Beta-ThalassemiaNCT03222453
Thalassemia Treatment Based on the Stem Cell Technology
A study testing Hematopoetic stem cells in people with Beta-Thalassemia, run by Xiaofang Sun.
Trial summary
In order to study the transplantation effect of hematopoetic stem cells from beta-thalassemia induced pluripotent stem cells. We applied clinical grade source of autologous hematopoietic stem cell for the treatment of beta-thalassemia patients, detecting the homing of hematopoietic stem cell transplantation, the… Read more about this study
- Who is it for?
- Ages 1 year to 18 years, men onlyHealthy volunteers can take part
- Conditions the study is for
- Beta-Thalassemia
- What is being tested?
- Hematopoetic stem cells
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- No sites listed yet
- Study dates
- 1 Jan 2015to 31 Dec 2017
- 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 1 year to 18 years
- Men only
- Healthy volunteers accepted
- Child, Adult
Who may be able to join
- The genetic diagnosis confirmed homozygous for beta to poverty or double heterozygote, clinical severe anemia;
- Aged 1 ~ 18 years old, no obvious iron overload cause organ damage;
- Has a suitable donor HLA high resolution;
- The informed consent
Who cannot join
- There has been a significant increase in the level of tumor markers (AFP/CEA/CA199 / CA125) in the past five years.
- Serious primary diseases such as cardiovascular, liver and hematopoietic systems; Those who have major organs with serious function; An adrenal disease or other disease that causes the organ failure of the organ;
- An autoimmune disease, a family history of genetic disease, and an abnormal thyroid function;
- Peripheral blood chromosome checking for nuclear aliens;
- HIV, hepatitis b or hepatitis c;
- A person with a history of severe drug allergies or an allergic person;
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- Those who do not expect to live for more than one year;
- The researchers suggest that the patient may have a potential or have a disorder (such as an uncontrolled infection, right heart failure, pulmonary hypertension) that is interfering with this study.
- In the first six months, alcohol and other substance abuse were not allowed;
- Subjects who participated in other clinical trials or participated in other clinical trials within 3 months.
About this study
Official title: The Third Affiliated Hospital of Guangzhou Medical University
In order to study the transplantation effect of hematopoetic stem cells from beta-thalassemia induced pluripotent stem cells. We applied clinical grade source of autologous hematopoietic stem cell for the treatment of beta-thalassemia patients, detecting the homing of hematopoietic stem cell transplantation, the differentiation of hematopoietic stem cells in vivo and the hemoglobin beta-chain (HBB) protein expression in the body of recovery, etc., as well as to make a research on the efficacy and safety of hematopoietic stem cells from beta-thalassemia induced pluripotent stem cells.
Read the detailed description
- On a Good Manufacturing Practice(GMP) condition, we establish non-exotic of different mutation types of beta-thalassemia -induced pluripotent stem cells（iPS） and make a comparisons of the stability and the differentiation of efficiency of these iPS cells inducing from different sources with foreign gene integration.
- Using the technique of artificial nuclease and in situ repairmen, we establish efficient system for different beta-thalassemia mutation site and in view of the security of these system.
- Establish a repaired beta-thalasemia gene mutated differentiation of iPS technology system.
- Build a functional gene therapy self-limiting slow viruses, optimizing the preparation system and to establish a virus preparation of infection of hematopoietic stem cell technology system under the GMP condition.
- Establish humanized beta-mice model, evaluate the safety of the iPS cell of gene therapy and efficiency before the clinical experiment.
- Improve the existing hematopoietic stem cell transplant(HSCT) clinical application solutions, detect rate of graft rejection, rate of transplantation and other indicators, finish the evaluation of application by clinical cases.
- Conditions
- Beta-Thalassemia
- Being tested
- Hematopoetic stem cells
- Keywords
- thalassemia, stem cell
- Medical subject headings
- beta-Thalassemia, Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Hematopoetic stem cellsBiological
Patient will inject with hematopoetic stem cells differentiated from beta-thalassemia induced pluripotent stem cells
| Study group | Role | What they receive |
|---|---|---|
| Intervention Patient | Experimental | Hematopoetic stem cells differentiated from beta-thalassemia induced pluripotent stem cells. Receives: Biological: Hematopoetic stem cells |
| non-intervention Patient | No intervention | No treatment |
What is being measured
The primary outcome is the main question the study is designed to answer.
The check of granulocyte transplantation-granulocyte plant living standards
Granulocyte plant living standards for three days in a row after transplantation granulocyte should be greater than 0.5 x 109 / L,
Measured: 3 days
The check of granulocyte transplantation-platelet plant living standards
Platelet plant living standard for seven consecutive days after transplantation the platelet should be greater than 20 x 109 / L and infusion.
Measured: seven consecutive days
Effect of cell transplantation
The granulocyte plant living standards and platelet plant living standards will be combined to measure the effect of cell transplantation.
Measured: seven days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Jul 2017
- Study start1 Jan 2015
- Primary completion14 Jun 2016
- Study completion31 Dec 2017 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Beta-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 N/A
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Xiaofang Sun
- Sponsor type
- University, hospital or other
- Collaborators
- Nanfang Hospital, Southern Medical University, Guangzhou Institutes of Biomedicine and Health Chinese Academy of Sciences, Third Affiliated Hospital, Sun Yat-Sen University
- Responsible party
- Sponsor-investigator, Xiaofang Sun, The Third Affiliated Hospital of Guangzhou Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03222453
- Sponsor's protocol ID
- 201508020258
- Status last verified
- Jul 2017
- First submitted
- 23 Jun 2017
- Last updated
- 19 Jul 2017
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 19 Jul 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Third Affiliated Hospital of Guangzhou Medical University
- ClinicalTrials.gov ID
- NCT03222453
- Acronym / sponsor ID
- 201508020258
- Conditions
- Beta-Thalassemia
- Keywords
- thalassemia, stem cell
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Xiaofang SunUniversity, hospital or other
- Collaborators
- Nanfang Hospital, Southern Medical University, Guangzhou Institutes of Biomedicine and Health Chinese Academy of Sciences, Third Affiliated Hospital, Sun Yat-Sen University
- Study start
- 1 Jan 2015 (actual)
- Primary completion
- 14 Jun 2016 (actual)
- Study completion
- 31 Dec 2017 (estimated)
- First posted
- 19 Jul 2017 (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.
In order to study the transplantation effect of hematopoetic stem cells from beta-thalassemia induced pluripotent stem cells. We applied clinical grade source of autologous hematopoietic stem cell for the treatment of beta-thalassemia patients, detecting the homing of hematopoietic stem cell transplantation, the differentiation of hematopoietic stem cells in vivo and the hemoglobin beta-chain (HBB) protein expression in the body of recovery, etc., as well as to make a research on the efficacy and safety of hematopoietic stem cells from beta-thalassemia induced pluripotent stem cells.
Detailed description
- On a Good Manufacturing Practice(GMP) condition, we establish non-exotic of different mutation types of beta-thalassemia -induced pluripotent stem cells（iPS） and make a comparisons of the stability and the differentiation of efficiency of these iPS cells inducing from different sources with foreign gene integration.
- Using the technique of artificial nuclease and in situ repairmen, we establish efficient system for different beta-thalassemia mutation site and in view of the security of these system.
- Establish a repaired beta-thalasemia gene mutated differentiation of iPS technology system.
- Build a functional gene therapy self-limiting slow viruses, optimizing the preparation system and to establish a virus preparation of infection of hematopoietic stem cell technology system under the GMP condition.
- Establish humanized beta-mice model, evaluate the safety of the iPS cell of gene therapy and efficiency before the clinical experiment.
- Improve the existing hematopoietic stem cell transplant(HSCT) clinical application solutions, detect rate of graft rejection, rate of transplantation and other indicators, finish the evaluation of application by clinical cases.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (participant)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Intervention Patient | Experimental | Biological: Hematopoetic stem cellsHematopoetic stem cells differentiated from beta-thalassemia induced pluripotent stem cells. |
| non-intervention Patient | No intervention | –No treatment |
Who may qualify
- Ages
- 1 year to 18 years
- Sex
- Male only
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- The genetic diagnosis confirmed homozygous for beta to poverty or double heterozygote, clinical severe anemia;
- Aged 1 ~ 18 years old, no obvious iron overload cause organ damage;
- Has a suitable donor HLA high resolution;
- The informed consent
Key exclusion criteria (10 listed)
- There has been a significant increase in the level of tumor markers (AFP/CEA/CA199 / CA125) in the past five years.
- Serious primary diseases such as cardiovascular, liver and hematopoietic systems; Those who have major organs with serious function; An adrenal disease or other disease that causes the organ failure of the organ;
- An autoimmune disease, a family history of genetic disease, and an abnormal thyroid function;
- Peripheral blood chromosome checking for nuclear aliens;
- HIV, hepatitis b or hepatitis c;
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 |
|---|---|---|
| Hematopoetic stem cellsPatient will inject with hematopoetic stem cells differentiated from beta-thalassemia induced pluripotent stem cells | Biological | Intervention Patient |
Primary outcome
The check of granulocyte transplantation-granulocyte plant living standards
Time frame: 3 days
Granulocyte plant living standards for three days in a row after transplantation granulocyte should be greater than 0.5 x 109 / L,
The check of granulocyte transplantation-platelet plant living standards
Time frame: seven consecutive days
Platelet plant living standard for seven consecutive days after transplantation the platelet should be greater than 20 x 109 / L and infusion.
Effect of cell transplantation
Time frame: seven days
The granulocyte plant living standards and platelet plant living standards will be combined to measure the effect of cell transplantation.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Jul 2017.
- Individual participant dataSharing planned: Undecided
Related trials for Beta-Thalassemia
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Active, not recruitingPhase 3
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)
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Investigating the Mechanistic Effects of Mitapivat in Subjects With Sickle Cell Disease
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A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)
Transfusion-dependent Alpha-Thalassemia, Transfusion-dependent Beta-Thalassemia
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.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A study testing Hematopoetic stem cells in people with Beta-Thalassemia, run by Xiaofang Sun.
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 10 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 19 Jul 2017. 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.
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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The check of granulocyte transplantation-granulocyte plant living standards (time frame: 3 days). The study lists 3 primary outcomes in all. 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 19 Jul 2017.