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

Thalassemia Clinical Trial

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT03728322
• Other study ID #: HBB HSC-01
• Status: Not yet recruiting
• Phase: Early Phase 1
• Start date: January 2019
• Est. completion date: January 2021

Study information

• Verified date: October 2018
• Source: Allife Medical Science and Technology Co., Ltd.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Description:

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

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 12
• Est. completion date: January 2021
• Est. primary completion date: January 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 60 Years

Eligibility

Inclusion Criteria:

1. Subjects = 2 and = 60 years of age

2. Subjects was confirmed the transfusion-dependent ß-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.

Related Conditions & MeSH terms

• beta-Thalassemia
• Thalassemia

Intervention

Biological:
• iHSCs treatment group
iHSCs intravenous injection

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Allife Medical Science and Technology Co., Ltd.

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	1 year