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iHSCs With the Gene Correction of HBB Intervent Subjests With β-thalassemia Mutations

Primary Purpose

Thalassemia

Status
Unknown status
Phase
Early Phase 1
Locations
Study Type
Interventional
Intervention
iHSCs treatment group
Sponsored by
Allife Medical Science and Technology Co., Ltd.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Thalassemia

Eligibility Criteria

2 Years - 60 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. Subjects ≥ 2 and ≤ 60 years of age
  2. Subjects was confirmed the transfusion-dependent β-thalassemia

  3. Adequate organ function, as defined by:

    Serum creatinine ≤ 1.5 mg/dl ； Serum ALT/AST）≤2.5×ULN；ALB≥25g/L； Serum total bilirubin < 1.5x ULN Left ventricular ejection fraction≥50%

  4. Chest X-ray and ecg test results were normal, no serious cardiopulmonary diseases
  5. Subjects survival was expected≥6 months
  6. Adult patients were willing to use reliable contraceptives (such as condoms) and not to donate sperm throughout the study period and within three months of discharge
  7. Subjects and the guardians able to undergo post-physical therapy/rehabilitation

Exclusion Criteria:

  1. Subjects allergic to macromolecular biological agents such as antibodies or cytokines
  2. Subjects receipt of any investigational clinical trials within 3 months.
  3. Subjects previous treatment with any hematopoietic stem cell transplantation or other organ transplantation
  4. Uncontrolled bleeding symptoms

  5. Severe cardiovascular disease is known, including any of the following:

    Myocardial infarction or thrombosis has occurred in the past six months Subjects with unstable angina pectoris Subjects with Class III/IV cardiovascular disability according to the New York Heart Association Classification

  6. Subjects have one kinds of tumors within 5 years
  7. Active hepatitis B (HBV DNA>1000copy/mL), hepatitis C or HIV infection.
  8. Subjects have an infectious diseases that cannot be controlled within 4 weeks
  9. subjects have severe central nervous system disease or epilepsy
  10. Subjects are Suffering from mental illness; Patients with alcohol dependence, drug abuse, drug addiction, and medical, psychological or social conditions that may interfere with research or have an impact on the evaluation of research results
  11. Women in pregnancy (positive urine/blood pregnancy test) or lactation
  12. Subjects who have other conditions that were not appropriate for the group determined by the researchers.

Sites / Locations

    Arms of the Study

    Arm 1

    Arm Type

    Experimental

    Arm Label

    iHSCs treatment group

    Arm Description

    Outcomes

    Primary Outcome Measures

    Occurrence of treatment related adverse events as assessed by CTCAE v4.0
    Defined as >= Grade 3 signs/symptoms, laboratory toxicities, and clinical events) that are possibly, likely, or definitely related to study treatment

    Secondary Outcome Measures

    Full Information

    First Posted
    October 31, 2018
    Last Updated
    November 1, 2018
    Sponsor
    Allife Medical Science and Technology Co., Ltd.
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    1. Study Identification

    Unique Protocol Identification Number
    NCT03728322
    Brief Title
    iHSCs With the Gene Correction of HBB Intervent Subjests With β-thalassemia Mutations
    Official Title
    A Safety and Efficacy Study of a Single Center, Open-label, Single Arm About the Gene Correction of HBB in Patient-specific iHSCs Using CRISPR/Cas9 That Intervent Subjests With β-thalassemia Mutations
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    October 2018
    Overall Recruitment Status
    Unknown status
    Study Start Date
    January 2019 (Anticipated)
    Primary Completion Date
    January 2020 (Anticipated)
    Study Completion Date
    January 2021 (Anticipated)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    Allife Medical Science and Technology Co., Ltd.

    4. Oversight

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

    5. Study Description

    Brief Summary
    This is a single centre、single arm、open-label study，to investigate the safety and efficacy of the gene correction of HBB in patient-specific iHSCs using CRISPR/Cas9.
    Detailed Description
    The purpose of this study is to evaluate the efficacy and safety of transplantation iHSCs intervent subjests with β-thalassemia mutations.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Thalassemia

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Early Phase 1
    Interventional Study Model
    Sequential Assignment
    Masking
    None (Open Label)
    Enrollment
    12 (Anticipated)

    8. Arms, Groups, and Interventions

    Arm Title
    iHSCs treatment group
    Arm Type
    Experimental
    Intervention Type
    Biological
    Intervention Name(s)
    iHSCs treatment group
    Intervention Description
    iHSCs intravenous injection
    Primary Outcome Measure Information:
    Title
    Occurrence of treatment related adverse events as assessed by CTCAE v4.0
    Description
    Defined as >= Grade 3 signs/symptoms, laboratory toxicities, and clinical events) that are possibly, likely, or definitely related to study treatment
    Time Frame
    1 year

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    2 Years
    Maximum Age & Unit of Time
    60 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    Inclusion Criteria: Subjects ≥ 2 and ≤ 60 years of age Subjects was confirmed the transfusion-dependent β-thalassemia Adequate organ function, as defined by: Serum creatinine ≤ 1.5 mg/dl ； Serum ALT/AST）≤2.5×ULN；ALB≥25g/L； Serum total bilirubin < 1.5x ULN Left ventricular ejection fraction≥50% Chest X-ray and ecg test results were normal, no serious cardiopulmonary diseases Subjects survival was expected≥6 months Adult patients were willing to use reliable contraceptives (such as condoms) and not to donate sperm throughout the study period and within three months of discharge Subjects and the guardians able to undergo post-physical therapy/rehabilitation Exclusion Criteria: Subjects allergic to macromolecular biological agents such as antibodies or cytokines Subjects receipt of any investigational clinical trials within 3 months. Subjects previous treatment with any hematopoietic stem cell transplantation or other organ transplantation Uncontrolled bleeding symptoms Severe cardiovascular disease is known, including any of the following: Myocardial infarction or thrombosis has occurred in the past six months Subjects with unstable angina pectoris Subjects with Class III/IV cardiovascular disability according to the New York Heart Association Classification Subjects have one kinds of tumors within 5 years Active hepatitis B (HBV DNA>1000copy/mL), hepatitis C or HIV infection. Subjects have an infectious diseases that cannot be controlled within 4 weeks subjects have severe central nervous system disease or epilepsy Subjects are Suffering from mental illness; Patients with alcohol dependence, drug abuse, drug addiction, and medical, psychological or social conditions that may interfere with research or have an impact on the evaluation of research results Women in pregnancy (positive urine/blood pregnancy test) or lactation Subjects who have other conditions that were not appropriate for the group determined by the researchers.

    12. IPD Sharing Statement

    Learn more about this trial

    iHSCs With the Gene Correction of HBB Intervent Subjests With β-thalassemia Mutations

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