF was defined as two or three cytopenias, absolute neutrophil count ≤ 1.5 × 109/L, platelet count ≤ 30 × 109/L, hemoglobin ≤ 85g/L, lasting for more than 14 consecutive weeks, in the presence of full donor chimerism and primary disease in remission without severe graft-versus- host disease (GVHD) and relapse.
Patients with the following conditions or diagnoses were excluded: allergic to decitabine or any components of frozen preservation of umbilical cord blood; active infections; uncontrolled GVHD; severe organ dysfunction; relapse of underlying malignancies; graft failure. Patients were also excluded if they had received decitabine or participated in other clinical trials within one month before screening.
Hematological improvement is defined as recovery of two or three blood lineages: absolute neutrophil count>1.5 × 109/L, platelet count>30 × 109/L, hemoglobin>85g/L, without G-CSF, red blood cell or platelet infusion.
Hematological response is defined as recovery of three blood lineages: absolute neutrophil count>2.5 × 109/L, platelet count>60 × 109/L, hemoglobin>100g/L, without G-CSF, red blood cell or platelet infusion.
No response: failed to achieve hematological improvement or response.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Arm A | Experimental | Drug: decitabine, Biological: umbilical cord blood, Drug: Granulocyte-colony stimulating factor, Drug: Recombinant human thrombopoietin / thrombopoietin receptor agonist, Drug: Recombinant human erythropoietindecitabine (Chia Tai Tianqing Pharma) 15 mg/m2 daily intravenously for consecutive 3 days (day 1 to day 3), combined with umbilical cord blood infusion (day 8) |
| Arm B | Active comparator | Drug: Granulocyte-colony stimulating factor, Drug: Recombinant human thrombopoietin / thrombopoietin receptor agonist, Drug: Recombinant human erythropoietinSupportive therapy: G-CSF for patients with absolute neutrophil count ≤ 1.5 × 109/L, rhTPO/TPO-R with platelet count ≤ 30 × 109/L, EPO with hemoglobin ≤ 85g/L. |
Who may qualify
- Ages
- 16 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Diagnosed as PGF at day 28 post-HSCT or later. PGF was defined as two or three cytopenias, absolute neutrophil count ≤ 1.5 × 109/L, platelet count ≤ 30 × 109/L, hemoglobin ≤ 85g/L, lasting for more than 2 consecutive weeks;
- Full donor chimerism;
- Primary disease in remission;
- No severe GVHD and relapse.
Key exclusion criteria (7 listed)
- Allergic to decitabine or any components of frozen preservation of umbilical cord blood;
- Active infections;
- Uncontrolled GVHD;
- Severe organ dysfunction;
- Relapse of underlying malignancies;
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 |
|---|---|---|
| decitabineAlso: Dec15 mg/m2 daily intravenously for consecutive 3 days | Drug | Arm A |
| umbilical cord bloodAlso: UCBMNC ≥ 3*108 cells; HLA compatibility ≥ 5/6 | Biological | Arm A |
| Granulocyte-colony stimulating factorAlso: G-CSFGranulocyte-colony stimulating factor will be used when absolute neutrophil count ≤ 1.5 × 109/L | Drug | Arm A, Arm B |
| Recombinant human thrombopoietin / thrombopoietin receptor agonistAlso: rhTPO/TPO-RARecombinant human thrombopoietin or thrombopoietin receptor agonist will be used when platelet count ≤ 30 × 109/L | Drug | Arm A, Arm B |
| Recombinant human erythropoietinAlso: EPORecombinant human erythropoietin will be used when hemoglobin ≤ 85 g/L | Drug | Arm A, Arm B |
Primary outcome
The treatment response
Time frame: day +28
The rate of hematological improvement and hematological response of 2 arms
Survival
Time frame: 1 year
The rate of overall survival
3 secondary outcomes
Bone marrow recovery
Time frame: day +28
relapse and GVHD
Time frame: 3-month
Event free survival
Time frame: 1-year
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 9 Jun 2023.
- Results publication: PubMed 27088376Alchalby H, Yunus DR, Zabelina T, Ayuk F, Kroger N. Incidence and risk factors of poor graft function after allogeneic stem cell transplantation for myelofibrosis. Bone Marrow Transplant. 2016 Sep;51(9):1223-7. doi: 10.1038/bmt.2016.98. Epub 2016 Apr 18.
- Results publication: PubMed 16818281Larocca A, Piaggio G, Podesta M, Pitto A, Bruno B, Di Grazia C, Gualandi F, Occhini D, Raiola AM, Dominietto A, Bregante S, Lamparelli T, Tedone E, Oneto R, Frassoni F, Van Lint MT, Pogliani E, Bacigalupo A. Boost of CD34+-selected peripheral blood cells without further conditioning in patients with poor graft function following allogeneic stem cell transplantation. Haematologica. 2006 Jul;91(7):935-40.
- Results publication: PubMed 34492685Prabahran A, Koldej R, Chee L, Ritchie D. Clinical features, pathophysiology, and therapy of poor graft function post-allogeneic stem cell transplantation. Blood Adv. 2022 Mar 22;6(6):1947-1959. doi: 10.1182/bloodadvances.2021004537.
- Results publication: PubMed 33646303Tang Y, Chen J, Liu Q, Chu T, Pan T, Liang J, He XF, Chen F, Yang T, Ma X, Wu X, Hu S, Cao X, Hu X, Hu J, Liu Y, Qi J, Shen Y, Ruan C, Han Y, Wu D. Low-dose decitabine for refractory prolonged isolated thrombocytopenia after HCT: a randomized multicenter trial. Blood Adv. 2021 Mar 9;5(5):1250-1258. doi: 10.1182/bloodadvances.2020002790.
- Results publication: PubMed 26181032Han Y, Tang Y, Chen J, Liang J, Ye C, Ruan C, Wu D. Low-Dose Decitabine for Patients With Thrombocytopenia Following Allogeneic Hematopoietic Stem Cell Transplantation: A Pilot Therapeutic Study. JAMA Oncol. 2015 May;1(2):249-51. doi: 10.1001/jamaoncol.2014.316. No abstract available.
- Individual participant dataSharing planned: No
Related trials for Poor Graft Function
Not yet recruitingPhase 1/2
Anakinra for Prevention of Poor Graft Function in MDS Allo-HSCT
Poor Graft Function, Myelodysplastic Syndromes
Recruiting
CD34+ Selected Stem Cell for Poor Graft Function or Graft Failure
Graft Failure, Poor Graft Function
RecruitingPhase 1
The Safety and Efficacy of Umbilical Cord Blood Mononuclear Cells in Patients With Secondary Poor Graft Function After Hematopoietic Stem Cell Transplantation
Poor Graft Function
Not yet recruitingPhase 1/2
REscuing Bone Marrow Function in Patients with AplaStic AnaEmia and Bone Marrow FaiLure Post AllogEneiC Transplantation 2
Poor Graft Function, Aplastic Anemia Idiopathic
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.
Yue Han principal investigator
The First Affiliated Hospital of Soochow University
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing decitabine, umbilical cord blood and Granulocyte-colony stimulating factor in people with Poor Graft Function, run by The First Affiliated Hospital of Soochow University.
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 4 inclusion and 7 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 9 Jun 2023. 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 Yaqiong Tang (18896588075, tangyaqiong@suda.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The treatment response (time frame: day +28). 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?
On the ClinicalTrials.gov record for NCT05669079, which also lists 5 linked publications.
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 9 Jun 2023; AzTrials last refreshed its copy on 30 Oct 2026.