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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT03728322
Recruitment Status : Unknown
Verified October 2018 by Allife Medical Science and Technology Co., Ltd..
Recruitment status was:  Not yet recruiting
First Posted : November 2, 2018
Last Update Posted : November 5, 2018
Information provided by (Responsible Party):
Allife Medical Science and Technology Co., Ltd.

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.

Condition or disease Intervention/treatment Phase
Thalassemia Biological: iHSCs treatment group Early Phase 1

Detailed Description:
The purpose of this study is to evaluate the efficacy and safety of transplantation iHSCs intervent subjests with β-thalassemia mutations.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 12 participants
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Estimated Study Start Date : January 2019
Estimated Primary Completion Date : January 2020
Estimated Study Completion Date : January 2021

Arm Intervention/treatment
Experimental: iHSCs treatment group Biological: iHSCs treatment group
iHSCs intravenous injection

Primary Outcome Measures :
  1. Occurrence of treatment related adverse events as assessed by CTCAE v4.0 [ Time Frame: 1 year ]
    Defined as >= Grade 3 signs/symptoms, laboratory toxicities, and clinical events) that are possibly, likely, or definitely related to study treatment

Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years to 60 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.
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Responsible Party: Allife Medical Science and Technology Co., Ltd. Identifier: NCT03728322    
Other Study ID Numbers: HBB HSC-01
First Posted: November 2, 2018    Key Record Dates
Last Update Posted: November 5, 2018
Last Verified: October 2018

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hematologic Diseases
Genetic Diseases, Inborn