Clinical trial NCT03222453: Thalassemia Treatment Based on the Stem Cell Technology
Official title: The Third Affiliated Hospital of Guangzhou Medical University
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: NA
Conditions: Beta-Thalassemia
Keywords: thalassemia
stem cell
Lead sponsor: The Third Affiliated Hospital of Guangzhou Medical University
Sponsor class: OTHER
Collaborators: name: Nanfang Hospital, Southern Medical University
name: Guangzhou Institutes of Biomedicine and Health Chinese Academy of Sciences
name: Third Affiliated Hospital, Sun Yat-Sen University
First posted: 2017-07-19
Enrollment: 2
Sex: MALE
Minimum age (years): 1
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: yes
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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 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: 1. 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.
2. 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.
3. Establish a repaired beta-thalasemia gene mutated differentiation of iPS technology system.
4. 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.
5. Establish humanized beta-mice model, evaluate the safety of the iPS cell of gene therapy and efficiency before the clinical experiment.
6. 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.

Eligibility criteria: Inclusion Criteria:

1. The genetic diagnosis confirmed homozygous for beta to poverty or double heterozygote, clinical severe anemia;
2. Aged 1 \~ 18 years old, no obvious iron overload cause organ damage;
3. Has a suitable donor HLA high resolution;
4. The informed consent

Exclusion Criteria:

1. There has been a significant increase in the level of tumor markers (AFP/CEA/CA199 / CA125) in the past five years.
2. 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;
3. An autoimmune disease, a family history of genetic disease, and an abnormal thyroid function;
4. Peripheral blood chromosome checking for nuclear aliens;
5. HIV, hepatitis b or hepatitis c;
6. A person with a history of severe drug allergies or an allergic person;
7. Those who do not expect to live for more than one year;
8. 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.
9. In the first six months, alcohol and other substance abuse were not allowed;
10. Subjects who participated in other clinical trials or participated in other clinical trials within 3 months.

Primary outcomes:
measure: The check of granulocyte transplantation-granulocyte plant living standards
timeFrame: 3 days
measure: The check of granulocyte transplantation-platelet plant living standards
timeFrame: seven consecutive days
measure: Effect of cell transplantation
timeFrame: seven days
Secondary outcomes:
Other outcomes:
Locations:
Study officials:
References: