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

Sponsor
Allife Medical Science and Technology Co., Ltd. (Industry)
Overall Status
Unknown status
CT.gov ID
NCT03728322
Collaborator
(none)
12
1
24

Study Details

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.

Condition or Disease Intervention/Treatment Phase
  • 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.

Study Design

Study Type:
Interventional
Anticipated 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
Anticipated Study Start Date :
Jan 1, 2019
Anticipated Primary Completion Date :
Jan 1, 2020
Anticipated Study Completion Date :
Jan 1, 2021

Arms and Interventions

Arm Intervention/Treatment
Experimental: iHSCs treatment group

Biological: iHSCs treatment group
iHSCs intravenous injection

Outcome Measures

Primary Outcome Measures

  1. Occurrence of treatment related adverse events as assessed by CTCAE v4.0 [1 year]

    Defined as >= Grade 3 signs/symptoms, laboratory toxicities, and clinical events) that are possibly, likely, or definitely related to study treatment

Eligibility Criteria

Criteria

Ages Eligible for Study:
2 Years to 60 Years
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%

  1. Chest X-ray and ecg test results were normal, no serious cardiopulmonary diseases

  2. Subjects survival was expected≥6 months

  3. 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

  4. 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

  1. Subjects have one kinds of tumors within 5 years

  2. Active hepatitis B (HBV DNA>1000copy/mL), hepatitis C or HIV infection.

  3. Subjects have an infectious diseases that cannot be controlled within 4 weeks

  4. subjects have severe central nervous system disease or epilepsy

  5. 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

  6. Women in pregnancy (positive urine/blood pregnancy test) or lactation

  7. Subjects who have other conditions that were not appropriate for the group determined by the researchers.

Contacts and Locations

Locations

No locations specified.

Sponsors and Collaborators

  • Allife Medical Science and Technology Co., Ltd.

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
Allife Medical Science and Technology Co., Ltd.
ClinicalTrials.gov Identifier:
NCT03728322
Other Study ID Numbers:
  • HBB HSC-01
First Posted:
Nov 2, 2018
Last Update Posted:
Nov 5, 2018
Last Verified:
Oct 1, 2018
Studies a U.S. FDA-regulated Drug Product:
No
Studies a U.S. FDA-regulated Device Product:
No
Additional relevant MeSH terms:

Study Results

No Results Posted as of Nov 5, 2018