Transfusion Dependent ThalassemiaPhase 2NCT06657391
Evaluation of Recombinant Humanized Anti-CD25 Monoclonal Antibody for Preventing Graft-versus-host Disease After Haploidentical/matched Unrelated Donor Hematopoietic Stem Cell Transplantation in Patients with Transfusion-dependent Thalassemia
A Phase 2 study testing recombinant humanized anti-CD25 monoclonal antibody in people with Transfusion Dependent Thalassemia, run by Rongrong Liu at 5 sites in China.
Trial summary
Graft-versus-host disease (GVHD) is a major factor affecting the efficacy and quality of life of alternative donor transplantation in thalassemia major (TM), severely limiting the clinical application of alternative donor transplantation in TM.The purpose of this clinical trial is to evaluate whether recombinant… Read more about this study
- Who is it for?
- Ages 3 to 20 years
- Conditions the study is for
- Transfusion Dependent Thalassemia
- What is being tested?
- recombinant humanized anti-CD25 monoclonal antibody
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 396 participants
- Where
- 5 sitesChina
- Study dates
- 1 Oct 2024to 30 Sep 2027
- Phase
- Phase 2
- 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 3 to 20 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- patients with transfusion-dependent thalassemia;
- patients who are planning to receive matched unrelated donor hematopoietic stem cell transplantation (MUD-HSCT) or HLA haploidentical donor hematopoietic stem cell transplantation (HID-HSCT);
- physical condition score (Lansky/Karnofsky score) ≥ 70%;
- patients (or legal guardians) voluntarily participate in the study and sign the informed consent form
Who cannot join
- patients with HLA-matched hematopoietic stem cell donors and willing to receive HLA-matched hematopoietic stem cell transplantation;
- patients with known infectious diseases such as hepatitis B, hepatitis C, AIDS, syphilis, human T-lymphotropic virus, etc.;
- patients with serious active bacterial, viral, fungal, malaria or parasitic infections;
- patients with autoimmune deficiency diseases;
- patients with a history of malignant tumors or current malignant tumors;
- patients with important organ diseases or abnormal laboratory tests, including but not limited to: 1) patients with cirrhosis, liver fibrosis or active hepatitis, and/or abnormal liver function tests (alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≥2.5×ULN; alkaline phosphatase ≥2.5×ULN); 2) patients with heart disease, or left ventricular ejection fraction (LVEF) <60%, or severe iron deposition in the heart; 3) kidney disease, or blood creatinine ≥1.5×ULN with creatinine clearance <30% of normal level; 4) patients with endocrine dysfunction;
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- patients with uncorrected bleeding disease;
- patients with severe mental illness (such as severe depression, schizophrenia, etc.) or cognitive dysfunction (dementia, delirium, etc.), which are unable to cooperate with the study;
- peripheral blood white blood cell (WBC) count <3×10^9/L or platelet count <100×10^9/L;
- patients having received thalidomide treatment within the past 3 months;
- patients having received any type of gene and/or cell therapy in the past;
- patients with severe allergies;
- female patients who are pregnant, breastfeeding, or planning to become pregnant within 1 year of participating in this trial;
- patients who are participating in other clinical trials;
- other situations that are not suitable for participation in this clinical trial as assessed by the investigator.
Trial locations and contacts
5 sites in 1 country. Contact the study team or a site near you to ask about taking part.
5 sites in 1 country
China5 sites, 5 recruiting
- Recruiting
Hainan Provincial People's Hospital
Haikou, Hainan, 570000
Guyun Wang
- Recruiting
Liuzhou Workers' Hospital
Liuzhou, Guangxi, 545007
Chunjie Qin, +86 0772 3805890, qinchunjie1@163.com
- Recruiting
Maoming People's Hospital
Maoming, Guangdong, 525000
Xiong Zhang
- Recruiting
The First Affiliated Hospital of Guangxi Medical University
Nanning, Guangxi, 530000
Rongrong Liu, +86 0771 5356510, liurongrong@stu.gxmu.edu.cn
- Recruiting
Yulin Red Cross Hospital
Yulin, Guangxi, 537000
Hua Zhang
About this study
Official title: Evaluation of the Clinical Efficacy and Safety of Recombinant Humanized Anti-CD25 Monoclonal Antibody in Preventing Graft-versus-host Disease After Haploidentical / Matched Unrelated Donor Hematopoietic Stem Cell Transplantation in Patients with Transfusion-dependent Thalassemia: a Prospective, Multicenter, Open-label, Randomized Controlled Clinical Trial
Graft-versus-host disease (GVHD) is a major factor affecting the efficacy and quality of life of alternative donor transplantation in thalassemia major (TM), severely limiting the clinical application of alternative donor transplantation in TM.The purpose of this clinical trial is to evaluate whether recombinant humanized anti-CD25 monoclonal antibody is effective in preventing GVHD and its safety after haploidentical/matched unrelated donor hematopoietic stem cell transplantation. The main questions it aims to answer are:
- Does recombinant humanized anti-CD25 monoclonal antibody reduce the incidence of GVHD disease after haploidentical/matched unrelated donor hematopoietic stem cell transplantation?
- What medical problems will participants experience when using the recombinant humanized anti-CD25 monoclonal antibody? What is the quality of life after 2 years follow-up? In this clinical trail, participants will be randomly assigned to the intervention group or the control group by researchers in a 2:1 ratio. The intervention group will be given recombinant humanized anti-CD25 monoclonal antibody (1mg/Kg) combined with the standard GVHD prophylaxis after transplantation, while the control group will only receive the standard GVHD prophylaxis. The incidence of GVHD after transplantation in the two groups will be observed. The main evaluation is the clinical efficacy of recombinant humanized anti-CD25 monoclonal antibody in preventing aGVHD.
- Conditions
- Transfusion Dependent Thalassemia
- Being tested
- recombinant humanized anti-CD25 monoclonal antibody
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
recombinant humanized anti-CD25 monoclonal antibodyDrug
The intervention group received 4 doses of recombinant humanized anti-CD25 monoclonal antibody on days +7, +14, +28, and +42 after transplantation, with a recommended dose of 1 mg/kg.
| Study group | Role | What they receive |
|---|---|---|
| intervention group | Experimental | The intervention group will receive 4 doses of recombinant humanized anti-CD25 monoclonal antibody (Sunshine Guojian Pharmaceutical(Shanghai) Co.,Ltd.) on days +7, +14, +28, and +42 after transplantation, with a recommended dose of 1 mg/kg. Receives: Drug: recombinant humanized anti-CD25 monoclonal antibody |
| control group | No intervention | The control group will receive no treatment at the same time points. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Grade III-IV aGVHD
the incidence of grade III-IV aGVHD within 100 days after HID-HSCT/MUD-HSCT was compared between the intervention group and the control group.
Measured: within 100 days after HID-HSCT/MUD-HSCT
Secondary outcomes (10)
Grade II-IV aGVHD
the incidence of grade II-IV aGVHD within 100 days after HID-HSCT/MUD-HSCT was compared between the intervention group and the control group.
Measured: within 100 days after HID-HSCT/MUD-HSCT
Chronic graft-versus-host disease (cGVHD)
The incidence of cGVHD after HID-HSCT/MUD-HSCT was compared between the intervention group and the control group.
Measured: 2 years
Overall survival (OS)
Comparison of 2-year OS between the intervention group and the control group.
Measured: 2 years
Thalassemia-free survival (TFS)
comparison of the 2-year TFS between the intervention group and the control group.
Measured: 2 years
Transplantation-related mortality (TRM)
comparison of the 2-year TRM between the intervention group and the control group.
Measured: 2 years
Transplantation-related complications
comparison of the 2-year transplantation-related complications between the intervention group and the control group.
Measured: 2 years
Infection
comparison of the infection during the transplantation between the intervention group and the control group.
Measured: 2 years
Immune reconstitution
comparison of the immune reconstitution after transplantation between the intervention group and the control group.
Measured: 2 years
Quality of life
comparison of the quality of life between the intervention group and the control group by using the Pediatric Quality of Life Inventory Version 4.0 (PedsQL4.0) scale. The scales are comprissed of parallel self-report and parent proxy-report formats:
- Self-report includes ages 8 to 12, 13 to 18, and 18 to 25, with a total of 23 items.
- Parent proxy-report assesses parent's perceptions of their child's health-related quality of life. Parent proxy-report (ages 5 to 7) has 23 items, and Parent proxy-report (ages 2 to 4) has 21 items.
A 5-point response scale is utilized across self-report for ages and parent proxy-report (0 = never a problem; 1 = almost never a problem; 2 = sometimes a problem; 3 = often a problem; 4 = almost always a problem). Items are reverse-scored and linearly transformed to a 0 to 100 scale (0 = 100, 1 = 75, 2 = 50, 3 =25, 4 = 0), so that higher scores indicate better health-related quality of life.
Measured: 2 years
Adverse events
comparison of the occurrence of adverse events between the intervention group and the control group.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Oct 2024
- Study start1 Oct 2024
- Primary completion30 Sep 2027 (estimated)
- Study completion30 Sep 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Transfusion Dependent ThalassemiaStudies 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
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 396 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Rongrong Liu
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Rongrong Liu, First Affiliated Hospital of Guangxi Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06657391
- Sponsor's protocol ID
- 2024-K387-01
- Status last verified
- Oct 2024
- First submitted
- 21 Oct 2024
- Last updated
- 28 Oct 2024
- Results posted
- No
- Data monitoring committee
- Yes
- 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 28 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of the Clinical Efficacy and Safety of Recombinant Humanized Anti-CD25 Monoclonal Antibody in Preventing Graft-versus-host Disease After Haploidentical / Matched Unrelated Donor Hematopoietic Stem Cell Transplantation in Patients with Transfusion-dependent Thalassemia: a Prospective, Multicenter, Open-label, Randomized Controlled Clinical Trial
- ClinicalTrials.gov ID
- NCT06657391
- Acronym / sponsor ID
- 2024-K387-01
- Conditions
- Transfusion Dependent Thalassemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 396 participants (estimated)
- Lead sponsor
- Rongrong LiuUniversity, hospital or other
- Study start
- 1 Oct 2024 (actual)
- Primary completion
- 30 Sep 2027 (estimated)
- Study completion
- 30 Sep 2027 (estimated)
- First posted
- 24 Oct 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.
Graft-versus-host disease (GVHD) is a major factor affecting the efficacy and quality of life of alternative donor transplantation in thalassemia major (TM), severely limiting the clinical application of alternative donor transplantation in TM.The purpose of this clinical trial is to evaluate whether recombinant humanized anti-CD25 monoclonal antibody is effective in preventing GVHD and its safety after haploidentical/matched unrelated donor hematopoietic stem cell transplantation. The main questions it aims to answer are:
- Does recombinant humanized anti-CD25 monoclonal antibody reduce the incidence of GVHD disease after haploidentical/matched unrelated donor hematopoietic stem cell transplantation?
- What medical problems will participants experience when using the recombinant humanized anti-CD25 monoclonal antibody? What is the quality of life after 2 years follow-up? In this clinical trail, participants will be randomly assigned to the intervention group or the control group by researchers in a 2:1 ratio. The intervention group will be given recombinant humanized anti-CD25 monoclonal antibody (1mg/Kg) combined with the standard GVHD prophylaxis after transplantation, while the control group will only receive the standard GVHD prophylaxis. The incidence of GVHD after transplantation in the two groups will be observed. The main evaluation is the clinical efficacy of recombinant humanized anti-CD25 monoclonal antibody in preventing aGVHD.
Study design
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| intervention group | Experimental | Drug: recombinant humanized anti-CD25 monoclonal antibodyThe intervention group will receive 4 doses of recombinant humanized anti-CD25 monoclonal antibody (Sunshine Guojian Pharmaceutical(Shanghai) Co.,Ltd.) on days +7, +14, +28, and +42 after transplantation, with a recommended dose of 1 mg/kg. |
| control group | No intervention | –The control group will receive no treatment at the same time points. |
Who may qualify
- Ages
- 3 to 20 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- patients with transfusion-dependent thalassemia;
- patients who are planning to receive matched unrelated donor hematopoietic stem cell transplantation (MUD-HSCT) or HLA haploidentical donor hematopoietic stem cell transplantation (HID-HSCT);
- physical condition score (Lansky/Karnofsky score) ≥ 70%;
- patients (or legal guardians) voluntarily participate in the study and sign the informed consent form
Key exclusion criteria (15 listed)
- patients with HLA-matched hematopoietic stem cell donors and willing to receive HLA-matched hematopoietic stem cell transplantation;
- patients with known infectious diseases such as hepatitis B, hepatitis C, AIDS, syphilis, human T-lymphotropic virus, etc.;
- patients with serious active bacterial, viral, fungal, malaria or parasitic infections;
- patients with autoimmune deficiency diseases;
- patients with a history of malignant tumors or current malignant tumors;
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 |
|---|---|---|
| recombinant humanized anti-CD25 monoclonal antibodyThe intervention group received 4 doses of recombinant humanized anti-CD25 monoclonal antibody on days +7, +14, +28, and +42 after transplantation, with a recommended dose of 1 mg/kg. | Drug | intervention group |
Primary outcome
Grade III-IV aGVHD
Time frame: within 100 days after HID-HSCT/MUD-HSCT
the incidence of grade III-IV aGVHD within 100 days after HID-HSCT/MUD-HSCT was compared between the intervention group and the control group.
10 secondary outcomes
Grade II-IV aGVHD
Time frame: within 100 days after HID-HSCT/MUD-HSCT
Chronic graft-versus-host disease (cGVHD)
Time frame: 2 years
Overall survival (OS)
Time frame: 2 years
Thalassemia-free survival (TFS)
Time frame: 2 years
Transplantation-related mortality (TRM)
Time frame: 2 years
Transplantation-related complications
Time frame: 2 years
Infection
Time frame: 2 years
Immune reconstitution
Time frame: 2 years
Quality of life
Time frame: 2 years
Adverse events
Time frame: 2 years
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 28 Oct 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.
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing recombinant humanized anti-CD25 monoclonal antibody in people with Transfusion Dependent Thalassemia, run by Rongrong Liu at 5 sites 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 4 inclusion and 15 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: looking for participants now, as of the sponsor's last update on 28 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites 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?
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 Rongrong Liu (+86 0771 5356510, liurongrong@stu.gxmu.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Grade III-IV aGVHD (time frame: within 100 days after HID-HSCT/MUD-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 28 Oct 2024.