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

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ClinicalTrials.gov 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
Sponsor:
Information provided by (Responsible Party):
Allife Medical Science and Technology Co., Ltd.

Tracking Information
First Submitted Date  ICMJE October 31, 2018
First Posted Date  ICMJE November 2, 2018
Last Update Posted Date November 5, 2018
Estimated Study Start Date  ICMJE January 2019
Estimated Primary Completion Date January 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: October 31, 2018)
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
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE iHSCs With the Gene Correction of HBB Intervent Subjests With β-thalassemia Mutations
Official Title  ICMJE 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
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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Early Phase 1
Study Design  ICMJE Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Thalassemia
Intervention  ICMJE Biological: iHSCs treatment group
iHSCs intravenous injection
Study Arms  ICMJE Experimental: iHSCs treatment group
Intervention: Biological: iHSCs treatment group
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Unknown status
Estimated Enrollment  ICMJE
 (submitted: October 31, 2018)
12
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE January 2021
Estimated Primary Completion Date January 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 2 Years to 60 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Not Provided
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03728322
Other Study ID Numbers  ICMJE HBB HSC-01
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Allife Medical Science and Technology Co., Ltd.
Study Sponsor  ICMJE Allife Medical Science and Technology Co., Ltd.
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Allife Medical Science and Technology Co., Ltd.
Verification Date October 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP