Thalassemia Majors (Beta-Thalassemia Major)NCT06734520
Clinical Study of Super Transplantation in the Treatment of Severe β-thalassemia
A study testing super-transplantation in 3 severe β -thalassemia pediatric patients. in people with Thalassemia Majors (Beta-Thalassemia Major) and Haplo-identical Donors, run by Guangzhou Women and Children's Medical Center at 2 sites in China.
Trial summary
This is a single-arm, open label, phase 1 study in subjects with beta-thalassemia. This study will evaluate the safety and efficacy of preconditioning-free super-transplantation on beta-thalassemia pediatric patients. Read more about this study
- Who is it for?
- Ages 7 to 10 years
- Conditions the study is for
- Thalassemia Majors (Beta-Thalassemia Major), Haplo-identical Donors
- What is being tested?
- super-transplantation in 3 severe β -thalassemia pediatric patients.
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 2 sitesChina
- Study dates
- 1 Oct 2024to 1 Jul 2029
- Phase
- Interventional
- 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 7 to 10 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Diagnosis of severe β -thalassemia
- Age between 7-10 years old, male and female; Weight < 40kg
- The patient has or does not have an HLA-compatible or semi-compatible donor, but unconditional transplantation or refusal of blood stem cell transplantation; Patients with thalassemia gene therapy without conditions or refusal;
- There are fully compatible or incompatible HLA donors, and the physical examination meets the donor conditions;
- The patient and family members agree to receive hypertransplant therapy and sign a written informed consent prior to the transplant trial.
Who cannot join
- Mental patients;
- Participants in other drug clinical trials within the past 1 month;
- There are no suitable HLA-incompatible donors.
- Other researchers decide that it is not suitable to participate in this researcher.
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
China2 sites, 1 recruiting
- Active, not recruiting
Guangzhou Women and Children's Medical Center Affiliated to Guangzhou Medical University
Guangzhou, Guangdong, 51000
- Recruiting
Guangzhou Women and Children's Medical Center Affiliated to Guangzhou Medical University
Guangzhou, Guangdong
About this study
This is a single-arm, open label, phase 1 study in subjects with beta-thalassemia. This study will evaluate the safety and efficacy of preconditioning-free super-transplantation on beta-thalassemia pediatric patients.
Read the detailed description
Patient screening:
- Two months before transplantation, pediatric patients are evaluated by our center, and the patients meeting the inclusion criteria will be screened into the group.
- Investigators and doctors will give detailed information about the benefits and risks of participating in the study, and the informed consents are further signed.
Transplant conduction:
- Donor mobilization: HLA-fully matched or haplo-identical donors are mobilized subcutaneously for five days with human granulocyte colony-stimulating factor (G-CSF) and the peripheral blood mononuclear cells (PBMCs) that contain a certain numbers of CD34+ stem cells and CD3+ lymphocytes will be extracted by blood cell separators.
- Transplant regimen: Hydroxycarbamide will be used to reduce the total white blood cells before transplant. However, no traditional transplantation conditioning will be performed, including but not limited to busulfan, fludarabine, Cyclophosphamide, ATG, and irradiation. Splenectomy should be performed if the longitudinal diameter of the spleen exceeds the normal value by 4 cm.
- Cell infusion: Pediatric patients will be infused with the PBMCs intravenously at day 0.
- GVHD prophylaxis: The dosage of immunosuppressants is half of the conventional post-transplantation level, but adjustments to the dosage may be necessary based on specific circumstances.
- Infection management: Antibacterial, antifungal medications, and anti-Pneumocystis medications need to be administered during the period of granulocytopenia. The preemptive treatment for cytomegalovirus will be performed when the virus serological test is positive.
- Being tested
- super-transplantation in 3 severe β -thalassemia pediatric patients.
- Medical subject headings
- beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
super-transplantation in 3 severe β -thalassemia pediatric patients.Drug
Haplo-identical donors are mobilized with G-CSF and PBMCs that contain CD34+ stem cells and CD3+ lymphocytes will be extracted by blood cell separators. Then the patients will be infused with the PBMC
| Study group | Role | What they receive |
|---|---|---|
| Severe β -thalassemia | Experimental | Children with β-thalassemia major received super transplantation, and chimerism tests were performed at 3 months, 6 months, 9 months, and 1 year after transplantation to evaluate the safety and effectiveness. Receives: Drug: super-transplantation in 3 severe β -thalassemia pediatric patients. |
What is being measured
The primary outcome is the main question the study is designed to answer.
chimeric status
Detecting the chimerism in recipients' peripheral blood and bone marrow.Chimerism is generally measured in percentages(%)
Measured: 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 posted16 Dec 2024
- Study start1 Oct 2024
- Primary completion1 Jul 2029 (estimated)
- Study completion1 Jul 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Thalassemia Majors (Beta-Thalassemia Major)Studies 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
- Masking
- None (open label)
- Enrolment
- 3 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Guangzhou Women and Children's Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Hua Jiang, PHD, Study director, Guangzhou Medical University Affiliated Women's and Children's Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06734520
- Sponsor's protocol ID
- 2024217A01
- Status last verified
- Dec 2024
- First submitted
- 16 Nov 2024
- Last updated
- 16 Dec 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Share IPD with 5th Medical Center ofA General Hospital
Publications
Ali S, Mumtaz S, Shakir HA, Khan M, Tahir HM, Mumtaz S, Mughal TA, Hassan A, Kazmi SAR, Sadia, Irfan M, Khan MA. Current status of beta-thalassemia and its treatment strategies. Mol Genet Genomic Med. 2021 Dec;9(12):e1788. doi: 10.1002/mgg3.1788. Epub 2021 Nov 5.
PubMed 34738740Shafique F, Ali S, Almansouri T, Van Eeden F, Shafi N, Khalid M, Khawaja S, Andleeb S, Hassan MU. Thalassemia, a human blood disorder. Braz J Biol. 2021 Sep 3;83:e246062. doi: 10.1590/1519-6984.246062. eCollection 2021.
PubMed 34495151Dever DP, Bak RO, Reinisch A, Camarena J, Washington G, Nicolas CE, Pavel-Dinu M, Saxena N, Wilkens AB, Mantri S, Uchida N, Hendel A, Narla A, Majeti R, Weinberg KI, Porteus MH. CRISPR/Cas9 beta-globin gene targeting in human haematopoietic stem cells. Nature. 2016 Nov 17;539(7629):384-389. doi: 10.1038/nature20134. Epub 2016 Nov 7.
PubMed 27820943
Study at a glance
From the registry record, last updated by the sponsor on 16 Dec 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Study of Super Transplantation in the Treatment of Severe β-thalassemia
- ClinicalTrials.gov ID
- NCT06734520
- Acronym / sponsor ID
- 2024217A01
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 3 participants (estimated)
- Lead sponsor
- Guangzhou Women and Children's Medical CenterUniversity, hospital or other
- Study start
- 1 Oct 2024 (actual)
- Primary completion
- 1 Jul 2029 (estimated)
- Study completion
- 1 Jul 2029 (estimated)
- First posted
- 16 Dec 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.
This is a single-arm, open label, phase 1 study in subjects with beta-thalassemia. This study will evaluate the safety and efficacy of preconditioning-free super-transplantation on beta-thalassemia pediatric patients.
Detailed description
Patient screening:
- Two months before transplantation, pediatric patients are evaluated by our center, and the patients meeting the inclusion criteria will be screened into the group.
- Investigators and doctors will give detailed information about the benefits and risks of participating in the study, and the informed consents are further signed.
Transplant conduction:
- Donor mobilization: HLA-fully matched or haplo-identical donors are mobilized subcutaneously for five days with human granulocyte colony-stimulating factor (G-CSF) and the peripheral blood mononuclear cells (PBMCs) that contain a certain numbers of CD34+ stem cells and CD3+ lymphocytes will be extracted by blood cell separators.
- Transplant regimen: Hydroxycarbamide will be used to reduce the total white blood cells before transplant. However, no traditional transplantation conditioning will be performed, including but not limited to busulfan, fludarabine, Cyclophosphamide, ATG, and irradiation. Splenectomy should be performed if the longitudinal diameter of the spleen exceeds the normal value by 4 cm.
- Cell infusion: Pediatric patients will be infused with the PBMCs intravenously at day 0.
- GVHD prophylaxis: The dosage of immunosuppressants is half of the conventional post-transplantation level, but adjustments to the dosage may be necessary based on specific circumstances.
- Infection management: Antibacterial, antifungal medications, and anti-Pneumocystis medications need to be administered during the period of granulocytopenia. The preemptive treatment for cytomegalovirus will be performed when the virus serological test is positive.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Severe β -thalassemia | Experimental | Drug: super-transplantation in 3 severe β -thalassemia pediatric patients.Children with β-thalassemia major received super transplantation, and chimerism tests were performed at 3 months, 6 months, 9 months, and 1 year after transplantation to evaluate the safety and effectiveness. |
Who may qualify
- Ages
- 7 to 10 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Diagnosis of severe β -thalassemia
- Age between 7-10 years old, male and female; Weight < 40kg
- The patient has or does not have an HLA-compatible or semi-compatible donor, but unconditional transplantation or refusal of blood stem cell transplantation; Patients with thalassemia gene therapy without conditions or refusal;
- There are fully compatible or incompatible HLA donors, and the physical examination meets the donor conditions;
- The patient and family members agree to receive hypertransplant therapy and sign a written informed consent prior to the transplant trial.
Key exclusion criteria (4 listed)
- Mental patients;
- Participants in other drug clinical trials within the past 1 month;
- There are no suitable HLA-incompatible donors.
- Other researchers decide that it is not suitable to participate in this researcher.
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 |
|---|---|---|
| super-transplantation in 3 severe β -thalassemia pediatric patients.Haplo-identical donors are mobilized with G-CSF and PBMCs that contain CD34+ stem cells and CD3+ lymphocytes will be extracted by blood cell separators. Then the patients will be infused with the PBMC | Drug | Severe β -thalassemia |
Primary outcome
chimeric status
Time frame: 1 year after transplantation.
Detecting the chimerism in recipients' peripheral blood and bone marrow.Chimerism is generally measured in percentages(%)
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 16 Dec 2024.
- Background: PubMed 34738740Ali S, Mumtaz S, Shakir HA, Khan M, Tahir HM, Mumtaz S, Mughal TA, Hassan A, Kazmi SAR, Sadia, Irfan M, Khan MA. Current status of beta-thalassemia and its treatment strategies. Mol Genet Genomic Med. 2021 Dec;9(12):e1788. doi: 10.1002/mgg3.1788. Epub 2021 Nov 5.
- Background: PubMed 34495151Shafique F, Ali S, Almansouri T, Van Eeden F, Shafi N, Khalid M, Khawaja S, Andleeb S, Hassan MU. Thalassemia, a human blood disorder. Braz J Biol. 2021 Sep 3;83:e246062. doi: 10.1590/1519-6984.246062. eCollection 2021.
- Background: PubMed 27820943Dever DP, Bak RO, Reinisch A, Camarena J, Washington G, Nicolas CE, Pavel-Dinu M, Saxena N, Wilkens AB, Mantri S, Uchida N, Hendel A, Narla A, Majeti R, Weinberg KI, Porteus MH. CRISPR/Cas9 beta-globin gene targeting in human haematopoietic stem cells. Nature. 2016 Nov 17;539(7629):384-389. doi: 10.1038/nature20134. Epub 2016 Nov 7.
- Individual participant dataSharing planned: Yes. Share IPD with 5th Medical Center ofA General Hospital
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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.
Hua Jiang, PHD study director
Guangzhou Medical University Affiliated Women's and Children's Medical Center
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing super-transplantation in 3 severe β -thalassemia pediatric patients. in people with Thalassemia Majors (Beta-Thalassemia Major) and Haplo-identical Donors, run by Guangzhou Women and Children's Medical Center at 2 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 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: looking for participants now, as of the sponsor's last update on 16 Dec 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 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.
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 Hua Jiang, Doctor (86-020-38076501, jiang_hua18@sina.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
chimeric status (time frame: 1 year after transplantation.). 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 NCT06734520, which also lists 3 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 16 Dec 2024.