search
Back to results

Thalassemia Treatment Based on the Stem Cell Technology

Primary Purpose

Beta-Thalassemia

Status
Unknown status
Phase
Not Applicable
Locations
Study Type
Interventional
Intervention
Hematopoetic stem cells
Sponsored by
Xiaofang Sun
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Beta-Thalassemia focused on measuring thalassemia, stem cell

Eligibility Criteria

1 Year - 18 Years (Child, Adult)MaleAccepts Healthy Volunteers

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.

Sites / Locations

    Arms of the Study

    Arm 1

    Arm 2

    Arm Type

    Experimental

    No Intervention

    Arm Label

    Intervention Patient

    non-intervention Patient

    Arm Description

    Hematopoetic stem cells differentiated from beta-thalassemia induced pluripotent stem cells.

    No treatment

    Outcomes

    Primary Outcome Measures

    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,
    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.
    Effect of cell transplantation
    The granulocyte plant living standards and platelet plant living standards will be combined to measure the effect of cell transplantation.

    Secondary Outcome Measures

    Full Information

    First Posted
    June 23, 2017
    Last Updated
    July 17, 2017
    Sponsor
    Xiaofang Sun
    Collaborators
    Nanfang Hospital, Southern Medical University, Guangzhou Institutes of Biomedicine and Health Chinese Academy of Sciences, Third Affiliated Hospital, Sun Yat-Sen University
    search

    1. Study Identification

    Unique Protocol Identification Number
    NCT03222453
    Brief Title
    Thalassemia Treatment Based on the Stem Cell Technology
    Official Title
    The Third Affiliated Hospital of Guangzhou Medical University
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    July 2017
    Overall Recruitment Status
    Unknown status
    Study Start Date
    January 1, 2015 (Actual)
    Primary Completion Date
    June 14, 2016 (Actual)
    Study Completion Date
    December 31, 2017 (Anticipated)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor-Investigator
    Name of the Sponsor
    Xiaofang Sun
    Collaborators
    Nanfang Hospital, Southern Medical University, Guangzhou Institutes of Biomedicine and Health Chinese Academy of Sciences, Third Affiliated Hospital, Sun Yat-Sen University

    4. Oversight

    Studies a U.S. FDA-regulated Drug Product
    No
    Studies a U.S. FDA-regulated Device Product
    No
    Data Monitoring Committee
    No

    5. Study Description

    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
    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.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Beta-Thalassemia
    Keywords
    thalassemia, stem cell

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Not Applicable
    Interventional Study Model
    Parallel Assignment
    Masking
    Participant
    Allocation
    Non-Randomized
    Enrollment
    2 (Actual)

    8. Arms, Groups, and Interventions

    Arm Title
    Intervention Patient
    Arm Type
    Experimental
    Arm Description
    Hematopoetic stem cells differentiated from beta-thalassemia induced pluripotent stem cells.
    Arm Title
    non-intervention Patient
    Arm Type
    No Intervention
    Arm Description
    No treatment
    Intervention Type
    Biological
    Intervention Name(s)
    Hematopoetic stem cells
    Intervention Description
    Patient will inject with hematopoetic stem cells differentiated from beta-thalassemia induced pluripotent stem cells
    Primary Outcome Measure Information:
    Title
    The check of granulocyte transplantation-granulocyte plant living standards
    Description
    Granulocyte plant living standards for three days in a row after transplantation granulocyte should be greater than 0.5 x 109 / L,
    Time Frame
    3 days
    Title
    The check of granulocyte transplantation-platelet plant living standards
    Description
    Platelet plant living standard for seven consecutive days after transplantation the platelet should be greater than 20 x 109 / L and infusion.
    Time Frame
    seven consecutive days
    Title
    Effect of cell transplantation
    Description
    The granulocyte plant living standards and platelet plant living standards will be combined to measure the effect of cell transplantation.
    Time Frame
    seven days

    10. Eligibility

    Sex
    Male
    Gender Based
    Yes
    Gender Eligibility Description
    Clinical diagnosis.
    Minimum Age & Unit of Time
    1 Year
    Maximum Age & Unit of Time
    18 Years
    Accepts Healthy Volunteers
    Accepts Healthy Volunteers
    Eligibility Criteria
    Inclusion Criteria: 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 Exclusion Criteria: 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; 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.

    12. IPD Sharing Statement

    Plan to Share IPD
    Undecided

    Learn more about this trial

    Thalassemia Treatment Based on the Stem Cell Technology

    We'll reach out to this number within 24 hrs